Next steps on FDA rare disease workshops

fda-logo-with-eagleLast week the FDA convened 3 days of workshops on developing drugs, biologics and devices for rare diseases with an emphasis on pediatric rare diseases. One 90-minute panel was on pediatric cancer. Among the topics covered were clinical trial designs, how to weigh benefit-risk and what might be done to develop drugs that might only be used in children with rare diseases. The FDA fulfilled its obligation under FDASIA (a law passed in 2012) to hold workshops on pediatric rare diseases to provide input for their overall strategic planning. Much of the workshop was a review of issues and deliberations that routinely occur in the childhood cancer community and in the rare disease community at large.  I left the discussion thinking of how we as advocates can promote more tangible action to realize the development of treatments for pediatric cancer. What are your thoughts?

As Children's Cause for Cancer Advocacy drafts further comments to submit as part of this process, we'd like to hear your take on some issues that were raised during the workshops:

  • How can trials be better designed to measure various endpoints? What trial endpoints should be measured besides survival?
  • How do we overcome a culture of risk-aversion and take into account what will happen if a certain clinical trial isn't conducted?
  • How can the FDA work better with advocacy groups to develop guidelines on how much risk families are willing to tolerate in clinical trials?
  • How do we take into account the long-term quality of life considerations for survivors of childhood cancer? Should survivors be included in trial design process?
  • How should differences in US vs. European regulations be reconciled? What changes might be needed to existing regulations to make them disease-focused instead of drug-focused?

These are just some of the questions we are considering. We'd love to hear your thoughts!