Small datasets

In life-threatening diseases, FDA is willing to accept increased uncertainty. Where do they draw the line?

 

Interpreting benefit-risk in a small program

FDA Committee: Oncologic Drugs Advisory Committee (ODAC)

Challenges for the sponsor

  • Accelerated timeline for preparation
  • Communicating clinical relevance of results in a single positive trial
  • Small core team with conflicting priorities
  • Reliant on outsourcing for various disciplines
  • Committee members not familiar with standard of care for the disease
  • Political and social sensitivity of the condition

How we helped

  • Brought in additional resources to bolster the team
  • Aligned the internal and external team on strategy
  • Educated the team on ODAC expectations
  • Supported speakers to bring out critically important aspects of disease state for the ODAC
  • Supported  Open Public Hearing speakers for live and remote testimony
Outcome Positive vote (10 – 3) on benefit-risk, leading to approval