Conference Program

Confirmed talks/speakers

• Robert Beckman (Georgetown University)
  Clinical Trial Designs for Rare Diseases: The Confirmatory Basket Trial and the Informational Design (slides)
• Ming-Hui Chen (University of Connecticut)
  Leveraging Historical Information: Methods and Applications
• Balram Gundapaneni (Pfizer)
  Results from a Pivotal Phase III Study Using Finkelstein-Schoenfeld, a Rank-Based Method to Combine Information Across Multiple Endpoints (slides)
• Rima Izem (Children's National Hospital)
  Comparative Effectiveness Using Natural History Data, A Case Study
• Ziliang Li, Chenkun Wang (Vertex Pharmaceuticals), Rui (Sammi) Tang (Servier)
  Designing a Clinical Trial for Rare Disease with Historical Control - A Practical Roadmap with Case Studies (slides)
• Qing Liu (Quantitative and Regulatory Medical Science, LLC)
  On Analysis of Single Arm Trial with Natural History Controls: A Virtual 1-to-Many Matched Control Method
• Peng Sun (Biogen)
  Adaptive Conditional Borrowing of Historical Data Using Propensity Score Matching in a Randomized Control Trial (RCT) in the Rare Disease Setting (slides)
• Feng Tai, Yanwei Zhang, Yanqiong Zhang (Agios Pharmaceuticals)
  Bayesian Dynamic Borrowing through Hierarchical Model to Assess Treatment Effect - A Phase I Case Study (slides)
• Lee-Jen Wei (Harvard University)
  Utilizing Multiple Endpoints and Multiple Studies for Assessing Totality of Evidence of Treatment Effect (slides)
• Jingjing Ye (CDER, FDA)
  Ahead of FDARA Sec. 504 Implementation: Years in Review and Years Ahead in Pediatric Cancer Clinical Trials (slides)