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  1. FDA Rare Disease Innovation Hub

FDA Rare Disease Innovation Hub Q&A

Q: What is the Rare Disease Innovation Hub?
A: The Rare Disease Innovation Hub is an FDA cross-center program that facilitates connection and engagement with outside parties and serves as a forum between CBER and CDER for consideration of cross-cutting rare disease-related issues. The Hub focuses on facilitating development of medical products for rare diseases, particularly those diseases for which we lack understanding of the pathophysiology or natural history and for which there are small patient populations with a high unmet need.

Q: Why was the Rare Disease Innovation Hub established?
A: The Hub was established to promote collaboration across FDA and advance a shared vision and comprehensive approach to address common challenges such as identifying and utilizing innovative approaches in the areas of novel endpoints, biomarker development and trial designs; and streamlining communications with the rare disease community. By functioning as a cross-center program, the Hub is able to take full advantage of our current clinical and scientific expertise across FDA.

Q: Who leads the Rare Disease Innovation Hub?
A: The Hub is co-led by the directors of the Center for Biologics Evaluation and Research (CBER) and the Center for Drug Evaluation and Research (CDER). They serve as co-chairs for the Rare Disease Innovation Steering Committee, which also includes relevant leadership from within the centers and other offices within FDA, such as the Center for Devices and Radiological Health (CDRH), the Oncology Center of Excellence (OCE), the Office of Orphan Products Development (OOPD) and the Office of Combination Products (OCP).

A critical role in the Hub is the newly created senior leadership position, Director of Strategic Coalitions, who is accountable to both center directors and promotes collaboration between the centers. In addition to acting as a single point of connection and engagement with outside parties, the Director of Strategic Coalitions works closely with the leads of CDER’s Accelerating Rare disease Cures (ARC) Program and CBER’s Rare Disease Program to leverage their activities and follow up with appropriate FDA staff involvement or appropriate settings for further external engagement. Through this engagement with outside parties, the Director of Strategic Coalitions helps to ensure the priorities of the Hub are informed by input from the rare disease community. The Director of Strategic Coalitions for the Hub is Amy Comstock Rick.

Q: How will the Rare Disease Innovation Hub benefit the rare disease community? 
A: The increased collaboration across FDA will help move forward approaches that will help to advance rare disease therapies, including cross-cutting regulatory science and policy issues. The Hub engaged with members of the rare disease community and other interested parties to develop a Strategic Agenda that outlines the Hub’s goals, priorities, and initiatives to advance treatments for rare diseases.

Q: What are the key functions of the Rare Disease Innovation Hub?
A: As outlined in the Strategic Agenda, the goals of the Hub are:

  • Goal 1: Serve as a primary point of connection and engagement with the rare disease community, including patient and caregiver groups, trade organizations, and scientific/academic organizations, for matters that intersect rare disease development within CDER and CBER.
  • Goal 2: Facilitate regular collaboration between CBER and CDER. Promote cross-Center information sharing and, where possible, alignment between Centers on review standards and decision-making. Where scientific reasons exist for differences in review procedures between CBER and CDER, promote clarity about those reasons with the rare disease community. Encourage transparency and communication between FDA Centers and drug developers throughout the review process.
  • Goal 3: Create and enhance opportunities for consideration of novel endpoints, biomarker development and assays, innovative trial design, real world evidence, and statistical methods. Initiate engagement with federal research partners and the broader rare disease research community with the purpose of sharing scientific innovations and enhancing collaboration in regulatory science.    

Throughout the year, there will be multiple ways for the rare disease community to engage with an offer suggestions to the Hub, both for its immediate use and for consideration for the 2026 Strategic Agenda

Q: Does the Rare Disease Innovation Hub have a role in reviewing the applications for approval? 
A: Decisions on approval of individual drugs and biological products will remain with the experts within the therapeutically aligned offices and divisions. The Hub provides a forum for considering cross-cutting regulatory science and policy issues to help address common challenges across applications. While we hope this effort will have a significant impact on the development of rare disease therapies, the Hub does not provide advice on individual applications. The Hub is also intended to help the rare disease community navigate important intersections across FDA that affect patients with rare diseases to ultimately improve outcomes for patients.

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