Friday, October 11, 2019

7:30 AM - 8:15 AM Registration and Breakfast
8:15 AM - 8:25 AM Welcome Remarks: Ouhong Wang (Vertex)
8:25 AM - 8:45 PM
  • Keynote Opening Session: Tyranny of Numbers
  • Speaker: Reshma Kewalramani (Chief Medical Officer, Vertex)
8:45 AM - 9:55 AM
  • Session I: Statistical Methods for Rare Disease Clinical Trials (I)
  • Chair: Yang Song (Vertex)
  • Speakers:
    • L.J. Wei (Harvard University) - Utilizing Multiple Endpoints and Multiple Studies for Assessing Totality of Evidence of Treatment Effect
    • Robert Beckman (Georgetown University) - Clinical Trial Designs for Rare Diseases: The Confirmatory Basket Trial and the Informational Design
  • Discussant: Kun Chen (University of Connecticut)
9:55 AM - 10:15 AM Coffee Break
10:15 AM - 12:00 PM
  • Session II: Review of Statistical Approaches for Developing Rare Disease Medicines: Regulatory, Industry and Academic Perspectives
  • Chair: Charlie Cao (Biogen)
  • Speakers:
    • Jingjing Ye (FDA) - Ahead of FDARA Sec. 504 Implementation: Years in Review and Years Ahead in Pediatric Cancer Clinical Trials
    • Ziliang Li (Vertex), Chenkun Wang (Vertex), Sammi Tang (Servier) - Designing a Clinical Trial for Rare Disease with Historical Control - A Practical Roadmap with Case Studies
    • Rima Izem (Children’s National) - Comparative Effectiveness Using Natural History Data, A Case Study
12:00 PM - 1:00 PM Lunch
1:00 PM - 2:10 PM
  • Session III: Statistical Methods for Rare Disease Clinical Trials (II)
  • Chair: Sammi Tang (Servier)
  • Speakers:
    • Ming-Hui Chen (University of Connecticut) - Leveraging Historical Information: Methods and Applications
    • 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
  • Discussant: Mike Hale (Takeda)
2:10 PM - 2:30 PM Coffee Break
2:30 PM - 4:15 PM
  • Session IV: Case Studies in Rare Disease Drug Development
  • Chair: Jeffery Palmer (Pfizer)
  • Speakers:
    • Balram Gundapaneni (Pfizer) - Results of the Pivotal Phase III ATTR- ACT Study Using a Novel Primary Endpoint / Rank-Based Method
    • Feng Tai (Agios), Yanwei Zhang (Agios), Yanqiong Zhang (Agios) - Bayesian Dynamic Borrowing through Hierarchical Model to Assess Treatment Effect - A Phase I Case Study
    • Peng Sun (Biogen) - Adaptive Historical Data Borrowing in a Rare Disease Setting
4:15 PM - 4:20 PM Closing Remarks: Yang Song & Sammi Tang (2019 NERDS workshop co-chairs)