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

Positive vote (8-4) on benefit-risk, leading to approval