Friday, October 27th
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8:00 a.m.
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 Welcome and Introduction
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Lynne Yao, M.D. (FDA) |
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8:10 - 8:25 a.m.
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Introductory Remarks
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Robert Temple, M.D. (FDA) |
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8:20 - 8:35 a.m.
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Opening presentation: General landscape of extrapolation of efficacy in pediatric drug development
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Lily Mulugeta, Pharm.D. (FDA) |
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8:35 - 8:50 a.m.
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Drug Development in Pediatric Heart Failure—EMA Perspective
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Dobromir Penkov, M.D. (European Medicines Agency) |
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Pediatric Heart Failure: Current State of Knowledge
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8:50 - 9:20 a.m.
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"Disease" Natural History in Adults and Children, and "Grouping" of Different Causes of Pediatric HF for Clinical Trials - Clinical Perspective
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Joseph Rossano, M.D. (Children's Hospital of Philadelphia) |
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9:20 - 9:50 a.m.
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Management of Pediatric Heart Failure - Clinical Perspective
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Daphne Hsu, M.D. (Montefiore Medical Center) |
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9:50 - 10:10 a.m.
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Break
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10:10 - 10:40 a.m.
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Role of Research Networks in Understanding the Natural History of the Disease and in Facilitating Clinical Trials— the Pediatric Heart Network perspective
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Kristin Burns, M.D. (National Heart, Lung, and Blood Institute) |
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10:40 - 10:45 a.m.
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Patient Perspective
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Callie Tansill-Suddath |
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10:45 - 11:30 a.m.
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Q&A and Moderated Panel Discussion
Moderators: Shari Targum, Lily Mulugeta
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Skip (Robert) Nelson, M.D.; Joseph Rossano, M.D.; Daphne Hsu, M.D.; Kristin Burns, M.D.; Steven Lipshultz, M.D.; Callie Tansill-Suddath; Preston Dunnmon; Dobromir Penkov, M.D.; Gilbert Burckart |
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11:30 - 12:30 p.m.
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Lunch
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Trial Design Considerations in Pediatric Heart Failure
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12:30 - 1:00 p.m.
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Biomarkers and Endpoint Selection and the Role of Registries
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Steven Lipshultz, M.D. (Wayne State) |
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1:00 - 1:20 p.m.
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Methodological and Statistical Considerations for Using External Controls in Clinical Trials
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Ruthanna Davi, Ph.D. (Medidata |
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1:20 - 1:40 p.m.
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The Use of Quantitative Tools Including Systems Pharmacology for Study Planning and Design Optimization
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Jeffrey Barrett, Ph.D. (Sanofi) |
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1:40 - 2:00 p.m.
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The Role of Bayesian Decision-Making in Pediatric Drug Development: Case Examples
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Andrew Thomson (European Medicines Agency) |
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Case Examples in Pediatric Heart Failure
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2:00 - 2:30 p.m.
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Current Approach When Limited Confidence in Inference from Existing Adult Efficiency Data: Case Example (Entresto)
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Fabian Chen, M.D. (Novartis) |
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2:30 - 2:45 p.m.
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Break
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2:45 - 3:15 p.m.
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Considerations for Trial Design When There is Greater Level of Certainty That Adult Data Can Be Considered for Extrapolation to Pediatric Patients
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Lisa Bollinger, M.D. (Amgen – Product developed and pediatric studies conducted by Servier)
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3:15 - 4:15 p.m.
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Moderated Panel Discussion
Moderator: Lynne Yao, M.D.
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Robert Temple, M.D.; Christoph Hornik; Joga Gobburu, Ph.D.; Andrew Thompson; Jeffrey Barrett; Fabian Chen; Lisa Bollinger; Norman Stockbridge, M.D.; Dobromir Penkov; Christoph Hornik, M.D. |
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4:15 - 4:30
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Closing Remarks & Future Direction
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Lynne Yao, M.D. (FDA) |
