Paradigm shift
Establishing a novel indication that could fundamentally change the treatment paradigm poses unique challenges.
New hematology/oncology indication
FDA Committee: Oncologic Drug Advisory Committee (ODAC)
Challenges for the Sponsor
- Complex clinical, regulatory and statistical issues
- Acceptance of novel endpoint
- Limitations of single-arm P2 study and historical comparator
- Potential confounding factors
- Clinical meaningfulness of results
- Lack of data in pediatric patients
- Uncertain regulatory pathway (accelerated or full approval)
How we helped
- Anticipated FDA/ODAC issues to develop clear, concise and compelling messaging
- Recruited tough mock panelists to prepare the team for the worst case
- Intensive Q&A rehearsal
- Coached the team to leverage every opportunity at ODAC
- Maintained energy and focus during emergency evacuation of facility
- Educated advocacy groups and speakers on Open Public Hearing expectations
Outcome
Positive vote (8-4) on benefit-risk, leading to approval