Advancing Rare Disease Therapies Through an FDA Rare Disease Innovation Hub
Hybrid Public Meeting: In-Person and Virtual
October 16, 2024 | 10am-2:30pm (eastern)
The Reagan-Udall Foundation for the FDA, in collaboration with FDA’s Rare Disease Innovation Hub, hosted a public meeting on October 16, 2024. The meeting brought together rare disease patient advocates, academic researchers, regulated industry, and other key stakeholders to discuss how the recently announced Rare Disease Innovation Hub can best engage with members of the rare disease community and prioritize its work.
During this public meeting, FDA sought to gather public comment in the following areas:
- Cross-cutting rare disease-related, scientific, regulatory, or policy issues (those not related to a particular disease or condition) that should be prioritized for consideration by the Rare Disease Innovation Hub
- Rare disease-specific (but not application-specific) scientific, regulatory, or policy issues that should be prioritized for consideration by the Rare Disease Innovation Hub
- Rare disease-related activities or initiatives currently being undertaken independently by CDER or CBER that you believe would benefit from being undertaken by the Rare Disease Innovation Hub as a joint activity
- Approaches that the Rare Disease Innovation Hub should follow for engagement with patients and caregiver groups, industry organizations, and scientific/academic organizations
Note the deadline to request to present stakeholder input was September 25.
Registrants were able to request to make oral presentations during the meeting when registering.
More about the Rare Disease Innovation Hub
An estimated 10,000+ rare diseases affect more than 30 million people – approximately one out of every 10 people – in the United States, and about half of these people are children. While there have been important advances in treatments for rare diseases, more needs to be done. Many rare diseases lack treatment options and therefore many patients have high unmet medical needs. This requires new approaches to expedite development and approval of safe and effective drugs and biologics. The FDA sees huge potential in establishing a new model, within the FDA, to leverage cross-agency expertise and greatly enhance our inter-center connectivity to spur the development of treatments for rare diseases.
To that end, the Center for Biologics Evaluation and Research (CBER) and the Center for Drug Evaluation and Research (CDER) are launching a Rare Disease Innovation Hub to advance rare disease therapy development through greater communication, collaboration, and coordination across CBER and CDER, in coordination with other Centers and Offices across FDA. Helmed by senior leadership from CBER and CDER, the Hub will work to develop and implement a comprehensive cross-center rare disease strategic agenda that takes full advantage of our current clinical and scientific expertise across both centers and is based on a shared vision and comprehensive approach to 1) align methods and approaches to rare disease programs, both during IND development, and in application review; 2) identify and enable innovative approaches in the areas of novel endpoints, biomarker development, and innovative trial designs; and 3) streamline communications with the rare disease community. The Hub will work across rare diseases but will especially focus on products intended for smaller populations or for diseases where the natural history is variable and not fully understood, as we recognize that development of therapies for these conditions can be particularly challenging.