Rare Disease Innovation Hub
Strengthening the infrastructure behind this collaboration between the FDA's Center for Drug Evaluation and Research, Center for Biologics Evaluation and Research, and Center for Devices and Radiological Health to streamline and accelerate treatments for rare diseases.
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The Rare Disease Innovation Hub is a cross-center initiative within the FDA designed to improve and accelerate the development of therapies for rare diseases by enhancing collaboration to identify and address the common scientific, clinical, and policy issues in rare disease product development.
The FDA has been instrumental in building the regulatory framework and institutional expertise needed to accelerate innovation and development of rare disease therapies. Leveraging key legislative milestones like the Orphan Drug Act of 1983 and the FDA Safety and Innovation Act of 2012, the FDA has enabled remarkable progress in which the number of approved orphan-designated products has grown to over 1300, serving about 400 different communities. Advancements in regulatory policy, the FDA’s embrace of patient-focused drug development, and the formation of the Rare Disease Innovation Hub are just a few of the examples that demonstrate the FDA’s commitment to creating a robust rare disease therapy development environment.
Unlike traditional FDA offices that operate within specific product “centers,” the Innovation Hub works across:
- The Center for Drug Evaluation and Research (CDER)
- The Center for Biologics Evaluation and Research (CBER)
- The Centers for Device and Radiological Health (CDRH)
- And others like the office that handles orphan drug designations
The Hub operates under shared FDA leadership, including the Center Directors from all three medical product centers. Its leadership model is intentionally collaborative – ensuring that rare disease expertise is shared across product center rather than siloed.
It’s mission is to make rare disease drug development more predictable, more scientifically rigorous, and more feasible for small and especially very small patient populations.
Check out the Hub’s 2026 agenda.
Strategic Goals for 2026
The Hub’s 2026 Strategic Plan lays out an ambitious agenda to grow its role in facilitating the dissemination and uptake of rare disease best practices across the Agency. They aim to:
- Host up to three additional RISE Workshops on timely topics that can catalyze therapy development
- Pilot a new enhanced approach for rare disease training for review staff
- Create additional opportunities for rare disease patient organizations and experts to inform the therapy development and evaluation process
- Streamline the navigation of FDA’s rare disease resources
Why it Matters
The Rare Disease Innovation Hub represents a shift in how the FDA thinks about its approach to rare diseases, but its activities and impact are limited by resources available to power their work.
When the Hub was created, it did not have dedicated funding from Congress. The early activities have been supported by resources “borrowed” from other areas within the agency. In 2026, each center is contributing some funding from their budgets, but the role of dedicated funding from Congress is critical to scaling and sustaining the Hub’s efforts.
With appropriate resources, the Rare Disease Innovation Hub can transform how rare disease community stakeholders, clinical and scientific experts, and product developers engage with the Agency and how knowledge and information are shared internally so that it translates into more predictable and consistent approaches to how rare disease therapies are evaluated.
Take Action
Encourage Your Member of Congress to Support the Rare Disease Innovation Hub
With appropriate resources, the Rare Disease Innovation Hub can continue to transform how rare disease community stakeholders, clinical and scientific experts, and product developers engage with the FDA.
The Rare Disease Innovation Hub: Pathways to Progress
March 2026: Third RISE Workshop
The Hub hosts the third RISE Workshop, “RISE Together: Data Sharing Across the Rare Disease Ecosystem.”
February 2026: Second Strategic Agenda
The Hub releases its second Strategic Agenda to communicate its goals and priorities for 2026.
November 2025: Second RISE Workshop
The second RISE Workshop is held, exploring the topic of “Individualized Therapies on the RISE.”
September 2025: First RISE Workshop
The Hub hosts the first RISE Workshop, “On the RISE: Controls in Rare Disease Clinical Trials for Small and Diminishing Populations.”
January 2025: First Strategic Agenda
The Rare Disease Innovation Hub releases its first Strategic Agenda, outlining its three core goals: advancing regulatory science, enhancing coordination, and creating a clear entry point for external stakeholders to navigate the FDA.
October 2024: Public Meeting
FDA, in partnership with the Reagan-Udall Foundation, hosts a public meeting to gather stakeholder input on the issues and initiatives the Rare Disease Innovation Hub should prioritize.
July 2024: FDA Rare Disease Innovation Hub Announcement
FDA announces the establishment of the first Rare Disease Innovation Hub to enhance and advance outcomes for patients.
May 2024: Center of Excellence Report Language
Thanks to advocacy from the rare disease community and Rare Disease Congressional Caucus leaders, Senate Appropriations Report Language calls for FDA to enhance intercenter collaboration.
March 2024 : FY25 FDA Rare Disease CoE Appropriations Letter
FY25 FDA Rare Disease CoE Appropriations Letter
Feb – April 2023: Center of Excellence: CDER Quantitative Medicine (QM)
FDA establishes the latest COE to facilitate and coordinate the continuous evolution and consistent application of quantitative medicine across CDER.
2021 – 2022: Congressional Rare Disease Caucus Leadership
Despite STAT Act not passing in the 117th Congress, members of the Caucus push for FDA coordination on RD challenges.
2020 : STAT ACT
EveryLife and rare disease partners advocate for the STAT Act, proposing the establishment of a COE.
2020: Center of Excellence: Digital Health
FDA creates Digital Health Center of Excellence to advance health care by fostering responsible and high-quality health innovation.
2020 : Rare Disease Week
Community calls for creation of Rare Disease COE during #RareDC2020.
2019: Center of Excellence: Compounding Quality
FDA creates the Compounding Quality Center of Excellence to support facilities and stakeholders involved in compounded medicines.
2018 : Scientific Workshop
Conceptualizes a framework for a rare disease COE.
2017: Center of Excellence: Oncology
FDA creates the first center of excellence to advance the development of medical products for people with cancer.
2016 : 21st Century Cures Act
Provides FDA the authority to create intercenter institutes (also called centers of excellence).
Meet Andrew and Jason
Andrew and Jason are brothers with Fragile X Syndrome that face anxiety, intellectual challenges, communication issues, and motor delays. The Rare Disease Innovation Hub aims to tackle therapy development complexities for cases like theirs.
Meet Harlie
Harlie, with a rare GNAO1 gene mutation and one of fewer than 100 known cases globally, is non-verbal and faces significant developmental delays.The Rare Diseases Innovation Hub can drive therapy development for ultra-rare conditions like hers.
