Biologics

Support, Suggestions for FDA Rare Disease Hub

David McFarland

— 2 min read
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Four stakeholders — St. Jude Children’s Research Hospital, the National Organization for Rare Disorders (NORD), the Muscular Dystrophy Association (MDA), and the Biotechnology Innovation Organization (BIO) — are voicing support for an FDA proposal to create an FDA Rare Disease Innovation Hub (RDI Hub). The four also respond to FDA questions about how the Hub could work and what it should address.

The St. Jude Pediatric Translational Neuroscience Initiative says an important issue for the Hub to address would be to refine the regulatory pathway for gene-targeted therapies to treat populations with more than a single patient but not enough patients to power a more traditional clinical study or garner commercial interest. It also suggests that FDA provide greater clarity on agency requirements or create alternative approaches to support the development of therapies.

NORD says it applauds FDA’s creation of an Innovation Hub rather than a Rare Disease Center of Excellence, saying it believes a Hub will provide the right organizational structure “to support effective, transparent, and patient-centered regulatory decision-making, and enhance collaboration internally between FDA Centers, as well as externally with the broader rare disease community.” It recommends that the Hub initially focus on three key priorities:

  • improving communication between the rare disease community and FDA;
  • strengthening alignment internally between CDER and CBER as well as CDRH and better explaining the rationale behind differences in decision-making; and
  • using learnings from past activities and establishing new ways to support meaningful patient group, academic investigator, and small biotech input.

It recommends that the following concepts be integrated into a strategic plan for the Hub:

  • ensuring patient representation in FDA’s RDI Hub;
  • rare disease academic regulatory assistance;
  • annual rare disease regulatory science meeting;
  • sustaining rare disease diagnostic innovation;
  • real-world data/real-world evidence partnership with NORD and other rate disease stakeholders;
  • facilitate robust patient access to safe and effective therapies through appropriate data collection; and
  • data element standardization for long-term cell and gene therapy follow-up from 10- and 15-year-long post-market commitments.

MDA calls for these Hub priorities: consistency and collaboration across review divisions, collaboration with the FDA Office of Patient Affairs, collaboration with other agencies impactful to the rare disease community, and advisory committee approaches and conduct.

Finally, BIO says the Hub’s priorities should be:

  • improved coordination and consistency within and across FDA Centers;
  • enhancing communication with external stakeholders;
  • facilitating expert discussions;
  • advancing scientific research and innovation;
  • hosting multi-stakeholder scientific workshops and forums;
  • incorporating continuous multi-stakeholder feedback; and
  • creating a strategic roadmap.

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