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)
- 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