Small But Mighty: Innovative Clinical Trial Designs in Patient Populations of Limited Size - Day 2

Chair(s)

• 

  Angela J. James, PhD

  Vice President, Head of Clinical Pharmacology
  MacroGenics, Inc.
• 

  Amy S. Y Cheung, PhD

  Senior Director
  Certara
• 

  Bernard Sebastien, PhD

  Local Head of Clinical Modeling and Evidence Integration France
  Sanofi

Facilitator(s)

• 

  Yin Edwards, PharmD, PhD

  Associate Director
  Certara
• 

  Yu-Wei Lin, PhD

  Co-Director of MTCPG, University of Malaya, Adjunct Researcher, Monash University, Associate Director, Certara
  Monash University
• FM

  Flora Musuamba Tshinanu
• 

  Marta Neve, PhD

  Director
  Certara
• TN

  Thao Nguyen Pham, MSc

  PhD Student
  Normandie University
• 

  Gina Patel, PhD

  President and CEO
  Patel Kwan Consultancy
• 

  Kunal S. Taskar, PhD

  Scientific Director
  GlaxoSmithKline
• 

  Daphne Williams, MS, PharmD, PhD

  Director
  BMS
• JY

  James Yates

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Session Description

Effective strategies to develop therapies used to treat rare diseases, pediatric indications or a subpopulation of oncology patients, (e.g., targeted therapies) are challenging mainly due to small patient populations. Innovative thinking is needed to design clinical trials for small patient populations that is both feasible, patient-centric and able to achieve maximum statistical benefit by regulatory requirements. The "Small But Mighty" workshop is designed to foster disruptive ideas and generate novel strategies to move beyond the "3+3" paradigm and help drug developers address this “unmet need”.

Day 2 will be the hands-on portion of the workshop where attendees will be randomized to project teams and tasked with designing a study for a small patient population with specified constraints that reflect real-world challenges. Scientists with all levels of drug development experience and different areas of expertise are invited to attend.

Learning Objectives

1. Collaborate with a cross-functional team to design clinical trials for small patient populations that have embedded patient-centric recruitment and retention strategies, novel clinical endpoints and a biomarker plan that is fit-for-purpose.

2. Compare and contrast similarities and dissimilarities of drug development strategies for large versus small patient populations and where current guidances may or may not be a "one-size fits all".

Session Schedule

8:00 AM - 8:15 AM ET
Introduce Clinical Development Strategy
Amy S. Y Cheung, PhD, and Bernard Sebastien, PhD

8:15 AM - 9:15 AM ET
Breakout Session #1
All Facilitators

9:15 AM - 9:30 AM ET
Breakout Session #1 Group Discussion
Amy S. Y Cheung, PhD, and Bernard Sebastien, PhD

9:30 AM - 10:30 AM ET
Breakout Session #2
All Facilitators

10:30 AM - 10:45 AM ET
BREAK

10:45 AM - 11:15 AM ET
Breakout Session #3 - Teams Wrap Up Presentations
All Facilitators

11:15 AM - 11:55 AM ET
Team Presentations

11:55 AM - 12:00 PM ET
Closing Remarks
Angela J. James, PhD