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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 will act as the single point of connection and engagement with outside parties and a forum between CBER and CDER for consideration of cross-cutting rare disease-related issues. The Hub will focus on facilitating development of medical products for these diseases, particularly for products being developed for rare diseases where we lack understanding of the pathophysiology or natural history, and where 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 shared strategic agenda for advancing therapies for rare diseases that will be adopted by the Hub will be 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 will be the newly created senior leadership position, the director of Strategic Coalitions, who will be accountable to both center directors and will promote collaboration between the centers. In addition to acting as a single point of connection and engagement with outside parties, the director of Strategic Coalitions will work 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 individual’s engagement with outside parties, the director of Strategic Coalitions will help to ensure the priorities of the Hub are informed by input from the rare disease community.  

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 intends to work with members of the rare disease community and other interested parties on developing a strategic and comprehensive agenda that will help to shape the priorities and initiatives. 

Q: What are the key functions of the Rare Disease Innovation Hub?

  • Serve as a single 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.   
  • Enhance collaboration across FDA to address common scientific, clinical and policy issues related to rare disease product development, including relevant cross-disciplinary approaches related to product review, and promote consistency across offices and centers.   
  • Advance regulatory science with dedicated workstreams for consideration of novel endpoints, biomarker development and assays, innovative trial design, real world evidence, and statistical methods.  

Q: Will the Rare Disease Innovation Hub have a role in reviewing the applications for approval? 
A: Decisions on approval of drugs and biological products will remain with the experts within the therapeutically aligned offices and divisions. The Hub will provide 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 will 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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