FDA Releases Strategic Agenda for Rare Disease Innovation Hub

Rare Daily Staff

The U.S. Food and Drug Administration released a strategic agenda for its newly created Rare Disease Innovation Hub, an outline of the hub’s actions during its first year highlighting three goals.

FDA created the Rare Disease Innovation Hub to serve as a point of collaboration and connectivity between the Center for Biologics Evaluation and Research and the Center for Drug Evaluation and Research, with the goal of improving patient outcomes. Although the Hub will work across rare diseases, it will focus on products intended for smaller populations or for diseases where the natural history is variable and not fully understood.

The Hub is co-led by the directors of Center for Biologics Evaluation and Research and the FDA’s Center for Drug Evaluation and Research, in close collaboration with the FDA’s Center for Devices and Radiological Health, Oncology Center of Excellence, Office of Orphan Products Development, and Office of Combination Products. The agenda follows input from and partnership with the larger rare disease community.

The plan calls for the Hub to advance the regulatory science of rare disease therapies, such as by considering novel endpoints, biomarker development and assays, innovative trial design, real-world evidence, and statistical methods.
It also seeks to strengthen coordination between various medical products centers within the agency, particularly the Center for Drug Evaluation and Research and the Center for Biologics Evaluation and Research. The hope is to create greater cross-center consistency on rare disease standards and decisions and facilitate regular collaboration between leadership and review teams.

And it calls for the creation of a centralized point of contact for external partners and to serve as the primary point of engagement with the rare disease community. This includes patient and caregiver groups, trade organizations, and scientific or academic organizations, for matters that intersect rare disease drug development.

“It is the hope and expectation of FDA leadership and the Hub that this agenda will evolve as the Hub evolves. It is also the expectation that the rare disease community will remain heavily engaged in this evolution,” the agency said. “Throughout the year, there will be multiple ways for the community to engage with and offer suggestions to the Hub, both for its immediate use and for the Hub to consider for the 2026 Strategic Agenda.”