Oncology Real World Evidence Program
Fostering Regulatory Science and Collaboration to Translate Real World Data into Real World Evidence
FDA defines real-world data (RWD) as data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources and real-world evidence (RWE) as the clinical evidence about the usage and potential benefits or risks of a medical product derived from analysis of RWD.1 [Framework]
In alignment with the 21st Century Cures Act, the goal of the Oncology Center of Excellence Real World Evidence Program is to engage in evidence development modernization through scientific collaboration and policy development to advance the appropriate fit-for-purpose application of RWD to generate RWE for regulatory purposes. Using a systematic approach to regulatory science, the OCE RWE program focuses on evolving scientific knowledge and rigorous methodological use of RWD to advance the development of oncology products in the pre- and post-approval settings.
RWE Mission Statement
Collaboratively advance the appropriate use of real-world evidence in oncology product development to facilitate patient-centered regulatory decision-making.
OCE RWE Strategic Priorities
- Optimize knowledge building through a centralized oncology RWD research portfolio that ensures study efficiency, transparency, and diversity.
- Advance the scientific development of resources, regulatory policy, and guidance on appropriate use of RWD informed by methodological research and collaboration.
- Collaborate through strategic partnerships that foster pragmatic, appropriate use of RWD across FDA, federal agencies and through public private partnerships.
- Accelerate the field of Oncology RWE through leadership and training in rigorous evaluation, methods development, and regulatory science.
Focus Areas
- Regulatory Review
- Regulatory Science Research & Collaboration
- Regulatory Policy
- Education & Engagement
Oncology RWE Program Leaders
Donna R. Rivera, PharmD, MSc, is Associate Director of Pharmacoepidemiology in the OCE and leads the Oncology RWE Program. Dr. Rivera has interests in the use of RWD to increase knowledge of unrepresented populations and advance health equity, observational study designs and RWD methodological approaches, and appropriate uses of RWD for drug development to increase access of effective therapies to patients.
Paul G. Kluetz, MD, is a medical oncologist and the Deputy Director of the OCE. He has a broad interest in trial design and endpoint selection to expedite drug development and define clinical benefit in oncology trials. Some of his initiatives include creating OCE’s patient-focused drug development program, and expanding OCE’s efforts to advance RWE, decentralized trial designs, and digital health technology.
Oncology RWE Program has a variety of mechanisms for collaboration to meet the objectives of the scientific interest areas within the OCE Scientific Collaborative.
Research Collaboration Agreements are binding agreements that allow for collaboration among designated parties to collaborate on a research project where no funding can be transferred.
| Projects | Collaborator |
|---|---|
|
Assessment of real-world endpoints including Real World Response Measuring and addressing health disparities to advance health equity |
Aetion |
|
Patient- and Clinician-reported Performance Status in Patients with Multiple Myeloma Treated in the United States Using Real World Data |
COTA Healthcare |
| Flatiron Health | |
| Syapse |
Centers of Excellence in Regulatory Science and Innovation (CERSI) Program hosted by FDA fosters robust and innovative approaches to advance regulatory science. CERSI projects aim to advance regulatory science through collaborative interactions with FDA scientific experts and funding offices.
| Building Capacity for Research on Cancer and Aging: The UMGCCC-Medicare Database | University of Maryland |
| Whole exome sequencing to determine risk of immune related adverse events and predict treatment response in patients on immune checkpoint inhibitors | UCSF Stanford |
| Real-world outcomes of novel PET imaging tracers for prostate cancer | Yale-Mayo |
U01 Grants
| Oncology Quality, Characterization, and Assessment of Real-world Data (Oncology QCARD) | Reagan-Udall Foundation |
FDA's Broad Agency Announcement is a specialized contract mechanism to spur innovation in regulatory science through extramural research to address FDA's challenges within scientific priority areas.
| Commensurate prior models accommodating historical controls for clinical trials | Medical College of Wisconsin |
| Development of a Novel Methodology for Endpoints Assessing Cardinal Health Response to Lymphoma Treatment in Real-World Studies | Cardinal Health |
| Calibrating RWE studies in oncology against randomized trials | Brigham and Women's Hospital/ Harvard Medical School |
|
Real World Collection of Radiographic Images and Treatment Emergent Adverse Event Data: Next Phase Development of ICAREdata Research Infrastructure |
Alliance NCTN Foundation |
| Analyzing clinical outcomes and genomic data of American Indian patient population treated with immune checkpoint inhibitors for various cancers | Stephenson Cancer Center |
Project Post COVIDity
In the COVID-19 era, there is a need to rapidly develop evidence to characterize and treat patients with COVID-19, especially patients in vulnerable populations such as those with cancer. To develop a longitudinal understanding of the impact of COVID-19 on patients with cancer post infection, including the impact of infection on treatment initiation or delays, sequalae (longitudinal), medical product safety, and vaccination.
- Choueiri TK, Labaki C, Bakouny Z, Hsu CY, Schmidt AL, [et al], Rivera DR, Shyr Y, Farmakiotis D, Warner JL. Breakthrough SARS-CoV-2 infections among patients with cancer following two and three doses of COVID-19 mRNA vaccines: a retrospective observational study from the COVID-19 and Cancer Consortium. Lancet Reg Health Am. 2023 Mar;19:100445. doi: 10.1016/j.lana.2023.100445. Epub 2023 Feb 13. PMID: 36818595; PMCID: PMC9925160.
- 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. Rapid real-world data analysis of patients with cancer, with and without COVID-19, across distinct health systems.Cancer Rep (Hoboken). 2021 May 20:e1388. doi: 10.1002/cnr2.138
Select RWE Publications
- 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. The FDA Oncology Center of Excellence Scientific Collaborative: Charting a Course for Applied Regulatory Science Research in Oncology. Clin Cancer Res. 2021 Apr 28. doi: 10.1158/1078-0432.CCR-20-4429. Online ahead of print.
- Rivera DR, Henk HJ, Garrett-Mayer E, Christian JB, Belli AJ, Bruinooge SS, Espirito JL, Sweetnam C, Izano MA, Natanzon Y, Robert NJ, Walker MS, Cohen AB, Boyd M, Enewold L, Hansen E, Honnold R, Kushi L, Mishra Kalyani PS, Pe Benito R, Sakoda LC, Sharon E, Tymejczyk O, Valice E, Wagner J, Lasiter L, Allen JD. The Friends of Cancer Research Real-World Data Collaboration Pilot 2.0: Methodological Recommendations from Oncology Case Studies. Clin Pharmacol Ther. 2021 Oct 18. doi: 10.1002/cpt.2453. Epub ahead of print. PMID: 34664259.
- Wood WA, Marks P, Plovnick RM, Hewitt K, Neuberg DS, Walters S, Dolan BK, Tucker EA, Abrams CS, Thompson AA, Anderson KC, Kluetz P, Farrell A, Rivera D, Gertzog M, Pappas G. ASH Research Collaborative: a real-world data infrastructure to support real-world evidence development and learning healthcare systems in hematology. Blood Adv. 2021 Dec 14;5(23):5429-5438. doi: 10.1182/bloodadvances.2021005902. PMID: 34673922; PMCID: PMC9153041.
- 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. External Control Arms in Oncology: Current Use and Future Directions. Ann Oncol. 2022 Jan 10:S0923-7534(22)00006-0. doi: 10.1016/j.annonc.2021.12.015. Epub ahead of print. PMID: 35026413.
- Rivera DR, Kluetz PG, Abdallah K, Agrawal S, Angus DC, Califf RM, Garrett-Mayer E, Hyer R, Lowy DR, Khleif SN. Clinical Evidence Generation During a Pandemic: Lessons Learned for Sustaining Progress. Cancer J. 2022 Mar-Apr 01;28(2):151-156. doi: 10.1097/PPO.0000000000000589. PMID: 35333502; PMCID: PMC9158733.
- Rivera DR, Larkins E, Kluetz PG. Blurring the Lines of Design: Prospective Observational Cohorts and Pragmatic Clinical Trials Characterizing Treatment Effects in Routine Cancer Care. Oncologist. 2022 Mar 4;27(2):79-81. doi: 10.1093/oncolo/oyab071. PMID: 35641221; PMCID: PMC8895745.
- Maignan K, Fashoyin-Aje LA, Torres AZ, Fernandes LL, Gwise T, Baxi SB, Roose JP, Rivera DR, Shen YL, Kluetz PG, Gormley NJ. Exploring racial disparities in treatment patterns and outcomes for patients with multiple myeloma using real world data. Blood Cancer J. 2022 Apr 19;12(4):65. doi: 10.1038/s41408-022-00665-x. PMID: 35440047; PMCID: PMC9018767.
- Benbow JH, Rivera DR, Lund JL, Feldman JE, Kim ES. Increasing Inclusiveness of Patient-Centric Clinical Evidence Generation in Oncology: Real-World Data and Clinical Trials. Am Soc Clin Oncol Educ Book. 2022 Apr;42:1-11. doi: 10.1200/EDBK_350574. PMID: 35561304.
Meet TEAM FoRWD
To advance the regulatory use of RWE, we have assembled TEAM FoRWD (Translational Evaluation and Assessment of Methods to Facilitate use of Oncology RWD).
TEAM FoRWD comprises FDA scientists with expertise in pharmacoepidemiology, hematology and oncology, epidemiology, biostatistics, and regulatory science to evaluate opportunities for RWD in regulatory contexts that can complement our understanding of medication risks and benefits for patients.
TEAM FoRWD Members 2022-2024
Donna Rivera, PharmD, MSc
Peter Bross, MD
Patrick DeMoss, MD
Jaleh Fallah, MD
Chaohong Fan, MD, PhD
Shabnam Ford, PhD
Michael Fusco, PharmD
Bernardo Goulart, MD
Vab Kumar, MD
Jennifer Lee, PharmD, RAC
Catherine Lerro, PhD
Gautam Mehta, MD
Pallavi Mishra-Kalyani, PhD
Abhilasha Nair, MD
Reena Philip, PhD
Elizabeth (Dianne) Pulte, MD
Fatima Rizvi, PharmD
Melanie Royce, MD, PhD
Deepti Telaraja, MD
Jonathon Vallejo, PhD
Joe Wynne, MD, PhD
Jay Zhao, PhD
Contact Information
Contact: OCERWE@fda.hhs.gov
Twitter: #OCERWE #OCETeamFoRWD
Further Information
- Oncology Quality, Characterization and Assessment of Real-World Data (QCARD) Initiative
- FDA Office of Medical Policy (OMP)
- FDA Center for Devices and Radiological Health
- OCE Scientific Collaborative Priorities
- OCE Project Pragmatica