Elizabeth S. Eaton

Published: July 17, 2024

Ref: FDA

The FDA launched its second initiative this year aimed at aiding the development of drugs for rare diseases. The agency’s new Innovation Hub, jointly managed by its Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER), will provide drugmakers with cross-agency expertise to accelerate rare disease treatments.

Over half of all the novel drugs and biologics approved last year were to prevent, diagnose or treat a rare disease or condition, but the agency noted that “more needs to be done.” The hub is intended to serve as a “new model” to further “expedite development and approval of safe and effective” therapies. 

According to the FDA, the hub will have three key functions. First, it will serve as the agency’s engagement arm to help the larger rare disease community navigate important topics, including medical devices, diagnostic tests and combination products. 

The hub is also intended to improve collaboration and consistency across the agency to address scientific, clinical and policy issues related to rare disease drug development, such as product reviews.

To streamline clinical development, hub employees will work to evaluate novel endpoints and biomarkers, and evaluate the use of real-world evidence and varying statistical methods as part of new, innovative trial designs.

The hub will also provide a forum to digest lessons learned from the FDA’s pilot programmes, including the Support for clinical Trials Advancing Rare disease Therapeutics (START) initiative. Under that programme, CDER selected three products for rare neurological diseases, and CBER chose four cell or gene therapies, with the intent of speeding up their development by enabling more frequent communication with the US regulator. All seven slots were filled last month.