FDA to Host Rare Disease Day 2026 as Agency Advances New Treatment Pathways

The U.S. Food and Drug Administration will host Rare Disease Day 2026 on February 23, aiming to engage with patients and the broader community to advance development of rare disease treatments. The posted agenda emphasizes patient viewpoints and accelerating cures, with panels scheduled to discuss patient-focused agency initiatives, patient engagement opportunities, the promises and perils of AI, and utilizing real-world data and real-world evidence at FDA.

In July 2024, FDA established its Rare Disease Hub to serve as a point of collaboration and connectivity between the Center for Biologics Evaluation and Research and Center for Drug Evaluation and Research with the goal of ultimately improving outcomes for patients. The hub will work across rare diseases, with a particular focus on products intended for smaller populations or for diseases where the natural history is variable and not fully understood.

On February 2, FDA released the 2026 Strategic Agenda for the Hub. The agenda touts three accomplishments from 2025: the Rare disease Innovation, Science, and Exploration workshop series, which focuses on issues common to development of therapies for multiple rare diseases and for which innovative methods and evolving regulatory science may offer valuable solutions; the Rare Disease Policy and Portfolio Council, a cross-Center collaborative body that meets monthly to convene senior medical product leadership and subject matter experts from CBER and CDER to align on regulatory approaches regarding complex challenges in rare disease drug development relevant to both Centers; and the creation of a public-facing email address and website to promote contact with the rare disease community and to facilitate the sharing of information about Hub initiatives.

Looking ahead, the Hub's strategic agenda clarifies that FDA will prioritize advancing regulatory science of rare disease therapies, including by enhancing opportunities for consideration of novel endpoints, biomarker development and assays, innovative trial design, real-world evidence, and statistical methods. Toward this end, FDA will continue to promote its RISE workshops, with the next one scheduled for March 30, 2026. The agency will also strengthen coordination between medical product Centers, including alignment on review standards and decision-making, with future RDPPC meetings to regularize cross-Center communication regarding the same or related diseases or issues. FDA plans to create a centralized point of contact for external partners, including an FDA website consolidation project, and publication of a GovDelivery rare disease focused newsletter.

In November 2025, FDA leaders published an article in the NEJM outlining a new "plausible mechanism pathway," under which a drug or biologic manufacturer may be able to obtain FDA marketing authorization for products targeting specific, well-understood genetic abnormalities. This approach is not intended to be limited to rare disease treatments. Instead, this approach intends to address areas of unmet need where traditional trials would be infeasible, and where data from such bespoke treatment can inform regulatory decisions regarding the product. A draft of a guidance document for this new pathway was recently circulated to White House for review.

In September 2025, FDA announced its "Rare Disease Evidence Principles," under which eligible drugs and biologics for ultra-rare diseases caused by known genetic defects will receive assurance of FDA consideration of additional supportive data that may be used to meet regulatory approval standards. For programs meeting the eligibility requirements, FDA said it will offer an additional meeting with the appropriate review team, which could help determine what data could be used to demonstrate "substantial evidence" of effectiveness.

Separately, the EveryLife Foundation for Rare Diseases will welcome advocates from 49 states, the District of Columbia, and Puerto Rico to Rare Disease Week on Capitol Hill, February 24 through February 26, 2026, in Washington, D.C. A free event, Rare Disease Week convenes patients, caregivers, advocates, and policymakers to elevate the rare disease patient voice and advance policies that improve the lives of millions of Americans living with rare conditions.

Rare Disease Week 2026 will introduce several updates designed to strengthen community connection, expand storytelling opportunities, and create a more intentional advocacy experience. The agenda will now include an additional half-day of programming on Tuesday, February 24. This added time will feature dedicated sessions led by community leaders, including 2025 RareVoice Award winners, focused on helping advocates craft, refine, and amplify their personal rare disease stories. Tuesday's programming will conclude with a Welcome Reception and dedicated meet-up opportunities for YARR participants, RDLA Advisory Committee members, and Community Scholarship recipients.

The Legislative Conference will take place on Wednesday, February 25 with a refreshed structure that provides attendees with more opportunities to connect and plan with advocates from their home states. The conference will also feature a gallery showcasing the work of the 2025 Rare Artists, highlighting the intersection of art, advocacy, and lived experience within the rare disease community.

On Thursday, February 26, the Senate Special Committee on Aging will hold a hearing at 9:30 a.m. ET to examine the FDA's role in rare disease therapy development. Then at 12:30 p.m. ET attendees will have the opportunity to attend the Rare Disease Congressional Caucus Briefing before meeting with their Members of Congress on Capitol Hill.

In 2025, more than 800 advocates from 49 states and representing over 184 patient organizations attended over 363 meetings with Members of Congress and/or staffers during Rare Disease Week. There is no charge for advocates to participate in Rare Disease Week, and advocacy training is provided for all who attend. The EveryLife Foundation provides travel reimbursements for advocates who may need assistance through the organization's Rare Giving program. In 2025, the Rare Giving program provided more than $220,000 in travel reimbursements to 209 advocates.