| Use of MRI of Joints as Potential Clinical Trial Endpoints in Patients with Rheumatoid Arthritis |
June 2013 |
| Use of Hyperpolarized Noble Gas MRI to Measure Lung Function in Patients with Cystic Fibrosis |
July 2013 |
| Use of Bone Scan Assessment as a Potential Clinical Trial Endpoint or for Qualification as a Biomarker |
July 2013 |
| Use of in vitro Testing to Predict Drug Sensitization Potential |
October 2013 |
| Potential Approaches in Drug Development for Adrenomyeloneuropathy |
December 2013 |
| Issues in the Laboratory Measurement of Dystrophin |
July 2014 |
| Bile Salt Export Pump Inhibition as a Biomarker |
May 2015 |
| Use of Disease Progression Modeling for Amyotrophic Lateral Sclerosis |
June 2015 |
| Use of a Database of Clinical Trial Data |
August 2015 |
| Use of a Database of Genomic and Clinical Data |
September 2015 |
| Patient Reported Outcome Assessment for Primary Sclerosing Cholangitis |
October 2015 |
| Drug Development for Mitochondrial Diseases |
October 2015 |
| Use of Circulating Tumor Biomarkers |
October 2015 |
| Patient Reported Outcome Assessment for early stage breast cancer |
October 2015 |
| Using Genetic Data to Advance Drug Development |
January 2016 |
| A Novel Analytic Method for Pharmacokinetic Data Interpretation |
January 2016 |
| A Diagnostic Tool to Advance Cancer Drug Development |
January 2016 |
| Approaches to Developing Therapies for Patients with Severely Debilitating and Life Threatening Conditions |
February 2016 |
| Biomarkers for Traumatic Brain Injury Clinical Trials |
March 2016 |
| Use of a Placebo Response Tool in Clinical Studies |
April 2016 |
| Digital Pathology Software System |
April 2016 |
| Mucolipidosis Type IV Translational Research |
May 2016 |
| Natural History Studies in Primary Sclerosing Cholangitis |
June 2016 |
| Development of Release Active Products |
July 2016 |
| Multi-System Assessment of Postnatal Development Following Extremely Preterm Birth |
July 2016 |
| Pediatric Cholestatic Liver Disease Endpoints |
July 2016 |
| Disease Interception Therapy in Rheumatoid Arthritis |
July 2016 |
| Symptom Measurement in Hepatocellular Carcinoma |
July 2016 |
| Targeting Shared Molecular Etiologies Underlying Multiple Rare Diseases (SaME) |
July 2016 |
| Natural History Studies in Angelman Syndrome |
July 2016 |
| Update on the Pediatric Trials Consortium |
August 2016 |
| Development of a Genomic Database |
September 2016 |
| Disease Interception Therapy in Type 1 Diabetes |
October 2016 |
| Atopic Dermatitis in Pediatric Patients |
October 2016 |
| Disease Interception Strategy in Cataracts and Presbyopia |
October 2016 |
| Registry for Pediatric Inflammatory Bowel Disease |
November 2016 |
| C1M Assessments in Rheumatoid Arthritis |
November 2016 |
| Tool to Evaluate Duplicates in Clinical Trials |
December 2016 |
| Raman Spectroscopy to Analyze Compounded Drugs |
December 2016 |
| Biomarkers to Identify Drug Induced Pancreatic Injury |
January 2017 |
| A Tool to Assess Clinical Trial Site Performance and Data Quality |
January 2017 |
| The Role of Inflammation and Immune Response in Duchenne |
January 2017 |
| Approaches to Advance Regulatory Science Training |
February 2017 |
| Novel Drug Development Tools and Biomarkers to Support Tuberculosis Drug Regimen |
March 2017 |
| Development of Innovative Novel Excipients |
March 2017 |
| Use of Biomarkers to Enrich for Cardiac Events in Cardiovascular Clinical Trials |
March 2017 |
| Use of Virtual Reality Platform in Clinical Trials |
March 2017 |
| Development of a Patient Reported Outcome Instrument for Alopecia Areata |
April 2017 |
| Pediatric Microdosing Research Using Accelerator Mass Spectrometry (AMS) |
May 2017 |
| Challenges Related to Drug Development for Cerebral Cavernous Malformations (CMM) Patient Community |
May 2017 |
| Health Related Quality of Life Assessment in Diabetes |
June 2017 |
| Physical Activity Accelermetry Assessment for Analgesic Clinical Trials (PAACT) |
June 2017 |
| Outcome Measures for Type 1 Diabetes |
July 2017 |
| 3D Bioprinted Human Tissue Models |
August 2017 |
| Outcome Measures for Type 1 Diabetes |
August 2017 |
| Chronic Obstructive Pulmonary Disease (COPD) Assessment Test |
August 2017 |
| The Use of Wearable Devices to Collect Endpoint Data in Clinical Trials |
September 2017 |
| Outcome Assessments in Acute Pancreatitis |
October 2017 |
| Bring Your Own Device PRO Assessments |
October 2017 |
| Adrenomyeloneuropathy (AMN) |
October 2017 |
| Drug Development Tools for Charcot-Marie Tooth Disease |
November 2017 |
| Genetic Prion Disease |
November 2017 |
| Clinical Assessment Tools for Alopecia Areata |
December 2017 |
| Disease Severity Score for Influenza and other Acute Respiratory Viruses |
January 2018 |
| Translational Pharmaceutics based drug development in the US |
February 2018 |
| Development of Prophylactic Treatments for Asthma and Other Allergic Diseases |
March 2018 |
| Hutchinson-Gilford Progeria Syndrome Biomarker Development |
April 2018 |
| Biomarkers and Clinical Outcome Assessments for Fragile X Syndrome |
April 2018 |
| A Patient-Centric Approach to Long-term Follow-up Studies |
May 2018 |
| Clinical Outcome Assessment tools for Batten Disease |
July 2018 |
| Clinical Research Networking Technology Platform |
October 2018 |
| Electronic product information in clinical trials |
October 2018 |
| A Patient-Centric Approach to Long-term Follow-up Studies |
October 2018 |
| Clinical Outcome Assessments for Prader-Willi Syndrome |
November 2018 |
| Definitions for clinically meaningful levels of hypoglycemia |
December 2018 |
| Drug development tools to aid rare disease natural history development |
December 2018 |
| Development of COA through personalized digital health coaching in clinical trials and drug development |
February 2019 |
| Voice activated PRO tools for Charcot-Marie-Tooth (CMT) Disease |
February 2019 |
| Challenges associated with the Development of Novel Immunosuppressive Drugs (ISD) for use in Kidney Transplantation |
March 2019 |
| Personalized oligonucleotide therapies for genetic neurodegenerative diseases |
May 2019 |
| Digital drug development tools for Parkinson’s Disease |
May 2019 |
| New approaches to analgesic drug development |
September 2019 |
| Statistical Approaches to Biosimilar Clinical Study Design |
October 2019 |
| Clinical Outcomes for Chronic Obstructive Pulmonary Disease |
October 2019 |
| Disease Interception in Non-Small Cell Lung Cancer (NSCLC) |
November 2019 |
| Self-Management Self-Test (SMST) in Psychiatric Disorders |
December 2019 |
| Master Protocol to Promote Expanded Access in Oncology |
February 2020 |
| The Use of the Sequential Parallel Comparison Design in the Development of treatments for Major Depressive Disorder |
February 2020 |
| Clinical Outcome Assessments of for Cutaneous Lupus Erythematosus |
February 2020 |
| Use of Artificial Intelligence (AI) to created Synthetic Controls in Clinical Trials |
March 2020 |
| Clinically Meaningful Endpoints for Wound Healing Clinical Trials |
June 2020 |
| Advancing Development of Therapies for Spinal Muscular Atrophy |
August 2020 |
| Goal Attainment Scaling (GAS) |
August 2020 |
| Mass-Action Law Pharmacodynamics (MAL-PD) |
October 2020 |
| Cyclin Dependent Kinase-like 5 (CDKL5) Deficiency Disorder (CDD) Observational Study |
December 2020 |
| The Use of Serum Neurofilament-light (sNfl) in Progressive Multiple Sclerosis (MS) Trials |
December 2020 |
| Embedding Qualitative Patient Interviews in Neurofibromatosis Clinical Trials to Facilitate Drug Development |
February 2021 |
| Down Syndrome Associated Alzheimer’s Disease (DS-AD) |
March 2021 |
| Fabry Disease Structural Endpoints for Assessment of Drug Efficacy in Fabry Nephropathy clinical Trials |
April 2021 |
| Optimizing pain measurements in neonates to expedite analgesic drug development |
June 2021 |
| Obtaining Reliability and Acceptance of Novel Endpoints using Digital Health Technologies |
July 2021 |
| Basket Trials to Study Sleep Wake Instability across Multiple Rare Disorders |
November 2021 |
| Innovative Conceptual Approaches to Clinical Trial Design and Analysis for an Integrated Research Platform in Non-Alcoholic Steatohepatitis (NASH) |
January 2022 |
| Rare Genetic Dilated Cardiomyopathy |
February 2022 |
| Extracellular RNA Splice Variant Biomarkers of Myotonic Dystrophy |
April 2022 |
| Use of Digital Pathology in Good Laboratory Practice (GLP) Environment for Nonclinical Studies |
June 2022 |
| Advancing Nocturnal Scratch as a Digital Endpoint for Atopic Dermatitis |
July 2022 |
| Transfer RNA-Readthrough Treatment for Rare Genetic Diseases |
September 2022 |
| Advanced Computational and in-vitro modeling in Drug Discovery & Drug Development |
January 2023 |
| Sjögren’s Tool for Assessing Response (STAR) |
February 2023 |
| Artificial Intelligence (AI) image analysis software for supporting the evaluation of oncology drug efficacy in clinical trials |
June 2023 |
| Precision Psychiatry Approach to Clinical Development |
September 2023 |
| Natural History Studies for Primary Sclerosing Cholangitis |
October 2023 |
| Oral Tyramine Challenge Testing of MAO Inhibitors |
January 2024 |
| Virtual Clinical Trials |
March 2024 |
| Electroencephalography (EEG)-based artificial intelligence (AI)-powered method to improve the efficiency of clinical trials of Major Depressive Disorder (MDD) |
July 2024 |
| Targeting Condensates to Treat Disease: Implications, Opportunities and Challenges |
September 2024 |
