A tentative schedule of events for Quantitative Assessment of Assumptions to Support Extrapolation of Efficacy in Pediatrics is provided below for the convenience of conference attendees.
This one-day workshop scheduled for Wednesday, June 1, 2016, will be held in the Great Room at the FDA's White Oak Campus, located at 10903 New Hampshire Avenue in Silver Spring, MD. It is open to the public. The goal of the workshop is to:
- Present quantitative and qualitative approaches for verifying assumptions around disease and response similarity in adults and children.
- Provide a forum for discussion on the use of modeling and simulation for systematic assessment of extrapolation assumptions.
| Time | Activity |
|---|---|
| 8:00-8:05 a.m. | Welcome UMD-CERSI |
| 8:05–8:15 a.m. | Introduction and Workshop Objectives Lily Mulugeta, PharmD Scientific Lead, Pediatrics Division of Pharmacometrics U.S. Food and Drug Administration Slides | Recording |
| 8:15–8:30 a.m. | Introductory Remarks by the Center Director Janet Woodcock, MD Center Director Center for Drug Evaluation and Research U.S. Food and Drug Administration |
| 8:30–8:45 a.m. | Introductory Remarks: Opportunities and Challenges in Pediatric Product Development Dianne Murphy, MD Director, Office of Pediatric Therapeutics U.S. Food and Drug Administration Recording |
| 8:45–9:00 a.m. | Extrapolation in Pediatric Product Development: Practical Application of the Principle of Scientific Necessity Skip Nelson, MD, PhD Deputy Director and Senior Pediatric Ethicist Office of Pediatric Therapeutics U.S. Food and Drug Administration Slides | Recording |
Assessment of Disease and Response Similarity Between Adults and Children -- Previous Experiences
| Time | Activity |
|---|---|
| 9:05-9:35 a.m. |
Data Related to Disease Similarity -- A Case Study: PEACE Initiative in Pediatric Epilepsy |
| 9:35–9:45 a.m. | Data Related to Disease Similarity in Partial Onset Seizures -- Regulatory Perspective Philip Sheridan, MD Medical Officer Division of Neurology Products U.S. Food and Drug Administration Slides | Recording |
| 9:45–10:00 a.m. | Quantitative Analysis to Support Full Extrapolation of Efficacy in Pediatrics for Partial Onset Seizures: FDA-PEACE Initiative Shailly Mehrotra, PhD (Atul Bhattaram, PhD; Angela Men, PhD) Office of Clinical Pharmacology U.S. Food and Drug Administration Slides | Recording |
| 10:00–10:15 a.m. | Break |
| 10:15—10:45 a.m. | Natural History and Predictors of Response/Remission in Pediatric and Adult Ulcerative Colitis Jeffrey Hyams, MD Division Head, Gastroenterology Connecticut Children's Medical Center Slides | Recording |
| 10:45—11:15 a.m. | Challenges in Conditions with Limited Pediatric Clinical Trial Data: Focus on Similarity of Response to TNFα Inhibitors in Ulcerative Colitis Richard Strauss, MD Senior Director, Clinical Research Janssen Pharmaceuticals Slides | Recording |
| 11:15–11:45 a.m. | Quantitative Assessment of Disease Similarity in RA and pJIA Stephen Wright, MD Group Medical Director, Lifecycle Leader Genentech/Roche Slides | Recording |
| 11:45-12:15 p.m. | Quantitative Assessment of Response/Exposure-Response Similarity in RA and pJIA Megan Gibbs, PhD Executive Director, Global Head Clinical Pharmacology, Modeling, and Simulation Amgen Slides | Recording |
| 12:15-12:30 p.m. | The Role of Non-clinical Data in Assessing Extrapolation Assumptions Tracy Behrsing, PhD Pharmacologist U.S. Food and Drug Administration Slides | Recording |
| 12:30-1:30 p.m. | Lunch |
| 1:30—2:15 p.m. |
Moderated Panel Discussion Panelists: Lynne Yao (FDA), Andrew Mulberg (FDA), Richard Vesely (EMA), Sarah Yim (FDA), Richard Strauss, Megan Gibbs, Stephen Wright, Barbara Buch (CBER,FDA), Jeffrey Hyams |
Alternative Approaches to the Assessment of Extrapolation Assumptions in Pediatric Drug Development
| Time | Activity |
|---|---|
| 2:15-2:35 p.m. | What Constitutes A Credible Disease Progression Model? Jeffrey Barrett, PhD Vice President, Global Head Translational Informatics Sanofi Slides | Recording |
| 2:35—3:05 p.m. | General Considerations for Developing Disease-Drug-Trial Models for Assessment of Extrapolation Assumptions Diane Mould, PhD President Projections Research, Inc. Slides | Recording |
| 3:05—3:15 p.m. | Break |
| 3:15—3:45 p.m. | The Role of Clinical Trial Simulation in Assessing Extrapolation Assumptions Marc Gastonguay, PhD President and Chief Executive Officer Metrum Research Slides | Recording |
| 3:45—4:05 p.m. | Leveraging Adult Data in Pediatric Product Development: The Role of Bayesian Approaches Freda Cooner, PhD Team Leader Office of Biostatistics U.S. Food and Drug Administration Slides | Recording |
| 4:05—4:50 p.m. |
Moderated Panel Discussion Panelists: Skip Nelson (FDA), Freda Cooner (FDA), Jeff Barrett (Sanofi), Andrew Thomson (EMA), Vasum Peiris (CDRH, FDA), Joga Gobburu (UMD), Diane Mould, Marc Gastonguay |
| 4:50—5:00 p.m. |
Closing Remarks/Future Direction |