OCE Publications
Journal publications by FDA oncology/hematology reviewers
FDA's Oncology Center of Excellence publishes numerous articles in peer-reviewed scientific journals, including original research papers, drug approval summaries, and regulatory perspectives.
Intramural research papers largely involve analysis of clinical trial data submitted with marketing applications. Such analyses have been particularly helpful to evaluate novel endpoints, including measures of surrogacy, and investigate safety concerns across trials. Other intramural research papers are produced by OCE’s Project NextGen, which leverages the expertise of the Oncology Translational Research Laboratory.
Extramural research papers address a wide variety of topics, including the scientific interest areas identified by the OCE Scientific Collaborative. These papers are typically produced through funded and unfunded (see Selected Currently Active Oncology RWE Collaborations) research. Extramural research projects may involve prospective or retrospective data analysis, as well as laboratory-based studies.
Open the sections below to browse publications.
Research Papers
- Integrating 4 methods to evaluate physical function in patients with cancer (In4M): protocol for a prospective cohort study. Thanarajasingam G, Kluetz P, Bhatnagar V, Brown A, Cathcart-Rake E, Diamond M, Faust L, Fiero MH, Huntington S, Jeffery MM, Jones L, Noble B, Paludo J, Powers B, Ross JS, Ritchie JD, Ruddy K, Schellhorn S, Tarver M, Dueck AC, Gross C. BMJ Open. 2024 Jan 10;14(1):e074030. doi: 10.1136/bmjopen-2023-074030. PMID: 38199641.
- Methodology for Good Machine Learning with Multi-Omics Data. Coroller T, Sahiner B, Amatya A, Gossmann A, Karagiannis K, Moloney C, Samala RK, Santana-Quintero L, Solovieff N, Wang C, Amiri-Kordestani L, Cao Q, Cha KH, Charlab R, Cross FH Jr, Hu T, Huang R, Kraft J, Krusche P, Li Y, Li Z, Mazo I, Paul R, Schnakenberg S, Serra P, Smith S, Song C, Su F, Tiwari M, Vechery C, Xiong X, Zarate JP, Zhu H, Chakravartty A, Liu Q, Ohlssen D, Petrick N, Schneider JA, Walderhaug M, Zuber E. Clin Pharmacol Ther. 2024 Apr;115(4):745-757. doi: 10.1002/cpt.3105. PMID: 37965805.
- Considering endpoints for comparative tolerability of cancer treatments using patient report given the estimand framework. Peipert JD, Breslin M, Basch E, Calvert M, Cella D, Smith ML, Thanarajasingam G, Roydhouse J. J Biopharm Stat. 2024 Feb 15:1-19. doi: 10.1080/10543406.2024.2313060. PMID: 38358291.
- Review of Racial and Ethnic Representation of Participants Enrolled in Pediatric Clinical Trials of Oncology Drugs Conducted Through FDA Written Requests. Fashoyin-Aje LA, Akalu AY, Boehmer J, Pazdur R, Donoghue M, Reaman GH. JAMA Oncol. 2024 Jan 4. doi: 10.1001/jamaoncol.2023.5781. PMID: 38175622.
- Correlations of response rate and progression-free survival with overall survival in immunotherapy trials for metastatic non-small-cell lung cancer: an FDA pooled analysis. Goulart BHL, Mushti SL, Chatterjee S, Larkins E, Mishra-Kalyani PS, Pazdur R, Kluetz PG, Singh H. Lancet Oncol. 2024 Mar 5:S1470-2045(24)00040-8. doi: 10.1016/S1470-2045(24)00040-8. PMID: 38458207.
- Efficacy of Poly(ADP-ribose) Polymerase Inhibitors by Individual Genes in Homologous Recombination Repair Gene-Mutated Metastatic Castration-Resistant Prostate Cancer: A US Food and Drug Administration Pooled Analysis. Fallah J, Xu J, Weinstock C, Gao X, Heiss BL, Maguire WF, Chang E, Agrawal S, Tang S, Amiri-Kordestani L, Pazdur R, Kluetz PG, Suzman DL. J Clin Oncol. 2024 Mar 14:JCO2302105. doi: 10.1200/JCO.23.02105. PMID: 38484203.
- TorchSurv: A Lightweight Package for Deep Survival Analysis. Monod M, Krusche P, Cao Q, Sahiner B, Petrick N, Ohlssen D, Coroller T. ArXive. 2024 Apr 16. doi: 10.48550/arXiv.2404.10761.
- Facets of physical function assessed by patient-reported outcome measures in oncology research. Coles T, Fillipo R, Plyler K, Hernandez A, Henke DM, Arizmendi C, Cantrell S, Lagoo-Deenadayalan S, Goodwin CR, LeBlanc TW, Horodniceanu EG, Bhatnagar V, Reeve BB, Weinfurt K. Qual Life Res. 2024 Jul;33(7):1819-1828. doi: 10.1007/s11136-024-03640-0. PMID: 38642217.
- What facets of physical function are most important to adults diagnosed with cancer? Coles T, Plyler K, Hernandez A, Fillipo R, Henke DM, Arizmendi C, Lagoo-Deenadayalan S, Goodwin CR, LeBlanc TW, Horodniceanu EG, Bhatnagar V, Reeve BB, Weinfurt KP. Qual Life Res. 2024 Jul;33(7):1829-1839. doi: 10.1007/s11136-024-03654-8. PMID: 38642219.
- Image registration based automated lesion correspondence pipeline for longitudinal CT data. Mukherjee S, Coroller T, Wang C, Samala RK, Hu T, Gokcay D, Petrick N, Sahiner B, Cao Q. ArXive. 2024 Apr 25. doi: 10.48550/arXiv.2404.1654.
- Comprehensive profiling cancer immunity with multimodal approaches for clinical management. Chen G, Shi Y, Xiao W, Kreil DP. Front Immunol. 2024 Apr 30; 15:1421576. doi: 10.3389/fimmu.2024.1421576. PMID: 38745672.
- Impact of Increasing PD-L1 Levels on Outcomes to PD-1/PD-L1 Inhibition in Patients With NSCLC: A Pooled Analysis of 11 Prospective Clinical Trials. Vallejo J, Singh H, Larkins E, Drezner N, Ricciuti B, Mishra-Kalyani P, Tang S, Beaver JA, Awad MM. Oncologist. 2024 Feb 13:oyae006. doi: 10.1093/oncolo/oyae006. PMID: 38349736.
- Evaluation of somatic copy number variation detection by NGS technologies and bioinformatics tools on a hyper-diploid cancer genome. Masood D, Ren L, Nguyen C, Brundu FG, Zheng L, Zhao Y, Jaeger E, Li Y, Cha SW, Halpern A, Truong S, Virata M, Yan C, Chen Q, Pang A, Alberto R, Xiao C, Yang Z, Chen W, Wang C, Cross F Jr, Catreux S, Shi L, Beaver JA, Xiao W, Meerzaman DM. Genome Biol. 2024 Jun 20;25(1):163. doi: 10.1186/s13059-024-03294-8. PMID: 38902799.
- Cytoreductive nephrectomy in the era of immune checkpoint inhibitors: a U.S. FDA pooled analysis. Fallah J, Gittleman H, Weinstock C, Chang E, Agrawal S, Tang S, Pazdur R, Kluetz PG, Suzman DL, Amiri-Kordestani L. J Natl Cancer Inst. 2024 Mar 14:djae066. doi: 10.1093/jnci/djae066. PMID: 38486364.
- Complete Remission with Partial Hematological Recovery as a Palliative Endpoint for Treatment of Acute Myeloid Leukemia. Le RQ, Przepiorka D, Chen H, Shen YL, Pulte ED, Norsworthy KJ, Theoret MR, De Claro RA. Blood. 2024 May 10:blood.2023023313. doi: 10.1182/blood.2023023313. PMID: 38728428.
- A Pooled Analysis of Treatment-Free Survival in Advanced Renal Cell Carcinoma. Chang E, Zhou J, Song C, Gittleman H, Fernandes L, Weinstock C, Atkins MB, Agrawal S, Sridhara R, Gormley N, Tang S, Suzman DL, Amiri-Kordestani L, Kluetz PG, Pazdur R, Rini BI, McDermott DF, Regan MM. Clin Cancer Res. 2024 Feb 28. doi: 10.1158/1078-0432.CCR-23-3719. PMID: 38416426.
- False-Positive Circulating Tumor DNA Results Do Not Explain Lack of Efficacy for PARP Inhibitors in Patients With Castration-Resistant Prostate Cancer Harboring ATM and CHEK2 Mutations. Jaleh Fallah et al. JCO Precis Oncol 8, e2400354(2024).
- Pharmacokinetic models for first-in-human dose selection of immune-activating products in oncology. Saber H, Thompson MD, Leighton JK. Regul Toxicol Pharmacol. 2024 Mar 30;149:105616. doi: 10.1016/j.yrtph.2024.105616. Epub ahead of print. PMID: 38561147.
- Remote Monitoring and Data Collection for Decentralized Clinical Trials. Fashoyin-Aje L, Smart VW, Chang IF, Tendler CL, Kim G, Fuchs CS, Beg MS, Zhang L, Legos JJ, Duran CO, Kalidas C, Qian J, Finnegan J, Pilarski P, Keane H, Shen J, Silverstein A, Wu YL, Pazdur R, Li BT. JAMA Netw Open. 2024 Apr 1;7(4):e246228. doi: 10.1001/jamanetworkopen.2024.6228. PMID: 38607626; PMCID: PMC11015350.
- Trends in new and persistent opioid use in older adults with and without cancer. Baum LVM, Kc M, Soulos PR, Jeffery MM, Ruddy KJ, Lerro CC, Lee H, Graham DJ, Rivera DR, Leapman MS, Jairam V, Dinan MA, Gross CP, Park HS. J Natl Cancer Inst. 2024 Feb 8;116(2):316-323. doi: 10.1093/jnci/djad206. PMID: 37802882.
- Most common symptomatic adverse reactions of cancer treatments from US drug labels (2015-2021) to inform selection of patient-reported outcomes. Horodniceanu EG, Datla T, Murugappan MN, Kanapuru B, Amiri-Kordestani L, Larkins E, Kluetz P, Bhatnagar V. Value Health. 2024 Oct 8:S1098-3015(24)02862-6. doi: 10.1016/j.jval.2024.09.009. PMID: 39389354.
- Recommendations to address respondent burden associated with patient-reported outcome assessment. Aiyegbusi OL, Cruz Rivera S, Roydhouse J, Kamudoni P, Alder Y, Anderson N, Baldwin RM, Bhatnagar V, Black J, Bottomley A, Brundage M, Cella D, Collis P, Davies EH, Denniston AK, Efficace F, Gardner A, Gnanasakthy A, Golub RM, Hughes SE, Jeyes F, Kern S, King-Kallimanis BL, Martin A, McMullan C, Mercieca-Bebber R, Monteiro J, Peipert JD, Quijano-Campos JC, Quinten C, Rantell KR, Regnault A, Sasseville M, Schougaard LMV, Sherafat-Kazemzadeh R, Snyder C, Stover AM, Verdi R, Wilson R, Calvert MJ. Nat Med. 2024 Mar;30(3):650-659. doi: 10.1038/s41591-024-02827-9. PMID: 38424214.
- Patient-reported frailty phenotype (PRFP) vs. International Myeloma Working Group frailty index (IMWG FI) proxy: A comparison between two approaches to measuring frailty. Murugappan MN, King-Kallimanis BL, Bhatnagar V, Kanapuru B, Farley JF, Seifert RD, Stenehjem DD, Chen TY, Horodniceanu EG, Kluetz PG. J Geriatr Oncol. 2024 Mar;15(2):101681. doi: 10.1016/j.jgo.2023.101681. PMID: 38104480.
- Investigating genetic predisposition to immune-related adverse events in patients with cancer treated with immune checkpoint inhibitors. Scott EC, Santana-Quintero LV, Kural KC, Azad NS, Marte JL, Overman MJ, Paul R, Philip R, Schoenfeld JD, Smith S, Wang V, Xiao W, Pazdur R, Sharon E, Theoret MR, Gulley JL. J Immunother Cancer. 2024;12. doi: 10.1136/jitc-2024-SITC2024.0480.
- Externally Controlled Studies Using Real-World Data in Patients With Hematological Cancers: A Systematic Review. Hermans SJF, van der Maas NG, van Norden Y, Dinmohamed AG, Berkx E, Huijgens PC, Rivera DR, de Claro RA, Pignatti F, Versluis J, Cornelissen JJ.JAMA Oncol. 2024 Oct 1;10(10):1426-1436. doi: 10.1001/jamaoncol.2024.3466.PMID: 39207765.
- Feasibility of structuring electronic health record data to facilitate real-world data research: ICAREdata methods applied to multicenter cancer clinical trials. George S, Campbell N, Hillman SL, Harlos ES, Stein DWJ, Chan MY, Chow SL, Elrahi CL, Quina AC, Kokolus MC, Casagni MD, Weiss M, Anderson DM, Stadler WM, Hoff OC, Rivera DR, Kluetz PG, Mandrekar SJ, Piantadosi S.Cancer. 2024 Aug 28. doi: 10.1002/cncr.35528. Online ahead of print.PMID: 39192753.
- Evaluation of Real-World Tumor Response Derived From Electronic Health Record Data Sources: A Feasibility Analysis in Patients With Metastatic Non-Small Cell Lung Cancer Treated With Chemotherapy. McKelvey BA, Garrett-Mayer E, Rivera DR, Alabaster A, Andrews HS, Bond EG, Brown TD, Bruno A, Damato L, Espirito JL, Fernandes LL, Hansen E, Kluetz P, Ma X, McCracken A, Mishra-Kalyani PS, Natanzon Y, Potter D, Robert NJ, Schwartz L, Schwind R, Sweetnam C, Wagner J, Stewart MD, Allen JD.JCO Clin Cancer Inform. 2024 Aug;8:e2400091. doi: 10.1200/CCI.24.00091.PMID: 39146509
Approval Summaries
- FDA approvals in 2023: biomarker-positive subsets, equipoise and verification of benefit. Norsworthy KJ, Lee-Alonzo RJ, Pazdur R. Nat Rev Clin Oncol. 2024 Feb 21. doi: 10.1038/s41571-024-00871-5. PMID: 38383759.
- FDA Approval Summary: Alpelisib for PIK3CA-Related Overgrowth Spectrum. Singh S, Bradford D, Li X, Mishra-Kalyani PS, Shen YL, Wang L, Zhao H, Xiong Y, Liu J, Charlab R, Kraft J, Khasar S, Miller CP, Rivera DR, Kluetz PG, Pazdur R, Beaver JA, Singh H, Donoghue M.Clin Cancer Res. 2024 Jan 5;30(1):23-28. doi: 10.1158/1078-0432.CCR-23-1270.PMID: 37624421.
- US Food and Drug Administration Approval Summary: Elacestrant for Estrogen Receptor-Positive, Human Epidermal Growth Factor Receptor 2-Negative, ESR1-Mutated Advanced or Metastatic Breast Cancer. Shah M, Lingam H, Gao X, Gittleman H, Fiero MH, Krol D, Biel N, Ricks TK, Fu W, Hamed S, Li F, Sun JJ, Fan J, Schuck R, Grimstein M, Tang L, Kalavar S, Abukhdeir A, Pathak A, Ghosh S, Bulatao I, Tilley A, Pierce WF, Mixter BD, Tang S, Pazdur R, Kluetz P, Amiri-Kordestani L. J Clin Oncol. 2024 Feb 21:JCO2302112. doi: 10.1200/JCO.23.02112. PMID: 38381994.
- FDA Approval Summary: Enfortumab Vedotin Plus Pembrolizumab for Cisplatin-Ineligible Locally Advanced or Metastatic Urothelial Carcinoma. Maguire WF, Lee D, Weinstock C, Gao X, Bulik CC, Agrawal S, Chang E, Hamed SS, Bloomquist EW, Tang S, Pazdur R, Kluetz PG, Amiri-Kordestani L, Suzman DL. Clin Cancer Res. 2024 Mar 5. doi: 10.1158/1078-0432.CCR-23-3738. PMID: 38441576.
- US Food and Drug Administration Approval Summary: Talazoparib in Combination With Enzalutamide for Treatment of Patients With Homologous Recombination Repair Gene-Mutated Metastatic Castration-Resistant Prostate Cancer. Heiss BL, Chang E, Gao X, Truong T, Brave MH, Bloomquist E, Shah A, Hamed S, Kraft J, Chiu HJ, Ricks TK, Tilley A, Pierce WF, Tang L, Abukhdeir A, Kalavar S, Philip R, Tang S, Pazdur R, Amiri-Kordestani L, Kluetz PG, Suzman DL. J Clin Oncol. 2024 Mar 7:JCO2302182. doi: 10.1200/JCO.23.02182. PMID: 38452327.
- FDA Approval Summary: Ivosidenib in Combination with Azacitidine for Treatment of Patients with Newly Diagnosed Acute Myeloid Leukemia with an IDH1 Mutation. Woods A, Norsworthy KJ, Wang X, Vallejo J, Chiu Yuen Chow E, Li RJ, Sun J, Charlab R, Jiang X, Pazdur R, Theoret MR, de Claro RA. Clin Cancer Res. 2024 Apr 1;30(7):1226-1231. doi: 10.1158/1078-0432.CCR-23-2234. PMID: 38010220; PMCID: PMC10984783.
- FDA Approval Summary: Ciltacabtagene Autoleucel for Relapsed or Refractory Multiple Myeloma. Natrajan K, Kaushal M, George B, Kanapuru B, Theoret MR. Clin Cancer Res. 2024 May 7. doi: 10.1158/1078-0432.CCR-24-0378. PMID: 38713595.
- FDA Approval Summary: Fruquintinib for the Treatment of Refractory Metastatic Colorectal Cancer. Fusco MJ, Casak SJ, Mushti SL, Cheng J, Christmas BJ, Thompson MD, Fu W, Wang H, Yoon M, Yang Y, Moore JN, Bi Y, Nan Y, Long CE, Auth D, Rahman NA, Tang S, Pazdur R, Fashoyin-Aje LA, Kluetz PG, Lemery SJ. Clin Cancer Res. 2024 May 29. doi: 10.1158/1078-0432.CCR-24-0281. PMID: 38809262.
- FDA Approval Summary: Lisocabtagene Maraleucel for Second-Line Treatment of Large B-Cell Lymphoma. Elmacken M, Peredo-Pinto H, Wang C, Xu Z, Tegenge M, Jaigirdar AA, Theoret MR, Purohit-Sheth T, Kasamon YL. Clin Cancer Res. 2024 Jun 3;30(11):2309-2316. doi: 10.1158/1078-0432.CCR-23-2967. PMID: 38324398
- FDA Approval Summary: Durvalumab and Pembrolizumab, Immune Checkpoint inhibitors for the Treatment of Biliary Tract Cancer. Casak SJ, Kumar V, Song C, Yuan M, Amatya AK, Cheng J, Mishra-Kalyani PS, Tang S, Lemery SJ, Auth D, Davis G, Kluetz PG, Pazdur R, Fashoyin-Aje L A. Clin Cancer Res. 2024 Jun 10. doi: 10.1158/1078-0432.CCR-24-0517. PMID: 38856639.
- FDA Approval Summary: Repotrectinib for locally advanced or metastatic ROS1-positive non-small cell lung cancer. Barbato MI, Bradford D, Ren Y, Aungst SL, Miller CP, Pan L, Fourie Zirkelbach J, Li Y, Bi Y, Fan J, Grimstein M, Dorff SE, Amatya AK, Mishra-Kalyani PS, Scepura B, Schotland P, Udoka O, Ojofeitimi I, Leighton JK, Rahman NA, Pazdur R, Singh H, Kluetz PG, Drezner N. Clin Cancer Res. 2024 Jun 14. doi: 10.1158/1078-0432.CCR-24-0949. PMID: 38875108.
- FDA Approval Summary: Eflornithine for High-Risk Neuroblastoma Following Prior Multiagent, Multimodality Therapy. Duke ES, Bradford D, Sinha AK, Mishra-Kalyani PS, Lerro CC, Rivera D, Wearne E, Miller CP, Leighton J, Sabit H, Zhao H, Lane A, Scepura B, Pazdur R, Singh H, Kluetz PG, Donoghue M, Drezner N. J Clin Oncol. 2024 Jun 25:101200JCO2400546. doi: 10.1200/JCO.24.00546. PMID: 38917371.
- FDA approval summary: fam-trastuzumab deruxtecan-nxki for unresectable or metastatic non-small cell lung cancer with activating HER2 mutations. Mehta GU, Vellanki PJ, Ren Y, Amatya AK, Mishra-Kalyani PS, Pan L, Zirkelbach JF, Pan Y, Liu J, Aungst SL, Miller CP, Shah M, Rahman NA, Theoret M, Kluetz P, Pazdur R, Beaver JA, Singh H. Oncologist. 2024 Jul 6:oyae151. doi: 10.1093/oncolo/oyae151. PMID: 38970465.
- US Food and Drug Administration Approval Summary: Capivasertib With Fulvestrant for Hormone Receptor-Positive, Human Epidermal Growth Factor Receptor 2-Negative Locally Advanced or Metastatic Breast Cancer With PIK3CA/AKT1/PTEN Alterations. Dilawari A, Buturla J, Osgood C, Gao X, Chen W, Ricks TK, Schaefer T, Avasarala S, Reyes Turcu F, Pathak A, Kalavar S, Bhatnagar V, Collazo J, Rahman NA, Mixter B, Tang S, Pazdur R, Kluetz P, Amiri-Kordestani L. J Clin Oncol. 2024 Aug 19:JCO2400427. doi: 10.1200/JCO.24.00427. PMID: 39159418.
- FDA Approval Summary: Belzutifan for Patients with Advanced Renal Cell Carcinoma. Fallah J, Heiss BL, Joeng HK, Weinstock C, Gao X, Pierce WF, Chukwurah B, Bhatnagar V, Fiero MH, Amiri-Kordestani L, Pazdur R, Kluetz PG, Suzman DL. Clin Cancer Res. 2024 Sep 9. doi: 10.1158/1078-0432.CCR-24-1199. PMID: 39248780.
- FDA Approval Summary: Capecitabine Labeling Update under Project Renewal. Agrawal S, Lee C, Pierce WF, Everhart E, King-Ducre A, Royce M, Osgood CL, Amiri-Kordestani L, Chiu HJ, Ricks TK, Pan L, Fourie Zirkelbach J, Charlab R, Pacanowski M, Kim T, Pazdur R, Kluetz PG, Gao JJ. Clin Cancer Res. 2024 Oct 8. doi: 10.1158/1078-0432.CCR-24-1708. PMID: 39377782.
- FDA Approval Summary: Polatuzumab Vedotin in the First-Line Treatment of Select Large B-cell Lymphomas. Sarraf Yazdy M, Kasamon YL, Gu W, Rodriguez LR, Jin S, Bhatnagar V, Richardson NC, Theoret MR, Pazdur R, Gormley NJ. Clin Cancer Res. 2024 Oct 15. doi: 10.1158/1078-0432.CCR-24-1729. PMID: 39404868.
- FDA Approval Summary: Teclistamab - A Bispecific CD3 T-cell Engager for Patients with Relapsed or Refractory Multiple Myeloma. Baines AC, Kanapuru B, Zhao J, Price LSL, Zheng N, Konicki R, Manning ML, Gehrke BJ, Theoret MR, Gormley NJ. Clin Cancer Res. 2024 Oct 16. doi: 10.1158/1078-0432.CCR-24-1872. PMID: 39412823.
- FDA Approval Summary: Asciminib for Ph+ CML in Chronic Phase Treated with Two or More Tyrosine Kinase Inhibitors and for the T315I Mutation. Pamuk GE, Chow ECY, Ionan AC, Chen H, Lee SL, Hsu V, Grimstein M, Zheng N, Sun J, Charlab R, Gehrke BJ, Vallejo J, Ehrlich LA, de Claro RA, Theoret MR. Clin Cancer Res. 2024 Oct 1;30(19):4266-4271. doi: 10.1158/1078-0432.CCR-24-1086. PMID: 39088257; PMCID: PMC11444873.
- FDA Approval Summary: Olutasidenib for Adult Patients with Relapsed or Refractory Acute Myeloid Leukemia with an Isocitrate Dehydrogenase-1 Mutation. Woods AC, Norsworthy KJ, Choe M, Gehrke BJ, Chen H, Vallejo J, Pan L, Jiang X, Li H, Kraft J, Liu J, Charlab R, Okusanya OO, Booth B, Pazdur R, Theoret MR, de Claro RA. Clin Cancer Res. 2024 Oct 30. doi: 10.1158/1078-0432.CCR-24-2196. PMID: 39475462.
- FDA Approval Summary: Olaparib in Combination With Abiraterone for Treatment of Patients With BRCA-Mutated Metastatic Castration-Resistant Prostate Cancer. Fallah J, Xu J, Weinstock C, Brave MH, Bloomquist E, Fiero MH, Schaefer T, Pathak A, Abukhdeir A, Bhatnagar V, Chiu HJ, Ricks T, John C, Hamed S, Lee C, Pierce WF, Kalavar S, Philip R, Tang S, Amiri-Kordestani L, Pazdur R, Kluetz PG, Suzman D. J Clin Oncol. 2024 Feb 10;42(5):605-613. doi: 10.1200/JCO.23.01868. PMID: 38127780.
- FDA Approval Summary: Dabrafenib in Combination with Trametinib for BRAFV600E Mutation-Positive Low-Grade Glioma. Barbato MI, Nashed J, Bradford D, Ren Y, Khasar S, Miller CP, Zolnik BS, Zhao H, Li Y, Bi Y, Shord SS, Amatya AK, Mishra-Kalyani PS, Scepura B, Al-Matari RA, Pazdur R, Kluetz PG, Donoghue M, Singh H, Drezner N. Clin Cancer Res. 2024 Jan 17;30(2):263-268. doi: 10.1158/1078-0432.CCR-23-1503.PMID: 37610803.
- FDA Approval Summary: Enfortumab Vedotin plus Pembrolizumab for Locally Advanced or Metastatic Urothelial Carcinoma. Brave MH, Maguire WF, Weinstock C, Zhang H, Gao X, Li F, Yu J, Fu W, Zhao H, Pierce WF, Chang E, Dinin J, Fiero MH, Rahman NA, Tang S, Pazdur R, Kluetz PG, Amiri-Kordestani L, Suzman DL. Clin Cancer Res. 2024 Nov 1;30(21):4815-4821. doi: 10.1158/1078-0432.CCR-24-1393. PMID: 39230571; PMCID: PMC11530298.
Regulatory Perspectives
- When less is more—reducing complexity in cancer trials. Patel TH, Rivera DR, Singh H, Kluetz PG. The Lancet Oncology 2024 January; V.25 No. 1, pp 10-12.
- Project Confirm: Accelerated Drug Approvals for CML-Response. Sweet KL, Cortes JE, Apperley JF, Mann M, Mauro MJ, Oehler VG, Ruiz C, Schiffer CA, Ehrlich LA, Pamuk GE, Wynne J, Mehta GU, Okusanya OO, de Claro RA, Theoret MR, Smith BD, Norsworthy KJ. Clin Cancer Res. 2024 Jan 5;30(1):237-238. doi: 10.1158/1078-0432.CCR-23-3234. PMID: 38178776; PMCID: PMC10783534.
- When can real-world data generate real-world evidence? Rahman M, Dal Pan G, Stein P, Levenson M, Kraus S, Chakravarty A, Rivera DR, Forshee R, Concato J.Pharmacoepidemiol Drug Saf. 2024 Jan;33(1):e5715. doi: 10.1002/pds.5715. PMID: 37855046.
- The Bar Is High: Evaluating Fit-for-Use Oncology Real-World Data for Regulatory Decision Making. Lerro CC, et al. JCO Clin Cancer Inform 8, e2300261(2024). DOI:10.1200/CCI.23.00261
- Considering Functional Outcomes as Efficacy Endpoints in Pediatric Low-Grade Glioma Clinical Trials: An FDA Educational Symposium. Fangusaro J, Avery RA, Fisher MJ, Packer RJ, Walsh KS, Schouten-van Meeteren A, Karres D, Bradford D, Bhatnagar V, Singh H, Kluetz PG, Donoghue M, Duke ES. Clin Cancer Res. 2024 Feb 15. doi: 10.1158/1078-0432.CCR-23-3386. PMID: 38358393.
- Pediatric Cancer Drug Development: Leveraging Insights in Cancer Biology and the Evolving Regulatory Landscape to Address Challenges and Guide Further Progress. Charlab R, Leong R, Shord SS, Reaman GH.Cold Spring Harb Perspect Med. 2024 Apr 1;14(4):a041656. doi: 10.1101/cshperspect.a041656. PMID: 38467448.
- Reflections and Roadmaps: Career Development Beyond the FDA-AACR Oncology Educational Fellowship. Patel T, Chen C, Al Hadidi S, Kadry Y, Sridhara R, Purcell E, Wisch L, Retzlaff J, Foti M, Pazdur R, Kluetz P, Gao J. Clin Cancer Res. 2024 Jun 10. doi: 10.1158/1078-0432.CCR-24-1444. PMID: 38856702.
- RE: is it time to reconsider the role of upfront cytoreductive nephrectomy in metastatic renal cell carcinoma? Fallah J, Gittleman H, Weinstock C, Chang E, Agrawal S, Tang S, Pazdur R, Kluetz PG, Suzman DL, Amiri-Kordestani L. J Natl Cancer Inst. 2024 Jul 1:djae158. doi: 10.1093/jnci/djae158. PMID: 38950174.
- Improving Collection and Analysis of Overall Survival Data. Rodriguez LR, Gormley NJ, Lu R, Amatya AK, Demetri GD, Flaherty KT, Mesa RA, Pazdur R, Sekeres MA, Shan M, Snapinn S, Theoret MR, Umoja R, Vallejo J, Warren NJH, Xu Q, Anderson KC. Clin Cancer Res. 2024 Jul 22. doi: 10.1158/1078-0432.CCR-24-0919. PMID: 39037364.
- Treatment of Acute Myeloid Leukemia in the Community Setting. Al-Juhaishi T, Dela Cruz S, Gupta R, Keiffer G, Morrison VA, Shapira I, Woods A, Norsworthy K, de Claro RA, Theoret MR, Garg R, Pulte ED. Oncologist. 2024 Aug 19:oyae051. doi: 10.1093/oncolo/oyae051. PMID: 39159003.
- Perspectives on Drug Development for the Treatment of Chronic Myeloid Leukemia in Pregnant Patients and Patients Who Are Breastfeeding. Cortes JE, Abruzzese E, Cardonick EH, Hernández-Díaz S, Gutierrez J, Sardegna MS, Torres-Chavez E, Dinatale M, Lerro CC, Gehrke BJ, Shord SS, De Claro RA, Theoret MR, DeMaria PJ, Norsworthy KJ. Clin Cancer Res. 2024 Sep 3;30(17):3658-3666. doi: 10.1158/1078-0432.CCR-24-0826. PMID: 38967550; PMCID: PMC11371510.
- Equipoise Lost? Trial Conduct Challenges in an Era of Breakthrough Therapies. Fallah J, Mulkey F, Fiero MH, Gittleman H, Song C, Puthiamadathil J, Amatya A, Agrawal S, Vellanki P, Suzman DL, Singh H, Amiri-Kordestani L, Mishra-Kalyani P, Pazdur R, Kluetz PG. J Clin Oncol. 2024 Sep 17:JCO2401200. doi: 10.1200/JCO-24-01200. PMID: 39288354.
- Post-approval evidence generation: a shared responsibility for healthcare. Abbasi A, Rivera D, Curtis LH, Califf RM. Nat Med. 2024 Sep 13. doi: 10.1038/s41591-024-03241-x. PMID: 39271845.
- Index of application status transparency and availability of public information for Project Orbis agencies. Lee SH, Hotaki LT, Oh K, Samuel J, de Villiers K, Eshetie K, Looi YH, Atiek E, Cudre-Maurous F, Rohr UP, Searle G, Santos LN, da. S. Andreoli SC, Aoi Y, Hirata M, Voltz-Girolt C, Aicardo C, Cain C, Naggan T, Boehm-Cagan A, Hirsch-Vexberg M, Luxenburg O, Ahmed N, Johnson L, Hunt M, Vu D, Theoret MT, Spillman D, de Claro RA. International Journal of Drug Regulatory Affairs. 2024;12(3):55-65.
- Dosage Optimization: A Regulatory Perspective for Developing Oncology Drugs. Rahman A, Shah M, Shord SS.Clin Pharmacol Ther. 2024 Sep;116(3):577-591. doi: 10.1002/cpt.3373. PMID: 39072758.
- Development of a Canadian Guidance for reporting real-world evidence for regulatory and health-technology assessment (HTA) decision-making. Tadrous M, Aves T, Fahim C, Riad J, Mittmann N, Prieto-Alhambra D, Rivera DR, Chan K, Lix LM, Kent S, Dawoud D, Guertin JR, McDonald JT, Round J, Klarenbach S, Stanojevic S, De Vera MA, Strumpf E, Platt RW, Husein F, Lambert L, Hayes KN.J Clin Epidemiol. 2024 Sep 23;176:111545. doi: 10.1016/j.jclinepi.2024.111545. PMID: 39322123.
- The Retrofit: Lessons From Sotorasib's Dosing Conundrum. Singh H, Vellanki PJ, Pazdur R. J Clin Oncol. 2024 Oct 7:JCO2400310. doi: 10.1200/JCO.24.00310. PMID: 39374478.
- Oncology Accelerated Approval Confirmatory Trials: When a Failed Trial Is Not a Failed Drug. Mehta GU, Pazdur R. J Clin Oncol. 2024 Oct 9:JCO2401654. doi: 10.1200/JCO-24-01654. PMID: 39383488.
- The Oncology QCARD Initiative: Fostering efficient evaluation of initial real-world data proposals. Rivera DR, Eckert JC, Rodriguez-Watson C, Lerro CC, Bertagnolli MM, Hubbard RA, Kushi LH, Lund JL, Schrag D, Wang SV, Wood WA, Lee JJ, Okafor C, Ghauri K, Winckler SC, Kluetz PG. Pharmacoepidemiol Drug Saf. 2024 Nov;33(11):e5818. doi: 10.1002/pds.5818. PMID: 39462868.
- An Overview of Myeloid Blast-Phase Chronic Myeloid Leukemia. Pamuk GE, Ehrlich LA. Cancers. 2024;16(21):3615. doi: 10.3390/cancers16213615.
- Advancing patient-centric care: integrating patient reported outcomes for tolerability assessment in early phase clinical trials - insights from an expert virtual roundtable. Yap C, Lee Aiyegbusi O, Alger E, Basch E, Bell J, Bhatnagar V, Cella D, Collis P, Dueck AC, Gilbert A, Gnanasakthy A, Greystoke A, Hansen AR, Kamudoni P, Kholmanskikh O, King-Kallimanis BL, Krumholz H, Minchom A, O'Connor D, Petrie J, Piccinin C, Rantell KR, Rauz S, Retzer A, Rizk S, Wagner L, Sasseville M, Seymour LK, Weber HA, Wilson R, Calvert M, Peipert JD. EClinicalMedicine. 2024 Sep 24;76:102838. doi: 10.1016/j.eclinm.2024.102838. eCollection 2024 Oct. PMID: 39386161.
- Administrative Aspects of Molecular Diagnostics-Oversight, Regulatory Approval Process, Clinical and Operational Workflows, and Payment Models. Wilfong L, Baggett L, Reena P, Murphy R, Singh H, Kluetz P, Byrd B, McDonough R, Reddy S, Brito RR. JCO Oncol Pract. 2024 Nov;20(11):1501-1507. doi: 10.1200/OP.23.00812. PMID: 39531846.
- Perspectives on drug development in chronic myelomonocytic leukemia: changing the paradigm. Hunter AM, Patnaik MM, Itzykson R, Mesa R, Karanes C, Li Y, de Claro RA, Norsworthy KJ, Theoret M, Pulte E, and Padron E. Blood. 2024 Nov 7;144(19):1987-1992. doi: 10.1182/blood.2024025648.
- Autopsy of a Drug Withdrawal - The Case of Melphalan Flufenamide. Kanapuru B, Carioti T, Gormley N, Pazdur R. N Engl J Med. 2024 Dec 7. doi: 10.1056/NEJMp2412068. PMID: 39651792.
Research Papers
- Use of Single-Arm Trials for US Food and Drug Administration Drug Approval in Oncology, 2002-2021. Agrawal S, Arora S, Amiri-Kordestani L, de Claro RA, Fashoyin-Aje L, Gormley N, Kim T, Lemery S, Mehta GU, Scott EC, Singh H, Tang S, Theoret MR, Pazdur R, Kluetz PG, Beaver JA. JAMA Oncol. 2023 Feb 1;9(2):266-272. doi: 10.1001/jamaoncol.2022.5985. PMID: 36580315.
- Differential Protein Citrullination in Human ER- and ER+ Tumor and Adjacent Healthy Breast Tissue. Biochemistry. Tillotson J, Aryal B, Lai L, Beaver JA, Rao VA. 2023 Feb 21;62(4):893-898. doi: 10.1021/acs.biochem.2c00551. PMID: 36757899; PMCID: PMC9948284.
- Abstract A014: An analysis comparing the geographic distribution of community cancer center providers to CRC prevalence rates among Medicare beneficiaries. Solarte M, Sacristan B, Boehmer L, Fashoyin-Aje L, Hobbs K. Cancer Epidemiology, Biomarkers & Prevention. 2023 Jan 1, A014-A014.
- Evaluating Pneumonitis Incidence in Patients with Non-small Cell Lung Cancer Treated with Immunotherapy and/or Chemotherapy Using Real-world and Clinical Trial Data. Liu Q, Zhang C, Huang Y, Huang R, Huang SM, Larkins E, Stapleford L, Rivera DR, Kluetz PG, Wang S, Zhu H, Weese J, Cromartie E, Teka M, Walters S, Wolf F, Brown TD. Cancer Res Commun. 2023 Feb 14;3(2):258-266. doi: 10.1158/2767-9764.CRC-22-0370. PMID: 36860658; PMCID: PMC9973394.
- Increasing Racial and Ethnic Equity, Diversity, and Inclusion in Cancer Treatment Trials: Evaluation of an ASCO-Association of Community Cancer Centers Site Self-Assessment. Guerra C, Pressman A, Hurley P, Garrett-Mayer E, Bruinooge SS, Howson A, Kaltenbaugh M, Hanley Williams J, Boehmer L, Bernick LA, Byatt L, Charlot M, Crews J, Fashoyin-Aje L, McCaskill-Stevens W, Merrill J, Nowakowski G, Patel MI, Ramirez A, Zwicker V, Oyer RA, Pierce LJ. JCO Oncol Pract. 2023 Apr;19(4):e581-e588. doi: 10.1200/OP.22.00560. PMID: 36630663.
- Transcriptomic analysis of pancreatic adenocarcinoma specimens obtained from Black and White patients. Biel TG, Petrovskaya S, Mascia F, Ju T, Fashoyin-Aje L, Herremans KM, Riner AN, Underwood PW, Gerber MH, Donoghue M, Trevino JG, Rao VA. PLoS One. 2023 Feb 22;18(2):e0281182. doi: 10.1371/journal.pone.0281182. PMID: 36812168; PMCID: PMC9946261.
- Recommendations on the use of item libraries for patient-reported outcome measurement in oncology trials: findings from an international, multidisciplinary working group. Piccinin C, Basch E, Bhatnagar V, Calvert M, Campbell A, Cella D, Cleeland CS, Coens C, Darlington AS, Dueck AC, Groenvold M, Herold R, King-Kallimanis BL, Kluetz PG, Kuliś D, O'Connor D, Oliver K, Pe M, Reeve BB, Reijneveld JC, Wang XS, Bottomley A. Lancet Oncol. 2023 Feb;24(2):e86-e95. doi: 10.1016/S1470-2045(22)00654.
- Measuring Frailty Using Patient-Reported Outcomes (PRO) Data: A Feasibility Study in Patients with Multiple Myeloma. Murugappan MN, King-Kallimanis BL, Bhatnagar V, Kanapuru B, Farley JF, Seifert RD, Stenehjem DD, Chen TY, Horodniceanu EG, Kluetz PG. Qual Life Res. 2023 Mar 20:1–12. doi: 10.1007/s11136-023-03390-5. PMID: 36935467; PMCID: PMC10025057.
- Integrating 4 Measures to Evaluate Physical Function in Patients with Cancer (In4M): Protocol for a prospective study. medRxiv [Preprint]. Thanarajasingam G, Kluetz PG, Bhatnagar V, Brown A, Cathcart-Rake E, Diamond M, Faust L, Fiero MH, Huntington SF, Jeffery MM, Jones L, Noble BN, Paludo J, Powers B, Ross JS, Ritchie JD, Ruddy KJ, Schellhorn SE, Tarver ME, Dueck AC, Gross CP. 2023 Mar 9:2023.03.08.23286924. doi: 10.1101/2023.03.08.23286924. PMID: 36945495; PMCID: PMC10029056.
- FDA Analysis of Ineligibility for Acute Myeloid Leukemia Clinical Trials by Race and Ethnicity. Pulte D, Fernandes L, Wei G, Woods A, Norsworthy KJ, Gormley N, Kanapuru B, Gwise TE, Pazdur R, Schneider J, Theoret MR, Fashoyin-Aje LA, de Claro RA. Clin Lymphoma Myeloma Leuk. 2023 Mar 30:S2152-2650(23)00109-X. doi: 10.1016/j.clml.2023.03.012. PMID: 37076368.
- The Association Between Baseline Hepatic or Renal Function and Clinical Outcomes for Patients With Non-Small Cell Lung Cancer Treated With a PD-1/PD-L1 Blocking Antibody Using Real-World and Trial Data. Liu Q, Mathur R, Xu Y, Torres AZ, Miksad RA, Liu C, Smithson H, Wang Y, Zhu H, Booth B, Huang SM, Zhi J, Sridhara R, Blumenthal GM, Larkins E, Mishra-Kalyani PS, Rivera DR, Kluetz PG, Sharon E. Clin Pharmacol Ther. 2023 May;113(5):1139-1149. doi: 10.1002/cpt.2874. PMID: 36790088.
- Eligibility criteria and Enrollment of a Diverse Racial and Ethnic population in Multiple Myeloma Clinical Trials. Blood. Kanapuru B, Fernandes L, Baines AC, Ershler R, Bhatnagar V, Pulte E, Gwise T, Theoret MR, Pazdur R, Fashoyin-Aje LA, Gormley N. Blood. 2023 May 4:blood.2022018657. doi: 10.1182/blood.2022018657. PMID: 37140031.
- Association between antibiotics and adverse oncological outcomes in patients receiving targeted or immune-based therapy for hepatocellular carcinoma. Pinato DJ, Li X, Mishra-Kalyani P, D'Alessio A, Fulgenzi CAM, Scheiner B, Pinter M, Wei G, Schneider J, Rivera DR, Pazdur R, Theoret MR, Casak S, Lemery S, Fashoyin-Aje L, Cortellini A, Pelosof L. JHEP Rep. 2023 Mar 30;5(6):100747. doi: 10.1016/j.jhepr.2023.100747. PMID: 37197442; PMCID: PMC10183666.
- Enrollment representation of age, race, and ethnicity in ovarian cancer registrational clinical trials (2010-2020): An evaluation by the U.S. Food and Drug Administration. Yang F, Huang Y, Lerro C, Liu W, Fiero M, Feng Z, Kanapuru B, Patel T, Amiri-Kordestani L, Ison G, Fashoyin-Aje L, Kluetz P, Singh H, Rivera D. Jour Clin Oncol. 2023 Jun 1.
- The Impact of Improved Treatments on Survival of Adult U.S. Leukemia Patients: 1990-2018. Howlader N, Sharon E, Bhattacharya M, Ehrlich LA, Richardson NC, Gormley NJ, de Claro RA, Wood AE, Mariotto AB, Cronin KA. Cancer Epidemiol Biomarkers Prev. 2023 Jun 1;32(6):744-747. doi: 10.1158/1055-9965.EPI-22-1171. PMID: 37012203.
- A U.S. Food and Drug Administration-pooled Analysis of Frontline Combination Treatment Survival Benefits by Risk Groups in Metastatic Renal Cell Carcinoma. Lee D, Gittleman H, Weinstock C, Suzman D, Bloomquist E, Agrawal S, Brave M, Brewer J, Fallah J, Singh H, Tang S, Ibrahim A, Pazdur R, Beaver JA, Amiri-Kordestani L. Eur Urol. 2023 Jun 2:S0302-2838(23)02875-0. doi: 10.1016/j.eururo.2023.05.030. PMID: 37271635.
- Trends in the approval of cancer therapies by the FDA in the twenty-first century. Scott EC, Baines AC, Gong Y, Moore R Jr, Pamuk GE, Saber H, Subedee A, Thompson MD, Xiao W, Pazdur R, Rao VA, Schneider J, Beaver JA. Nat Rev Drug Discov. 2023 Jun 21. doi: 10.1038/s41573-023-00723-4. PMID: 37344568.
- Assessing the Needs of Those Who Serve the Underserved: A Qualitative Study among US Oncology Clinicians. Cancers (Basel). Patel MI, Hinyard L, Hlubocky FJ, Merrill JK, Smith KT, Kamaraju S, Carrizosa D, Kalwar T, Fashoyin-Aje L, Gomez SL, Jeames S, Florez N, Kircher SM, Tap WD. 2023 Jun 23;15(13):3311. doi: 10.3390/cancers15133311. PMID: 37444421; PMCID: PMC10341104.
- Drug combinations identified by high-throughput screening promote cell cycle transition and upregulate Smad pathways in myeloma. Peat TJ, Gaikwad SM, Dubois W, Gyabaah-Kessie N, Zhang S, Gorjifard S, Phyo Z, Andres M, Hughitt VK, Simpson RM, Miller MA, Girvin AT, Taylor A, Williams D, D'Antonio N, Zhang Y, Rajagopalan A, Flietner E, Wilson K, Zhang X, Shinn P, Klumpp-Thomas C, McKnight C, Itkin Z, Chen L, Kazandijian D, Zhang J, Michalowski AM, Simmons JK, Keats J, Thomas CJ, Mock BA. Cancer Lett. 2023 Jun 23:216284. doi:10.1016/j.canlet.2023.216284. PMID: 37356470.
- Atezolizumab Induces Necroptosis and Contributes to Hepatotoxicity of Human Hepatocytes. Endo Y, Winarski KL, Sajib MS, Ju A, Wu WJ. Int J Mol Sci. 2023 Jul 20;24(14):11694. doi: 10.3390/ijms241411694. PMID: 37511454; PMCID: PMC10380327.
- Landscape Analysis of Generic Availability for Oncologic Drugs. Kumar V, Wang F, Hu M, Kluetz P, Zhao L. Landscape Analysis of Generic Availability for Oncologic Drugs. Ther Innov Regul Sci. 2023 Aug 10. doi: 10.1007/s43441-023-00562-w. PMID: 37561261.
- Researcher Experience and Comfort With Telemedicine and Remote Patient Monitoring in Cancer Treatment Trials. Lichtenstein MRL, Levit LA, Schenkel C, Kirkwood K, Fashoyin-Aje LA, Bruinooge SS, Kelley MJ, Mailman JA, Magnuson A, Mirda DP, Natesan D, Hershman DL. Oncologist. 2023 Sep 7:oyad237. doi: 10.1093/oncolo/oyad237. PMID: 37676048.
- Quartet RNA reference materials improve the quality of transcriptomic data through ratio-based profiling. Yu Y, Hou W, Liu Y, Wang H, Dong L, Mai Y, et al. Nat Biotechnol. 2023 Sep 7. doi: 10.1038/s41587-023-01867-9. PMID: 37679545.
- The Quartet Data Portal: integration of community-wide resources for multiomics quality control. Yang J, Liu Y, Shang J, Chen Q, Chen Q, Ren L, et al. Genome Biol. 2023 Oct 26;24(1):245. doi: 10.1186/s13059-023-03091-9. PMID: 37884999.
- Quartet DNA reference materials and datasets for comprehensively evaluating germline variant calling performance. Ren L, Duan X, Dong L, Zhang R, Yang J, Gao Y, et al. Genome Biol. 2023 Nov 27;24(1):270. doi: 10.1186/s13059-023-03109-2. PMID: 38012772.
- Analysis and therapeutic targeting of the IL-1R pathway in anaplastic large cell lymphoma. Song Z, Wu W, Wei W, Xiao W, Lei M, Cai KQ, et al. Blood. 2023 Oct 12;142(15):1297-1311. doi: 10.1182/blood.2022019166. PMID: 37339580.
- Multi-omics data integration using ratio-based quantitative profiling with Quartet reference materials. Zheng Y, Liu Y, Yang J, Dong L, Zhang R, Tian S, et al. Nat Biotechnol. 2023 Sep 7. doi: 10.1038/s41587-023-01934-1. PMID: 37679543.
- Multi-omics investigation on the prognostic and predictive factors in metastatic breast cancer using data from Phase III ribociclib clinical trials: A statistical and machine learning analysis plan. Coroller T, Sahiner B, Amatya A, Gossmann A, Karagiannis K, Samala RK, et al. MedRxiv. 2023 Aug 31. doi: https://doi.org/10.1101/2023.08.30.23294367.
- Trends in New and Persistent Opioid Use in Older Adults with and without Cancer. Van Metre Baum L, K C M, Soulos PR, Jeffery MM, Ruddy KJ, Lerro CC, Lee H, Graham DJ, Rivera DR, Leapman MS, Jairam V, Dinan MA, Gross CP, Park HS. J Natl Cancer Inst. 2023 Oct 6:djad206. doi: 10.1093/jnci/djad206. PMID: 37802882.
- Methodology for good machine learning with multi-omics data. Coroller T, Sahiner B, Amatya A, Gossmann A, Karagiannis K, Moloney C, Samala RK, Santana-Quintero L, Solovieff N, Wang C, Amiri-Kordestani L, Cao Q, Cha KH, Charlab R, Cross FH Jr, Hu T, Huang R, Kraft J, Krusche P, Li Y, Li Z, Mazo I, Paul R, Schnakenberg S, Serra P, Smith S, Song C, Su F, Tiwari M, Vechery C, Xiong X, Zarate JP, Zhu H, Chakravartty A, Liu Q, Ohlssen D, Petrick N, Schneider JA, Walderhaug M, Zuber E. Clin Pharmacol Ther. 2023 Nov 15. doi: 10.1002/cpt.3105. PMID: 37965805.
- Use of patient-reported outcomes (PRO) data to complement exposure-response analysis in early clinical cancer drug development. Xia H, Booth BP, Wang Y, Fan C, Bhatnagar V, Kluetz P, Fourie Zirkelbach J. J Patient Rep Outcomes. 2023 Nov 17;7(1):116. doi: 10.1186/s41687-023-00651-2. PMID: 37975967; PMCID: PMC10656371.
Approval Summaries
- FDA Approval Summary: Belzutifan for VHL Disease Tumors-Response. Fallah J, Weinstock C, Mehta GU, Brave MH, Pierce WF, Pazdur R, Nair A, Suzman DL, Amiri-Kordestani L. Clin Cancer Res. 2023 Feb 1;29(3):685. doi: 10.1158/1078-0432.CCR-22-3428. PMID: 36722139.
- FDA Approval Summary: Mobocertinib for Metastatic Non-Small Cell Lung Cancer with EGFR Exon 20 Insertion Mutations. Duke ES, Stapleford L, Drezner N, Amatya AK, Mishra-Kalyani PS, Shen YL, Maxfield K, Zirkelbach JF, Bi Y, Liu J, Zhang X, Wang H, Yang Y, Zheng N, Reece K, Wearne E, Glen JJ, Ojofeitimi I, Scepura B, Nair A, Bikkavilli RK, Ghosh S, Philip R, Pazdur R, Beaver JA, Singh H, Donoghue M. Clin Cancer Res. 2023 Feb 1;29(3):508-512. doi: 10.1158/1078-0432.CCR-22-2072. PMID: 36112541; PMCID: PMC9898076.
- US Food and Drug Administration Approval Summary: Fam-Trastuzumab Deruxtecan-nxki for Human Epidermal Growth Factor Receptor 2-Low Unresectable or Metastatic Breast Cancer. Narayan P, Dilawari A, Osgood C, Feng Z, Bloomquist E, Pierce WF, Jafri S, Kalavar S, Kondratovich M, Jha P, Ghosh S, Tang S, Pazdur R, Beaver JA, Amiri-Kordestani L. J Clin Oncol. 2023 Feb 13:JCO2202447. doi: 10.1200/JCO.22.02447. PMID: 36780610.
- Approvals in 2022: overall survival, dose optimization, new approvals and beyond. Telaraja D, Gormley N, Pazdur R. Nat Rev Clin Oncol. 2023 Feb 23. doi: 10.1038/s41571-023-00741-6. PMID: 36823362.
- FDA Approval Summary: Pemigatinib for Previously Treated, Unresectable Locally Advanced or Metastatic Cholangiocarcinoma with FGFR2 Fusion or Other Rearrangement. Patel TH, Marcus L, Horiba MN, Donoghue M, Chatterjee S, Mishra-Kalyani PS, Schuck RN, Li Y, Zhang X, Fourie Zirkelbach J, Charlab R, Liu J, Yang Y, Lemery SJ, Pazdur R, Theoret MR, Fashoyin-Aje LA. Clin Cancer Res. 2023 Mar 1;29(5):838-842. doi: 10.1158/1078-0432.CCR-22-2036. PMID: 36206041; PMCID: PMC9991984.
- FDA Approval Summary: Atezolizumab as Adjuvant Treatment Following Surgical Resection and Platinum-Based Chemotherapy for Stage II to IIIA NSCLC. Mathieu LN, Larkins E, Sinha AK, Mishra-Kalyani PS, Jafri S, Kalavar S, Ghosh S, Goldberg KB, Pazdur R, Beaver JA, Singh H. Clin Cancer Res. 2023 Mar 23:CCR-22-3699. doi: 10.1158/1078-0432.CCR-22-3699. PMID: 36951523.
- US Food and Drug Administration Approval Summary: Nivolumab Plus Platinum-Doublet Chemotherapy for the Neoadjuvant Treatment of Patients With Resectable Non-Small-Cell Lung Cancer. Akinboro O, Drezner N, Amatya A, Runyan J, Fourie-Zirkelbach J, Zhao M, Bi Y, Korsah K, Mixter B, Tang S, Larkins E, Pazdur R, Beaver JA, Singh H. J Clin Oncol. 2023 May 4:JCO2202509. doi: 10.1200/JCO.22.02509. PMID: 37141544.
- FDA Approval Summary: Lutetium Lu 177 Vipivotide Tetraxetan for Patients with Metastatic Castration-Resistant Prostate Cancer. Fallah J, Agrawal S, Gittleman H, Fiero MH, Subramaniam S, John C, Chen W, Ricks TK, Niu G, Fotenos A, Wang M, Chiang K, Pierce WF, Suzman DL, Tang S, Pazdur R, Amiri-Kordestani L, Ibrahim A, Kluetz PG. Clin Cancer Res. 2023 May 1;29(9):1651-1657. doi: 10.1158/1078-0432.CCR-22-2875. PMID: 36469000; PMCID: PMC10159870.
- FDA Approval Summary: Selpercatinib for the treatment of advanced RET fusion-positive solid tumors. Duke ES, Bradford D, Marcovitz M, Amatya AK, Mishra-Kalyani PS, Nguyen E, Price LSL, Fourie Zirkelbach J, Li Y, Bi Y, Kraft J, Dorff SE, Scepura B, Stephenson M, Ojofeitimi I, Nair A, Han Y, Tezak Z, Lemery SJ, Pazdur R, Larkins E, Singh H. Clin Cancer Res. 2023 May 23:CCR-23-0459. doi: 10.1158/1078-0432.CCR-23-0459. PMID: 37219515.
- FDA Approval Summary: Mirvetuximab soravtansine-gynx for FRα-positive, Platinum-Resistant Ovarian Cancer. Dilawari A, Shah M, Ison G, Gittleman H, Fiero MH, Shah A, Hamed SS, Qiu J, Yu J, Manheng W, Ricks TK, Pragani R, Arudchandran A, Patel P, Zaman S, Roy A, Kalavar S, Ghosh S, Pierce WF, Rahman NA, Tang S, Mixter BD, Kluetz PG, Pazdur R, Amiri-Kordestani L. Clin Cancer Res. 2023 May 22:CCR-23-0991. doi: 10.1158/1078-0432.CCR-23-0991. PMID: 37212825.
- FDA Approval Summary: Ripretinib for Advanced Gastrointestinal Stromal Tumor. Kumar V, Doros L, Thompson M, Mushti SL, Charlab R, Spehalski EI, Zhao H, Thompson MD, Tang S, Pazdur R, Lemery SJ, Theoret MR, Fashoyin-Aje LA. Clin Cancer Res. 2023 Jun 1;29(11):2020-2024. doi: 10.1158/1078-0432.CCR-22-2400. PMID: 36485007; PMCID: PMC10238554.
- FDA Approval Summary: Futibatinib for Unresectable Advanced or Metastatic, Chemotherapy Refractory Intrahepatic Cholangiocarcinoma with FGFR2 Fusions or Other Rearrangements. Gandhy SU, Casak SJ, Mushti SL, Cheng J, Subramaniam S, Zhao H, Zhao M, Bi Y, Liu G, Fan J, Adeniyi O, Charlab R, Kufrin D, Thompson MD, Jarrell K, Auth D, Lemery SJ, Pazdur R, Kluetz PG, Fashoyin-Aje LA. Clin Cancer Res. 2023 Jun 8:CCR-23-1042. doi: 10.1158/1078-0432.CCR-23-1042. PMID: 37289037.
- FDA Approval Summary: Tucatinib with trastuzumab for advanced unresectable or metastatic, chemotherapy refractory, HER2 positive RAS wild type colorectal cancer. Casak SJ, Horiba MN, Yuan M, Cheng J, Lemery SJ, Shen YL, Fu W, Moore JN, Li Y, Bi Y, Auth D, Fesenko N, Kluetz PG, Pazdur R, Fashoyin-Aje LA. Clin Cancer Res. 2023 Jun 15:CCR-23-1041. doi: 10.1158/1078-0432.CCR-23-1041. Epub ahead of print. PMID: 37318379.
- FDA Approval Summary: Axicabtagene Ciloleucel for Second-Line Treatment of Large B-cell Lymphoma. Sharma P, Kasamon YL, Lin X, Xu Z, Theoret MR, Purohit-Sheth T. Clin Cancer Res. 2023 Jul 5:CCR-23-0568. doi: 10.1158/1078-0432.CCR-23-0568. PMID: 37405396.
- FDA Approval Summary: Ribociclib Indicated for Male Patients with Hormone Receptor-Positive, HER2-Negative Advanced or Metastatic Breast Cancer. Gao JJ, Osgood CL, Feng Z, Bloomquist EW, Tang S, Chang CJG, Ricks TK, Hou SC, Pierce WF, Rivera DR, Pazdur R, Kluetz PG, Amiri-Kordestani L. Clin Cancer Res. 2023 Aug 18:CCR-23-1133. doi: 10.1158/1078-0432.CCR-23-1133. PMID: 37594723.
- FDA Approval Summary: Dabrafenib in combination with trametinib for BRAF V600E mutation-positive low-grade glioma. Barbato MI, Nashed J, Bradford D, Ren Y, Khasar S, Miller CP, Zolnik BS, Zhao H, Li Y, Bi Y, Shord SS, Amatya AK, Mishra-Kalyani PS, Scepura B, Al-Matari RA, Pazdur R, Kluetz PG, Donoghue M, Singh H, Drezner N. Clin Cancer Res. 2023 Aug 23:CCR-23-1503. doi: 10.1158/1078-0432.CCR-23-1503. PMID: 37610803.
- FDA Approval Summary: Alpelisib for PIK3CA-related Overgrowth Spectrum (PROS). Singh S, Bradford D, Li X, Mishra-Kalyani PS, Shen YL, Wang L, Zhao H, Xiong Y, Liu J, Charlab R, Kraft J, Khasar S, Miller CP, Rivera DR, Kluetz PG, Pazdur R, Beaver JA, Singh H, Donoghue M. Clin Cancer Res. 2023 Aug 25:CCR-23-1270. doi: 10.1158/1078-0432.CCR-23-1270. PMID: 37624421.
- FDA Approval Summary: Amivantamab for the Treatment of Patients with Non-Small Cell Lung Cancer with EGFR Exon 20 Insertion Mutations. Chon K, Larkins E, Chatterjee S, Mishra-Kalyani PS, Aungst S, Wearne E, Subramaniam S, Li Y, Liu J, Sun J, Charlab R, Zhao H, Saritas-Yildirim B, Bikkavilli RK, Ghosh S, Philip R, Beaver JA, Singh H. Clin Cancer Res. 2023 Sep 1;29(17):3262-3266. doi: 10.1158/1078-0432.CCR-22-3713. PMID: 37022784; PMCID: PMC10523842.
- FDA Approval Summary: Tremelimumab in combination with durvalumab for the treatment of patients with unresectable hepatocellular carcinoma. Patel TH, Brewer JR, Fan J, Cheng J, Shen YL, Xiang Y, Zhao H, Lemery SJ, Pazdur R, Kluetz PG, Fashoyin-Aje LA. Clin Cancer Res. 2023 Sep 7. doi: 10.1158/1078-0432.CCR-23-2124. PMID: 37676259.
- FDA Approval Summary: Pirtobrutinib for Relapsed or Refractory Mantle Cell Lymphoma. Telaraja D, Kasamon YL, Collazo JS, Leong R, Wang K, Li P, Dahmane E, Yang Y, Earp J, Grimstein M, Rodriguez LR, Theoret MR, Gormley NJ. Clin Cancer Res. 2023 Aug 25:CCR-23-1272. doi: 10.1158/1078-0432.CCR-23-1272. PMID: 37624619.
- FDA Approval Summary: Ivosidenib in Combination with Azacitidine for Treatment of Patients with Newly Diagnosed Acute Myeloid Leukemia with an IDH1 Mutation. Woods A, Norsworthy KJ, Wang X, Vallejo J, Chow ECY, Li RJ, Sun J, Charlab R, Jiang X, Pazdur R, Theoret MR, de Claro RA. Clin Cancer Res. 2023 Nov 27. doi: 10.1158/1078-0432.CCR-23-2234. PMID: 38010220.
- FDA Approval Summary: Olaparib in Combination With Abiraterone for Treatment of Patients With BRCA-Mutated Metastatic Castration-Resistant Prostate Cancer. Fallah J, Xu J, Weinstock C, Brave MH, Bloomquist E, Fiero MH, Schaefer T, Pathak A, Abukhdeir A, Bhatnagar V, Chiu HJ, Ricks T, John C, Hamed S, Lee C, Pierce WF, Kalavar S, Philip R, Tang S, Amiri-Kordestani L, Pazdur R, Kluetz PG, Suzman D. J Clin Oncol. 2023 Dec 21:JCO2301868. doi: 10.1200/JCO.23.01868. PMID: 38127780.
- Highlights of FDA Oncology Approvals in 2023: Bispecific T-cell Engagers, Pediatric Indications, and Inclusive Drug Development. Donaldson JM, Seddiq M, Fusco MJ, Singla S, Pamuk GE, Lee-Alonso RJ, Mixter BD, Goldberg KB, Amiri-Kordestani L, de Claro RA, Drezner N, Gormley NJ, Kanapuru B, Lemery SJ, Fashoyin-Aje LA, Richardson NC, Singh H, Suzman DL, Theoret MR, Kluetz PG, Pazdur R. Cancer Discov. 2023 Dec 12;13(12):2515-2524. doi: 10.1158/2159-8290.CD-23-1226. PMID: 38084090.
Regulatory Perspectives
- FDA regulatory considerations for the review of drugs intended to treat pediatric cancers and rare tumors. Bradford D, Singh H, Donoghue M. Curr Opin Pediatr. 2023 Feb 1;35(1):48-54. doi: 10.1097/MOP.0000000000001201. PMID: 36592026.
- FDA Oncology Center of Excellence Seeks Applications for New Research Funding Opportunities. Schneider JA, Gao JJ, Donoghue MB, Fashoyin-Aje L, Summers J, Kluetz PG, Theoret MR, Pazdur R. The ASCO Post. January 24, 2023.
- Regulatory implications of ctDNA in immuno-oncology for solid tumors. J Immunother Cancer. Vellanki PJ, Ghosh S, Pathak A, Fusco MJ, Bloomquist EW, Tang S, Singh H, Philip R, Pazdur R, Beaver JA. J Immunother Cancer. 2023 Feb;11(2):e005344. doi: 10.1136/jitc-2022-005344. PMID: 36796877; PMCID: PMC9936292.
- The Development of the Oncology Center of Excellence Patient-Friendly Language Glossary of Oncology Clinical Trial Terms. Krol D, Kim J, Gao J, Amiri-Kordestani L, Beaver JA, Kluetz P. Oncologist. 2023 Mar 7:oyad034. doi: 10.1093/oncolo/oyad034. PMID: 36882084.
- Minimal Residual Disease Data in Hematologic Malignancy Drug Applications and Labeling: An FDA Perspective. Baines AC, Sarraf Yazdy M, Kasamon YL, Ershler R, Jen EY, Kanapuru B, Richardson NC, Lane A, Carioti T, Theoret MR, Pazdur R, Gormley NJ. Clin Cancer Res. 2023 Mar 9:CCR-22-3579. doi: 10.1158/1078-0432.CCR-22-3579. PMID: 36892497.
- Irreconcilable Differences: The Divorce Between Response Rates, Progression-Free Survival, and Overall Survival. Margret Merino, Yvette Kasamon, Marc Theoret, Richard Pazdur, Paul Kluetz, and Nicole Gormley. J Clin Oncol. 2023 Mar 17;JCO2300225. doi: 10.1200/JCO.23.00225.
- Generating evidence for racial and ethnic minorities during development of oncologic therapeutics. Fashoyin-Aje L, et al. RF Quarterly. 2023;3(1)23-30. Published online 22 March 2023.
- Summary of ASA BIOP Section’s Virtual Discussion with Regulators on Cancer Clinical Trial Design Considerations when Accepting Foreign Data from a Single Country. Barksdale E, Sridhara R, Marchenko O, Jiang Q, Pazdur R. American Statistical Association’s Biopharmaceutical Report Spring 2023, pp 40-42.
- Summary of ASA BIOP Section’s Virtual Discussion with Regulators on Cancer Clinical Trial Design Considerations in Evaluating Treatment Effect in Marker Negative Population. Jiang Q, Sridhara R, Marchenko O, Barksdale E, Theoret M, Pazdur R. American Statistical Association’s Biopharmaceutical Report Spring 2023, pp 43-45.
- Summary of ASA BIOP Section’s Virtual Discussion with Regulators on Impact on Type 1 Error with Unplanned Analyses in Cancer Clinical Trials. Marchenko O, Sridhara R, Jiang Q, Barksdale E, Pazdur R, Theoret M. American Statistical Association’s Biopharmaceutical Report Spring 2023, pp 46-48.
- Summary of ASA BIOP Section’s Virtual Discussion with Regulators on Consideration in the Evaluation and Interpretation of Interim Overall Survival Results from Randomized Cancer Clinical Trials in Chronic Diseases. Sridhara R, Marchenko O, Jiang Q, Barksdale E, Theoret M. American Statistical Association’s Biopharmaceutical Report Spring 2023, pp 49-51.
- Summary of ASA BIOP Section’s Virtual Discussion with Regulators on Considerations in the Evaluation of Progression-Free Survival with Informative Censoring in Cancer Clinical Trials. Sridhara R, Marchenko O, Jiang Q, Barksdale E, Theoret M. American Statistical Association’s Biopharmaceutical Report Spring 2023, pp 52-54.
- FDA Oncology Center of Excellence crowdsourcing initiative: outreach to the scientific community to identify research questions for pooled analyses of oncology clinical trial data. Schneider JA, Eckstein J, Goldberg KB, Ascione MC, Bailey T, Taylor K, Coffey AM, Satchi D, Philips H, Sridhara R, Nair A, Pazdur R, Theoret MR. Clin Cancer Res. 2023 Apr 3:CCR-22-3240. doi: 10.1158/1078-0432.CCR-22-3240. Epub ahead of print. PMID: 37011149.
- Comparative Expedited Regulatory Programs of U.S Food & Drug Administration and Project Orbis Partners. Hotaki LT, Shrestha A, Bennett MP, Valdes IL, Lee SH, Wang Y, Spillman D, MacAulay T, Hunt M, Gervais J, Mafi M, Panetta V, Looi YH, Shum M, Atiek E, Meincke R, Rohr UP, Ainbinder D, Boehm-Cagan A, Luxenburg O, Cerqueira MR, Mouawad LS, Thees MFRES, Prasad K, de Claro RA. Ther Innov Regul Sci. 2023 Apr 18. doi: 10.1007/s43441-023-00522-4. Epub ahead of print. PMID: 37072651.
- Equitable Access to Clinical Trials: How Do We Achieve It? Ana Acuña-Villaorduña, Joaquina Celebre Baranda, Jessica Boehmer, Lola Fashoyin-Aje, and Steven D. Gore. ASCO Educational Book. Volume 43.
- Study design considerations to assess the impact of potential drug-drug interactions in first-in-human studies in oncology drug development. Subramaniam S, Shord SS, Leong R, Norsworthy K, Rahman A, Booth B, Okusanya O. Clin Transl Sci. 2023 May;16(5):719-722. doi: 10.1111/cts.13496. PMID: 36823411; PMCID: PMC10176012.
- The opportunity for greater patient and public involvement and engagement in drug development and regulation. Aiyegbusi OL, Cruz Rivera S, Oliver K, Manna E, Collis P, King-Kallimanis BL, Bhatnagar V, Herold R, Hopkins J, Campbell L, Croker A, Leach M, Calvert MJ. Nat Rev Drug Discov. 2023 May;22(5):337-338. doi: 10.1038/d41573-023-00031-x. PMID: 36854837.
- Landscape and Regulatory Perspective on Oncology Drugs in Pregnancy. Leong R, Grimstein M, DeMaria P, Norsworthy KJ, Fletcher EP, Shord S. J Clin Pharmacol. 2023 Jun;63 Suppl 1:S170-S175. doi: 10.1002/jcph.2224. PMID: 37317488.
- Continuation of Third-Generation Tyrosine Kinase Inhibitors in Second-Line Trials for EGFR-Mutated Non-Small-Cell Lung Cancer: Regulatory Considerations. Goulart BHL, Larkins E, Beaver JA, Singh H. J Clin Oncol. 2023 Jun 8:JCO2300154. doi: 10.1200/JCO.23.00154. PMID: 37290026.
- Project Confirm: Accelerated Drug Approvals for Chronic Myeloid Leukemia. Sweet KL, Cortes JE, Apperley JF, Mann M, Mauro MJ, Oehler VG, Ruiz C, Schiffer CA, Ehrlich LA, Pamuk GE, Wynne J, Mehta GU, de Claro RA, Theoret MR, Smith BD, Norsworthy KJ. Clin Cancer Res. 2023 Jun 13;29(12):2179-2183. doi: 10.1158/1078-0432.CCR-22-2628. PMID: 36547666; PMCID: PMC10272032.
- Driving Diversity and Inclusion in Cancer Drug Development - Industry and Regulatory Perspectives, Current Practices, Opportunities, and Challenges. Fashoyin-Aje LA, Tendler C, Lavery B, Ghiorghiu S, Gerald B, Kalidas C, Richie N, Winson K, Warren NJH, Tellman TV, Retzlaff J, Foti M, Pazdur R. Clin Cancer Res. 2023 Jun 28:OF1-OF7. doi: 10.1158/1078-0432.CCR-23-1391. PMID: 37378578.
- Advancing Therapies for Asian Americans, Native Hawaiians, and Other Pacific Islanders With Cancer: OCE's Project ASIATICA. Gao JJ, Pazdur R, Kim T. JCO Oncol Pract. 2023 Jun 29:OP2300198. doi: 10.1200/OP.23.00198. PMID: 37384849.
- Summary of ASA BIOP Section’s Virtual Discussion with Regulators on Assessing Bias in Cancer Studies with Real-World Data Elements. Sridhara R, Marchenko O, Jiang Q, Barksdale E, Chen Y, Kluetz P. American Statistical Association’s Biopharmaceutical Report Summer 2023, pp 55-56.
- Summary of ASA BIOP Section’s Virtual Discussion with Regulators on Statistical Considerations in the Early Interim Overall Survival Analysis in Indolent Cancers for Evaluation Risk. Sridhara R, Marchenko O, Jiang Q, Barksdale E, Chen Y, Theoret M. American Statistical Association’s Biopharmaceutical Report Summer 2023, pp 57-59.
- Summary of ASA BIOP Section’s Virtual Discussion with Regulators on Non-inferiority Cancer Clinical Trial Design Considerations When Data from a Single Foreign Country in Available. Sridhara R, Marchenko O, Jiang Q, Barksdale E, Chen Y, Theoret M. American Statistical Association’s Biopharmaceutical Report Summer 2023, pp 60-61.
- Summary of ASA BIOP Section’s Virtual Discussion with Regulators on Considerations of Innovative Cancer Clinical Trial Designs for Post-Market Dose Optimization Studies. Sridhara R, Marchenko O, Jiang Q, Barksdale E, Chen Y, Theoret M. American Statistical Association’s Biopharmaceutical Report Summer 2023, pp 62-64.
- Considerations for Drug Development in Myelodysplastic Syndromes. Sekeres MA, Kim N, DeZern AE, Norsworthy KJ, Garcia JS, de Claro RA, Theoret MR, Jen EY, Ehrlich LA, Zeidan AM, Komrokji RS. Clin Cancer Res. 2023 Jul 14;29(14):2573-2579. doi: 10.1158/1078-0432.CCR-22-3348. PMID: 36688922; PMCID: PMC10349686.
- Handbook for Designing and Conducting Clinical and Translational Research: Translational Radiation Oncology. Chapters 77 and 88 coauthored by Shruti U. Gandhy, Sandra J. Casak, and Jennifer J. Lee. Elsevier. Published April 2023.
- FDA Aims to Broaden Access to New Therapies. Cancer Discov. 2023 Aug 11:OF1. doi: 10.1158/2159-8290.CD-NB2023-0058. PMID: 37565721.
- US Food and Drug Administration embraces using innovation to identify optimized dosages for patients with cancer. CPT Pharmacometrics Syst Pharmacol. Shord SS, Zhu H, Liu J, Rahman A, Booth B, Zineh I. 2023 Aug 28. doi: 10.1002/psp4.13033.PMID: 37641498.
- Advancing Clinical Trial Design for Non-Muscle Invasive Bladder Cancer. Chang, Elaine et al.; Bladder Cancer, 8 September 2023.
- Drug Development and Regulatory Considerations. Hsu V, Thompson MD, Przepiorka D, Okusanya OO. In Cancer Pharmacology 2nd Edition, Emadi A and Karp JE, eds. Springer Publishing Company, Danvers, MA, October 2023. DOI: 10.1891/9780826149336.
- Differentiation Syndrome in Acute Leukemia: APL and Beyond. Cancers (Basel). Woods AC, Norsworthy KJ. 2023 Sep 28;15(19):4767. doi: 10.3390/cancers15194767. PMID: 37835461; PMCID: PMC10571864.
- Sponsor Perspectives on the Impact of the COVID-19 Pandemic on Interventional Cancer Clinical Trial Protocols and Data Quality. Unger JM, Stires H, Levit LA, Stewart M, McKelvey BA, Canin B, Dressler E, Flaherty K, Fredette P, Jones L, McCann P, Miller T, Onitilo AA, Palmieri F, Patel T, Paul R, Smith GL, Bruinooge SS, Garrett-Mayer E, Lei XJ, Alva A, Schenkel C. JCO Oncol Pract. 2023 Oct;19(10):907-916. doi: 10.1200/OP.23.00185. PMID: 37643386; PMCID: PMC10615547.
- Developing Therapy for Every Kid With Cancer - Summary of a Scientific Session at the 2023 ASCPT Meeting. Moore JN, Campagne O, Bhojwani D, Crowe D, Stewart CF. Clin Pharmacol Ther. 2023 Nov 24. doi: 10.1002/cpt.3101. PMID: 37997700.
- Summary of ASA BIOP Section’s Virtual Discussion with Regulators on Consideration of Criteria for Evaluation of Surrogate Endpoints. Sridhara R, Marchenko O, Jiang Q, Barksdale E, Chen Y, Theoret M. American Statistical Association’s Biopharmaceutical Report Winter 2023, pp 49-51.
- Summary of ASA BIOP Section’s Virtual Discussion with Regulators on Cancer Clinical Trial Design and Analysis Considerations in Evaluating Treatment Effect in Market Negative Population-Part 2. Sridhara R, Marchenko O, Jiang Q, Barksdale E, Chen Y, Theoret M. American Statistical Association’s Biopharmaceutical Report Winter 2023, pp 52-54.
- Utility of Physiologically Based Pharmacokinetic Modeling to Investigate the Impact of Physiological Changes of Pregnancy and Cancer on Oncology Drug Pharmacokinetics. Yang X, Grimstein M, Pressly M, Fletcher EP, Shord S, Leong R. Pharmaceutics. 2023 Dec 4;15(12):2727. doi: 10.3390/pharmaceutics15122727. PMID: 38140068; PMCID: PMC10748010.
- Statistical Considerations in Pediatric Cancer Trials: Report of American Statistical Association Biopharmaceutical Section Open Forum Discussions, Statistics in Biopharmaceutical Research. Sridhara R, Marchenko O, Jiang Q, Barksdale E, Alonzo T, Amatya A, Arons D, Bliu A, Choo Q, Coory M, Donoghue M, Ehrlich L, Filho L, Fox E, Freidlin B, Goodman N, Hawkins D, Häring D, Karres D, Kolb E, Mao H, Kalyani P, Naranjo A, Pappo A, Posch M, Price K, Raven A, Rantell K, Renfro L, Rivera D, Roy P, Shan M, Simon R, Singh S, Smith M, Theoret M, Thomas M, Thomas Z, Thompson A, Tian H, Tymofyeyev Y, Vallejo J, Wathen K, Ye J, Pazdur R, Reaman G (2023), 15:4, 845-851, DOI: 10.1080/19466315.2023.2238650
Research Papers
- Abstract PO-084: The pharmaceutical industry in action: 2021 clinical research diversity and inclusion survey. Cancer Epidemiology, Biomarkers & Prevention, 31(1_Supplement), PO-084 Regnante JM, Fashoyin-Aje L, Sonet EM, Highsmith Q, Gonzales M, Amaro S, Davis A, Murray MS, Karmo M, Bierer B. Cancer Epidemiol Biomarkers Prev (2022) 31 (1_Supplement): PO-084. January 1, 2022.
- Nonproportional Hazards – An evaluation of the MaxCombo test in cancer clinical trials. Shen YL, Wang X, Mushti S, Mulkey F, Zhou J, Gao X, Zhang L, Gwise T, Tang S, Theoret M, Pazdur R, Sridhara R. Statistics in Biopharmaceutical Research 2022. Published online: Jan. 4, 2022
- Exploring the Potential of External Control Arms created from Patient Level Data: A case study in non-small cell lung cancer. Yin X, Mishra-Kalyan PS, Sridhara R, Stewart MD, Stuart EA, Davi RC. J Biopharm Stat. 2022 Jan 2;32(1):204-218. doi: 10.1080/10543406.2021.2011901. PMID: 34986069.
- In search of autophagy biomarkers in breast cancer: Receptor status and drug agnostic transcriptional changes during autophagy flux in cell lines. Mascia F, Mazo I, Alterovitz WL, Karagiannis K, Wu WW, Shen RF, Beaver JA, Rao VA. PLoS One. 2022 Jan 6;17(1):e0262134. doi: 10.1371/journal.pone.0262134. PMID: 34990474; PMCID: PMC8735604.
- Achieving robust somatic mutation detection with deep learning models derived from reference data sets of a cancer sample. Sahraeian SME, Fang LT, Karagiannis K, Moos M, Smith S, Santana-Quintero L, Xiao C, Colgan M, Hong H, Mohiyuddin M, Xiao W. Genome Biol. 2022 Jan 7;23(1):12. doi: 10.1186/s13059-021-02592-9. PMID: 34996510; PMCID: PMC8740374.
- Association of Radiation Therapy With Risk of Adverse Events in Patients Receiving Immunotherapy: A Pooled Analysis of Trials in the US Food and Drug Administration Database. Anscher MS, Arora S, Weinstock C, Amatya A, Bandaru P, Tang C, Girvin AT, Fiero MH, Tang S, Lubitz R, Amiri-Kordestani L, Theoret MR, Pazdur R, Beaver JA. JAMA Oncol. 2022 Feb 1;8(2):232-240. doi: 10.1001/jamaoncol.2021.6439. Erratum in: JAMA Oncol. 2022 Feb 1;8(2):306. PMID: 34989781; PMCID: PMC8739815.
- Floor and ceiling effects in the EORTC QLQ-C30 Physical Functioning Subscale among patients with advanced or metastatic breast cancer. Murugappan MN, King-Kallimanis BL, Mangir C, Howie L, Bhatnagar V, Beaver JA, Basch EM, Henson SR, Kluetz PG. Cancer. 2022 Feb 15;128(4):808-818. doi: 10.1002/cncr.33959. PMID: 34634139.
- Reply to "Another look at floor and ceiling effects in the EORTC QLQ-C30 Physical Functioning subscale and possible solutions". Murugappan MN, King-Kallimanis BL, Mangir C, Howie L, Bhatnagar V, Beaver JA, Basch EM, Henson SR, Kluetz PG. Cancer. 2022 Jun 15;128(12):2386-2387. doi: 10.1002/cncr.34197. PMID: 35389509.
- Exploring racial disparities in treatment patterns and outcomes for patients with multiple myeloma using real world data. Maignan K, Fashoyin-Aje LA, Torres AZ, Fernandes LL, Gwise T, Baxi SB, Roose JP, Rivera DR, Shen YL, Kluetz PG, Gormley NJ. Blood Cancer J. 2022 Apr 19;12(4):65. doi: 10.1038/s41408-022-00665-x. PMID: 35440047; PMCID: PMC9018767.
- Response Rate, Event-Free Survival, and Overall Survival in Newly Diagnosed Acute Myeloid Leukemia: US Food and Drug Administration Trial-Level and Patient-Level Analyses. Norsworthy KJ, Gao X, Ko CW, Pulte ED, Zhou J, Gong Y, Shen YL, Vallejo J, Gwise TE, Sridhara R, Deisseroth AB, Farrell AT, de Claro RA, Blumenthal GM, Pazdur R. J Clin Oncol. 2022 Mar 10;40(8):847-854. doi: 10.1200/JCO.21.01548. PMID: 34890212; PMCID: PMC8906455.
- Analysis of racial and ethnic disparities in multiple myeloma US FDA drug approval trials. Kanapuru B, Fernandes LL, Fashoyin-Aje LA, Baines AC, Bhatnagar V, Ershler R, Gwise T, Kluetz P, Pazdur R, Pulte E, Shen YL, Gormley N. Blood Adv. 2022 Mar 22;6(6):1684-1691. doi: 10.1182/bloodadvances.2021005482. PMID: 35114691; PMCID: PMC8941450.
- Time to deterioration of symptoms or function using patient-reported outcomes in cancer trials. Fiero MH, Roydhouse JK, Bhatnagar V, Chen TY, King-Kallimanis BL, Tang S, Kluetz PG. Lancet Oncol. 2022 May;23(5):e229-e234. doi: 10.1016/S1470-2045(22)00021-3. PMID: 35489354.
- US Food and Drug Administration Analysis of Patient-Reported Diarrhea and Its Impact on Function and Quality of Life in Patients Receiving Treatment for Breast Cancer. Chen TY, King-Kallimanis BL, Merzoug L, Horodniceanu EG, Fiero MH, Gao JJ, Beaver JA, Bhatnagar V, Kluetz P. Value Health. 2022 Apr;25(4):566-570. doi: 10.1016/j.jval.2021.09.007. PMID: 35365300.
- Updated FDA pooled analysis of pain medication use in trial participants with HR+, HER2-negative metastatic breast cancer treated with endocrine therapy and a CDK 4/6 inhibitor. Gao JL, Girvin A, Hodgdon C, Osgood C, Bhatnagar V, Kluetz P, Pazdur R, Amiri-Kordestani L, Beaver JA. DOI: 10.1200/JCO.2022.40.16_suppl.e24101 Journal of Clinical Oncology 40, no. 16_suppl (June 01, 2022) e24101-e24101.
- Analysis of Association of Radiation Therapy With Risk of Adverse Events in Patients Receiving Immunotherapy Using Pooled Trial Data Matched by Propensity Score-Reply. Weinstock C, Amatya A, Beaver JA. JAMA Oncol. 2022 Jul 1;8(7):1073. doi: 10.1001/jamaoncol.2022.0880. PMID: 35511133.
- Personalized genome assembly for accurate cancer somatic mutation discovery using tumor-normal paired reference samples. Xiao C, Chen Z, Chen W, Padilla C, Colgan M, Wu W, Fang LT, Liu T, Yang Y, Schneider V, Wang C, Xiao W.Genome Biol. 2022 Nov 9;23(1):237. doi: 10.1186/s13059-022-02803-x. PMID: 36352452; PMCID: PMC9648002.
- Burden of Cardiovascular Disease in Immune Checkpoint Inhibitor–Treated Patients: Reconciling Adjudicated and Coded Outcomes. Kondapalli L, Hsia J, Miller R, Flaig TW, Bonaca MP. J Am Coll Cardiol CardioOnc. 2022 Dec, 4 (5) 649–656
- Structural variant analysis of a cancer reference cell line sample using multiple sequencing technologies. Talsania K, Shen TW, Chen X, Jaeger E, Li Z, Chen Z, Chen W, Tran B, Kusko R, Wang L, Pang AWC, Yang Z, Choudhari S, Colgan M, Fang LT, Carroll A, Shetty J, Kriga Y, German O, Smirnova T, Liu T, Li J, Kellman B, Hong K, Hastie AR, Natarajan A, Moshrefi A, Granat A, Truong T, Bombardi R, Mankinen V, Meerzaman D, Mason CE, Collins J, Stahlberg E, Xiao C, Wang C, Xiao W, Zhao Y. Genome Biol. 2022 Dec 13;23(1):255. doi: 10.1186/s13059-022-02816-6. PMID: 36514120; PMCID: PMC9746098.
Approval Summaries
- FDA Approval Summary: Capmatinib and Tepotinib for the Treatment of Metastatic NSCLC Harboring MET Exon 14 Skipping Mutations or Alterations. Mathieu LN, Larkins E, Akinboro O, Roy P, Amatya AK, Fiero MH, Mishra-Kalyani PS, Helms WS, Myers CE, Skinner AM, Aungst S, Jin R, Zhao H, Xia H, Zirkelbach JF, Bi Y, Li Y, Liu J, Grimstein M, Zhang X, Woods S, Reece K, Abukhdeir AM, Ghosh S, Philip R, Tang S, Goldberg KB, Pazdur R, Beaver JA, Singh H. Clin Cancer Res. 2022 Jan 15;28(2):249-254. doi: 10.1158/1078-0432.CCR-21-1566. PMID: 34344795.
- FDA Approval Summary: Abemaciclib With Endocrine Therapy for High-Risk Early Breast Cancer.Royce M, Osgood C, Mulkey F, Bloomquist E, Pierce WF, Roy A, Kalavar S, Ghosh S, Philip R, Rizvi F, Mixter BD, Tang S, Pazdur R, Beaver JA, Amiri-Kordestani L. J Clin Oncol. 2022 Apr 10;40(11):1155-1162. doi: 10.1200/JCO.21.02742. PMID: 35084948; PMCID: PMC8987222.
- FDA Approval Summary: Nivolumab in Combination with Ipilimumab for the Treatment of Unresectable Malignant Pleural Mesothelioma. Nakajima EC, Vellanki PJ, Larkins E, Chatterjee S, Mishra-Kalyani PS, Bi Y, Qosa H, Liu J, Zhao H, Biable M, Hotaki LT, Shen YL, Pazdur R, Beaver JA, Singh H, Donoghue M. Clin Cancer Res. 2022 Feb 1;28(3):446-451. doi: 10.1158/1078-0432.CCR-21-1466. Epub 2021 Aug 30. PMID: 34462287; PMCID: PMC8810571.
- FDA Approval Summary: Revised Indication and Dosing Regimen for Ponatinib Based on the Results of the OPTIC Trial. Pulte ED, Chen H, Price LSL, Gudi R, Li H, Okusanya OO, Ma L, Rodriguez L, Vallejo J, Norsworthy KJ, de Claro RA, Theoret MR, Pazdur R. Oncologist. 2022 Mar 4;27(2):149-157. doi: 10.1093/oncolo/oyab040. PMID: 35641211; PMCID: PMC8895737.
- FDA approval summary: Crizotinib for pediatric and young adult patients with relapsed or refractory systemic anaplastic large cell lymphoma. Merino M, Kasamon Y, Li H, Ma L, Leong R, Zhou J, Reaman G, Chambers W, Richardson N, Theoret M, Pazdur R, Gormley N. Pediatr Blood Cancer. 2022 Aug;69(8):e29602. doi: 10.1002/pbc.29602. Epub 2022 May 13. PMID: 35561013.
- FDA Approval Summary: Idecabtagene Vicleucel for Relapsed or Refractory Multiple Myeloma. Sharma P, Kanapuru B, George B, Lin X, Xu Z, Bryan WW, Pazdur R, Theoret MR. Clin Cancer Res. 2022 May 2;28(9):1759-1764. doi: 10.1158/1078-0432.CCR-21-3803. PMID: 35046063; PMCID: PMC9064878.
- FDA Approval Summary: Pembrolizumab, Atezolizumab, and Cemiplimab-rwlc as Single Agents for First-Line Treatment of Advanced/Metastatic PD-L1-High NSCLC. Akinboro O, Larkins E, Pai-Scherf LH, Mathieu LN, Ren Y, Cheng J, Fiero MH, Fu W, Bi Y, Kalavar S, Jafri S, Mishra-Kalyani PS, Fourie Zirkelbach J, Li H, Zhao H, He K, Helms WS, Chuk MK, Wang M, Bulatao I, Herz J, Osborn BL, Xu Y, Liu J, Gong Y, Sickafuse S, Cohen R, Donoghue M, Pazdur R, Beaver JA, Singh H. Clin Cancer Res. 2022 Jun 1;28(11):2221-2228. doi: 10.1158/1078-0432.CCR-21-3844. PMID: 35101885.
- FDA Approval Summary: Ruxolitinib for Treatment of Chronic Graft-Versus-Host Disease after Failure of One or Two Lines of Systemic Therapy. Le RQ, Wang X, Zhang H, Li H, Przepiorka D, Vallejo J, Leong R, Ma L, Goldberg KB, Pazdur R, Theoret MR, De Claro A. Oncologist. 2022 Jun 8;27(6):493-500. doi: 10.1093/oncolo/oyac042. PMID: 35363318; PMCID: PMC9177119.
- FDA Approval Summary: Belumosudil for Adult and Pediatric Patients 12 Years and Older with Chronic GvHD after Two or More Prior Lines of Systemic Therapy. Przepiorka D, Le RQ, Ionan A, Li RJ, Wang YH, Gudi R, Mitra S, Vallejo J, Okusanya OO, Ma L, Yang Y, Patel P, Mezaache D, Shah R, Banerjee A, McLamore S, Maung AN, Goldberg KB, Pazdur R, Theoret MR, De Claro RA. Clin Cancer Res. 2022 Jun 13;28(12):2488-2492. doi: 10.1158/1078-0432.CCR-21-4176. PMID: 35135839; PMCID: PMC9197942.
- FDA Approval Summary: Ivosidenib for the Treatment of Patients with Advanced Unresectable or Metastatic, Chemotherapy Refractory Cholangiocarcinoma with an IDH1 Mutation. Casak SJ, Pradhan S, Fashoyin-Aje LA, Ren Y, Shen YL, Xu Y, Chow ECY, Xiong Y, Zirklelbach JF, Liu J, Charlab R, Pierce WF, Fesenko N, Beaver JA, Pazdur R, Kluetz PG, Lemery SJ. Clin Cancer Res. 2022 Jul 1;28(13):2733-2737. doi: 10.1158/1078-0432.CCR-21-4462. PMID: 35259259; PMCID: PMC9250596.
- FDA Approval Summary: Axicabtagene Ciloleucel for Relapsed or Refractory Follicular Lymphoma. Bouchkouj N, Zimmerman M, Kasamon YL, Wang C, Dai T, Xu Z, Wang X, Theoret M, Purohit-Sheth T, George B. Oncologist. 2022 Jul 5;27(7):587-594. doi: 10.1093/oncolo/oyac054. PMID: 35403693; PMCID: PMC9255972.
- FDA Approval Summary: Decitabine and Cedazuridine Tablets for Myelodysplastic Syndromes. Kim N, Norsworthy KJ, Subramaniam S, Chen H, Manning ML, Kitabi E, Earp J, Ehrlich LA, Okusanya OO, Vallejo J, Gehrke BJ, de Claro RA, Pazdur R. Clin Cancer Res. 2022 Aug 15;28(16):3411-3416. doi: 10.1158/1078-0432.CCR-21-4498. PMID: 35435961; PMCID: PMC9378483.
- FDA Approval Summary: Tivozanib for Relapsed or Refractory Renal Cell Carcinoma. Chang E, Weinstock C, Zhang L, Fiero MH, Zhao M, Zahalka E, Ricks TK, Fourie Zirkelbach J, Qiu J, Yu J, Chen XH, Bhatnagar V, Goldberg KB, Tang S, Kluetz PG, Pazdur R, Ibrahim A, Beaver JA, Amiri-Kordestani L. Clin Cancer Res. 2022 Feb 1;28(3):441-445. doi: 10.1158/1078-0432.CCR-21-2334. Epub 2021 Aug 20. PMID: 34417198; PMCID: PMC8810577.
- FDA Approval Summary: Mobocertinib for Metastatic Non-Small Cell Lung Cancer with EGFR Exon 20 Insertion Mutations. Duke ES, Stapleford L, Drezner N, Amatya AK, Mishra-Kalyani PS, Shen YL, Maxfield K, Fourie Zirkelbach J, Bi Y, Liu J, Zhang X, Wang H, Yang Y, Zheng N, Reece K, Wearne E, Glen JJ, Ojofeitimi I, Scepura B, Nair A, Bikkavilli RK, Ghosh S, Philip R, Pazdur R, Beaver JA, Singh H, Donoghue M. Clin Cancer Res. 2022 Sep 16:CCR-22-2072. doi: 10.1158/1078-0432.CCR-22-2072. PMID: 36112541.
- FDA Approval Summary: Cabozantinib for Differentiated Thyroid Cancer. Duke ES, Barone AK, Chatterjee S, Mishra-Kalyani PS, Shen YL, Isikwei E, Zhao H, Bi Y, Liu J, Rahman NA, Wearne E, Leighton JK, Stephenson M, Ojofeitimi I, Scepura B, Nair A, Pazdur R, Beaver JA, Singh H. Clin Cancer Res. 2022 Oct 3;28(19):4173-4177. doi: 10.1158/1078-0432.CCR-22-0873. PMID: 35679021; PMCID: PMC9529996.
- FDA Approval Summary: Pemigatinib for Previously Treated, Unresectable Locally Advanced or Metastatic Cholangiocarcinoma with FGFR2 fusion or other rearrangement. Patel TH, Marcus L, Horiba MN, Donoghue M, Chatterjee S, Mishra-Kalyani PS, Schuck RN, Li Y, Zhang X, Fourie Zirkelbach J, Charlab R, Liu J, Yang Y, Lemery SJ, Pazdur R, Theoret MR, Fashoyin-Aje LA. Clin Cancer Res. 2022 Oct 7:CCR-22-2036. doi: 10.1158/1078-0432.CCR-22-2036. PMID: 36206041.
- FDA Approval Summary: Belantamab Mafodotin for Patients with Relapsed or Refractory Multiple Myeloma. Baines AC, Ershler R, Kanapuru B, Xu Q, Shen G, Li L, Ma L, Okusanya OO, Simpson NE, Nguyen W, Theoret MR, Pazdur R, Gormley NJ. Clin Cancer Res. 2022 Nov 1;28(21):4629-4633. doi: 10.1158/1078-0432.CCR-22-0618. PMID: 35736811; PMCID: PMC9633344.
- FDA Approval Summary: Belzutifan for von Hippel-Lindau Disease-Associated Tumors. Fallah J, Brave MH, Weinstock C, Mehta GU, Bradford D, Gittleman H, Bloomquist EW, Charlab R, Hamed SS, Miller CP, Dorff SE, Chambers WA, Mixter BD, Dinin J, Pierce WF, Ricks TK, Tang S, Donoghue M, Pazdur R, Amiri-Kordestani L, Ibrahim A, Beaver JA. Clin Cancer Res. 2022 Nov 14;28(22):4843-4848. doi: 10.1158/1078-0432.CCR-22-1054. PMID: 35727604; PMCID: PMC9669093.
- FDA Approval Summary: lutetium Lu 177 vipivotide tetraxetan for patients with metastatic castration-resistant prostate cancer. Fallah J, Agrawal S, Gittleman H, Fiero MH, Subramaniam S, John C, Chen W, Ricks TK, Niu G, Fotenos A, Wang M, Chiang K, Pierce WF, Suzman DL, Tang S, Pazdur R, Amiri-Kordestani L, Ibrahim A, Kluetz PG. Clin Cancer Res. 2022 Dec 5:CCR-22-2875. doi: 10.1158/1078-0432.CCR-22-2875. PMID: 36469000.
- FDA Approval Summary: Ripretinib for advanced gastrointestinal stromal tumor. Kumar V, Doros L, Thompson M, Mushti SL, Charlab R, Spehalski EI, Zhao H, Thompson MD, Tang S, Pazdur R, Lemery SJ, Theoret MR, Fashoyin-Aje LA. Clin Cancer Res. 2022 Dec 9:CCR-22-2400. doi: 10.1158/1078-0432.CCR-22-2400. PMID: 36485007.
- FDA Approval Summary: Pembrolizumab for Neoadjuvant and Adjuvant Treatment of Patients with High-Risk Early-Stage Triple-Negative Breast Cancer. Shah M, Osgood CL, Amatya AK, Fiero MH, Pierce WF, Nair A, Herz J, Robertson KJ, Mixter BD, Tang S, Pazdur R, Beaver JA, Amiri-Kordestani L. Clin Cancer Res. 2022 Dec 15;28(24):5249-5253. doi: 10.1158/1078-0432.CCR-22-1110. PMID: 35925043.
- FDA Approval Summary: Nivolumab for the Adjuvant Treatment of Adults with Completely Resected Esophageal/Gastroesophageal Junction Cancer and Residual Pathologic Disease. Horiba MN, Casak SJ, Mishra-Kalyani PS, Roy P, Beaver JA, Pazdur R, Kluetz PG, Lemery SJ, Fashoyin-Aje LA. Clin Cancer Res. 2022 Dec 15;28(24):5244-5248. doi: 10.1158/1078-0432.CCR-22-0617. PMID: 35960160; PMCID: PMC9771915.
Regulatory Perspectives
- Perspectives on Drug Development in Multiple Myeloma-Looking Forward to 2025. Voorhees PM, Jakubowiak AJ, Kumar SK, Kanapuru B, Baines AC, Bhatnagar V, Ershler R, Theoret MR, Gormley NJ, Pazdur R. Clin Cancer Res. 2022 Jan 1;28(1):23-26. doi: 10.1158/1078-0432.CCR-21-1069. PMID: 34315721.
- Call to Action for Improving Oral Anticancer Agent Adherence. Levit LA, Arora S, Kluetz PG, Magnuson A, Rahman A, Harvey RD. J Clin Oncol. 2022 Apr 1;40(10):1036-1040. doi: 10.1200/JCO.21.02529. PMID: 34990218.
- External control arms in oncology: current use and future directions. Mishra-Kalyani PS, Amiri Kordestani L, Rivera DR, Singh H, Ibrahim A, DeClaro RA, Shen Y, Tang S, Sridhara R, Kluetz PG, Concato J, Pazdur R, Beaver JA. Ann Oncol. 2022 Apr;33(4):376-383. doi: 10.1016/j.annonc.2021.12.015. PMID: 35026413.
- Patient-Reported Outcomes in Pediatric Cancer Registration Trials: A US Food and Drug Administration Perspective. Murugappan MN, King-Kallimanis BL, Reaman GH, Bhatnagar V, Horodniceanu EG, Bouchkouj N, Kluetz PG. J Natl Cancer Inst. 2022 Jan 11;114(1):12-19. doi: 10.1093/jnci/djab087. PMID: 33930159; PMCID: PMC8755487.
- Importing oncology trials from China: a bridge over troubled waters? Singh H, Pazdur R. Lancet Oncol. 2022 Mar;23(3):323-325. doi: 10.1016/S1470-2045(22)00071-7. PMID: 35131039.
- Better by design: a regulatory perspective on rationalizing combination selection and development. Immuno-Oncology Insights 2022; 3(5), 57–63. 10.18609/ioi.2022.007. Published: 28 February 2022. Interview with Elaine Chang.
- Accelerated Approval Is Not Conditional Approval: Insights From International Expedited Approval Programs. Mehta GU, de Claro RA, Pazdur R. JAMA Oncol. 2022 Mar 1;8(3):335-336. doi: 10.1001/jamaoncol.2021.6854. PMID: 35050302.
- Blurring the Lines of Design: Prospective Observational Cohorts and Pragmatic Clinical Trials Characterizing Treatment Effects in Routine Cancer Care. Rivera DR, Larkins E, Kluetz PG. Oncologist. 2022 Mar 4;27(2):79-81. doi: 10.1093/oncolo/oyab071. PMID: 35641221; PMCID: PMC8895745.
- Project Confirm: An Initiative to Promote Transparency of the Accelerated Approval Program for Oncology Indications. The ASCO Post. Mehta GU, Rizvi F, Kim T. March 10, 2022.
- Optimizing Clinical Trial Design for Patients with Non-muscle-invasive Bladder Cancer. Weinstock C, Agrawal S, Chang E. Eur Urol. 2022 Mar 15:S0302-2838(22)01655-4. doi: 10.1016/j.eururo.2022.02.014. PMID: 35304001.
- U.S. FDA Drug Approvals for Breast Cancer: A Decade in Review. Arora S, Narayan P, Osgood CL, Wedam S, Prowell TM, Gao JJ, Shah M, Krol D, Wahby S, Royce M, Ghosh S, Philip R, Ison G, Berman T, Brus C, Bloomquist EW, Fiero MH, Tang S, Pazdur R, Ibrahim A, Amiri-Kordestani L, Beaver JA. Clin Cancer Res. 2022 Mar 15;28(6):1072-1086. doi: 10.1158/1078-0432.CCR-21-2600. PMID: 34711632; PMCID: PMC8923912.
- U.S. FDA Drug Approvals for Gynecological Malignancies: A Decade in Review. Arora S, Narayan P, Ison G, Berman T, Suzman DL, Wedam S, Prowell TM, Ghosh S, Philip R, Osgood CL, Gao JJ, Shah M, Krol D, Wahby S, Royce M, Brus C, Bloomquist EW, Fiero MH, Tang S, Pazdur R, Ibrahim A, Amiri-Kordestani L, Beaver JA. Clin Cancer Res. 2022 Mar 15;28(6):1058-1071. doi: 10.1158/1078-0432.CCR-21-2599. PMID: 34711631; PMCID: PMC8923904.
- Clinical Evidence Generation During a Pandemic: Lessons Learned for Sustaining Progress. Rivera DR, Kluetz PG, Abdallah K, Agrawal S, Angus DC, Califf RM, Garrett-Mayer E, Hyer R, Lowy DR, Khleif SN. Cancer J. 2022 Mar-Apr 01;28(2):151-156. doi: 10.1097/PPO.0000000000000589. PMID: 35333502.
- Summary of ASA Virtual Discussion with Regulators on Statistical Considerations in Clinical Trials for Rare Pediatric Cancers. Sridhara R, Marchenko O, Jiang Q, et.al. Biopharmaceutical Report, Spring 2022, Vol. 29, No 1, pp 30-32.
- Increasing Inclusiveness of Patient-Centric Clinical Evidence Generation in Oncology: Real-World Data and Clinical Trials. Benbow JH, Rivera DR, Lund JL, Feldman JE, Kim ES. Am Soc Clin Oncol Educ Book. 2022 Apr;42:1-11. doi: 10.1200/EDBK_350574. PMID: 35561304.
- The saga of PI3K inhibitors in haematological malignancies: survival is the ultimate safety endpoint. Richardson NC, Kasamon Y, Pazdur R, Gormley N. Lancet Oncol. 2022 May;23(5):563-566. doi: 10.1016/S1470-2045(22)00200-5. Epub 2022 Apr 14. PMID: 35429996.
- Reimagining patient-centric cancer clinical trials: a multi-stakeholder international coalition. Li BT, Daly B, Gospodarowicz M, Bertagnolli MM, Brawley OW, Chabner BA, Fashoyin-Aje L, de Claro RA, Franklin E, Mills J, Legos J, Kaucic K, Li M, The L, Hou T, Wu TH, Albrecht B, Shao Y, Finnegan J, Qian J, Shahidi J, Gasal E, Tendler C, Kim G, Yan J, Morrow PK, Fuchs CS, Zhang L, LaCaze R, Oelrich S, Murphy MJ, Pazdur R, Rudd K, Wu YL. Nat Med. 2022 Apr;28(4):620-626. doi: 10.1038/s41591-022-01775-6. PMID: 35440725.
- Development of Tissue-Agnostic Treatments for Patients with Cancer. Annual Review of Cancer Biology. Lemery S, Fashoyin-Aje L, Marcus L, Casak S, Schneider J, Theoret M, Kluetz P, Pazdur R, Beaver J. April 2022.
- Reaching beyond maximum grade: progress and future directions for modernising the assessment and reporting of adverse events in haematological malignancies. Thanarajasingam G, Minasian LM, Bhatnagar V, Cavalli F, De Claro RA, Dueck AC, El-Galaly TC, Everest N, Geissler J, Gisselbrecht C, Gormley N, Gribben J, Horowitz M, Ivy SP, Jacobson CA, Keating A, Kluetz PG, Kwong YL, Little RF, Matasar MJ, Mateos MV, McCullough K, Miller RS, Mohty M, Moreau P, Morton LM, Nagai S, Nair A, Nastoupil L, Robertson K, Sidana S, Smedby KE, Sonneveld P, Tzogani K, van Leeuwen FE, Velikova G, Villa D, Wingard JR, Seymour JF, Habermann TM. Lancet Haematol. 2022 May;9(5):e374-e384. doi: 10.1016/S2352-3026(22)00045-X. PMID: 35483398; PMCID: PMC9241484.
- How to Get the Dose Right. The ASCO Post. Mirat Shah, Antiqur Rahman, Marc R. Theoret, and Richard Pazdur. May 10, 2022.
- Timing is everything: The importance of patient-reported outcome assessment frequency when characterizing symptomatic adverse events. King-Kallimanis BL, Bhatnagar V, Horodniceanu EG, Chen TY, Kluetz PG. Clin Trials. 2022 Jun;19(3):267-273. doi: 10.1177/17407745221093935. Epub 2022 May 15. PMID: 35575012.
- Ethical Considerations for the Inclusion of Patient-Reported Outcomes in Clinical Research: The PRO Ethics Guidelines. Cruz Rivera S, Aiyegbusi OL, Ives J, Draper H, Mercieca-Bebber R, Ells C, Hunn A, Scott JA, Fernandez CV, Dickens AP, Anderson N, Bhatnagar V, Bottomley A, Campbell L, Collett C, Collis P, Craig K, Davies H, Golub R, Gosden L, Gnanasakthy A, Haf Davies E, von Hildebrand M, Lord JM, Mahendraratnam N, Miyaji T, Morel T, Monteiro J, Zwisler AO, Peipert JD, Roydhouse J, Stover AM, Wilson R, Yap C, Calvert MJ. JAMA. 2022 May 17;327(19):1910-1919. doi: 10.1001/jama.2022.6421. PMID: 35579638.
- Increasing Racial and Ethnic Diversity in Cancer Clinical Trials: An American Society of Clinical Oncology and Association of Community Cancer Centers Joint Research Statement. Oyer RA, Hurley P, Boehmer L, Bruinooge SS, Levit K, Barrett N, Benson A, Bernick LA, Byatt L, Charlot M, Crews J, DeLeon K, Fashoyin-Aje L, Garrett-Mayer E, Gralow JR, Green S, Guerra CE, Hamroun L, Hardy CM, Hempstead B, Jeames S, Mann M, Matin K, McCaskill-Stevens W, Merrill J, Nowakowski GS, Patel MI, Pressman A, Ramirez AG, Segura J, Segarra-Vasquez B, Hanley Williams J, Williams JE Jr, Winkfield KM, Yang ES, Zwicker V, Pierce LJ. J Clin Oncol. 2022 Jul 1;40(19):2163-2171. doi: 10.1200/JCO.22.00754. PMID: 35588469.
- Project Facilitate: Innovation and advancement in FDA’s oncology expanded access program. J Chan M, Antony R, Boehmer J, Kim I, Pazdur R. DOI: 10.1200/JCO.2022.40.16_suppl.e18556 Journal of Clinical Oncology 40, no. 16_suppl (June 01, 2022) e18556-e18556.
- Encouraging Rigorous Patient-Generated Data All Along the Drug Development Continuum. Bhatnagar V, Kluetz PG. JNCI: Journal of the National Cancer Institute, 2022; djac 129. July 28, 2022.
- Action for Increasing Diversity, Market Access, and Capacity in Oncology Registration Trials-Is Africa the Answer? Report From a Satellite Session of the Accelerating Anti-Cancer Agent Development and Validation Workshop. Kizub D, Manner CK, Graef K, Abubakar B, Orem J, Odedina F, Adeyeye MC, Nakigudde G, Ayalew K, Kalidas C, Lyerly HK, Norman T, Fashoyin-Aje L, Freedman J, Dent J, Cance B, Gralow J. JCO Glob Oncol. 2022 Jun;8:e2200117. doi: 10.1200/GO.22.00117. Erratum in: JCO Glob Oncol. 2022 Aug;8:e2200230. PMID: 35714309; PMCID: PMC9232363.
- Changes in Circulating Tumor DNA Reflect Clinical Benefit Across Multiple Studies of Patients With Non–Small-Cell Lung Cancer Treated With Immune Checkpoint Inhibitors. Beaver JA. Philip R. DOI: 10.1200/PO.21.00372 JCO Precision Oncology no. 6 (2022) e2100372. Published online August 11, 2022. PMID: 35952319.
- Summary of American Statistical Association Biopharmaceutical Section’s Virtual Discussion with Regulators on Time-to Event Endpoints in Cancer Trials in the Presence of Non-proportional Hazards. Sridhara R, Marchenko O, Jiang Q, et. al., Biopharmaceutical Report, Summer 2022, Vol. 29, No. 2, pp 42-45.
- A New Approach to Simplifying and Harmonizing Cancer Clinical Trials-Standardizing Eligibility Criteria. Gerber DE, Singh H, Larkins E, Ferris A, Forde PM, Selig W, Basu Roy U. JAMA Oncol. 2022 Sep 1;8(9):1333-1339. doi: 10.1001/jamaoncol.2022.1664. PMID: 35925576.
- Improving Dose-Optimization Processes Used in Oncology Drug Development to Minimize Toxicity and Maximize Benefit to Patients. Fourie Zirkelbach J, Shah M, Vallejo J, Cheng J, Ayyoub A, Liu J, Hudson R, Sridhara R, Ison G, Amiri-Kordestani L, Tang S, Gwise T, Rahman A, Pazdur R, Theoret MR. J Clin Oncol. 2022 Sep 12:JCO2200371. doi: 10.1200/JCO.22.00371. PMID: 36095296.
- Summary of ASA BIOP Section’s Virtual Discussion with Regulators on Consideration of Bayesian Approaches in Pediatric Cancer Clinical Trials. Rajeshwari Sridhara, Olga Marchenko, Qi Jang, et. al. Biopharmaceutical Report, Vol. 20, No. 3, Fall 2022 pp45-46.
- Summary of ASA BIOP Section’s Virtual Discussion with Regulators on Considerations for Data Monitoring Committee and Regulator Direct Interactions in Ongoing Randomized Cancer Clinical Trials. Olga Marchenko, Rajeshwari Sridhara, Qi Jang, et. al. Biopharmaceutical Report, Vol. 20, No. 3, Fall 2022 pp47-49.
- Assessing the needs of those who serve the underserved: A national survey among cancer care clinicians. Patel M, Merrill J, Smith K, Carrizosa D, Florez N, Fashoyin-Aje L, Gomez S, Giuliani M, Hinyard L, Hlubocky F, Jeames S, Kalwar T, Kamaraju S, Kircher S, Tap W. (2022). DOI: 10.1200/JCO.2022.40.28_suppl.175 Journal of Clinical Oncology 40, no. 28_suppl (October 01, 2022) 175-175.
- The Critical Role of Academic Clinical Trials in Pediatric Cancer Drug Approvals: Design, Conduct, and Fit for Purpose Data for Positive Regulatory Decisions. De Wilde B, Barry E, Fox E, Karres D, Kieran M, Manlay J, Ludwinski D, Reaman G, Kearns P. DOI: 10.1200/JCO.22.00033 Journal of Clinical Oncology 40, no. 29 (October 10, 2022) 3456-.
- The On- and Off-Ramps of Oncology Accelerated Approval. Fashoyin-Aje LA, Mehta GU, Beaver JA, Pazdur R. N Engl J Med. 2022 Oct 20;387(16):1439-1442. doi: 10.1056/NEJMp2208954. Epub 2022 Sep 21. PMID: 36129992.
- Journal of Clinical Oncology: Addressing Barriers to Clinical Trial Participation for Transgender People With Cancer to Improve Access and Generate Data. Ash B. Alpert, Jamie Renee Brewer, Spencer Adams, Lexis Rivers, Sunshine Orta, John R. Blosnich, Susanne Miedlich, Charles Kamen, Don S. Dizon, Richard Pazdur, Julia A. Beaver, and Lola Fashoyin-Aje. DOI: 10.1200/JCO.22.01174. E-Pub 27 October 2022. PMID: 36302204
- Accelerated approvals hit the target in precision oncology. Subbiah V, Wirth LJ, Kurzrock R, Pazdur R, Beaver JA, Singh H, Mehta GU. Nat Med. 2022 Oct;28(10):1976-1979. doi: 10.1038/s41591-022-01984-z. PMID: 36192554.
- Evaluation of Treatment Effect in Underrepresented Population in Cancer Trials: Discussion with International Regulators, Statistics in Biopharmaceutical Research. Sridhara R, Marchenko O, Jiang Qi, Barksdale E, Chen J, Dreyer N, Fashoyin-Aje L, Garrett-Mayer E, Gormley N, Gwise T, Hess L, Mandrekar S, Pignatti F, Rantell K, Raven A, Shen YL, Singh H, Tendler C, Theoret M, Pazdur R. Statistics in Biopharmaceutical Research. doi: 10.1080/19466315.2022.2128404. 31 Oct 2022.
- Proceedings From the ASCO/College of American Pathologists Immune Checkpoint Inhibitor Predictive Biomarker Summit. Hayes DF, Herbst RS, Myles JL, Topalian SL, Yohe SL, Aronson N, Bellizzi AM, Basu Roy U, Bradshaw G, Edwards RH, El-Gabry EA, Elvin J, Gajewski TF, McShane LM, Oberley M, Philip R, Rimm DL, Rosenbaum JN, Rubin EH, Schlager L, Sherwood SW, Stewart M, Taube JM, Thurin M, Vasalos P, Laser J. JCO Precis Oncol. 2022 Nov;6:e2200454. doi: 10.1200/PO.22.00454. PMID: 36446042.
- Real world treatment patterns among patients with newly diagnosed acute myeloid leukemia in the United States. Pulte D, Norsworthy KJ, Fernandes L, Kanapuru B, Lerro C, Rizvi F, Vallejo J, Wang K, Wynne J, Barcellos A, Belli AJ, Hansen E, Gwise T, Kluetz P, De Claro A, Wang CK, Rivera D. Blood 2022;Suppl 1 5100-02; November 15, 2022
- An FDA Oncology Perspective of Juvenile Toxicity Studies to Support Pediatric Drug Development. IN: Pediatric Cancer Therapeutics Development, Pediatric Oncology. Leighton JK, Helms W. J DiMartino, GH Reaman FO Smith eds. Springer Nature Switzerland AG 2022. Pp 25-30, November 19, 2022
- ACCELERATE Paediatric Strategy Forums: an advance for oncological drug development? Pearson ADJ, Rojas T, Karres D, Reaman G, Scobie N, Fox E, Lesa G, Ligas F, Norga K, Nysom K, Pappo A, Weigel B, Weiner S, Vassal G. Lancet Oncol. 2022 Nov;23(11):1354-1357. doi: 10.1016/S1470-2045(22)00619-2. PMID: 36328007.
- Highlights of FDA Oncology Approvals in 2022: Tissue-Agnostic Indications, Dosage Optimization, and Diversity in Drug Development. Duke ES, J Fusco M, DeMoss P, Dilawari A, Pamuk GE, Boehmer J, Mixter B, Goldberg KB, Kluetz P, Pazdur R. Cancer Discov. 2022 Dec 2;12(12):2739-2746. doi: 10.1158/2159-8290.CD-22-1185. PMID: 36458428.
- To Adjudicate or Not Adjudicate: That is the Question. Hicks KA, MD, Fashoyin-Aje L, Amiri-Kordestani L. J Am Coll Cardiol CardioOnc. 2022 Dec, 4 (5) 657–659
- Mobilizing the clinical trial ecosystem to drive adoption of master protocols. Bronson A, Chase MK, Fisher K, Millar D, Perlmutter J, Richardson N. Clin Trials. 2022 Dec;19(6):690-696. doi: 10.1177/17407745221110199. PMID: 36086812; PMCID: PMC9679560.
- Project Confirm: Accelerated Drug Approvals for Chronic Myeloid Leukemia. Sweet KL, Cortes JE, Apperley JF, Mann M, Mauro MJ, Oehler VG, Ruiz C, Schiffer CA, Ehrlich LA, Pamuk GE, Wynne J, Mehta GU, de Claro RA, Theoret MR, Smith BD, Norsworthy KJ. Clin Cancer Res. 2022 Dec 22:CCR-22-2628. doi: 10.1158/1078-0432.CCR-22-2628. PMID: 36547666.
- Use of Single-Arm Trials for US Food and Drug Administration Drug Approval in Oncology, 2002-2021. Agrawal S, Arora S, Amiri-Kordestani L, de Claro RA, Fashoyin-Aje L, Gormley N, Kim T, Lemery S, Mehta GU, Scott EC, Singh H, Tang S, Theoret MR, Pazdur R, Kluetz PG, Beaver JA. JAMA Oncol. 2022 Dec 29. doi: 10.1001/jamaoncol.2022.5985. PMID: 36580315.
Research Papers
- Radiographic read paradigms and the roles of the central imaging laboratory in neuro-oncology clinical trials. Ellingson BM, Brown MS, Boxerman JL, Gerstner ER, Kaufmann TJ, Cole PE, Bacha JA, Leung D, Barone A, Colman H, van den Bent MJ, Wen PY, Alfred Yung WK, Cloughesy TF, Goldin JG. Neuro Oncol. 2021 Feb 25;23(2):189-198. doi: 10.1093/neuonc/noaa253. PMID: 33130879; PMCID: PMC7906061.
- Does Knowledge of Treatment Assignment Affect Patient Report of Symptoms, Function, and Health Status? An Evaluation Using Multiple Myeloma Trials. Roydhouse JK, Mishra-Kalyani PS, Bhatnagar V, Gutman R, King-Kallimanis BL, Sridhara R, Kluetz PG. Value Health. 2021 Jun;24(6):822-829. doi: 10.1016/j.jval.2020.12.015. PMID: 34119080.
- Rapid real-world data analysis of patients with cancer, with and without COVID-19, across distinct health systems. Hwang C, Izano MA, Thompson MA, Gadgeel SM, Weese JL, Mikkelsen T, Schrag A, Teka M, Walters S, Wolf FM, Hirsch J, Rivera DR, Kluetz PG, Singh H, Brown TD. Cancer Rep (Hoboken). 2021 Oct;4(5):e1388. doi: 10.1002/cnr2.1388. PMID: 34014037; PMCID: PMC8209944.
- Model Development of CDK4/6 Predicted Efficacy in Patients With Hormone Receptor-Positive, Human Epidermal Growth Factor Receptor 2-Negative Advanced or Metastatic Breast Cancer. Mason J, Gong Y, Amiri-Kordestani L, Wedam S, Gao JJ, Prowell TM, Singh H, Amatya A, Tang S, Pazdur R, Kuhn P, Blumenthal GM, Beaver JA. JCO Clin Cancer Inform. 2021 Jun;5:758-767. doi: 10.1200/CCI.21.00025. PMID: 34297598.
- Patient-Reported Outcomes After Treatment Discontinuation: Commercial Clinical Trial Data From Four Cancer Types. Value Health. King-Kallimanis BL, Lederer NM, Kim J, Nair A, Horodniceanu E, Bhatnagar V, Kluetz PG. 2021 Sep;24(9):1302-1307. doi: 10.1016/j.jval.2021.04.1279. PMID: 34452710.
- Survival outcomes in older men with non-metastatic castration-resistant prostate cancer treated with androgen receptor inhibitors: a US Food and Drug Administration pooled analysis of patient-level data from three randomised trials. Fallah J, Zhang L, Amatya A, Gong Y, King-Kallimanis B, Bhatnagar V, Weinstock C, Suzman DL, Agrawal S, Chang E, Anscher MS, Chi DC, Xu JX, Brewer JR, Brave MH, Hadadi M, Theoret MR, Kluetz PG, Goldberg KB, Ibrahim A, Tang S, Pazdur R, Beaver JA, Amiri-Kordestani L, Singh H. Lancet Oncol. 2021 Sep;22(9):1230-1239. doi: 10.1016/S1470-2045(21)00334-X. PMID: 34310904.
- Review of patient-reported outcomes in multiple myeloma registrational trials: highlighting areas for improvement. Fernandes LL, Zhou J, Kanapuru B, Horodniceanu E, Gwise T, Kluetz PG, Bhatnagar V. Blood Cancer J. 2021 Aug 31;11(8):148. doi: 10.1038/s41408-021-00543-y. PMID: 34465728; PMCID: PMC8408214.
- Arterial aneurysm and dissection with systemic vascular endothelial growth factor inhibitors: A review of cases reported to the FDA Adverse Event Reporting System and published in the literature. Cheng C, Nguyen MN, Nayernama A, Jones SC, Brave M, Agrawal S, Amiri-Kordestani L, Woronow D. Vasc Med. 2021 Oct;26(5):526-534. doi: 10.1177/1358863X211006470. PMID: 33840328.
- Floor and ceiling effects in the EORTC QLQ-C30 Physical Functioning Subscale among patients with advanced or metastatic breast cancer. Murugappan MN, King-Kallimanis BL, Mangir C, Howie L, Bhatnagar V, Beaver JA, Basch EM, Henson SR, Kluetz PG. Cancer. 2021 Oct 11. doi: 10.1002/cncr.33959. PMID: 34634139.
- Overall survival in patients with hormone receptor-positive, HER2-negative, advanced or metastatic breast cancer treated with a cyclin-dependent kinase 4/6 inhibitor plus fulvestrant: a US Food and Drug Administration pooled analysis. Gao JJ, Cheng J, Prowell TM, Bloomquist E, Tang S, Wedam SB, Royce M, Krol D, Osgood C, Ison G, Sridhara R, Pazdur R, Beaver JA, Amiri-Kordestani L. Lancet Oncol. 2021 Nov;22(11):1573-1581. doi: 10.1016/S1470-2045(21)00472-1. Erratum in: Lancet Oncol. 2021 Nov;22(11):e472. PMID: 34656225.
- Treatment-related toxicity and improved outcome from immunotherapy in hepatocellular cancer: Evidence from an FDA pooled analysis of landmark clinical trials with validation from routine practice. Pinato DJ, Marron TU, Mishra-Kalyani PS, Gong Y, Wei G, Szafron D, Sharon E, Saeed A, Jun T, Dharmapuri S, Naqash AR, Peeraphatdit T, Gampa A, Wang Y, Khan U, Muzaffar M, Navaid M, Lee CJ, Lee PC, Bulumulle A, Yu B, Paul S, Nimkar N, Bettinger D, Hildebrand H, Abugabal YI, Pressiani T, Personeni N, D'Alessio A, Kaseb AO, Huang YH, Ang C, Schneider J, Pillai A, Rimassa L, Goldberg KB, Pazdur R, Theoret M, Lemery S, Fashoyin-Aje ', Cortellini A, Pelosof L. Eur J Cancer. 2021 Nov;157:140-152. doi: 10.1016/j.ejca.2021.08.020. PMID: 34508996.
- Response Rate, Event-Free Survival, and Overall Survival in Newly Diagnosed Acute Myeloid Leukemia: US Food and Drug Administration Trial-Level and Patient-Level Analyses. Norsworthy KJ, Gao X, Ko CW, Pulte ED, Zhou J, Gong Y, Shen YL, Vallejo J, Gwise TE, Sridhara R, Deisseroth AB, Farrell AT, de Claro RA, Blumenthal GM, Pazdur R. J Clin Oncol. 2021 Dec 10:JCO2101548. doi: 10.1200/JCO.21.01548. PMID: 34890212.
- Toward best practice in cancer mutation detection with whole-genome and whole-exome sequencing. Xiao W, Ren L, Chen Z, Fang LT, Zhao Y, Lack J, Guan M, Zhu B, Jaeger E, Kerrigan L, Blomquist TM, Hung T, Sultan M, Idler K, Lu C, Scherer A, Kusko R, Moos M, Xiao C, Sherry ST, Abaan OD, Chen W, Chen X, Nordlund J, Liljedahl U, Maestro R, Polano M, Drabek J, Vojta P, Kõks S, Reimann E, Madala BS, Mercer T, Miller C, Jacob H, Truong T, Moshrefi A, Natarajan A, Granat A, Schroth GP, Kalamegham R, Peters E, Petitjean V, Walton A, Shen TW, Talsania K, Vera CJ, Langenbach K, de Mars M, Hipp JA, Willey JC, Wang J, Shetty J, Kriga Y, Raziuddin A, Tran B, Zheng Y, Yu Y, Cam M, Jailwala P, Nguyen C, Meerzaman D, Chen Q, Yan C, Ernest B, Mehra U, Jensen RV, Jones W, Li JL, Papas BN, Pirooznia M, Chen YC, Seifuddin F, Li Z, Liu X, Resch W, Wang J, Wu L, Yavas G, Miles C, Ning B, Tong W, Mason CE, Donaldson E, Lababidi S, Staudt LM, Tezak Z, Hong H, Wang C, Shi L. Nat Biotechnol. 2021 Sep;39(9):1141-1150. doi: 10.1038/s41587-021-00994-5. Epub 2021 Sep 9. PMID: 34504346; PMCID: PMC8506910.
- Whole genome and exome sequencing reference datasets from a multi-center and cross-platform benchmark study. Zhao Y, Fang LT, Shen TW, Choudhari S, Talsania K, Chen X, Shetty J, Kriga Y, Tran B, Zhu B, Chen Z, Chen W, Wang C, Jaeger E, Meerzaman D, Lu C, Idler K, Ren L, Zheng Y, Shi L, Petitjean V, Sultan M, Hung T, Peters E, Drabek J, Vojta P, Maestro R, Gasparotto D, Kõks S, Reimann E, Scherer A, Nordlund J, Liljedahl U, Foox J, Mason CE, Xiao C, Hong H, Xiao W. Sci Data. 2021 Nov 9;8(1):296. doi: 10.1038/s41597-021-01077-5. PMID: 34753956; PMCID: PMC8578599.
Approval Summaries
- Oncology approvals in 2020: a year of firsts in the midst of a pandemic. Amiri-Kordestani L, Pazdur R. Nat Rev Clin Oncol. 2021 Mar;18(3):129-130. doi: 10.1038/s41571-021-00477-1. PMID: 33514911; PMCID: PMC7844812.
- FDA Approval Summary: Rucaparib for the Treatment of Patients with Deleterious BRCA-Mutated Metastatic Castrate-Resistant Prostate Cancer. Anscher MS, Chang E, Gao X, Gong Y, Weinstock C, Bloomquist E, Adeniyi O, Charlab R, Zimmerman S, Serlemitsos-Day M, Ning YM, Mayrosh R, Fuller B, Trentacosti AM, Gallagher P, Bijwaard K, Philip R, Ghosh S, Fahnbulleh F, Diggs F, Arora S, Goldberg KB, Tang S, Amiri-Kordestani L, Pazdur R, Ibrahim A, Beaver JA. Oncologist. 2021 Feb;26(2):139-146. doi: 10.1002/onco.13585. PMID: 33145877; PMCID: PMC7873319.
- FDA Approval Summary: Lurbinectedin for the Treatment of Metastatic Small Cell Lung Cancer. Singh S, Jaigirdar AA, Mulkey F, Cheng J, Hamed SS, Li Y, Liu J, Zhao H, Goheer A, Helms WS, Wang X, Agarwal R, Pragani R, Korsah K, Tang S, Leighton J, Rahman A, Beaver JA, Pazdur R, Theoret MR, Singh H. Clin Cancer Res. 2021 May 1;27(9):2378-2382. doi: 10.1158/1078-0432.CCR-20-3901. PMID: 33288660.
- FDA Approval Summary: Olaparib Monotherapy or in Combination with Bevacizumab for the Maintenance Treatment of Patients with Advanced Ovarian Cancer. Arora S, Balasubramaniam S, Zhang H, Berman T, Narayan P, Suzman D, Bloomquist E, Tang S, Gong Y, Sridhara R, Turcu FR, Chatterjee D, Saritas-Yildirim B, Ghosh S, Philip R, Pathak A, Gao JJ, Amiri-Kordestani L, Pazdur R, Beaver JA. Oncologist. 2021 Jan;26(1):e164-e172. doi: 10.1002/onco.13551. PMID: 33017510; PMCID: PMC7794199.
- U.S. FDA Drug Approvals for Gynecological Malignancies: A Decade in Review. Arora S, Narayan P, Ison G, Berman T, Suzman DL, Wedam S, Prowell TM, Ghosh S, Philip R, Osgood CL, Gao JJ, Shah M, Krol D, Wahby S, Royce M, Brus C, Bloomquist EW, Fiero MH, Tang S, Pazdur R, Ibrahim A, Amiri-Kordestani L, Beaver JA. Clin Cancer Res. 2021 Oct 28. doi: 10.1158/1078-0432.CCR-21-2599. PMID: 34711631.
- U.S. FDA Drug Approvals for Breast Cancer - A Decade in Review. Arora S, Narayan P, Osgood CL, Wedam S, Prowell TM, Gao JJ, Shah M, Krol D, Wahby S, Royce M, Ghosh S, Philip R, Ison G, Berman T, Brus C, Bloomquist EW, Fiero MH, Tang S, Pazdur R, Ibrahim A, Amiri-Kordestani L, Beaver JA. Clin Cancer Res. 2021 Oct 28:clincanres.2600.2021. doi: 10.1158/1078-0432.CCR-21-2600. PMID: 34711632.
- FDA Approval Summary: Selpercatinib for the Treatment of Lung and Thyroid Cancers with RET Gene Mutations or Fusions. Bradford D, Larkins E, Mushti SL, Rodriguez L, Skinner AM, Helms WS, Price LSL, Zirkelbach JF, Li Y, Liu J, Charlab R, Turcu FR, Liang D, Ghosh S, Roscoe D, Philip R, Zack-Taylor A, Tang S, Kluetz PG, Beaver JA, Pazdur R, Theoret MR, Singh H. Clin Cancer Res. 2021 Apr 15;27(8):2130-2135. doi: 10.1158/1078-0432.CCR-20-3558. PMID: 33239432.
- FDA Approval Summary: Atezolizumab Plus Bevacizumab for the Treatment of Patients with Advanced Unresectable or Metastatic Hepatocellular Carcinoma. Casak SJ, Donoghue M, Fashoyin-Aje L, Jiang X, Rodriguez L, Shen YL, Xu Y, Jiang X, Liu J, Zhao H, Pierce WF, Mehta S, Goldberg KB, Theoret MR, Kluetz PG, Pazdur R, Lemery SJ. Clin Cancer Res. 2021 Apr 1;27(7):1836-1841. doi: 10.1158/1078-0432.CCR-20-3407. PMID: 33139264.
- FDA Approval Summary: Pembrolizumab for the First-line Treatment of Patients with MSI-H/dMMR Advanced Unresectable or Metastatic Colorectal Carcinoma. Casak SJ, Marcus L, Fashoyin-Aje L, Mushti SL, Cheng J, Shen YL, Pierce WF, Her L, Goldberg KB, Theoret MR, Kluetz PG, Pazdur R, Lemery SJ. Clin Cancer Res. 2021 Sep 1;27(17):4680-4684. doi: 10.1158/1078-0432.CCR-21-0557. PMID: 33846198; PMCID: PMC8416693.
- FDA Approval Summary: Selumetinib for Plexiform Neurofibroma. Casey D, Demko S, Sinha A, Mishra-Kalyani PS, Shen YL, Khasar S, Goheer MA, Helms WS, Pan L, Xu Y, Fan J, Leong R, Liu J, Yang Y, Windsor K, Ou M, Stephens O, Oh B, Reaman GH, Nair A, Shord SS, Bhatnagar V, Daniels SR, Sickafuse S, Goldberg KB, Theoret MR, Pazdur R, Singh H. Clin Cancer Res. 2021 Aug 1;27(15):4142-4146. doi: 10.1158/1078-0432.CCR-20-5032. PMID: 33712511.
- FDA Approval Summary: Enfortumab Vedotin for Locally Advanced or Metastatic Urothelial Carcinoma. Chang E, Weinstock C, Zhang L, Charlab R, Dorff SE, Gong Y, Hsu V, Li F, Ricks TK, Song P, Tang S, Waldron PE, Yu J, Zahalka E, Goldberg KB, Pazdur R, Theoret MR, Ibrahim A, Beaver JA. Clin Cancer Res. 2021 Feb 15;27(4):922-927. doi: 10.1158/1078-0432.CCR-20-2275. PMID: 32962979.
- FDA Approval Summary: Tivozanib for Relapsed or Refractory Renal Cell Carcinoma. Chang E, Weinstock C, Zhang L, Fiero MH, Zhao M, Zahalka E, Ricks TK, Fourie Zirkelbach J, Qiu J, Yu J, Chen XH, Bhatnagar V, Goldberg KB, Tang S, Kluetz PG, Pazdur R, Ibrahim A, Beaver JA, Amiri-Kordestani L. Clin Cancer Res. 2021 Aug 20. doi: 10.1158/1078-0432.CCR-21-2334. PMID: 34417198.
- FDA Approval Summary: Pertuzumab, Trastuzumab, and Hyaluronidase-zzxf Injection for Subcutaneous Use in Patients with HER2-positive Breast Cancer. Gao JJ, Osgood CL, Gong Y, Zhang H, Bloomquist EW, Jiang X, Qiu J, Yu J, Song P, Rahman NA, Chiu HJ, Ricks TK, Rizvi F, Hou S, Wilson W, Abukhdeir AM, Seidman J, Ghosh S, Philip R, Pierce WF, Bhatnagar V, Kluetz PG, Pazdur R, Beaver JA, Amiri-Kordestani L. Clin Cancer Res. 2021 Apr 15;27(8):2126-2129. doi: 10.1158/1078-0432.CCR-20-3474. PMID: 33188141.
- FDA Approval Summary: Selinexor for Relapsed or Refractory Diffuse Large B-Cell Lymphoma. Kasamon YL, Price LSL, Okusanya OO, Richardson NC, Li RJ, Ma L, Wu YT, Theoret M, Pazdur R, Gormley NJ. Oncologist. 2021 Oct;26(10):879-886. doi: 10.1002/onco.13859. PMID: 34132444; PMCID: PMC8488790.
- FDA Approval Summary: Pralsetinib for the Treatment of Lung and Thyroid Cancers With RET Gene Mutations or Fusions. Kim J, Bradford D, Larkins E, Pai-Scherf LH, Chatterjee S, Mishra-Kalyani PS, Wearne E, Helms WS, Ayyoub A, Bi Y, Sun J, Charlab R, Liu J, Zhao H, Liang D, Ghosh S, Philip R, Pazdur R, Theoret MR, Beaver JA, Singh H. Clin Cancer Res. 2021 Oct 15;27(20):5452-5456. doi: 10.1158/1078-0432.CCR-21-0967. PMID: 34045295.
- FDA Approval Summary: Osimertinib for Adjuvant Treatment of Surgically Resected Non-Small Cell Lung Cancer, a Collaborative Project Orbis Review. Koch AL, Vellanki PJ, Drezner N, Li X, Mishra-Kalyani PS, Shen YL, Xia H, Li Y, Liu J, Zirkelbach JF, Palazov E, Gamarian A, Choo Q, Girčys A, Rohr UP, Fesenko N, Spillman D, Pazdur R, Beaver JA, Singh H. Clin Cancer Res. 2021 Jul 22. doi: 10.1158/1078-0432.CCR-21-1034. PMID: 34301748.
- FDA Approval Summary: Entrectinib for the Treatment of NTRK gene Fusion Solid Tumors. Marcus L, Donoghue M, Aungst S, Myers CE, Helms WS, Shen G, Zhao H, Stephens O, Keegan P, Pazdur R. Clin Cancer Res. 2021 Feb 15;27(4):928-932. doi: 10.1158/1078-0432.CCR-20-2771. PMID: 32967940.
- FDA Approval Summary: Pembrolizumab for the Treatment of Tumor Mutational Burden-High Solid Tumors. Marcus L, Fashoyin-Aje LA, Donoghue M, Yuan M, Rodriguez L, Gallagher PS, Philip R, Ghosh S, Theoret MR, Beaver JA, Pazdur R, Lemery SJ. Clin Cancer Res. 2021 Sep 1;27(17):4685-4689. doi: 10.1158/1078-0432.CCR-21-0327. PMID: 34083238; PMCID: PMC8416776.
- FDA Approval Summary: Capmatinib and Tepotinib for the Treatment of Metastatic NSCLC Harboring MET Exon 14 Skipping Mutations or Alterations. Mathieu LN, Larkins E, Akinboro O, Roy P, Amatya AK, Fiero MH, Mishra-Kalyani PS, Helms WS, Myers CE, Skinner AM, Aungst S, Jin R, Zhao H, Xia H, Zirkelbach JF, Bi Y, Li Y, Liu J, Grimstein M, Zhang X, Woods S, Reece K, Abukhdeir AM, Ghosh S, Philip R, Tang S, Goldberg KB, Pazdur R, Beaver JA, Singh H. Clin Cancer Res. 2021 Aug 3. doi: 10.1158/1078-0432.CCR-21-1566. PMID: 34344795.
- FDA Approval Summary: Atezolizumab and Durvalumab in Combination with Platinum-Based Chemotherapy in Extensive Stage Small Cell Lung Cancer. Mathieu L, Shah S, Pai-Scherf L, Larkins E, Vallejo J, Li X, Rodriguez L, Mishra-Kalyani P, Goldberg KB, Kluetz PG, Theoret MR, Beaver JA, Pazdur R, Singh H. Oncologist. 2021 May;26(5):433-438. doi: 10.1002/onco.13752. PMID: 33687763; PMCID: PMC8100557.
- FDA Supplemental Approval Summary: Lenvatinib for the Treatment of Unresectable Hepatocellular Carcinoma. Nair A, Reece K, Donoghue MB, Yuan WV, Rodriguez L, Keegan P, Pazdur R. Oncologist. 2021 Mar;26(3):e484-e491. doi: 10.1002/onco.13566. PMID: 33044793; PMCID: PMC7930400.
- FDA Approval Summary: Nivolumab in Combination with Ipilimumab for the Treatment of Unresectable Malignant Pleural Mesothelioma. Nakajima EC, Vellanki PJ, Larkins E, Chatterjee S, Mishra-Kalyani PS, Bi Y, Qosa H, Liu J, Zhao H, Biable M, Hotaki LT, Shen YL, Pazdur R, Beaver JA, Singh H, Donoghue M. Clin Cancer Res. 2021 Aug 30. doi: 10.1158/1078-0432.CCR-21-1466. PMID: 34462287.
- FDA Approval Summary: Sotorasib for KRAS G12C Mutated Metastatic NSCLC. Nakajima EC, Drezner N, Li X, Mishra-Kalyani PS, Liu Y, Zhao H, Bi Y, Liu J, Rahman A, Wearne E, Ojofeitimi I, Hotaki LT, Spillman D, Pazdur R, Beaver JA, Singh H. Clin Cancer Res. 2021 Dec 13:clincanres.3074.2021. doi: 10.1158/1078-0432.CCR-21-3074. PMID: 34903582.
- FDA Approval Summary: Fam-Trastuzumab Deruxtecan-Nxki for the Treatment of Unresectable or Metastatic HER2-Positive Breast Cancer. Narayan P, Osgood CL, Singh H, Chiu HJ, Ricks TK, Chiu Yuen Chow E, Qiu J, Song P, Yu J, Namuswe F, Guiterrez-Lugo M, Hou S, Pierce WF, Goldberg KB, Tang S, Amiri-Kordestani L, Theoret MR, Pazdur R, Beaver JA. Clin Cancer Res. 2021 Aug 15;27(16):4478-4485. doi: 10.1158/1078-0432.CCR-20-4557. PMID: 33753456; PMCID: PMC8570919.
- FDA Approval Summary: Alpelisib Plus Fulvestrant for Patients with HR-positive, HER2-negative, PIK3CA-mutated, Advanced or Metastatic Breast Cancer. Narayan P, Prowell TM, Gao JJ, Fernandes LL, Li E, Jiang X, Qiu J, Fan J, Song P, Yu J, Zhang X, King-Kallimanis BL, Chen W, Ricks TK, Gong Y, Wang X, Windsor K, Rhieu SY, Geiser G, Banerjee A, Chen X, Reyes Turcu F, Chatterjee DK, Pathak A, Seidman J, Ghosh S, Philip R, Goldberg KB, Kluetz PG, Tang S, Amiri-Kordestani L, Theoret MR, Pazdur R, Beaver JA. Clin Cancer Res. 2021 Apr 1;27(7):1842-1849. doi: 10.1158/1078-0432.CCR-20-3652. PMID: 33168657; PMCID: PMC8535764.
- Benefit-Risk Summary of Nivolumab for the Treatment of Patients with Unresectable Advanced, Recurrent, or Metastatic Esophageal Squamous Cell Carcinoma After Prior Fluoropyrimidine- and Platinum-Based Chemotherapy. Pelosof L, Saung MT, Donoghue M, Casak S, Mushti S, Cheng J, Jiang X, Liu J, Zhao H, Khazraee M, Goldberg KB, Theoret M, Lemery S, Pazdur R, Fashoyin-Aje L. Oncologist. 2021 Apr;26(4):318-324. doi: 10.1002/onco.13646. PMID: 33345396; PMCID: PMC8018317.
- FDA Approval Summary: Gilteritinib for Relapsed or Refractory Acute Myeloid Leukemia with a FLT3 Mutation. Pulte ED, Norsworthy KJ, Wang Y, Xu Q, Qosa H, Gudi R, Przepiorka D, Fu W, Okusanya OO, Goldberg KB, De Claro RA, Farrell AT, Pazdur R. Clin Cancer Res. 2021 Jul 1;27(13):3515-3521. doi: 10.1158/1078-0432.CCR-20-4271. PMID: 33632926; PMCID: PMC8506653.
- FDA Approval Summary: Margetuximab plus chemotherapy for advanced or metastatic HER2-positive breast cancer. Royce M, Osgood CL, Amatya AK, Fiero MH, Chang CJG, Ricks TK, Shetty KA, Kraft J, Qiu J, Song P, Charlab R, Yu J, King KE, Rastogi A, Janelsins B, Weinberg WC, Clouse K, Borders-Hemphill V, Brown L, Gomez-Broughton C, Li Z, Nguyen TT, Qiu Z, Ly AT, Chang S, Gao T, Tu CM, King-Kallimanis B, Pierce WF, Chiang K, Lee C, Goldberg KB, Leighton JK, Tang S, Pazdur R, Beaver JA, Amiri-Kordestani L. Clin Cancer Res. 2021 Dec 16:clincanres.3247.2021. doi: 10.1158/1078-0432.CCR-21-3247. PMID: 34916216.
- FDA Approval Summary: Nivolumab Plus Ipilimumab for the Treatment of Patients with Hepatocellular Carcinoma Previously Treated with Sorafenib. Saung MT, Pelosof L, Casak S, Donoghue M, Lemery S, Yuan M, Rodriguez L, Schotland P, Chuk M, Davis G, Goldberg KB, Theoret MR, Pazdur R, Fashoyin-Aje L. Oncologist. 2021 Sep;26(9):797-806. doi: 10.1002/onco.13819. PMID: 33973307; PMCID: PMC8417871.
- FDA Approval Summary: Tucatinib for the Treatment of Patients with Advanced or Metastatic HER2-positive Breast Cancer. Shah M, Wedam S, Cheng J, Fiero MH, Xia H, Li F, Fan J, Zhang X, Yu J, Song P, Chen W, Ricks TK, Chen XH, Goldberg KB, Gong Y, Pierce WF, Tang S, Theoret MR, Pazdur R, Amiri-Kordestani L, Beaver JA. Clin Cancer Res. 2021 Mar 1;27(5):1220-1226. doi: 10.1158/1078-0432.CCR-20-2701. PMID: 33055172.
- FDA Approval Summary: Nivolumab with Ipilimumab and Chemotherapy for Metastatic Non-small Cell Lung Cancer, A Collaborative Project Orbis Review. Vellanki PJ, Mulkey F, Jaigirdar AA, Rodriguez L, Wang Y, Xu Y, Zhao H, Liu J, Howe G, Wang J, Choo Q, Golding SJ, Mansell V, Korsah K, Spillman D, de Claro RA, Pazdur R, Beaver JA, Singh H. Clin Cancer Res. 2021 Jul 1;27(13):3522-3527. doi: 10.1158/1078-0432.CCR-20-4338. Epub 2021 Feb 25. PMID: 33632925; PMCID: PMC8254731.
- FDA Approval Summary: Accelerated Approval of Sacituzumab Govitecan-hziy for Third-line Treatment of Metastatic Triple-negative Breast Cancer. Wahby S, Fashoyin-Aje L, Osgood CL, Cheng J, Fiero MH, Zhang L, Tang S, Hamed SS, Song P, Charlab R, Dorff SE, Ricks TK, Barnett-Ringgold K, Dinin J, Goldberg KB, Theoret MR, Pazdur R, Amiri-Kordestani L, Beaver JA. Clin Cancer Res. 2021 Apr 1;27(7):1850-1854. doi: 10.1158/1078-0432.CCR-20-3119. Epub 2020 Nov 9. PMID: 33168656.
Regulatory Perspectives
- Subgroup Analyses in Oncology Trials: Regulatory Considerations and Case Examples. Amatya AK, Fiero MH, Bloomquist EW, Sinha AK, Lemery SJ, Singh H, Ibrahim A, Donoghue M, Fashoyin-Aje LA, de Claro RA, Gormley NJ, Amiri-Kordestani L, Sridhara R, Theoret MR, Kluetz PG, Pazdur R, Beaver JA, Tang S. Clin Cancer Res. 2021 Nov 1;27(21):5753-5756. doi: 10.1158/1078-0432.CCR-20-4912. PMID: 34117032; PMCID: PMC8563387.
- Minimal Residual Disease in Myeloma: Application for Clinical Care and New Drug Registration. Anderson KC, Auclair D, Adam SJ, Agarwal A, Anderson M, Avet-Loiseau H, Bustoros M, Chapman J, Connors DE, Dash A, Di Bacco A, Du L, Facon T, Flores-Montero J, Gay F, Ghobrial IM, Gormley NJ, Gupta I, Higley H, Hillengass J, Kanapuru B, Kazandjian D, Kelloff GJ, Kirsch IR, Kremer BE, Landgren O, Lightbody ED, Lomas OC, Lonial S, Mateos MV, Montes de Oca R, Mukundan L, Munshi NC, O'Donnell EK, Orfao A, Paiva B, Patel R, Pugh TJ, Ramasamy K, Ray J, Roshal M, Ross JA, Sigman CC, Thoren KL, Trudel S, Ulaner GA, Valente N, Weiss BM, Zamagni E, Kumar SK. Clin Cancer Res. 2021 Jul 28:clincanres.1059.2021. doi: 10.1158/1078-0432.CCR-21-1059. PMID: 34321279.
- "Dangling" Accelerated Approvals in Oncology. Beaver JA, Pazdur R. N Engl J Med. 2021 May 6;384(18):e68. doi: 10.1056/NEJMp2104846. PMID: 33882220.
- The Wild West of Checkpoint Inhibitor Development. Beaver JA, Pazdur R. N Engl J Med. 2021 Dec 15. doi: 10.1056/NEJMp2116863. PMID: 34910860.
- Refining neoadjuvant therapy clinical trial design for muscle-invasive bladder cancer before cystectomy: a joint US Food and Drug Administration and Bladder Cancer Advocacy Network workshop. Chang E, Apolo AB, Bangs R, Chisolm S, Duddalwar V, Efstathiou JA, Goldberg KB, Hansel DE, Kamat AM, Kluetz PG, Lerner SP, Plimack E, Prowell T, Singh H, Suzman D, Yu EY, Zhang H, Beaver JA, Pazdur R, Weinstock C, Galsky MD. Nat Rev Urol. 2021 Sep 10. doi: 10.1038/s41585-021-00505-w. Epub ahead of print. PMID: 34508246.
- Systematic Review of PD-1/PD-L1 Inhibitors in Oncology: From Personalized Medicine to Public Health. Chang E, Pelosof L, Lemery S, Gong Y, Goldberg KB, Farrell AT, Keegan P, Veeraraghavan J, Wei G, Blumenthal GM, Amiri-Kordestani L, Singh H, Fashoyin-Aje L, Gormley N, Kluetz PG, Pazdur R, Beaver JA, Theoret MR. Oncologist. 2021 Oct;26(10):e1786-e1799. doi: 10.1002/onco.13887. PMID: 34196068; PMCID: PMC8488782.
- U.S. Food and Drug Administration: Initial Experience with the Real-Time Oncology Review Program. de Claro RA, Gao JJ, Kim T, Kluetz PG, Theoret MR, Beaver JA, Pazdur R. Clin Cancer Res. 2021 Jan 1;27(1):11-14. doi: 10.1158/1078-0432.CCR-20-2220. PMID: 32816899.
- Intercontinental collaboration in clinical trials for children and adolescents with cancer—A systematic review by ACCELERATE. De Rojas T, Pearson AJ, Scobie N, Knox L, Wariabharaj D, Kearns P, Vassal G, Reaman G. Cancer Med. 2021; 10: 8462– 8474. doi:10.1002/cam4.4356
- Promoting Inclusion of Members of Racial and Ethnic Minority Groups in Cancer Drug Development. Fashoyin-Aje L, Beaver JA, Pazdur R. JAMA Oncol. 2021 Oct 1;7(10):1445-1446. doi: 10.1001/jamaoncol.2021.2137. PMID: 34264279.
- Rethinking Cancer Clinical Trial Conduct Induced by COVID-19: An Academic Center, Industry, Government, and Regulatory Agency Perspective. Flaherty KT, Doroshow JH, Galbraith S, Ribas A, Kluetz PG, Pazdur R, Theoret MR. Cancer Discov. 2021 Aug;11(8):1881-1885. doi: 10.1158/2159-8290.CD-21-0850. PMID: 34290074; PMCID: PMC8340848.
- Bringing safe and effective therapies to premenopausal women with breast cancer: efforts to broaden eligibility criteria. Gao JJ, Krol D, Narayan P, Cardoso F, Regan MM, Goetz MP, Hurvitz SA, Mauro L, Hodgdon C, Miller CP, Booth B, Bloomquist E, Ison G, Osgood C, Bhatnagar V, Fashoyin-Aje L, Pazdur R, Amiri-Kordestani L, Beaver JA. Ann Oncol. 2021 Aug;32(8):950-953. doi: 10.1016/j.annonc.2021.05.356. Epub 2021 May 13. PMID: 33991601.
- FDA Oncology Center of Excellence During COVID-19-Working for Patients With Cancer. Gao JJ, Pazdur R. JAMA Oncol. 2020 Dec 23. doi: 10.1001/jamaoncol.2020.6783. Epub ahead of print. PMID: 33355627.
- Recommendations on eliminating racial disparities in multiple myeloma therapies: a step toward achieving equity in healthcare.Gormley N, Fashoyin-Aje L, Locke T, Unger J, Little RF, Nooka A, Mezzi K, Popa-McKiver M, Kobos R, Biru Y, Williams TH, Anderson KC. Blood Cancer Discov. 2021 Mar;2(2):119-124. doi: 10.1158/2643-3230.bcd-20-0123. PMID: 34179821; PMCID: PMC8224821.
- US Food and Drug Administration Support for Oncology Drug Development During COVID-19. Kadakia KT, Pazdur R, Shah A. JAMA Oncol. 2021 Jan 1;7(1):27-28. doi: 10.1001/jamaoncol.2020.4975. PMID: 33030504.
- Continuing to Broaden Eligibility Criteria to Make Clinical Trials More Representative and Inclusive: ASCO-Friends of Cancer Research Joint Research Statement. Kim ES, Uldrick TS, Schenkel C, Bruinooge SS, Harvey RD, Magnuson A, Spira A, Wade JL, Stewart MD, Vega DM, Beaver JA, Denicoff AM, Ison G, Ivy SP, George S, Perez RP, Spears PA, Tap WD, Schilsky RL. Clin Cancer Res. 2021 May 1;27(9):2394-2399. doi: 10.1158/1078-0432.CCR-20-3852. PMID: 33563632.
- The FDA's patient-focused drug development initiative. Kluetz PG, Bhatnagar V. Clin Adv Hematol Oncol. 2021 Feb;19(2):70-72. PMID: 33596186.
- FDA Oncology Center of Excellence Project Renewal: Engaging the Oncology Community to Update Product Labeling for Older Oncology Drugs. Kluetz PG, Keegan P, Demetri GD, Thornton K, Sul J, Kim J, Katzen H, Burke LB, Harvey RD, Alebachew E, Agrawal S, Nair A, Donoghue M, Pierce WF, Shord SS, Gao JJ, Pazdur R. Clin Cancer Res. 2021 Feb 15;27(4):916-921. doi: 10.1158/1078-0432.CCR-20-3213. PMID: 33257426.
- Advancing Assessment, Analysis, and Reporting of Safety and Tolerability in Cancer Trials. Kluetz PG, King-Kallimanis BL, Suzman D, Chang E, Brave MM, Weinstock C, Bhatnagar V, Beaver JA, Chuk MK. J Natl Cancer Inst. 2021 May 4;113(5):507-508. doi: 10.1093/jnci/djaa135. PMID: 32857840; PMCID: PMC8096361.
- Report of National Brain Tumor Society roundtable workshop on innovating brain tumor clinical trials: building on lessons learned from COVID-19 experience. Lee EQ, Selig W, Meehan C, Bacha J, Barone A, Bloomquist E, Chang SM, de Groot JF, Galanis E, Hassan I, Kalidas C, Khasraw M, Kvedar JC, Lassman AB, Puduvalli V, Sahebjam S, Schwamm LH, Tamir S, Welch M, Yung WKA, Zadeh G, Arons D, Wen PY. Neuro Oncol. 2021 Aug 2;23(8):1252-1260. doi: 10.1093/neuonc/noab082. PMID: 33822177; PMCID: PMC8083574.
- Roadmap to 2030 for Drug Evaluation in Older Adults. Liu Q, Schwartz JB, Slattum PW, Lau SWJ, Guinn D, Madabushi R, Burckart G, Califf R, Cerreta F, Cho C, Cook J, Gamerman J, Goldsmith P, van der Graaf PH, Gurwitz JH, Haertter S, Hilmer S, Huang SM, Inouye SK, Kanapuru B, Pirmohamed M, Posner P, Radziszewska B, Keipp Talbot H, Temple R. Clin Pharmacol Ther. 2021 Oct 16. doi: 10.1002/cpt.2452. PMID: 34656074.
- Modernizing Clinical Trial Eligibility Criteria: Recommendations of the ASCO-Friends of Cancer Research Performance Status Work Group. Magnuson A, Bruinooge SS, Singh H, Wilner KD, Jalal S, Lichtman SM, Kluetz PG, Lyman GH, Klepin HD, Fleury ME, Hirsch B, Melemed A, Arnaldez FI, Basu Roy U, Schenkel C, Sherwood S, Garrett-Mayer E. Clin Cancer Res. 2021 May 1;27(9):2424-2429. doi: 10.1158/1078-0432.CCR-20-3868. PMID: 33563633; PMCID: PMC8102305.
- Evaluating Cancer Drugs in Patients With Central Nervous System Metastases. Marur S, Beaver JA, Pazdur R. JAMA Oncol. 2021 Apr 15. doi: 10.1001/jamaoncol.2021.0180. PMID: 33856413.
- US Food and Drug Administration regulatory updates in neuro-oncology. Mehta GU, Barone AK, Bradford D, Larkins E, Kim J, Pai-Scherf L, Jaigirdar A, Shah M, Wedam S, Amiri-Kordestani L, Theoret MR, Pazdur R, Beaver JA, Singh H. J Neurooncol. 2021 Jul;153(3):375-381. doi: 10.1007/s11060-021-03789-5. PMID: 34156585; PMCID: PMC8218275.
- Neoadjuvant Therapy for Melanoma: A U.S. Food and Drug Administration-Melanoma Research Alliance Public Workshop. Mueller KL, Theoret MR, Lemery SJ, Amiri-Kordestani L, Ariyan CE, Atkins MB, Berry DA, Blank CU, DeMichele AM, Forde PM, Ibrahim N, Keegan P, Mitchell TC, Moss RA, Robert C, Sridhara R, Taube JM, Tetzlaff MT, Wargo JA, Flaherty KT, Kaplan MJ, Topalian SL, Ward AF, Hurlbert MS. Clin Cancer Res. 2021 Jan 15;27(2):394-401. doi: 10.1158/1078-0432.CCR-20-3285. PMID: 33188142.
- Patient Reported Outcomes in Pediatric Cancer Registration Trials: A U.S. Food and Drug Administration Perspective. Murugappan MN, King-Kallimanis BL, Reaman GH, Bhatnagar V, Horodniceanu EG, Bouchkouj N, Kluetz PG. J Natl Cancer Inst. 2021 Apr 30:djab087. doi: 10.1093/jnci/djab087. PMID: 33930159.
- Modernizing Clinical Trial Eligibility Criteria: Recommendations of the ASCO-Friends of Cancer Research Prior Therapies Work Group. Osarogiagbon RU, Vega DM, Fashoyin-Aje L, Wedam S, Ison G, Atienza S, De Porre P, Biswas T, Holloway JN, Hong DS, Wempe MM, Schilsky RL, Kim ES, Wade JL 3rd. Clin Cancer Res. 2021 May 1;27(9):2408-2415. doi: 10.1158/1078-0432.CCR-20-3854. PMID: 33563637; PMCID: PMC8170959.
- FDA and Its Role in Drug Development. Pazdur R. J Adv Pract Oncol. 2021 Apr;12(3):293-295. doi: 10.6004/jadpro.2021.12.3.15. PMID: 34084578; PMCID: PMC8087239.
- Project 2025: Proposals for the Continued Success of Drug Development in Acute Myeloid Leukemia. Pollyea DA, Barrett J, DiNardo CD, Michaelis LC, Roboz GJ, Le RQ, Norsworthy KJ, de Claro RA, Theoret MR, Pazdur R. Clin Cancer Res. 2021 Nov 9:clincanres.2124.2021. doi: 10.1158/1078-0432.CCR-21-2124. PMID: 34753779.
- The Role of Acute Myeloid Leukemia Minimal Residual Disease in Regulatory Decision-making. Przepiorka D, de Claro RA, Pazdur R. JAMA Oncol. 2021 May 1;7(5):784. doi: 10.1001/jamaoncol.2021.0122. PMID: 33764395.
- Oncology Expanded Access and FDA's Project Facilitate. Scepura B, Chan M, Kim T, Boehmer J, Goldberg KB, Pazdur R. Oncologist. 2021 Oct;26(10):e1880-e1882. doi: 10.1002/onco.13910. PMID: 34288259; PMCID: PMC8488788.
- The FDA Oncology Center of Excellence Scientific Collaborative: Charting a Course for Applied Regulatory Science Research in Oncology. Schneider JA, Gong Y, Goldberg KB, Kluetz PG, Theoret MR, Amiri-Kordestani L, Beaver JA, Fashoyin-Aje L, Gormley NJ, Jaigirdar AA, Lemery SJ, Mishra-Kalyani PS, Reaman GH, Rivera DR, Rubinstein WS, Singh H, Sridhara R, Pazdur R. Clin Cancer Res. 2021 Apr 28:10.1158/1078-0432.CCR-20-4429. doi: 10.1158/1078-0432.CCR-20-4429. PMID: 33910935; PMCID: PMC8551300.
- The Oncology Center of Excellence Encourages Submissions of Applied Regulatory Science Research Proposals. Schneider JA, Gao JJ, Rivera DR, Donoghue MB, Reaman GH, Sridhara R, Beaver JA, Kluetz PG, Theoret MR, Pazdur R. The ASCO Post. 2021, Dec 25.
- The Drug-Dosing Conundrum in Oncology - When Less Is More. Shah M, Rahman A, Theoret MR, Pazdur R. N Engl J Med. 2021 Oct 14;385(16):1445-1447. doi: 10.1056/NEJMp2109826. PMID: 34623789.
- Immunotherapy for Patients With Small-Cell Lung Cancer-2 Gains, 2 Losses. Singh H, Beaver JA, Pazdur R. JAMA Oncol. 2021 Oct 28. doi: 10.1001/jamaoncol.2021.4956. PMID: 34709353.
- Contribution of Early Clinical Benefit End Points to Decreased Lung Cancer Mortality Rates. Singh H, Pazdur R. JAMA Oncol. 2021 Jun 1;7(6):829-830. doi: 10.1001/jamaoncol.2020.8090. PMID: 33630046.
- Modernizing Clinical Trial Eligibility Criteria: Recommendations of the ASCO-Friends of Cancer Research Laboratory Reference Ranges and Testing Intervals Work Group. Spira AI, Stewart MD, Jones S, Chang E, Fielding A, Richie N, Wood LS, Thompson MA, Jones L, Nair A, Mahal BA, Gerber DE. Clin Cancer Res. 2021 May 1;27(9):2416-2423. doi: 10.1158/1078-0432.CCR-20-3853. PMID: 33563636; PMCID: PMC8102342.
- Perspectives on Drug Development in Multiple Myeloma-Looking Forward to 2025. Voorhees PM, Jakubowiak AJ, Kumar SK, Kanapuru B, Baines AC, Bhatnagar V, Ershler R, Theoret MR, Gormley NJ, Pazdur R. Clin Cancer Res. 2021 Jul 27. doi: 10.1158/1078-0432.CCR-21-1069. PMID: 34315721.
- Updated Standardized Definitions for Efficacy End Points (STEEP) in Adjuvant Breast Cancer Clinical Trials: STEEP Version 2.0. Tolaney SM, Garrett-Mayer E, White J, Blinder VS, Foster JC, Amiri-Kordestani L, Hwang ES, Bliss JM, Rakovitch E, Perlmutter J, Spears PA, Frank E, Tung NM, Elias AD, Cameron D, Denduluri N, Best AF, DiLeo A, Baizer L, Butler LP, Schwartz E, Winer EP, Korde LA. J Clin Oncol. 2021 Aug 20;39(24):2720-2731. doi: 10.1200/JCO.20.03613. Epub 2021 May 18. PMID: 34003702.
Research Papers
- Idelalisib for Treatment of Relapsed Follicular Lymphoma and Chronic Lymphocytic Leukemia: A Comparison of Treatment Outcomes in Clinical Trial Participants vs Medicare Beneficiaries. Bird ST, Tian F, Flowers N, Przepiorka D, Wang R, Jung TH, Kessler Z, Woods C, Kim B, Miller BW, Wernecke M, Kim C, McKean S, Gelperin K, MaCurdy TE, Kelman JA, Graham DJ. JAMA Oncol. 2020 Feb 1;6(2):248-254. doi: 10.1001/jamaoncol.2019.3994.
- CDK4/6 inhibitor treatment for patients with hormone receptor-positive, HER2-negative, advanced or metastatic breast cancer: a US Food and Drug Administration pooled analysis. Gao JJ, Cheng J, Bloomquist E, Sanchez J, Wedam SB, Singh H, Amiri-Kordestani L, Ibrahim A, Sridhara R, Goldberg KB, Theoret MR, Kluetz PG, Blumenthal GM, Pazdur R, Beaver JA, Prowell TM. Lancet Oncol. 2020 Feb;21(2):250-260. doi: 10.1016/S1470-2045(19)30804-6.
- Comparison of iRECIST versus RECIST V.1.1 in patients treated with an anti-PD-1 or PD-L1 antibody: pooled FDA analysis. Mulkey F, Theoret MR, Keegan P, Pazdur R, Sridhara R. J Immunother Cancer. 2020 Feb;8(1):e000146. doi: 10.1136/jitc-2019-000146.
- An FDA Analysis of Survival Outcomes Comparing an Adjuvant Paclitaxel and Trastuzumab Trial to an External Control from Historical Clinical Trials. Amiri-Kordestani L, Xie D, Tolaney SM, Bloomquist E, Tang S, Ibrahim A, Goldberg KB, Theoret MR, Pazdur R, Sridhara R, Winer EP, Beaver JA. Ann Oncol. 2020 Aug 28:S0923-7534(20)42171-4. doi: 10.1016/j.annonc.2020.08.2106.
- Differentiation Syndrome with Ivosidenib and Enasidenib Treatment in Patients with Relapsed or Refractory IDH-Mutated AML: A U.S. Food and Drug Administration Systematic Analysis. Norsworthy KJ, Mulkey F, Scott EC, Ward AF, Przepiorka D, Charlab R, Dorff SE, Deisseroth A, Kazandjian D, Sridhara R, Beaver JA, Farrell AT, de Claro RA, Pazdur R. Clin Cancer Res. 2020 Aug 15;26(16):4280-4288. doi: 10.1158/1078-0432.CCR-20-0834.
- An Empirical Investigation of Bayesian Clinical Trial Design in Metastatic Breast Cancer. Bloomquist E, Jin S, Zhou J, Tang S, Sridhara R. Ther Innov Regul Sci. 2020 Jul;54(4):861-869. doi: 10.1007/s43441-019-00002-8.
- Weighty choices: selecting optimal G-CSF doses for stem cell mobilization to optimize yield. Farhadfar N, Hsu JW, Logan BR, Sees JA, Chitphakdithai P, Sugrue MW, Abdel-Azim H, Anderlini PN, Bredeson C, Chhabra S, Diaz MA, Ganguly S, Hematti P, Kamble RT, Kasow KA, Lazarus HM, Lynch DK, Murthy HS, Olsson RF, Papari M, Przepiorka D, Savani BN, Schears R, Seo S, Solh MM, Spitzer T, Yared JA, Pulsipher MA, Shah NN, Switzer GE, Confer DL, Shaw BE, Wingard JR. Blood Adv. 2020 Feb 25;4(4):706-716. doi: 10.1182/bloodadvances.2019000923.
- Minimum Technical Data Elements for Liquid Biopsy Data Submitted to Public Databases. Febbo PG, Martin AM, Scher HI, Barrett JC, Beaver JA, Beresford PJ, Blumenthal GM, Bramlett K, Compton C, Dittamore R, Eberhard DA, Edelstein D, Godsey J, Gruen A, Hanlon SE, Hicks J, Hovelson D, Hullings M, Johann D, Johnson J, Kolatkar A, Kuhn P, Levine R, Martini JF, Miller DP, Moore C, Moy B, Pathak A, Philip R, Reese D, Royalty W, Ryder M, Sakul H, Salvatore LM, Schade A, Silvestro A, Simmons JK, Simons J, Singh Bhan S, Smalley MD, Somiari SB, Talasaz A, Tewari M, Tseng HR, Vinson J, Wells W, Welsh A, Grossman RL, Lee JSH, Leiman LC. Clin Pharmacol Ther. 2020 Apr;107(4):730-734. doi: 10.1002/cpt.1747.
- Demystifying the estimand framework: a case study using patient-reported outcomes in oncology. Fiero MH, Pe M, Weinstock C, King-Kallimanis BL, Komo S, Klepin HD, Gray SW, Bottomley A, Kluetz PG, Sridhara R. Lancet Oncol. 2020 Oct;21(10):e488-e494. doi: 10.1016/S1470-2045(20)30319-3.
- US Food and Drug Administration review of statistical analysis of patient-reported outcomes in lung cancer clinical trials approved between January 2008 and December 2017. Fiero MH, Roydhouse JK, Vallejo J, King-Kallimanis BL, Kluetz PG, Sridhara R. Lancet Oncol. 2019 Oct;20(10):e582-e589. doi: 10.1016/S1470-2045(19)30335-3.
- Assessment of Discordance Among Smoldering Multiple Myeloma Risk Models. Hill E, Dew A, Morrison C, Yuan C, Stetler-Stevenson M, Landgren O, Kazandjian D. JAMA Oncol. 2020 Nov 19:e205585. doi: 10.1001/jamaoncol.2020.5585.
- Diagnostic performance of 18F‐FDG‐PET/CT compared to standard skeletal survey for detecting bone destruction in smouldering multiple myeloma: time to move forward. Hill, E., Mena, E., Morrison, C., Dew, A., Choyke, P., Lindenberg, L. and Kazandjian, D. (2020), Br J Haematol. 2020 Sep 23. doi: 10.1111/bjh.17088.
- Evaluation of Serious Postmarket Safety Signals Within 2 Years of FDA Approval for New Cancer Drugs. Kim J, Nair A, Keegan P, Beaver JA, Kluetz PG, Pazdur R, Chuk M, Blumenthal GM. Oncologist. 2020 Apr;25(4):348-354. doi: 10.1634/theoncologist.2019-0653.
- An FDA analysis of clinical hold deficiencies affecting investigational new drug applications for oncology products. Manning ML, Thompson MD, Saber H, Maher VE, Crich JZ, Leighton JK. Regul Toxicol Pharmacol. 2020 Feb;110:104511. doi: 10.1016/j.yrtph.2019.104511.
- Extrapolation of Adult Efficacy to Pediatric Patients With Chemotherapy-Induced Nausea and Vomiting. Momper JD, Heinrichs MT, Krudys K, Griebel D, Kumar S, Kim I, Mehrotra N, Mulberg AE, Garimella N, Nelson R, Reaman G, Sinha V, Yao L, Zineh I, Burckart G, Sachs H, Mulugeta Y. J Clin Pharmacol. 2020 Jun;60(6):775-784. doi: 10.1002/jcph.1577.
- Immune Response Evaluation and Treatment with Immune Checkpoint Inhibitors Beyond Clinical Progression: Response Assessments for Cancer Immunotherapy. Mushti SL, Mulkey F, Tang S, Singh H, Lemery SJ, Goldberg KB, Sridhara R, Keegan P, Kluetz PG, Pazdur R, Theoret MR, Beaver JA. Curr Oncol Rep. 2020 Aug 27;22(11):116. doi: 10.1007/s11912-020-00974-z.
- Proteasome inhibitor-associated thrombotic microangiopathy: A review of cases reported to the FDA adverse event reporting system and published in the literature. Nguyen MN, Nayernama A, Jones SC, Kanapuru B, Gormley N, Waldron PE. Am J Hematol. 2020 Apr 15. doi: 10.1002/ajh.25832.
- Second cancers in adults with acute promyelocytic leukemia treated with or without arsenic trioxide: a SEER-Medicare analysis. Norsworthy KJ, Avagyan A, Bird ST, Li Y, Akhtar S, Liao J, Wernecke M, Deisseroth AB, Chuk M, MaCurdy TE, Swain R, Kelman JA, Farrell AT, de Claro RA, Pazdur R, Blumenthal G, Graham DJ. Leukemia. 2020 Jun 9. doi: 10.1038/s41375-020-0905-y.
- BCR-ABL tyrosine kinase inhibitor-associated thyroid dysfunction: A review of cases reported to the FDA Adverse Event Reporting System and published in the literature. Patel S, Nayernama A, Jones SC, de Claro RA, Waldron PE. Am J Hematol. 2020 Sep 11. doi: 10.1002/ajh.25997.
- Using a Benefit-Risk Analysis Approach to Capture Regulatory Decision Making: Renal Cell Carcinoma. Raju GK, Gurumurthi K, Domike R, Singh H, Weinstock C, Kluetz P, Pazdur R, Woodcock J. Clin Pharmacol Ther. 2020 Mar;107(3):495-506. doi: 10.1002/cpt.1589.
- Exploration of baseline patient-reported side effect bother from cancer therapy. Roydhouse JK, King-Kallimanis BL, Roy P, Weinstock C, Krol D, Daniels SR, Suzman DL, Beaver JA, Kluetz PG. Clin Trials. 2020 Jun;17(3):332-337. doi: 10.1177/1740774520910389.
- Concomitant botanical medicine use among patients participating in commercial prostate cancer trials. Roydhouse JK, Menapace LA, Xia H, Song P, Berman T, Agarwal R, Suzman DL, Wright K, Beaver JA, Kluetz PG. Complement Ther Med. 2020 Nov;54:102549. doi: 10.1016/j.ctim.2020.102549.
- An Analysis of Recent FDA Oncology Scientific Publications. Schneider JA, Miklos AC, Onken J, Gong Y, Calcagno AM, Blumenthal GM, Aragon R, Pazdur R. Oncologist. 2020 Mar;25(3):266-270. doi: 10.1634/theoncologist.2019-0503.
- Fatal neutropenic enterocolitis associated with docetaxel use: A review of cases reported to the United States Food and Drug Administration Adverse Event Reporting System. Singh P, Nayernama A, Christopher Jones S, Amiri Kordestani L, Fedenko K, Prowell T, Bersoff-Matcha SJ. J Oncol Pharm Pract. 2020 Jun;26(4):923-928. doi: 10.1177/1078155219879494.
- NGS Evaluation of Colorectal Cancer Reveals Interferon Gamma Dependent Expression of Immune Checkpoint Genes and Identification of Novel IFNγ Induced Genes. Xu L, Pelosof L, Wang R, McFarland HI, Wu WW, Phue JN, Lee CT, Shen RF, Juhl H, Wu LH, Alterovitz WL, Petricon E, Rosenberg AS. Front Immunol. 2020 Mar 19;11:224. doi: 10.3389/fimmu.2020.00224. eCollection 2020.PMID: 32265897
Approval Summaries
- FDA Approval Summary: Rucaparib for the Treatment of Patients with Deleterious BRCA-Mutated Metastatic Castrate-Resistant Prostate Cancer. Anscher MS, Chang E, Gao X, Gong Y, Weinstock C, Bloomquist E, Adeniyi O, Charlab R, Zimmerman S, Serlemitsos-Day M, Ning YM, Mayrosh R, Fuller B, Trentacosti AM, Gallagher P, Bijwaard K, Philip R, Ghosh S, Fahnbulleh F, Diggs F, Arora S, Goldberg KB, Tang S, Amiri-Kordestani L, Pazdur R, Ibrahim A, Beaver JA. Oncologist. 2020 Nov 4. doi: 10.1002/onco.13585.
- FDA Approval Summary: Olaparib monotherapy or in combination with bevacizumab for the maintenance treatment of patients with advanced ovarian cancer. Arora S, Balasubramaniam S, Zhang H, Berman T, Narayan P, Suzman D, Bloomquist E, Tang S, Gong Y, Sridhara R, Turcu FR, Chatterjee D, Saritas-Yildirim B, Ghosh S, Philip R, Pathak A, Gao JJ, Amiri-Kordestani L, Pazdur R, Beaver JA. Oncologist. 2020 Oct 5. doi: 10.1002/onco.13551.
- FDA review summary of patient-reported outcome results for ibrutinib in the treatment of chronic graft versus host disease. King-Kallimanis BL, Wroblewski T, Kwitkowski V, De Claro RA, Gwise T, Bhatnagar V, Farrell AT, Kluetz PG. Qual Life Res. 2020 Jul;29(7):1903-1911. doi: 10.1007/s11136-020-02448-y.
- FDA Approval Summary: Pembrolizumab plus Lenvatinib for Endometrial Carcinoma, a Collaborative International Review under Project Orbis. Arora S, Balasubramaniam S, Zhang W, Zhang L, Sridhara R, Spillman D, Mathai JP, Scott B, Golding SJ, Coory M, Pazdur R, Beaver JA. Clin Cancer Res. 2020 Oct 1;26(19):5062-5067. doi: 10.1158/1078-0432.CCR-19-3979.
- FDA Accelerated Approval of Pembrolizumab for Recurrent Locally Advanced or Metastatic Merkel Cell Carcinoma. Bradford D, Demko S, Jin S, Mishra-Kalyani P, Beckles AR, Goldberg KB, Lemery S, Ward A, Keegan P, Pazdur R. Oncologist. 2020 Jul;25(7):e1077-e1082. doi: 10.1634/theoncologist.2020-0184.
- FDA Approval Summary: Selpercatinib for the Treatment of Lung and Thyroid cancers with RET Gene Mutations or Fusions. Bradford D, Larkins E, Mushti SL, Rodriguez L, Skinner AM, Helms WS, Price LSL, Fourie Zirkelbach J, Li Y, Liu J, Charlab R, Reyes Turcu F, Liang D, Ghosh S, Roscoe D, Philip R, Zack-Taylor A, Tang S, Kluetz PG, Beaver JA, Pazdur R, Theoret MR, Singh H. Clin Cancer Res. 2020 Nov 25:clincanres.3558.2020. doi: 10.1158/1078-0432.CCR-20-3558.
- FDA Approval Summary: Atezolizumab plus bevacizumab for the treatment of patients with advanced unresectable or metastatic hepatocellular carcinoma. Casak SJ, Donoghue M, Fashoyin-Aje L, Jiang X, Rodriguez L, Shen YL, Xu Y, Jiang X, Liu J, Zhao H, Pierce WF, Mehta S, Goldberg KB, Theoret MR, Kluetz PG, Pazdur R, Lemery SJ. Clin Cancer Res. 2020 Nov 2:clincanres.3407.2020. doi: 10.1158/1078-0432.CCR-20-3407.
- FDA Approval Summary: Enfortumab Vedotin for Locally Advanced or Metastatic Urothelial Carcinoma. Chang E, Weinstock C, Zhang L, Charlab R, Dorff SE, Gong Y, Hsu V, Li F, Ricks TK, Song P, Tang S, Waldron PE, Yu J, Zahalka E, Goldberg KB, Pazdur R, Theoret MR, Ibrahim A, Beaver JA. Clin Cancer Res. 2020 Sep 22:clincanres.2275.2020. doi: 10.1158/1078-0432.CCR-20-2275.
- FDA Approval Summary: Pertuzumab, Trastuzumab, and Hyaluronidase-zzxf injection for subcutaneous use in Patients with HER2-Positive Breast Cancer. Gao JJ, Osgood CL, Gong Y, Zhang H, Bloomquist EW, Jiang X, Qiu J, Yu J, Song P, Rahman A, Chiu HJ, Ricks TK, Rizvi F, Hou S, Wilson W, Abukhdeir AM, Seidman J, Ghosh S, Philip R, Pierce WF, Bhatnagar V, Kluetz PG, Pazdur R, Beaver JA, Amiri-Kordestani L. Clin Cancer Res. 2020 Nov 13:clincanres.3474.2020. doi: 10.1158/1078-0432.CCR-20-3474.
- FDA Approval Summary: Entrectinib for the treatment of NTRK-gene fusion solid tumors. Marcus L, Donoghue M, Aungst S, Myers CE, Helms WS, Shen G, Zhao H, Stephens O, Keegan P, Pazdur R. Clin Cancer Res. 2020 Sep 23:clincanres.2771.2020. doi: 10.1158/1078-0432.CCR-20-2771.
- FDA approval summary: Dalteparin for the treatment of symptomatic venous thromboembolism in pediatric patients. Merino M, Richardson N, Reaman G, Ande A, Zvada S, Liu C, Hariharan S, De Claro RA, Farrell A, Pazdur R. Pediatr Blood Cancer. 2020 Sep 8:e28688. doi: 10.1002/pbc.28688.
- FDA Supplemental Approval Summary: Lenvatinib for the Treatment of Unresectable Hepatocellular Carcinoma. Nair A, Reece K, Donoghue MB, Yuan WV, Rodriguez L, Keegan P, Pazdur R. Oncologist. 2020 Oct 12. doi: 10.1002/onco.13566.
- FDA Approval Summary: Alpelisib plus fulvestrant for patients with HR-positive, HER2-negative, PIK3CA-mutated, advanced or metastatic breast cancer. Narayan P, Prowell TM, Gao JJ, Fernandes LL, Li E, Jiang X, Qiu J, Fan J, Song P, Yu J, Zhang X, King-Kallimanis BL, Chen W, Ricks TK, Gong Y, Wang X, Windsor K, Rhieu SY, Gieser G, Banerjee A, Chen X, Reyes Turcu F, Chatterjee DK, Pathak A, Seidman J, Ghosh S, Philip R, Goldberg KB, Kluetz PG, Tang S, Amiri-Kordestani L, Theoret MR, Pazdur R, Beaver JA. Clin Cancer Res. 2020 Nov 9:clincanres.3652.2020. doi: 10.1158/1078-0432.CCR-20-3652.
- FDA Approval Summary: Atezolizumab Plus Paclitaxel Protein-bound for the Treatment of Patients with Advanced or Metastatic TNBC Whose Tumors Express PD-L1. Narayan P, Wahby S, Gao JJ, Amiri-Kordestani L, Ibrahim A, Bloomquist E, Tang S, Xu Y, Liu J, Fu W, Song P, King-Kallimanis BL, Hou S, Gong Y, Kalavar S, Ghosh S, Philip R, Goldberg KB, Theoret MR, Blumenthal GM, Kluetz PG, Sridhara R, Pazdur R, Beaver JA. Clin Cancer Res. 2020 May 15;26(10):2284-2289. doi: 10.1158/1078-0432.CCR-19-3545.
- Benefit-Risk Summary of Nivolumab for the Treatment of Patients with Unresectable Advanced, Recurrent, or Metastatic Esophageal Squamous Cell Carcinoma After Prior Fluoropyrimidine- and Platinum-Based Chemotherapy. Pelosof L, Saung MT, Donoghue M, Casak S, Mushti S, Cheng J, Jiang X, Liu J, Zhao H, Khazraee M, Goldberg KB, Theoret M, Lemery S, Pazdur R, Fashoyin-Aje L. Oncologist. 2020 Dec 20. doi: 10.1002/onco.13646.
- FDA Approval Summary: Ruxolitinib for Treatment of Steroid-Refractory Acute Graft-Versus-Host Disease. Przepiorka D, Luo L, Subramaniam S, Qiu J, Gudi R, Cunningham LC, Nie L, Leong R, Ma L, Sheth C, Deisseroth A, Goldberg KB, Blumenthal GM, Pazdur R. Oncologist. 2020 Feb;25(2):e328-e334. doi: 10.1634/theoncologist.2019-0627.
- FDA Approval Summary: Tucatinib for the Treatment of Patients with Advanced or Metastatic HER2-Positive Breast Cancer. Shah M, Wedam S, Cheng J, Fiero MH, Xia H, Li F, Fan J, Zhang X, Yu J, Song P, Chen W, Ricks TK, Chen XH, Goldberg KB, Gong Y, Pierce WF, Tang S, Theoret MR, Pazdur R, Amiri-Kordestani L, Beaver JA. Clin Cancer Res 2020 Oct 14;clincanres.2701.2020.
- Approvals in 2019: international review and a new agnostic molecular entity. Singh H, Blumenthal G, Pazdur R. Nat Rev Clin Oncol. 2020 Mar;17(3):130-131. doi: 10.1038/s41571-020-0336-8.
- FDA Approval Summary: Lurbinectedin for the Treatment of Metastatic Small Cell Lung Cancer. Singh S, Jaigirdar AA, Mulkey F, Cheng J, Hamed SS, Li Y, Liu J, Zhao H, Goheer A, Helms WS, Wang X, Agarwal R, Pragani R, Korsah K, Tang S, Leighton J, Rahman A, Beaver JA, Pazdur R, Theoret MR, Singh H. Clin Cancer Res. 2020 Dec 7:clincanres.3901.2020. doi: 10.1158/1078-0432.CCR-20-3901.
- FDA Approval Summary: Accelerated Approval of Sacituzumab Govitecan-hziy for Third Line Treatment of Metastatic Triple-negative Breast Cancer (mTNBC). Wahby S, Fashoyin-Aje L, Osgood CL, Cheng J, Fiero MH, Zhang L, Tang S, Hamed SS, Song P, Charlab R, Dorff SE, Ricks TK, Barnett-Ringgold K, Dinin J, Goldberg KB, Theoret MR, Pazdur R, Amiri-Kordestani L, Beaver JA. Oncologist. 2021 Feb;26(2):139-146. doi: 10.1002/onco.13585. Epub 2020 Nov 17. PMID: 33145877; PMCID: PMC7873319.
- FDA Approval Summary: Palbociclib for Male Patients with Metastatic Breast Cancer. Wedam S, Fashoyin-Aje L, Bloomquist E, Tang S, Sridhara R, Goldberg KB, Theoret MR, Amiri-Kordestani L, Pazdur R, Beaver JA. Clin Cancer Res. 2020 Mar 15;26(6):1208-1212. doi: 10.1158/1078-0432.CCR-19-2580.
- Approval Summary: Ado-Trastuzumab Emtansine for the Adjuvant Treatment of HER2-positive Early Breast Cancer. Wedam S, Fashoyin-Aje L, Gao X, Bloomquist E, Tang S, Sridhara R, Goldberg KB, King-Kallimanis BL, Theoret MR, Ibrahim A, Amiri-Kordestani L, Pazdur R, Beaver JA. Clin Cancer Res. 2020 Aug 15;26(16):4180-4185. doi: 10.1158/1078-0432.CCR-19-3980.
Regulatory Perspectives
- Pivotal Considerations for Optimal Deployment of Healthy Volunteers in Oncology Drug Development. Ahmed MA, Patel C, Drezner N, Helms W, Tan W, Stypinski D. Clin Transl Sci. 2020 Jan;13(1):31-40. doi: 10.1111/cts.12703.
- Public-private partnerships in transplant drug development. Albrecht R, Papadopoulos EJ, Campbell M, Daniels S, Kluetz PG, Parekh A, Wang Y. Am J Transplant. 2020 Feb;20(2):377-381. doi: 10.1111/ajt.15604.
- Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data Consortium. International standards for the analysis of quality-of-life and patient-reported outcome endpoints in cancer randomised controlled trials: recommendations of the SISAQOL Consortium. Coens C, Pe M, Dueck AC, Sloan J, Basch E, Calvert M, Campbell A, Cleeland C, Cocks K, Collette L, Devlin N, Dorme L, Flechtner HH, Gotay C, Griebsch I, Groenvold M, King M, Kluetz PG, Koller M, Malone DC, Martinelli F, Mitchell SA, Musoro JZ, O'Connor D, Oliver K, Piault-Louis E, Piccart M, Quinten C, Reijneveld JC, Schürmann C, Smith AW, Soltys KM, Taphoorn MJB, Velikova G, Bottomley A. Lancet Oncol. 2020 Feb;21(2):e83-e96. doi: 10.1016/S1470-2045(19)30790-9.
- A review of the experience with pediatric written requests issued for oncology drug products. Akalu AY, Meng X, Reaman GH, Ma L, Yuan W, Ye J. Pediatr Blood Cancer. 2020 Nov 27:e28828. doi: 10.1002/pbc.28828.
- Update on Data Required to Build a Learning Health System. Bertagnolli MM, Anderson B, Norsworthy K, Piantadosi S, Quina A, Schilsky RL, Miller RS, Khozin S. J Clin Oncol. 2020 May 10;38(14):1602-1607. doi: 10.1200/JCO.19.03094.
- Patient-Reported Outcomes in Oncology Clinical Trials: Stakeholder Perspectives from the Accelerating Anticancer Agent Development and Validation Workshop 2019. Bhatnagar V, Hudgens S, Piault-Louis E, Jones L, Beaver JA, Lyerly HK, Reaman G, Fleming T, Kluetz PG. Oncologist. 2020 May 28. doi: 10.1634/theoncologist.2020-0062.
- An FDA Review of Drug Development in Nonmetastatic Castration-resistant Prostate Cancer. Brave M, Weinstock C, Brewer JR, Chi DC, Suzman DL, Cheng J, Zhang L, Sridhara R, Ibrahim A, Kluetz PG, Pazdur R, Beaver JA. Clin Cancer Res. 2020 Sep 15;26(18):4717-4722. doi: 10.1158/1078-0432.CCR-19-3835.
- Regulatory Considerations for Contribution of Effect of Drugs used in Combination Regimens: Renal Cell Cancer Case Studies. Brewer JR, Chang E, Agrawal S, Singh H, Suzman DL, Xu J, Weinstock C, Fernandes LL, Cheng J, Zhang L, Xie D, Goldberg KB, Bloomquist EW, Tang S, Sridhara R, Theoret MR, Pazdur R, Ibrahim A, Beaver JA. Clin Cancer Res. 2020 Jul 30:clincanres.4229.2020. doi: 10.1158/1078-0432.CCR-19-4229.
- Disparities in Cancer Prevention in the COVID-19 Era. Carethers JM, Sengupta R, Blakey R, Ribas A, D'Souza G. Cancer Prev Res (Phila). 2020 Sep 17. doi: 10.1158/1940-6207.CAPR-20-0447.
- U.S. Food and Drug Administration: Initial Experience with the Real-Time Oncology Review Program. de Claro RA, Gao JJ, Kim T, Kluetz PG, Theoret MR, Beaver JA, Pazdur R. Clin Cancer Res. 2020 Aug 19:clincanres.2220.2020. doi: 10.1158/1078-0432.CCR-20-2220.
- Project Orbis: Global Collaborative Review Program. de Claro RA, Spillman D, Hotaki LT, Shum M, Mouawad LS, Santos GML, Robinson K, Hunt M, Healy C, Chan A, Looi YH, Rodrigues C, Rohr UP, Walther C, Pazdur R. Clin Cancer Res. 2020 Oct 9. doi: 10.1158/1078-0432.CCR-20-3292.
- The RACE to Develop New Targeted Therapies for Children With CNS Tumors. Fletcher EP, Burckart GJ, Robinson GW, Reaman GH, Stewart CF. Clin Pharmacol Ther. 2020 Jul 7. doi: 10.1002/cpt.1937.
- Expanding Access to Lung Cancer Clinical Trials by Reducing the Use of Restrictive Exclusion Criteria: Perspectives of a Multistakeholder Working Group. Forde PM, Bonomi P, Shaw A, Blumenthal GM, Ferris A, Patel C, Melemed A, Basu Roy U, Ramamoorthy A, Liu Q, Burns T, Gainor JF, Lovly C, Piotrowska Z, Lehman J, Selig W. Clin Lung Cancer. 2020 Jul;21(4):295-307. doi: 10.1016/j.cllc.2020.02.008.
- Ethnicity-based differences in breast cancer features and responsiveness to CDK4/6 inhibitors combined with endocrine therapy - Authors' reply. Gao JJ, Cheng J, Beaver JA, Prowell TM. Lancet Oncol. 2020 Mar;21(3):e131. doi: 10.1016/S1470-2045(20)30095-4.
- Engaging the Oncology Community to Advanced Regulatory Science – FDA’s Project Renewal and Project Socrates. Gao JJ, Keegan P, Kluetz P, Pazdur R. AACR Cancer Research Catalyst. July 28, 2020.
- FDA Oncology Center of Excellence Review Processes and Tools. Gao JJ, Kluetz PG, Pazdur R. Clin Pharmacol Ther. 2020 Jul 12. doi: 10.1002/cpt.1945.
- FDA Oncology Center of Excellence During COVID-19-Working for Patients With Cancer. Gao JJ, Pazdur R. JAMA Oncol. 2020 Dec 23. doi: 10.1001/jamaoncol.2020.6783.
- Before It's Too Late: Multistakeholder Perspectives on Compassionate Access to Investigational Drugs for Pediatric Patients With Cancer. Gerasimov E, Donoghue M, Bilenker J, Watt T, Goodman N, Laetsch TW. Am Soc Clin Oncol Educ Book. 2020 May;40:1-10. doi: 10.1200/EDBK_278995. PMID: 32412804.
- Novel Therapeutic Interventions Early in the Disease Trajectory: Drug Development Beyond the Refractory Setting. Hafez N, Rugo HS, Tempero MA, Fox E, Reaman GH, Lyerly HK, Walker D, LoRusso PM. Clin Cancer Res. 2020 Sep 15;26(18):4743-4747. doi: 10.1158/1078-0432.CCR-19-4035.
- Summary of the 2019 Blood and Marrow Transplant Clinical Trials Network Myeloma Intergroup Workshop on Minimal Residual Disease and Immune Profiling. Holstein SA, Howard A, Avigan D, Bhutani M, Cohen AD, Costa LJ, Dhodapkar MV, Gay F, Gormley N, Green DJ, Hillengass J, Korde N, Li Z, Mailankody S, Neri P, Parekh S, Pasquini MC, Puig N, Roodman GD, Samur MK, Shah N, Shah UA, Shi Q, Spencer A, Suman VJ, Usmani SZ, McCarthy PL. Biol Blood Marrow Transplant. 2020 Oct;26(10):e247-e255. doi: 10.1016/j.bbmt.2020.06.011.
- US Food and Drug Administration Support for Oncology Drug Development During COVID-19. Kadakia KT, Pazdur R, Shah A. JAMA Oncol. 2020 Oct 8. doi: 10.1001/jamaoncol.2020.4975.
- Older adults in hematologic malignancy trials: Representation, barriers to participation and strategies for addressing underrepresentation. Kanapuru B, Singh H, Kwitkowski V, Blumenthal G, Farrell AT, Pazdur R. Blood Rev. 2020 Sep;43:100670. doi: 10.1016/j.blre.2020.100670.
- The Globalization of Geriatric Oncology: From Data to Practice. Kanesvaran R, Mohile S, Soto-Perez-de-Celis E, Singh H. Am Soc Clin Oncol Educ Book. 2020 Mar;40:1-9. doi: 10.1200/EDBK_279513. PMID: 32347757.
- The changing role of high dose melphalan with stem cell rescue in the treatment of newly diagnosed multiple myeloma in the era of modern therapies-back to the future! Kazandjian D, Dew A, Hill E. Best Pract Res Clin Haematol. 2020 Mar;33(1):101150. doi: 10.1016/j.beha.2020.101150.
- Application of the Lung Immune Prognostic Index From Research to Clinical Practice-Reply. Kazandjian D, Gong Y, Blumenthal GM. JAMA Oncol. 2020 Feb 1;6(2):300-301. doi: 10.1001/jamaoncol.2019.5157.
- The role of high-dose melphalan with autologous stem-cell transplant in multiple myeloma: is it time for a paradigm shift? Kazandjian D, Mo CC, Landgren O, Richardson PG. Br J Haematol. 2020 Jun 5. doi: 10.1111/bjh.16764.
- FDA Oncology Center of Excellence Project Renewal: Engaging the Oncology Community to Update Product Labeling for Older Oncology Drugs. Kluetz PG, Keegan P, Demetri GD, Thornton KA, Sul J, Kim J, Katzen H, Burke LB, Harvey RD, Alebachew E, Agrawal S, Nair A, Donoghue M, Pierce WF, Shord SS, Gao JJ, Pazdur R. Clin Cancer Res. 2020 Nov 30:clincanres.3213.2020. doi: 10.1158/1078-0432.CCR-20-3213.
- Advancing Assessment, Analysis, and Reporting of Safety and Tolerability in Cancer Trials. Kluetz PG, King-Kallimanis BL, Suzman D, Chang E, Brave MM, Weinstock C, Bhatnagar V, Beaver JA, Chuk MK. J Natl Cancer Inst. 2020 Aug 28:djaa135. doi: 10.1093/jnci/djaa135.
- Preparing Fellows for Graduation: Perspectives on Career Guidance. LaCasce A, Graff S, Gao J, Close J, Boulmay B. Am Soc Clin Oncol Educ Book. 2019 Jan;39:609-614. doi: 10.1200/EDBK_242603.
- Treatments for newly diagnosed multiple myeloma: when endurance is interrupted. Landgren O, Siegel D, Kazandjian D, Costa L, Jakubowiak A. Lancet Oncol. 2020 Dec;21(12):e540. doi: 10.1016/S1470-2045(20)30635-5.
- Cancer and aging activities at the American Society of Clinical Oncology and beyond: Reflections on the legacy of Dr. Arti Hurria. Levit LA, Singh H, Klepin HD. J Geriatr Oncol. 2020 Mar;11(2):151-153. doi: 10.1016/j.jgo.2019.07.007.
- Second malignancies in multiple myeloma; emerging patterns and future directions. Maclachlan K, Diamond B, Maura F, Hillengass J, Turesson I, Landgren CO, Kazandjian D. Best Pract Res Clin Haematol. 2020 Mar;33(1):101144. doi: 10.1016/j.beha.2020.101144.
- Summary of a workshop on preclinical and translational safety assessment of CD3 bispecifics. Manning ML, Thompson MD, Saber H, Maher VE, Crich JZ, Leighton JK. An FDA analysis of clinical hold deficiencies affecting investigational new drug applications for oncology products. Regul Toxicol Pharmacol. 2020 Feb;110:104511. doi: 10.1016/j.yrtph.2019.104511. Kamperschroer C, Shenton J, Lebrec H, Leighton JK, Moore PA, Thomas O. J Immunotoxicol. 2020 Dec;17(1):67-85. doi: 10.1080/1547691X.2020.1729902.
- Neoadjuvant Therapy for Melanoma: A US Food and Drug Administration - Melanoma Research Alliance Public Workshop. Mueller KL, Theoret MR, Lemery SJ, Amiri-Kordestani L, Ariyan CE, Atkins MB, Berry DA, Blank CU, DeMichele A, Forde PM, Ibrahim N, Keegan P, Mitchell TC, Moss RA, Robert C, Sridhara R, Taube JM, Tetzlaff MT, Wargo JA, Flaherty K, Kaplan MJ, Topalian SL, Ward AF, Hurlbert MS. Clin Cancer Res. 2020 Nov 13:clincanres.3285.2020. doi: 10.1158/1078-0432.CCR-20-3285.
- State of the Science and Future Directions for Liquid Biopsies in Drug Development. Narayan P, Ghosh S, Philip R, Barrett JC, McCormack RT, Odegaard JI, R Oxnard G, Pracht LJ, Williams PM, Kelloff GJ, Beaver JA. Oncologist. 2020 Jun 8;25(9):730–2. doi: 10.1634/theoncologist.2020-0246.
- Paediatric Strategy Forum for medicinal product development of epigenetic modifiers for children: ACCELERATE in collaboration with the European Medicines Agency with participation of the Food and Drug Administration. Pearson AD, Stegmaier K, Bourdeaut F, Reaman G, Heenen D, Meyers ML, Armstrong SA, Brown P, De Carvalho D, Jabado N, Marshall L, Rivera M, Smith M, Adamson PC, Barone A, Baumann C, Blackman S, Buenger V, Donoghue M, Duncan AD, Fox E, Gadbaw B, Hattersley M, Ho P, Jacobs I, Kelly MJ, Kieran M, Lesa G, Ligas F, Ludwinski D, McDonough J, Nikolova Z, Norga K, Senderowicz A, Taube T, Weiner S, Karres D, Vassal G. Eur J Cancer. 2020 Sep 25;139:135-148. doi: 10.1016/j.ejca.2020.08.014.
- The RACE to accelerate drug development for children with cancer. Pearson ADJ, Karres D, Reaman G, DuBois SG, Knox L, Scobie N, Vassal G. Lancet Child Adolesc Health. 2020 Oct;4(10):714-716. doi: 10.1016/S2352-4642(20)30247-9.
- ACCELERATE and European Medicines Agency Paediatric Strategy Forum for medicinal product development of checkpoint inhibitors for use in combination therapy in paediatric patients. Pearson ADJ, Rossig C, Lesa G, Diede SJ, Weiner S, Anderson J, Gray J, Geoerger B, Minard-Colin V, Marshall LV, Smith M, Sondel P, Bajars M, Baldazzi C, Barry E, Blackman S, Blanc P, Capdeville R, Caron H, Cole PD, Jiménez JC, Demolis P, Donoghue M, Elgadi M, Gajewski T, Galluzzo S, Ilaria R Jr, Jenkner A, Karres D, Kieran M, Ligas F, Lowy I, Meyers M, Oprea C, Peddareddigari VGR, Sterba J, Stockman PK, Suenaert P, Tabori U, van Tilburg C, Yancey T, Weigel B, Norga K, Reaman G, Vassal G. Eur J Cancer. 2020 Mar;127:52-66. doi: 10.1016/j.ejca.2019.12.029.
- Paediatric Strategy Forum for medicinal product development for acute myeloid leukaemia in children and adolescents: ACCELERATE in collaboration with the European Medicines Agency with participation of the Food and Drug Administration. Pearson ADJ, Zwaan CM, Kolb EA, Karres D, Guillot J, Kim SY, Marshall L, Tasian SK, Smith M, Cooper T, Adamson PC, Barry E, Benettaib B, Binlich F, Borgman A, Brivio E, Capdeville R, Delgado D, Faller D, Fogelstrand L, Fraenkel PG, Hasle H, Heenen D, Kaspers G, Kieran M, Klusmann JH, Lesa G, Ligas F, Mappa S, Mohamed H, Moore A, Morris J, Nottage K, Reinhardt D, Scobie N, Simko S, Winkler T, Norga K, Reaman G, Vassal G. Eur J Cancer. 2020 Sep;136:116-129. doi: 10.1016/j.ejca.2020.04.038.
- Broadening Eligibility Criteria for Oncology Clinical Trials: Current Advances and Future Directions. Rahman NA, Ison G, Beaver JA. Clin Pharmacol Ther. 2020 Jul 8. doi: 10.1002/cpt.1919.
- Accelerating the Global Development of Pediatric Cancer Drugs: A Call to Coordinate the Submissions of Pediatric Investigation Plans and Pediatric Study Plans to the European Medicines Agency and US Food and Drug Administration. Reaman G, Karres D, Ligas F, Lesa G, Casey D, Ehrlich L, Norga K, Pazdur R. J Clin Oncol. 2020 Sep 18:JCO2002152. doi: 10.1200/JCO.20.02152.
- Crossing Oceans: Preclinical Collaboration to Improve Pediatric Drug Development. Reaman G, Stancato L, Vassal G, Maris JM. Am Soc Clin Oncol Educ Book. 2020 Mar;40:1-8. doi:10.1200/EDBK_278893.
- Operational strategies in US cancer centers of excellence that support the successful accrual of racial and ethnic minorities in clinical trials. Regnante JM, Richie N, Fashoyin-Aje L, Hall LL, Highsmith Q, Louis J, Turner K, Hoover S, Lee SC, González E, Williams E, Adams H 3rd, Obasaju C, Sargeant I, Spinner J, Reddick C, Gandee M, Geday M, Dang J, Watson R, Chen MS Jr. Contemp Clin Trials Commun. 2020 Jan 22;17:100532. doi: 10.1016/j.conctc.2020.100532.
- Antibody-Drug Conjugates: Simple Idea, Complicated Matter. Saber H, Leighton JK. DIA Global Forum. 2020 October.
- The Evolution of Clinical Trials in Metastatic Breast Cancer: Design Features and Endpoints That Matter. Seidman AD, Maues J, Tomlin T, Bhatnagar V, Beaver JA. Am Soc Clin Oncol Educ Book. 2020 Mar;40:1-11. doi: 10.1200/EDBK_280451.
- Summary of American Statistical Association Biopharmaceutical Section’s virtual discussion with regulators on type 1 error considerations in master protocols with common control in oncology. Sridhara R, Marchenko O, Jiang Q, Pazdur R, Binkowitz B. 2020 Biopharmaceutical Report. 27;3:39-40.
- Development, Validation, and Regulatory Considerations for a Liquid Biopsy Test. Tsui DWY, Blumenthal GM, Philip R, Barrett JC, Montagut C, Bramlett K, Ladanyi M, Ghosh S. Clin Chem. 2020 Mar 1;66(3):408-414. doi: 10.1093/clinchem/hvaa010.
- A Bayesian approach in design and analysis of pediatric cancer clinical trials. Ye J, Reaman G, De Claro RA, Sridhara R. Pharm Stat. 2020 Jun 14. doi: 10.1002/pst.2039.
- Reducing Uninformative IND Safety Reports: A List of Serious Adverse Events anticipated to Occur in Patients with Lung Cancer. Bonomi P, Stuccio N, Delgra CJ, Chuk MK, Spira A, Deitz AC, Blumenthal GM, Ferris A, Gong Y, He J, Basu Roy U, Selig W. Ther Innov Regul Sci. 2020 Sep;54(5):1208-1214. doi: 10.1007/s43441-020-00145-z.
Research Papers
- Patient reported outcomes in anti-PD-1/PD-L1 inhibitor immunotherapy registration trials: FDA analysis of data submitted and future directions. King-Kallimanis BL, Howie LJ, Roydhouse JK, Singh H, Theoret MR, Blumenthal GM, Kluetz PG. Clin Trials. 2019 Jun;16(3):322-326. doi: 10.1177/1740774519836991.
- Analysis of time-to-treatment discontinuation of targeted therapy, immunotherapy, and chemotherapy in clinical trials of patients with non-small-cell lung cancer. Blumenthal GM, Gong Y, Kehl K, Mishra-Kalyani P, Goldberg KB, Khozin S, Kluetz PG, Oxnard GR, Pazdur R. Ann Oncol. 2019 May 1;30(5):830-838. doi: 10.1093/annonc/mdz060.
- Blinding and Patient-Reported Outcome Completion Rates in US Food and Drug Administration Cancer Trial Submissions, 2007-2017. Roydhouse JK, King-Kallimanis BL, Howie LJ, Singh H, Kluetz PG. J Natl Cancer Inst. 2019 May 1;111(5):459-464. doi: 10.1093/jnci/djy181. PMID: 30561711.
- Analysis of the Association Between Adverse Events and Outcome in Patients Receiving a Programmed Death Protein 1 or Programmed Death Ligand 1 Antibody. Maher VE, Fernandes LL, Weinstock C, Tang S, Agarwal S, Brave M, Ning YM, Singh H, Suzman D, Xu J, Goldberg KB, Sridhara R, Ibrahim A, Theoret M, Beaver JA, Pazdur R. J Clin Oncol. 2019 Oct 20;37(30):2730-2737. doi: 10.1200/JCO.19.00318.
- Analyzing patient-reported outcome data when completion differs between arms in open-label trials: an application of principal stratification. Roydhouse JK, Gutman R, Bhatnagar V, Kluetz PG, Sridhara R, Mishra- Kalyani PS. Pharmacoepidemiol Drug Saf. 2019 Oct;28(10):1386-1394. doi: 10.1002/pds.4875.
- Machine-Learning and Stochastic Tumor Growth Models for Predicting Outcomes in Patients With Advanced Non-Small-Cell Lung Cancer. Siah KW, Khozin S, Wong CH, Lo AW. JCO Clin Cancer Inform. 2019 Sep;3:1-11. doi: 10.1200/CCI.19.00046.
- An Analysis of Recent FDA Oncology Scientific Publications. Schneider JA, Miklos AC, Onken J, Gong Y, Calcagno AM, Blumenthal GM, Aragon R, Pazdur R. Oncologist. 2019 Nov 26. pii: theoncologist.2019-0503. doi: 10.1634/ theoncologist.2019-0503.
- Eligibility and Radiologic Assessment for Adjuvant Clinical Trials in Kidney Cancer. Agrawal S, Haas NB, Bagheri M, Lane BR, Coleman J, Hammers H, Bratslavsky G, Chauhan C, Kim L, Krishnasamy VP, Marko J, Maher VE, Ibrahim A, Cross F Jr, Liu K, Beaver JA, Pazdur R, Blumenthal GM, Singh H, Plimack ER, Choueiri TK, Uzzo R, Apolo AB. JAMA Oncol. 2019 Nov 21. doi: 10.1001/jamaoncol.2019.4117.
- An FDA analysis of clinical hold deficiencies affecting investigational new drug applications for oncology products. Manning ML, Thompson MD, Saber H, Maher VE, Crich JZ, Leighton JK. Regul Toxicol Pharmacol. 2019 Oct 31;110:104511. doi: 10.1016/j.yrtph.2019.104511.
- Eligibility and Radiologic Assessment in Adjuvant Clinical Trials in Bladder Cancer. Apolo AB, Milowsky MI, Kim L, Inman BA, Kamat AM, Steinberg G, Bagheri M, Krishnasamy VP, Marko J, Dinney CP, Bangs R, Sweis RF, Maher VE, Ibrahim A, Liu K, Werntz R, Cross F, Beaver JA, Singh H, Pazdur R, Blumenthal GM, Lerner SP, Bajorin DF, Rosenberg JE, Agrawal S. JAMA Oncol. 2019 Oct 31:1-9. doi: 10.1001/jamaoncol.2019.4114.
- Fatal neutropenic enterocolitis associated with docetaxel use: A review of cases reported to the United States Food and Drug Administration Adverse Event Reporting System. Singh P, Nayernama A, Christopher Jones S, Amiri Kordestani L, Fedenko K, Prowell T, Bersoff-Matcha SJ. J Oncol Pharm Pract. 2019 Oct 8:1078155219879494. doi: 10.1177/1078155219879494.
- US Food and Drug Administration review of statistical analysis of patient-reported outcomes in lung cancer clinical trials approved between January, 2008, and December, 2017. Fiero MH, Roydhouse JK, Vallejo J, King-Kallimanis BL, Kluetz PG, Sridhara R. Lancet Oncol. 2019 Oct;20(10):e582-e589. doi: 10.1016/S1470- 2045(19)30335-3.
- Outcomes of Older Women With Hormone Receptor-Positive, Human Epidermal Growth Factor Receptor-Negative Metastatic Breast Cancer Treated With a CDK4/6 Inhibitor and an Aromatase Inhibitor: An FDA Pooled Analysis. Howie LJ, Singh H, Bloomquist E, Wedam S, Amiri-Kordestani L, Tang S, Sridhara R, Sanchez J, Prowell TM, Kluetz PG, King-Kallimanis BL, Gao JJ, Ibrahim A, Goldberg KB, Theoret M, Pazdur R, Beaver JA. J Clin Oncol. 2019 Sep 27:JCO1802217. doi: 10.1200/JCO.18.02217.
- Association Between FDA Label Restriction and Immunotherapy and Chemotherapy Use in Bladder Cancer. Parikh RB, Adamson BJS, Khozin S, Galsky MD, Baxi SS, Cohen A, Mamtani R. JAMA. 2019 Sep 24;322(12):1209- 1211. doi: 10.1001/jama.2019.10650.
- Cytokine Effects on the Entry of Filovirus Envelope Pseudotyped Virus-Like Particles into Primary Human Macrophages. Stantchev TS, Zack-Taylor A, Mattson N, Strebel K, Broder CC, Clouse KA. Viruses. 2019 Sep 23;11(10). pii: E889. doi: 10.3390/v11100889.
- Exposure to TNF antagonist therapies induces variations of the gut microbiota in an in vivo model using healthy mice. Gabay O, Vicenty J, Zack-Taylor A, Tiffany L, Wunderlin G, Smith D, Reyes-Munoz L, Edwards V, Wu WW, Phue JN, Santana-Quintero L, Lam PV, Clouse KA. Joint Bone Spine. 2019 Sep 9. pii: S1297-319X(19)30119-8. doi: 10.1016/j.jbspin.2019.08.001.
- Global Variation in Opioid Use in Prostate Cancer Trials. Roydhouse JK, Suzman DL, Menapace LA, Mishra-Kalyani PS, Sridhara R, Blumenthal GM, Beaver JA, Pazdur R, Kluetz PG. JAMA Oncol. 2019 Sep 12:e192971. doi: 10.1001/ jamaoncol.2019.2971.
- Novel reference genes in colorectal cancer identify a distinct subset of high stage tumors and their associated histologically normal colonic tissues. Xu L, Luo H, Wang R, Wu WW, Phue JN, Shen RF, Juhl H, Wu L, Alterovitz WL, Simonyan V, Pelosof L, Rosenberg AS. BMC Med Genet. 2019 Aug 13;20(1):138. doi: 10.1186/s12881-019-0867-y.
- Characterizing the Feasibility and Performance of Real-World Tumor Progression End Points and Their Association With Overall Survival in a Large Advanced Non-Small-Cell Lung Cancer Data Set. Griffith SD, Miksad RA, Calkins G, You P, Lipitz NG, Bourla AB, Williams E, George DJ, Schrag D, Khozin S, Capra WB, Taylor MD, Abernethy AP. JCO Clin Cancer Inform. 2019 Aug;3:1-13. doi: 10.1200/CCI.19.00013.
- Analyzing patient-reported outcome data when completion differs between arms in open-label trials: an application of principal stratification. Roydhouse JK, Gutman R, Bhatnagar V, Kluetz PG, Sridhara R, Mishra- Kalyani PS. Pharmacoepidemiol Drug Saf. 2019 Oct;28(10):1386-1394. doi: 10.1002/pds.4875.
- Novel reference genes in colorectal cancer identify a distinct subset of high stage tumors and their associated histologically normal colonic tissues. Xu L, Luo H, Wang R, Wu WW, Phue JN, Shen RF, Juhl H, Wu L, Alterovitz WL, Simonyan V, Pelosof L, Rosenberg AS. BMC Med Genet. 2019 Aug 13;20(1):138. doi: 10.1186/s12881-019-0867-y.
- Characterizing the Feasibility and Performance of Real-World Tumor Progression End Points and Their Association With Overall Survival in a Large Advanced Non-Small-Cell Lung Cancer Data Set. Griffith SD, Miksad RA, Calkins G, You P, Lipitz NG, Bourla AB, Williams E, George DJ, Schrag D, Khozin S, Capra WB, Taylor MD, Abernethy AP. JCO Clin Cancer Inform. 2019 Aug;3:1-13. doi: 10.1200/CCI.19.00013.
- Real-world progression, treatment, and survival outcomes during rapid adoption of immunotherapy for advanced non-small cell lung cancer. Khozin S, Miksad RA, Adami J, Boyd M, Brown NR, Gossai A, Kaganman I, Kuk D, Rockland JM, Pazdur R, Torres AZ, Zhi J, Abernethy AP. Cancer. 2019 Nov 15;125(22):4019-4032. doi: 10.1002/ cncr.32383.
- Prognostic Value of the Lung Immune Prognostic Index for Patients Treated for Metastatic Non-Small Cell Lung Cancer. Kazandjian D, Gong Y, Keegan P, Pazdur R, Blumenthal GM. JAMA Oncol. 2019 Jul 25. doi: 10.1001/ jamaoncol.2019.1747.
- An Exploratory Analysis of Real-World End Points for Assessing Outcomes Among Immunotherapy-Treated Patients With Advanced Non-Small-Cell Lung Cancer. Stewart M, Norden AD, Dreyer N, Henk HJ, Abernethy AP, Chrischilles E, Kushi L, Mansfield AS, Khozin S, Sharon E, Arunajadai S, Carnahan R, Christian JB, Miksad RA, Sakoda LC, Torres AZ, Valice E, Allen J. JCO Clin Cancer Inform. 2019 Jul;3:1-15. doi: 10.1200/CCI.18.00155.
- An FDA oncology analysis of toxicities associated with PBD-containing antibody-drug conjugates. Saber H, Simpson N, Ricks TK, Leighton JK. Regul Toxicol Pharmacol. 2019 Oct;107:104429. doi: 10.1016/j. yrtph.2019.104429.
- Generating Real-World Tumor Burden Endpoints from Electronic Health Record Data: Comparison of RECIST, Radiology-Anchored, and Clinician-Anchored Approaches for Abstracting Real-World Progression in Non-Small Cell Lung Cancer. Griffith SD, Tucker M, Bowser B, Calkins G, Chang CJ, Guardino E, Khozin S, Kraut J, You P, Schrag D, Miksad RA. Adv Ther. 2019 Aug;36(8):2122-2136. doi: 10.1007/s12325-019-00970-1.
- Identification and quantification of defective virus genomes in high throughput sequencing data using DVG-profiler, a novel post-sequence alignment processing algorithm. Bosma TJ, Karagiannis K, Santana-Quintero L, Ilyushina N, Zagorodnyaya T, Petrovskaya S, Laassri M, Donnelly RP, Rubin S, Simonyan V, Sauder CJ. PLoS One. 2019 May 17;14(5):e0216944. doi: 10.1371/journal.pone.0216944.
- Investigating Potential Bias in Patient-Reported Outcomes in Open-label Cancer Trials. Roydhouse JK, Fiero MH, Kluetz PG. JAMA Oncol. 2019 Apr 1;5(4):457-458. doi: 10.1001/jamaoncol.2018.6205.
- CDK4/6 inhibitor treatment for patients with hormone receptor-positive, HER2-negative, advanced or metastatic breast cancer: a US Food and Drug Administration pooled analysis. Gao JJ, Cheng J, Bloomquist E, Sanchez J, Wedam SB, Singh H, Amiri-Kordestani L, Ibrahim A, Sridhara R, Goldberg KB, Theoret MR, Kluetz PG, Blumenthal GM, Pazdur R, Beaver JA, Prowell TM. Lancet Oncol. 2019 Dec 16. pii: S1470-2045(19)30804-6. doi: 10.1016/ S1470-2045(19)30804-6.
- Idelalisib for Treatment of Relapsed Follicular Lymphoma and Chronic Lymphocytic Leukemia: A Comparison of Treatment Outcomes in Clinical Trial Participants vs Medicare Beneficiaries. Bird ST, Tian F, Flowers N, Przepiorka D, Wang R, Jung TH, Kessler Z, Woods C, Kim B, Miller BW, Wernecke M, Kim C, McKean S, Gelperin K, MaCurdy TE, Kelman JA, Graham DJ. JAMA Oncol. 2019 Dec 19. doi: 10.1001/jamaoncol.2019.3994.
- Evaluation of Serious Postmarket Safety Signals Within 2 Years of FDA Approval for New Cancer Drugs. Kim J, Nair A, Keegan P, Beaver JA, Kluetz PG, Pazdur R, Chuk M, Blumenthal GM. Oncologist. 2019 Dec 17. pii: theoncologist.2019-0653. doi: 10.1634/theoncologist.2019-0653.
- End points for sickle cell disease clinical trials: patient-reported outcomes, pain, and the brain. Farrell AT, Panepinto J, Carroll CP, Darbari DS, Desai AA, King AA, Adams RJ, Barber TD, Brandow AM, DeBaun MR, Donahue MJ, Gupta K, Hankins JS, Kameka M, Kirkham FJ, Luksenburg H, Miller S, Oneal PA, Rees DC, Setse R, Sheehan VA, Strouse J, Stucky CL, Werner EM, Wood JC, Zempsky WT. Blood Adv. 2019 Dec 10;3(23):3982-4001. doi: 10.1182/bloodadvances.2019000882.
- End points for sickle cell disease clinical trials: renal and cardiopulmonary, cure, and low-resource settings. Farrell AT, Panepinto J, Desai AA, Kassim AA, Lebensburger J, Walters MC, Bauer DE, Blaylark RM, DiMichele DM, Gladwin MT, Green NS, Hassell K, Kato GJ, Klings ES, Kohn DB, Krishnamurti L, Little J, Makani J, Malik P, McGann PT, Minniti C, Morris CR, Odame I, Oneal PA, Setse R, Sharma P, Shenoy S. Blood Adv. 2019 Dec 10;3(23):4002- 4020. doi: 10.1182/bloodadvances.2019000883.
Approval Summaries
- Approvals in 2018: a histology-agnostic new molecular entity, novel end points and real-time review. Blumenthal GM, Pazdur R. Nat Rev Clin Oncol. 2019 Mar;16(3):139-141. doi: 10.1038/s41571-019-0170-z.
- FDA Approval Summary: Palbociclib for Male Patients with Metastatic Breast Cancer. Wedam S, Fashoyin-Aje L, Bloomquist E, Tang S, Sridhara R, Goldberg KB, Theoret MR, Amiri-Kordestani L, Pazdur R, Beaver JA. Clin Cancer Res. 2019 Oct 24. pii: clincanres.2580.2019. doi: 10.1158/1078-0432.CCR-19-2580.
- FDA Approval Summary: Ruxolitinib for Treatment of Steroid-Refractory Acute Graft-Versus-Host Disease. Przepiorka D, Luo L, Subramaniam S, Qiu J, Gudi R, Cunningham LC, Nie L, Leong R, Ma L, Sheth C, Deisseroth A, Goldberg KB, Blumenthal GM, Pazdur R. Oncologist. 2019 Oct 22. pii: theoncologist.2019-0627. doi: 10.1634/ theoncologist.2019-0627.
- FDA Approval Summary: Tagraxofusp-erzs For Treatment of Blastic Plasmacytoid Dendritic Cell Neoplasm. Jen EY, Gao X, Li L, Zhuang L, Simpson NE, Aryal B, Wang R, Przepiorka D, Shen YL, Leong R, Liu C, Sheth CM, Bowen S, Goldberg KB, Farrell AT, Blumenthal GM, Pazdur R. Clin Cancer Res. 2019 Sep 23. doi: 10.1158/1078-0432.CCR- 19-2329.
- Recent drug approvals for acute myeloid leukemia. Lai C, Doucette K, Norsworthy K. J Hematol Oncol. 2019 Sep 18;12(1):100. doi: 10.1186/s13045-019-0774-x.
- FDA Approval Summary: Calaspargase Pegol-mknl For Treatment of Acute Lymphoblastic Leukemia in Children and Young Adults. Li RJ, Jin R, Liu C, Cao X, Manning ML, Di XM, Przepiorka D, Namuswe F, Deisseroth A, Goldberg KB, Blumenthal GM, Pazdur R. Clin Cancer Res. 2019 Aug 23. doi: 10.1158/1078-0432.CCR-19-1255.
- FDA Approval Summary: Mogamulizumab-kpkc for Mycosis Fungoides and Sézary Syndrome. Kasamon YL, Chen H, de Claro RA, Nie L, Ye J, Blumenthal GM, Farrell AT, Pazdur R. Clin Cancer Res. 2019 Jul 31. doi: 10.1158/1078-0432.CCR-19-2030.
- FDA Approval Summary: Glasdegib for Newly Diagnosed Acute Myeloid Leukemia. Norsworthy KJ, By K, Subramaniam S, Zhuang L, Del Valle PL, Przepiorka D, Shen YL, Sheth CM, Liu C, Leong R, Goldberg KB, Farrell AT, Pazdur R. Clin Cancer Res. 2019 Oct 15;25(20):6021-6025. doi: 10.1158/1078-0432.CCR-19-0365.
- U.S. Food and Drug Administration Benefit-Risk Assessment of Nilotinib Treatment Discontinuation in Patients with Chronic Phase Chronic Myeloid Leukemia in a Sustained Molecular Remission. Pulte ED, Wroblewski T, Bloomquist E, Tang S, Farrell A, Deisseroth A, McKee AE, Pazdur R. Oncologist. 2019 May;24(5):e188-e195. doi: 10.1634/theoncologist.2018-0759. Epub 2019 Apr 24.
- FDA Approval Summary: Brentuximab Vedotin in First-Line Treatment of Peripheral T-Cell Lymphoma. Richardson NC, Kasamon YL, Chen H, de Claro RA, Ye J, Blumenthal GM, Farrell AT, Pazdur R. Oncologist. 2019 May;24(5):e180-e187. doi: 10.1634/theoncologist.2019-0098.
- FDA Approval Summary: Pembrolizumab for the Treatment of Microsatellite Instability-High Solid Tumors. Marcus L, Lemery SJ, Keegan P, Pazdur R. Clin Cancer Res. 2019 Jul 1;25(13):3753-3758. doi: 10.1158/1078- 0432.CCR-18-4070.
- FDA Approval Summary: Ivosidenib for Relapsed or Refractory Acute Myeloid Leukemia with an Isocitrate Dehydrogenase-1 Mutation. Norsworthy KJ, Luo L, Hsu V, Gudi R, Dorff SE, Przepiorka D, Deisseroth A, Shen YL, Sheth CM, Charlab R, Williams GM, Goldberg KB, Farrell AT, Pazdur R. Clin Cancer Res. 2019 Jun 1;25(11):3205- 3209. doi: 10.1158/1078-0432.CCR-18-3749.
Regulatory Perspectives
- Application of the Lung Immune Prognostic Index From Research to Clinical Practice-Reply. Kazandjian D, Gong Y, Blumenthal GM. JAMA Oncol. 2019 Nov 27. doi: 10.1001/jamaoncol.2019.5157.
- Reply to H. McLeod et al. Weinstock C, Fernandes LL, Theoret M, Tang S, Sridhara R, Beaver JA, Pazdur R. J Clin Oncol. 2019 Nov 1:JCO1902429. doi: 10.1200/JCO.19.02429.
- Pivotal Considerations for Optimal Deployment of Healthy Volunteers in Oncology Drug Development. Ahmed MA, Patel C, Drezner N, Helms W, Tan W, Stypinski D. Clin Transl Sci. 2019 Oct 31. doi: 10.1111/cts.12703. Review.
- Assessing an ASCO Decision Aid for Improving the Accuracy and Attribution of Serious Adverse Event Reporting From Investigators to Sponsors. Mileham KF, Schenkel C, Chuk MK, Buchmeier A, Perez RP, Hurley P, Levit LA, Garrett-Mayer E, Davis C, Bruinooge SS, Vose J. J Oncol Pract. 2019 Oct 24:JOP1900366. doi: 10.1200/ JOP.19.00366.
- Public-private partnerships in transplant drug development. Albrecht R, Papadopoulos EJ, Campbell M, Daniels S, Kluetz PG, Parekh A, Wang Y. Am J Transplant. 2019 Sep 25. doi: 10.1111/ajt.15604.
- Development of Treatments for Localized Prostate Cancer in Patients Eligible for Active Surveillance: U.S. Food and Drug Administration Oncology Center of Excellence Public Workshop. Weinstock C, Suzman D, Kluetz P, Baxley J, Viviano C, Ibrahim A, Jarow J, Sridhara R, Liu K, Carroll P, Eggener S, Hu JC, Hussain M, King M, Klein E, Kungel T, Makarov D, Pinto PA, Rini B, Roach M, Sandler H, Schlegel PN, Song D, Goldberg K, Pazdur R, Beaver JA. J Urol. 2020 Jan;203(1):115-119. doi: 10.1097/JU.0000000000000532.
- Radiologic Considerations and Standardization of Malignant Pleural Mesothelioma Imaging Within Clinical Trials: Consensus Statement from the NCI Thoracic Malignancy Steering Committee - International Association for the Study of Lung Cancer - Mesothelioma Applied Research Foundation Clinical Trials Planning Meeting. Gill RR, Tsao AS, Kindler HL, Richards WG, Armato SG 3rd, Francis RJ, Gomez DR, Dahlberg S, Rimner A, Simone CB 2nd, de Perrot M, Blumenthal G, Adjei AA, Bueno R, Harpole DH Jr, Hesdorffer M, Hirsch FR, Pass HI, Yorke E, Rosenzweig K, Burt B, Fennell DA, Lindwasser W, Malik S, Peikert T, Mansfield AS, Salgia R, Yang H, Rusch VW, Nowak AK. J Thorac Oncol. 2019 Oct;14(10):1718-1731. doi: 10.1016/j.jtho.2019.08.012.
- Myocarditis in the Setting of Cancer Therapeutics: Proposed Case Definitions for Emerging Clinical Syndromes in Cardio-Oncology Bonaca MP, Olenchock BA, Salem JE, Wiviott SD, Ederhy S, Cohen A, Stewart GC, Choueiri TK, Di Carli M, Allenbach Y, Kumbhani DJ, Heinzerling L, Amiri-Kordestani L, Lyon AR, Thavendiranathan P, Padera R, Lichtman A, Liu PP, Johnson DB, Moslehi J. Circulation. 2019 Jul 2;140(2):80-91.
- Artificial intelligence and machine learning in clinical development: a translational perspective. Shah P, Kendall F, Khozin S, Goosen R, Hu J, Laramie J, Ringel M, Schork N. NPJ Digit Med. 2019 Jul 26;2:69. doi: 10.1038/s41746- 019-0148-3. eCollection 2019.
- Using a Benefit Risk Analysis Approach To Capture Regulatory Decision-Making: Renal Cell Carcinoma. Raju GK, Gurumurthi K, Domike R, Weinstock C, Singh H, Kluetz P, Pazdur R, Woodcock J. Clin Pharmacol Ther. 2019 Jul 29. doi: 10.1002/cpt.1589.
- Cancer and aging activities at the American Society of Clinical Oncology and beyond: Reflections on the legacy of Dr. Arti Hurria. Levit LA, Singh H, Klepin HD. J Geriatr Oncol. 2019 Jul 15. pii: S1879-4068(19)30302-9. doi: 10.1016/j.jgo.2019.07.007.
- Erratum: Author Correction: Developing and adopting safe and effective digital biomarkers to improve patient outcomes. Coravos A, Khozin S, Mandl KD. NPJ Digit Med. 2019 May 10;2:40. doi: 10.1038/s41746-019-0119-8. eCollection 2019.
- Assessing cardiac safety in oncology drug development. Seltzer JH, Gintant G, Amiri-Kordestani L, Singer J, Koplowitz LP, Moslehi JJ, Barac A, Yu AF. Am Heart J. 2019 Aug;214:125-133. doi: 10.1016/j.ahj.2019.04.010. Epub 2019 May 4. Review. No abstract available. Erratum in: Am Heart J. 2019 Dec;218:133.
- Cancer drugs approved for use in children: Impact of legislative initiatives and future opportunities. Barone A, Casey D, McKee AE, Reaman G. Pediatr Blood Cancer. 2019 Aug;66(8):e27809. doi: 10.1002/pbc.27809.
- Advancing Drug Development in Gynecologic Malignancies. Beaver JA, Coleman RL, Arend RC, Armstrong DK, Bala S, Mills GB, Sood AK, Herzog TJ. Clin Cancer Res. 2019 Aug 15;25(16):4874-4880. doi: 10.1158/1078-0432. CCR-19-0619.
- Preparing Fellows for Graduation: Perspectives on Career Guidance. LaCasce A, Graff S, Gao J, Close J, Boulmay B. Am Soc Clin Oncol Educ Book. 2019 Jan;39:609-614. doi: 10.1200/EDBK_242603.
- Patient-Friendly Language to Facilitate Treatment Choice for Patients with Cancer. Kim J, Gao J, Amiri-Kordestani L, Beaver JA, Kluetz P. Oncologist. 2019 Aug;24(8):1011-1012. doi: 10.1634/theoncologist.2018-0761.
- Decentralized Trials in the Age of Real-World Evidence and Inclusivity in Clinical Investigations. Khozin S, Coravos A. Clin Pharmacol Ther. 2019 Jul;106(1):25-27. doi: 10.1002/cpt.1441. Epub 2019 Apr 23. No abstract available.
- Using a Benefit-Risk Analysis Approach to Capture Regulatory Decision Making: Melanoma. Raju GK, Gurumurthi K, Domike R, Theoret MR, Pazdur R, Woodcock J. Clin Pharmacol Ther. 2019 Jul;106(1):123-135. doi: 10.1002/ cpt.1461.
- Current Landscape of Immunotherapy in Breast Cancer: A Review. Adams S, Gatti-Mays ME, Kalinsky K, Korde LA, Sharon E, Amiri-Kordestani L, Bear H, McArthur HL, Frank E, Perlmutter J, Page DB, Vincent B, Hayes JF, Gulley JL, Litton JK, Hortobagyi GN, Chia S, Krop I, White J, Sparano J, Disis ML, Mittendorf EA. JAMA Oncol. 2019 Apr 11. doi: 10.1001/jamaoncol.2018.7147.
- Proceedings of a Workshop: US Food and Drug Administration-International Society of Pharmacometrics Model- Informed Drug Development in Oncology. Bruno R, Jin JY, Maxfield K, Milligan L, Liu C, Wang Y, McKee AE, Zineh I. Clin Pharmacol Ther. 2019 Jul;106(1):81-83. doi: 10.1002/cpt.1421.
- Developing and adopting safe and effective digital biomarkers to improve patient outcomes. Coravos A, Khozin S, Mandl KD. NPJ Digit Med. 2019;2(1). pii: 14. doi: 10.1038/s41746-019-0090-4. Epub 2019 Mar 11. Erratum in: NPJ Digit Med. 2019 May 10;2:40.
- US Cancer Centers of Excellence Strategies for Increased Inclusion of Racial and Ethnic Minorities in Clinical Trials. Regnante JM, Richie NA, Fashoyin-Aje L, Vichnin M, Ford M, Roy UB, Turner K, Hall LL, Gonzalez E, Esnaola N, Clark LT, Adams HC 3rd, Alese OB, Gogineni K, McNeill L, Petereit D, Sargeant I, Dang J, Obasaju C, Highsmith Q, Lee SC, Hoover SC, Williams EL, Chen MS Jr. J Oncol Pract. 2019 Apr;15(4):e289-e299. doi: 10.1200/ JOP.18.00638. Epub 2019 Mar 4.
- ACCELERATE and European Medicine Agency Paediatric Strategy Forum for medicinal product development for mature B-cell malignancies in children. Pearson ADJ, Scobie N, Norga K, Ligas F, Chiodin D, Burke A, Minard-Colin V, Adamson P, Marshall LV, Balakumaran A, Benettaib B, Bhargava P, Bollard CM, Bolotin E, Bomken S, Buechner J, Burkhardt B, Caron H, Copland C, Demolis P, Egorov A, Farhan M, Zugmaier G, Gross T, Horton-Taylor D, Klapper W, Lesa G, Marcus R, Miles RR, Nottage K, Pacaud L, Ricafort R, Schrappe M, Sterba J, Vezan R, Weiner S, Kim SY, Reaman G, Vassal G. Eur J Cancer. 2019 Mar;110:74-85. doi: 10.1016/j.ejca.2019.01.013. Epub 2019 Feb 14.
- Residual Disease after Neoadjuvant Therapy - Developing Drugs for High-Risk Early Breast Cancer. Prowell TM, Beaver JA, Pazdur R. N Engl J Med. 2019 Feb 14;380(7):612-615. doi: 10.1056/NEJMp1900079. No abstract available.
- Model-Informed Drug Development Approach to Expedite Approval: Case of Alectinib in First-Line Anaplastic Lymphoma Kinase + Non-Small Cell Lung Cancer. Morcos PN, Liu J, Blumenthal GM, Zhao H. Clin Pharmacol Ther. 2019 Apr;105(4):826-828. doi: 10.1002/cpt.1340.
- Challenges with Novel Clinical Trial Designs: Master Protocols. Cecchini M, Rubin EH, Blumenthal GM, Ayalew K, Burris HA, Russell-Einhorn M, Dillon H, Lyerly HK, Reaman GH, Boerner S, LoRusso PM. Clin Cancer Res. 2019 Apr 1;25(7):2049-2057. doi: 10.1158/1078-0432.CCR-18-3544.
Research Papers
- Evaluation of Overall Response Rate and Progression-Free Survival as Potential Surrogate Endpoints for Overall Survival in Immunotherapy Trials. Mushti SL, Mulkey F, Sridhara R. Clin Cancer Res. 2018 May 15;24(10):2268-2275.
- Patients with melanoma treated with an anti-PD-1 antibody beyond RECIST progression: a US Food and Drug Administration pooled analysis. Beaver JA, Hazarika M, Mulkey F, Mushti S, Chen H, He K, Sridhara R, Goldberg KB, Chuk MK, Chi DC, Chang J, Barone A, Balasubramaniam S, Blumenthal GM, Keegan P, Pazdur R, Theoret MR. Lancet Oncol. 2018 Feb;19(2):229-239. doi: 10.1016/S1470-2045(17)30846-X.
- Real-World Outcomes of Patients with Metastatic Non-Small Cell Lung Cancer Treated with Programmed Cell Death Protein 1 Inhibitors in the Year Following U.S. Regulatory Approval. Khozin S, Carson KR, Zhi J, Tucker M, Lee SE, Light DE, Curtis MD, Bralic M, Kaganman I, Gossai A, Hofmeister P, Torres AZ, Miksad RA, Blumenthal GM, Pazdur R, Abernethy AP. Oncologist. 2018 Dec 27. pii: theoncologist.2018-0307. doi: 10.1634/theoncologist.2018-0307.
- FDA analyses of survival in older adults with metastatic non-small cell lung cancer in controlled trials of PD-1/PD-L1 blocking antibodies. Marur S, Singh H, Mishra-Kalyani P, Larkins E, Keegan P, Sridhara R, Blumenthal GM, Pazdur R. Semin Oncol. 2018 Aug;45(4):220-225. doi: 10.1053/j.seminoncol.2018.08.007.
- Upper gastrointestinal acute graft-versus-host disease adds minimal prognostic value in isolation or with other graft-versus-host disease symptoms as currently diagnosed and treated. Nikiforow S, Wang T, Hemmer M, Spellman S, Akpek G, Antin JH, Choi SW, Inamoto Y, Khoury HJ, MacMillan M, Marks DI, Meehan K, Nakasone H, Nishihori T, Olsson R, Paczesny S, Przepiorka D, Reddy V, Reshef R, Schoemans H, Waller N, Weisdorf D, Wirk B, Horowitz M, Alousi A, Couriel D, Pidala J, Arora M, Cutler C; GV12-02 Writing Committee on behalf of the CIBMTR® Graft-versus-Host Disease Working Committee. Haematologica. 2018 Oct;103(10):1708-1719. doi: 10.3324/haematol.2017.182550.
- Wound healing complications with lenvatinib identified in a pharmacovigilance database. Cheng C, Nayernama A, Christopher Jones S, Casey D, Waldron PE. J Oncol Pharm Pract. 2018 Nov 30:1078155218817109. doi: 10.1177/1078155218817109.
- Ipilimumab plus nivolumab and DNA-repair defects in AR-V7-expressing metastatic prostate cancer. Boudadi K, Suzman DL, Anagnostou V, Fu W, Luber B, Wang H, Niknafs N, White JR, Silberstein JL, Sullivan R, Dowling D, Harb R, Nirschl TR, Veeneman BA, Tomlins SA, Wang Y, Jendrisak A, Graf RP, Dittamore R, Carducci MA, Eisenberger MA, Haffner MC, Meeker AK, Eshleman JR, Luo J, Velculescu VE, Drake CG, Antonarakis ES. Oncotarget. 2018 Jun 19;9(47):28561-28571. doi: 10.18632/oncotarget.25564.
- In-Depth Comparative Analysis of Illumina® MiSeq Run Metrics: Development of a Wet-lab Quality Assessment Tool. Kastanis GJ, Santana-Quintero LV, Sanchez-Leon M, Lomonaco S, Brown EW, Allard MW. Mol Ecol Resour. 2018 Dec 2. doi: 10.1111/1755-0998.12973.
- Phase I/II Trial of Vandetanib and Bortezomib in Adults with Locally Advanced or Metastatic Medullary Thyroid Cancer. Del Rivero J, Edgerly M, Ward J, Madan RA, Balasubramaniam S, Fojo T, Gramza AW. Oncologist. 2019 Jan;24(1):16-e14. doi: 10.1634/theoncologist.2018-0452.
- Target-Adverse Event Profiles to Augment Pharmacovigilance: A Pilot Study With Six New Molecular Entities. Schotland P, Racz R, Jackson D, Levin R, Strauss DG, Burkhart K. CPT Pharmacometrics Syst Pharmacol. 2018 Dec;7(12):809-817. doi: 10.1002/psp4.12356.
- A population pharmacokinetic/toxicity model for the reduction of platelets during a 48-h continuous intravenous infusion of the histone deacetylase inhibitor belinostat. Peer CJ, Hall OM, Sissung TM, Piekarz R, Balasubramaniam S, Bates SE, Figg WD. Cancer Chemother Pharmacol. 2018 Sep;82(3):565-570. doi: 10.1007/s00280-018-3631-7.
- Ibrutinib-associated ventricular arrhythmia in the FDA Adverse Event Reporting System. Cheng C, Woronow D, Nayernama A, Wroblewski T, Jones SC. Leuk Lymphoma. 2018 Dec;59(12):3016-3017. doi: 10.1080/10428194.2018.1457149.
- Exploration of a Novel Intermediate Response Endpoint in Immunotherapy Clinical Studies. Gao X, Zhang L, Sridhara R. Clin Cancer Res. 2018 May 15;24(10):2262-2267.
- US Food and Drug Administration Pooled Analysis to Assess the Impact of Bone-Only Metastatic Breast Cancer on Clinical Trial Outcomes and Radiographic Assessments. Wedam SB, Beaver JA, Amiri-Kordestani L, Bloomquist E, Tang S, Goldberg KB, Sridhara R, Ibrahim A, Kim G, Kluetz P, McKee A, Pazdur R. J Clin Oncol. 2018 Apr 20;36(12):1225-1231. doi: 10.1200/JCO.2017.74.6917.
- Survival of ethnic and racial minority patients with multiple myeloma treated with newer medications. Pulte ED, Nie L, Gormley N, Goldberg KB, McKee A, Farrell A, Pazdur R. Blood Adv. 2018 Jan 23;2(2):116-119. doi: 10.1182/bloodadvances.2017010512.
- A multistate investigation of health care-associated Burkholderia cepacia complex infections related to liquid docusate sodium contamination, January-October 2016. Glowicz J, Crist M, Gould C, Moulton-Meissner H, Noble-Wang J, de Man TJB, Perry KA, Miller Z, Yang WC, Langille S, Ross J, Garcia B, Kim J, Epson E, Black S, Pacilli M, LiPuma JJ, Fagan R; B. cepacia Investigation Workgroup. Am J Infect Control. 2018 Jun;46(6):649-655. doi: 10.1016/j.ajic.2017.11.018.
- Characteristics of Real-World Metastatic Non-Small Cell Lung Cancer Patients Treated with Nivolumab and Pembrolizumab During the Year Following Approval. Khozin S, Abernethy AP, Nussbaum NC, Zhi J, Curtis MD, Tucker M, Lee SE, Light DE, Gossai A, Sorg RA, Torres AZ, Patel P, Blumenthal GM, Pazdur R. Oncologist. 2018 Mar;23(3):328-336. doi: 10.1634/theoncologist.2017-0353.
Approval Summaries
- Approvals in 2017: gene therapies and site-agnostic indications. Blumenthal GM, Pazdur R. Nat Rev Clin Oncol. 2018 Mar;15(3):127-128. doi: 10.1038/nrclinonc.2018.11.
- Overview of Oncology and Hematology Drug Approvals at US Food and Drug Administration Between 2008 and 2016. Zhou J, Vallejo J, Kluetz P, Pazdur R, Kim T, Keegan P, Farrell A, Beaver JA, Sridhara R. J Natl Cancer Inst. 2018 Aug 4. doi: 10.1093/jnci/djy130.
- FDA Approval Summary: Pertuzumab for adjuvant treatment of HER2-positive early breast cancer. Howie LJ, Scher NS, Amiri-Kordestani L, Zhang L, King-Kallimanis BL, Choudhry Y, Schroeder J, Goldberg KB, Kluetz PG, Ibrahim A, Sridhara R, Blumenthal GM, Pazdur R, Beaver JA. Clin Cancer Res. 2018 Dec 14. pii: clincanres.3003.2018. doi: 10.1158/1078-0432.CCR-18-3003.
- FDA Approval Summary: Atezolizumab or Pembrolizumab for the Treatment of Patients with Advanced Urothelial Carcinoma Ineligible for Cisplatin-Containing Chemotherapy. Suzman DL, Agrawal S, Ning YM, Maher VE, Fernandes LL, Karuri S, Tang S, Sridhara R, Schroeder J, Goldberg KB, Ibrahim A, McKee AE, Pazdur R, Beaver JA. Oncologist. 2018 Dec 12. pii: theoncologist.2018-0084. doi: 10.1634/theoncologist.2018-0084.
- FDA Approval Summary: (Daunorubicin and Cytarabine) Liposome for Injection for the Treatment of Adults with High-Risk Acute Myeloid Leukemia. Krauss AC, Gao X, Li L, Manning ML, Patel P, Fu W, Janoria KG, Gieser G, Bateman DA, Przepiorka D, Shen YL, Shord SS, Sheth CM, Banerjee A, Liu J, Goldberg KB, Farrell AT, Blumenthal GM, Pazdur R. Clin Cancer Res. 2018 Dec 12. pii: clincanres.2990.2018. doi: 10.1158/1078-0432.CCR-18-2990.
- FDA Approval Summary: Axicabtagene Ciloleucel for Relapsed or Refractory Large B-Cell Lymphoma. Bouchkouj N, Kasamon YL, de Claro RA, George B, Lin X, Lee S, Blumenthal GM, Bryan W, McKee AE, Pazdur R. Clin Cancer Res. 2018 Nov 9. pii: clincanres.2743.2018. doi: 10.1158/1078-0432.CCR-18-2743.
- FDA Approval Summary: Tisagenlecleucel for Treatment of Patients with Relapsed or Refractory B-cell Precursor Acute Lymphoblastic Leukemia. O'Leary MC, Lu X, Huang Y, Lin X, Mahmood I, Przepiorka D, Gavin D, Lee S, Liu K, George B, Bryan W, Theoret MR, Pazdur R. Clin Cancer Res. 2018 Oct 11. doi: 10.1158/1078-0432.CCR-18-2035.
- FDA Approval: Blinatumomab for Patients with B-cell Precursor Acute Lymphoblastic Leukemia in Morphologic Remission with Minimal Residual Disease. Jen EY, Xu Q, Schetter A, Przepiorka D, Shen YL, Roscoe D, Sridhara R, Deisseroth A, Philip R, Farrell AT, Pazdur R. Clin Cancer Res. 2018 Sep 25. doi: 10.1158/1078-0432.CCR-18-2337.
- FDA Approval Summary: Pembrolizumab for Recurrent Locally Advanced or Metastatic Gastric or Gastroesophageal Junction Adenocarcinoma Expressing PD-L1. Fashoyin-Aje L, Donoghue M, Chen H, He K, Veeraraghavan J, Goldberg KB, Keegan P, McKee AE, Pazdur R. Oncologist. 2019 Jan;24(1):103-109. doi: 10.1634/theoncologist.2018-0221.
- FDA Approval Summary: Vemurafenib for the Treatment of Patients with Erdheim-Chester Disease with the BRAFV600 Mutation. Oneal PA, Kwitkowski V, Luo L, Shen YL, Subramaniam S, Shord S, Goldberg KB, McKee AE, Kaminskas E, Farrell A, Pazdur R. Oncologist. 2018 Aug 17. pii: theoncologist.2018-0295. doi: 10.1634/theoncologist.2018-0295.
- FDA Approval Summary: Midostaurin for the Treatment of Advanced Systemic Mastocytosis. Kasamon YL, Ko CW, Subramaniam S, Ma L, Yang Y, Nie L, Shord S, Przepiorka D, Farrell AT, McKee AE, Pazdur R. Oncologist. 2018 Aug 16. pii: theoncologist.2018-0222. doi: 10.1634/theoncologist.2018-0222.
- FDA Supplemental Approval: Blinatumomab for Treatment of Relapsed and Refractory Precursor B-Cell Acute Lymphoblastic Leukemia. Pulte ED, Vallejo J, Przepiorka D, Nie L, Farrell AT, Goldberg KB, McKee AE, Pazdur R. Oncologist. 2018 Nov;23(11):1366-1371. doi: 10.1634/theoncologist.2018-0179.
- FDA's Approval of the First Biosimilar to Bevacizumab. Casak SJ, Lemery SJ, Chung J, Fuchs C, Schrieber SJ, Chow ECY, Yuan W, Rodriguez L, Gwise T, Rowzee A, Lim S, Keegan P, McKee AE, Pazdur R. Clin Cancer Res. 2018 Sep 15;24(18):4365-4370. doi: 10.1158/1078-0432.CCR-18-0566.
- FDA Approval Summary: Niraparib for the Maintenance Treatment of Patients with Recurrent Ovarian Cancer in Response to Platinum-Based Chemotherapy. Ison G, Howie LJ, Amiri-Kordestani L, Zhang L, Tang S, Sridhara R, Pierre V, Charlab R, Ramamoorthy A, Song P, Li F, Yu J, Manheng W, Palmby TR, Ghosh S, Horne HN, Lee EY, Philip R, Dave K, Chen XH, Kelly SL, Janoria KG, Banerjee A, Eradiri O, Dinin J, Goldberg KB, Pierce WF, Ibrahim A, Kluetz PG, Blumenthal GM, Beaver JA, Pazdur R. Clin Cancer Res. 2018 Sep 1;24(17):4066-4071. doi: 10.1158/1078-0432.CCR-18-0042.
- FDA Approval Summary: Mylotarg for Treatment of Patients with Relapsed or Refractory CD33-Positive Acute Myeloid Leukemia. Norsworthy KJ, Ko CW, Lee JE, Liu J, John CS, Przepiorka D, Farrell AT, Pazdur R. Oncologist. 2018 Sep;23(9):1103-1108. doi: 10.1634/theoncologist.2017-0604.
- FDA Approval Summary: Tocilizumab for Treatment of Chimeric Antigen Receptor T Cell-Induced Severe or Life-Threatening Cytokine Release Syndrome. Le RQ, Li L, Yuan W, Shord SS, Nie L, Habtemariam BA, Przepiorka D, Farrell AT, Pazdur R. Oncologist. 2018 Aug;23(8):943-947. doi: 10.1634/theoncologist.2018-0028.
- U.S. Food and Drug Administration Approval: Neratinib for the Extended Adjuvant Treatment of Early-Stage HER2-Positive Breast Cancer. Singh H, Walker AJ, Amiri-Kordestani L, Cheng J, Tang S, Balcazar P, Barnett-Ringgold K, Palmby TR, Cao X, Zheng N, Liu Q, Yu J, Pierce WF, Daniels SR, Sridhara R, Ibrahim A, Kluetz PG, Blumenthal GM, Beaver JA, Pazdur R. Clin Cancer Res. 2018 Aug 1;24(15):3486-3491. doi: 10.1158/1078-0432.CCR-17-3628.
- FDA Approval: Gemtuzumab Ozogamicin for the Treatment of Adults with Newly Diagnosed CD33-Positive Acute Myeloid Leukemia. Jen EY, Ko CW, Lee JE, Del Valle PL, Aydanian A, Jewell C, Norsworthy KJ, Przepiorka D, Nie L, Liu J, Sheth CM, Shapiro M, Farrell AT, Pazdur R. Clin Cancer Res. 2018 Jul 15;24(14):3242-3246. doi: 10.1158/1078-0432.CCR-17-3179.
- FDA Approval Summary: Lenalidomide as Maintenance Therapy After Autologous Stem Cell Transplant in Newly Diagnosed Multiple Myeloma. Pulte ED, Dmytrijuk A, Nie L, Goldberg KB, McKee AE, Farrell AT, Pazdur R. Oncologist. 2018 Jun;23(6):734-739. doi: 10.1634/theoncologist.2017-0440.
- FDA Approval Summary: Dabrafenib and Trametinib for the Treatment of Metastatic Non-Small Cell Lung Cancers Harboring BRAF V600E Mutations. Odogwu L, Mathieu L, Blumenthal G, Larkins E, Goldberg KB, Griffin N, Bijwaard K, Lee EY, Philip R, Jiang X, Rodriguez L, McKee AE, Keegan P, Pazdur R. Oncologist. 2018 Jun;23(6):740-745. doi: 10.1634/theoncologist.2017-0642.
- FDA Approval: Ribociclib for the Treatment of Postmenopausal Women with Hormone Receptor-Positive, HER2-Negative Advanced or Metastatic Breast Cancer. Shah A, Bloomquist E, Tang S, Fu W, Bi Y, Liu Q, Yu J, Zhao P, Palmby TR, Goldberg KB, Chang CJG, Patel P, Alebachew E, Tilley A, Pierce WF, Ibrahim A, Blumenthal GM, Sridhara R, Beaver JA, Pazdur R. Clin Cancer Res. 2018 Jul 1;24(13):2999-3004. doi: 10.1158/1078-0432.CCR-17-2369.
- Benefit-Risk Summary of Regorafenib for the Treatment of Patients with Advanced Hepatocellular Carcinoma That Has Progressed on Sorafenib. Pelosof L, Lemery S, Casak S, Jiang X, Rodriguez L, Pierre V, Bi Y, Liu J, Zirkelbach JF, Patel A, Goldberg KB, McKee AE, Keegan P, Pazdur R. Oncologist. 2018 Apr;23(4):496-500. doi: 10.1634/theoncologist.2017-0422.
- FDA Benefit-Risk Assessment of Osimertinib for the Treatment of Metastatic Non-Small Cell Lung Cancer Harboring Epidermal Growth Factor Receptor T790M Mutation. Odogwu L, Mathieu L, Goldberg KB, Blumenthal GM, Larkins E, Fiero MH, Rodriguez L, Bijwaard K, Lee EY, Philip R, Fan I, Donoghue M, Keegan P, McKee A, Pazdur R. Oncologist. 2018 Mar;23(3):353-359. doi: 10.1634/theoncologist.2017-0425.
Regulatory Perspectives
- Seamless Designs: Current Practice and Considerations for Early-Phase Drug Development in Oncology. Hobbs BP, Barata PC, Kanjanapan Y, Paller CJ, Perlmutter J, Pond GR, Prowell TM, Rubin EH, Seymour LK, Wages NA, Yap TA, Feltquate D, Garrett-Mayer E, Grossman W, Hong DS, Ivy SP, Siu LL, Reeves SA, Rosner GL. J Natl Cancer Inst. 2018 Dec 17. doi: 10.1093/jnci/djy196.
- Clinical Development of Novel Drug-Radiotherapy Combinations. Ahmad SS, Crittenden MR, Tran PT, Kluetz PG, Blumenthal GM, Bulbeck H, Baird RD, Williams KJ, Illidge T, Hahn S, Lawrence TS, Spears PA, Walker AJ, Sharma RA. Clin Cancer Res. 2018 Nov 29. pii: clincanres.2466.2018. doi: 10.1158/1078-0432.CCR-18-2466.
- Assessing function of electronic health records for real-world data generation. Guinn D, Wilhelm EE, Lieberman G, Khozin S. BMJ Evid Based Med. 2018 Nov 26. pii: bmjebm-2018-111111. doi: 10.1136/bmjebm-2018-111111.
- Summary of the Second Annual BMT CTN Myeloma Intergroup Workshop on Minimal Residual Disease and Immune Profiling. Holstein SA, Ye JC, Howard A, Bhutani M, Gormley N, Hahn T, Hillengass J, Krishnan A, Landgren CO, Munshi NC, Oliva S, Owen RG, Pasquini MC, Puig N, Weinhold N, Weisel K, McCarthy PL. Biol Blood Marrow Transplant. 2018 Nov 5. pii: S1083-8791(18)30694-3. doi: 10.1016/j.bbmt.2018.11.001.
- Tumour-targeting bacteria engineered to fight cancer. Zhou S, Gravekamp C, Bermudes D, Liu K. Nat Rev Cancer. 2018 Dec;18(12):727-743. doi: 10.1038/s41568-018-0070-z.
- Immunotherapy Combinations in Multiple Myeloma - Known Unknowns. Gormley NJ, Pazdur R. N Engl J Med. 2018 Nov 8;379(19):1791-1795. doi: 10.1056/NEJMp1803602.
- Learning from Patients: Reflections on Use of Patient-Reported Outcomes in Lung Cancer Trials. Basu Roy U, King-Kallimanis BL, Kluetz PG, Selig W, Ferris A. J Thorac Oncol. 2018 Dec;13(12):1815-1817. doi: 10.1016/j.jtho.2018.09.003.
- Expanding the Evidence Base in Geriatric Oncology: Action Items From an FDA-ASCO Workshop. Levit LA, Singh H, Klepin HD, Hurria A. J Natl Cancer Inst. 2018 Nov 1;110(11):1163-1170. doi: 10.1093/jnci/djy169.
- Current Status and Future Perspectives on Neoadjuvant Therapy in Lung Cancer. Blumenthal GM, Bunn PA Jr, Chaft JE, McCoach CE, Perez EA, Scagliotti GV, Carbone DP, Aerts HJWL, Aisner DL, Bergh J, Berry DA, Jarkowski A, Botwood N, Cross DAE, Diehn M, Drezner NL, Doebele RC, Blakely CM, Eberhardt WEE, Felip E, Gianni L, Keller SP, Leavey PJ, Malik S, Pignatti F, Prowell TM, Redman MW, Rizvi NA, Rosell R, Rusch V, de Ruysscher D, Schwartz LH, Sridhara R, Stahel RA, Swisher S, Taube JM, Travis WD, Keegan P, Wiens JR, Wistuba II, Wynes MW, Hirsch FR, Kris MG. J Thorac Oncol. 2018 Dec;13(12):1818-1831. doi: 10.1016/j.jtho.2018.09.017.
- Current and Future Management of Malignant Mesothelioma: A Consensus Report from the National Cancer Institute Thoracic Malignancy Steering Committee, International Association for the Study of Lung Cancer, and Mesothelioma Applied Research Foundation. Tsao AS, Lindwasser OW, Adjei AA, Adusumilli PS, Beyers ML, Blumenthal GM, Bueno R, Burt BM, Carbone M, Dahlberg SE, de Perrot M, Fennell DA, Friedberg J, Gill RR, Gomez DR, Harpole DH Jr, Hassan R, Hesdorffer M, Hirsch FR, Hmeljak J, Kindler HL, Korn EL, Liu G, Mansfield AS, Nowak AK, Pass HI, Peikert T, Rimner A, Robinson BWS, Rosenzweig KE, Rusch VW, Salgia R, Sepesi B, Simone CB 2nd, Sridhara R, Szlosarek P, Taioli E, Tsao MS, Yang H, Zauderer MG, Malik SM. J Thorac Oncol. 2018 Nov;13(11):1655-1667. doi: 10.1016/j.jtho.2018.08.2036.
- No Decision Is Final: Career Planning and Career Transitions. Boulmay B, Prowell T, Blaya M, Pietanza MC. Am Soc Clin Oncol Educ Book. 2018 May 23;(38):881-886. doi: 10.1200/EDBK_200983.
- Progress Through Collaboration: An ASCO and U.S. Food and Drug Administration Workshop to Improve the Evidence Base for Treating Older Adults With Cancer. Singh H, Hurria A, Klepin HD. Am Soc Clin Oncol Educ Book. 2018 May 23;(38):392-399. doi: 10.1200/EDBK_201133.
- Complexity of Delivering Precision Medicine: Opportunities and Challenges. Davis AA, McKee AE, Kibbe WA, Villaflor VM. Am Soc Clin Oncol Educ Book. 2018 May 23;(38):998-1007. doi: 10.1200/EDBK_200279.
- Assessing the Risks of Breast Implants and FDA's Vision for the National Breast Implant Registry. Ashar BS. Ann Surg. 2019 Jan;269(1):37-38. doi: 10.1097/SLA.0000000000003052.
- National Cancer Institute Breast Cancer Steering Committee Working Group Report on Meaningful and Appropriate End Points for Clinical Trials in Metastatic Breast Cancer. Seidman AD, Bordeleau L, Fehrenbacher L, Barlow WE, Perlmutter J, Rubinstein L, Wedam SB, Hershman DL, Hayes JF, Butler LP, Smith ML, Regan MM, Beaver JA, Amiri-Kordestani L, Rastogi P, Zujewski JA, Korde LA. J Clin Oncol. 2018 Sep 13:JCO1800242. doi: 10.1200/JCO.18.00242.
- Moving forward toward standardizing analysis of quality of life data in randomized cancer clinical trials. Bottomley A, Pe M, Sloan J, Basch E, Bonnetain F, Calvert M, Campbell A, Cleeland C, Cocks K, Collette L, Dueck AC, Devlin N, Flechtner HH, Gotay C, Greimel E, Griebsch I, Groenvold M, Hamel JF, King M, Kluetz PG, Koller M, Malone DC, Martinelli F, Mitchell SA, Moinpour CM, Musoro JZ, O'Connor D, Oliver K, Piault-Louis E, Piccart M, Pimentel FL, Quinten C, Reijneveld JC, Schürmann C, Smith AW, Soltys KM, Sridhara R, Taphoorn MJB, Velikova G, Coens C. Clin Trials. 2018 Dec;15(6):624-630. doi: 10.1177/1740774518795637.
- White paper on microbial anti-cancer therapy and prevention. Forbes NS, Coffin RS, Deng L, Evgin L, Fiering S, Giacalone M, Gravekamp C, Gulley JL, Gunn H, Hoffman RM, Kaur B, Liu K, Lyerly HK, Marciscano AE, Moradian E, Ruppel S, Saltzman DA, Tattersall PJ, Thorne S, Vile RG, Zhang HH, Zhou S, McFadden G. J Immunother Cancer. 2018 Aug 6;6(1):78. doi: 10.1186/s40425-018-0381-3.
- Determination of Similarity Margin in Comparative Clinical Studies to Support the Development of Biosimilar Products of Neupogen® (Filgrastim, Granulocyte Colony-Stimulating Factor [G-CSF]). Nie L, Przepiorka D, Deisseroth A, Sridhara R, Gwise TE. BioDrugs. 2018 Jul 13. doi: 10.1007/s40259-018-0288-z. [Epub ahead of print]
- Clinical trial design for cutaneous neurofibromas. Cannon A, Jarnagin K, Korf B, Widemann BC, Casey D, Ko HS, Blakeley JO, Verma SK, Pichard DC. Neurology. 2018 Jul 10;91(2 Supplement 1):S31-S37. doi: 10.1212/WNL.0000000000005790.
- Metastasis-free Survival - A New End Point in Prostate Cancer Trials. Beaver JA, Kluetz PG, Pazdur R. N Engl J Med. 2018 Jun 28;378(26):2458-2460. doi: 10.1056/NEJMp1805966.
- Informing the Tolerability of Cancer Treatments Using Patient-Reported Outcome Measures: Summary of an FDA and Critical Path Institute Workshop. Kluetz PG, Kanapuru B, Lemery S, Johnson LL, Fiero MH, Arscott K, Barbachano Y, Basch E, Campbell M, Cappelleri JC, Cella D, Cleeland C, Coens C, Daniels S, Denlinger CS, Fairclough DL, Hillard JR, Minasian L, Mitchell SA, O'Connor D, Patel S, Rubin EH, Ryden A, Soltys K, Sridhara R, Thanarajasingam G, Velikova G, Coons SJ. Value Health. 2018 Jun;21(6):742-747. doi: 10.1016/j.jval.2017.09.009.
- Beyond maximum grade: modernising the assessment and reporting of adverse events in haematological malignancies. Thanarajasingam G, Minasian LM, Baron F, Cavalli F, De Claro RA, Dueck AC, El-Galaly TC, Everest N, Geissler J, Gisselbrecht C, Gribben J, Horowitz M, Ivy SP, Jacobson CA, Keating A, Kluetz PG, Krauss A, Kwong YL, Little RF, Mahon FX, Matasar MJ, Mateos MV, McCullough K, Miller RS, Mohty M, Moreau P, Morton LM, Nagai S, Rule S, Sloan J, Sonneveld P, Thompson CA, Tzogani K, van Leeuwen FE, Velikova G, Villa D, Wingard JR, Wintrich S, Seymour JF, Habermann TM. Lancet Haematol. 2018 Nov;5(11):e563-e598. doi: 10.1016/S2352-3026(18)30051-6.
- Prevention of cisplatin-induced hearing loss in children: Informing the design of future clinical trials. Minasian LM, Frazier AL, Sung L, O'Mara A, Kelaghan J, Chang KW, Krailo M, Pollock BH, Reaman G, Freyer DR. Cancer Med. 2018 May 30. doi: 10.1002/cam4.1563.
- Myocarditis Associated with Immune Checkpoint Inhibitors: An Expert Consensus on Data Gaps and a Call to Action. Neilan TG, Rothenberg ML, Amiri-Kordestani L, Sullivan RJ, Steingart RM, Gregory W, Hariharan S, Hammad TA, Lindenfeld J, Murphy MJ, Moslehi JJ; Checkpoint Inhibitor Safety Working Group. Oncologist. 2018 Aug;23(8):874-878. doi: 10.1634/theoncologist.2018-0157.
- Treatment of Nonmetastatic Unilateral Retinoblastoma in Children. Pérez V, Sampor C, Rey G, Parareda-Salles A, Kopp K, Dabezies AP, Dufort G, Zelter M, López JP, Urbieta M, Alcalde-Ruiz E, Catala-Mora J, Suñol M, Ossandon D, Fandiño AC, Croxatto JO, de Dávila MTG, Reaman G, Ravindranath Y, Chantada GL. JAMA Ophthalmol. 2018 Jul 1;136(7):747-752. doi: 10.1001/jamaophthalmol.2018.1501.
- Making Lung Cancer Clinical Trials More Inclusive: Recommendations for Expanding Eligibility Criteria. Bonomi P, Blumenthal G, Ferris AS, Stewart DJ, Selig WKD, Krug LM, Allen J, Ison G, Langer CJ, Melemed A, Odogwu L, Basu Roy U, Sandler A. J Thorac Oncol. 2018 Jun;13(6):748-751. doi: 10.1016/j.jtho.2018.02.013.
- Accelerating Pediatric Cancer Drug Development: Challenges and Opportunities for Pediatric Master Protocols. Khan T, Stewart M, Blackman S, Rousseau R, Donoghue M, Cohen K, Seibel N, Fleury M, Benettaib B, Malik R, Vassal G, Reaman G. Ther Innov Regul Sci. 2018 Jan 1:2168479018774533. doi: 10.1177/2168479018774533.
- Real-World Evidence In Support Of Precision Medicine: Clinico-Genomic Cancer Data As A Case Study. Agarwala V, Khozin S, Singal G, O'Connell C, Kuk D, Li G, Gossai A, Miller V, Abernethy AP. Health Aff (Millwood). 2018 May;37(5):765-772. doi: 10.1377/hlthaff.2017.1579.
- Incorporating the patient experience into regulatory decision making in the USA, Europe, and Canada. Kluetz PG, O'Connor DJ, Soltys K. Lancet Oncol. 2018 May;19(5):e267-e274. doi: 10.1016/S1470-2045(18)30097-4.
- Anti-PD-1 antibody treatment for melanoma - Authors' reply. Beaver JA, Keegan P, Lemery S, Pazdur R, Theoret MR. Lancet Oncol. 2018 May;19(5):e220. doi: 10.1016/S1470-2045(18)30251-1.
- Breakthrough-Therapy Designation - An FDA Perspective. Corrigan-Curay J, McKee AE, Stein P. N Engl J Med. 2018 Apr 12;378(15):1457-1458. doi: 10.1056/NEJMc1801222.
- INFORMED: an incubator at the US FDA for driving innovations in data science and agile technology. Khozin S, Pazdur R, Shah A. Nat Rev Drug Discov. 2018 Aug;17(8):529-530. doi: 10.1038/nrd.2018.34.
- Hepatotoxicity of immune checkpoint inhibitors: An evolving picture of risk associated with a vital class of immunotherapy agents. Suzman DL, Pelosof L, Rosenberg A, Avigan MI. Liver Int. 2018 Jun;38(6):976-987. doi: 10.1111/liv.13746.
- A 25-Year Experience of US Food and Drug Administration Accelerated Approval of Malignant Hematology and Oncology Drugs and Biologics: A Review. Beaver JA, Howie LJ, Pelosof L, Kim T, Liu J, Goldberg KB, Sridhara R, Blumenthal GM, Farrell AT, Keegan P, Pazdur R, Kluetz PG. JAMA Oncol. 2018 Jun 1;4(6):849-856. doi: 10.1001/jamaoncol.2017.5618.
- Leveraging the Success of HIV Drug Development Paradigms for Cancer. Blumenthal GM, Birnkrant D, Pazdur R. Clin Cancer Res. 2018 Jun 1;24(11):2491-2492. doi: 10.1158/1078-0432.CCR-18-0544.
- Considerations for Developing Targeted Therapies in Low-Frequency Molecular Subsets of a Disease. Schuck RN, Woodcock J, Zineh I, Stein P, Jarow J, Temple R, Permutt T, LaVange L, Beaver JA, Charlab R, Blumenthal GM, Dorff SE, Leptak C, Lemery S, Rogers H, Chowdhury B, Litwack ED, Pacanowski M. Clin Pharmacol Ther. 2018 Aug;104(2):282-289. doi: 10.1002/cpt.1041.
- Guidelines for Inclusion of Patient-Reported Outcomes in Clinical Trial Protocols: The SPIRIT-PRO Extension. Calvert M, Kyte D, Mercieca-Bebber R, Slade A, Chan AW, King MT; the SPIRIT-PRO Group, Hunn A, Bottomley A, Regnault A, Chan AW, Ells C, O'Connor D, Revicki D, Patrick D, Altman D, Basch E, Velikova G, Price G, Draper H, Blazeby J, Scott J, Coast J, Norquist J, Brown J, Haywood K, Johnson LL, Campbell L, Frank L, von Hildebrand M, Brundage M, Palmer M, Kluetz P, Stephens R, Golub RM, Mitchell S, Groves T. JAMA. 2018 Feb 6;319(5):483-494. doi: 10.1001/jama.2017.21903.
- Quantitative Relationship Between AUEC of Absolute Neutrophil Count and Duration of Severe Neutropenia for G-CSF in Breast Cancer Patients. Li L, Ma L, Schrieber SJ, Rahman NA, Deisseroth A, Farrell AT, Wang Y, Sinha V, Marathe A. Clin Pharmacol Ther. 2018 Oct;104(4):742-748. doi: 10.1002/cpt.991.
- The US Food and Drug Administration's Approach for Safe Innovation of Medical Devices in Dermatology. Chang CJ, Ashar BS, Marquart LN. JAMA Dermatol. 2018 Mar 1;154(3):261-263. doi: 10.1001/jamadermatol.2017.5758.
- The FDA NIH Biomarkers, EndpointS, and other Tools (BEST) resource in neuro-oncology. Cagney DN, Sul J, Huang RY, Ligon KL, Wen PY, Alexander BM. Neuro Oncol. 2018 Aug 2;20(9):1162-1172. doi: 10.1093/neuonc/nox242.
- Use of PRO Measures to Inform Tolerability in Oncology Trials: Implications for Clinical Review, IND Safety Reporting, and Clinical Site Inspections. Kim J, Singh H, Ayalew K, Borror K, Campbell M, Johnson LL, Karesh A, Khin NA, Less JR, Menikoff J, Minasian L, Mitchell SA, Papadopoulos EJ, Piekarz RL, Prohaska KA, Thompson S, Sridhara R, Pazdur R, Kluetz PG. Clin Cancer Res. 2018 Apr 15;24(8):1780-1784. doi: 10.1158/1078-0432.CCR-17-2555.
- Pediatric Development of Molecularly Targeted Oncology Drugs. Patel SK, Leong R, Zhao H, Barone A, Casey D, Liu Q, Burckart GJ, Reaman G. Clin Pharmacol Ther. 2018 Aug;104(2):384-389. doi: 10.1002/cpt.942.
- Accelerating Early Access to Immunotherapies for Advanced Urothelial Carcinoma. Ning YM, Maher VE, Beaver JA, Goldberg KB, Blumenthal GM, Pazdur R. Oncologist. 2018 Feb;23(2):139-142. doi: 10.1634/theoncologist.2017-0415.
- The FDA Oncology Center of Excellence and precision medicine. Goldberg KB, Blumenthal GM, McKee AE, Pazdur R. Exp Biol Med (Maywood). 2018 Feb;243(3):308-312. doi: 10.1177/1535370217740861.
- A Benefit-Risk Analysis Approach to Capture Regulatory Decision-Making: Multiple Myeloma. Raju GK, Gurumurthi K, Domike R, Kazandjian D, Landgren O, Blumenthal GM, Farrell A, Pazdur R, Woodcock J. Clin Pharmacol Ther. 2018 Jan;103(1):67-76. doi: 10.1002/cpt.871.
- Join FDA for the First National Hispanic/Latino Family Cancer Awareness Week, Sept. 20-26. Mathieu L, Rivera DR, Blakey R. FDA.gov: FDA Voices, 2024 Sept 12.
- Join Us in Recognizing National Black Family Cancer Awareness Week. Pazdur R, Blakey R. FDA.gov: FDA Voices, 2024 June 4.
- FDA Enhances Global Patient and Regulatory Collaborations in Oncology. Kumar V, Blakey R. FDA.gov: FDA Voices, 2024 Jan 30.
- Facilitating Review of Real-World Data Studies: The Oncology QCARD Initiative. Rivera DR, Kluetz PG. FDA.gov: FDA Voices, 2023 June 27.
- 50 Years of Progress in Treating Patients with Cancer. Pazdur R. FDA.gov: From Our Perspective, 2022 Jan 19.
- FDA’s Oncology Center of Excellence Launches National Black Family Cancer Awareness Week. Pazdur R, Blakey R. FDA.gov: FDA Voices 2021 June 1.
- Project Orbis: Strengthening International Collaboration for Oncology Product Reviews, Faster Patient Access to Innovative Therapies. Guram J, de Claro RA, Pazdur R. FDA.gov: FDA Voices, 2020 Dec 8.
- The FDA's Oncology Center of Excellence — Quantifying the Patient Experience. Kluetz PK. FDA.gov: FDA Voices, 2019 Feb 22.