FDA Rare Disease Innovation Moves Forward

FDA’s Patrizia Cavazzoni, director, Center for Drug Evaluation and Research (CDER), and Peter Marks, director, Center for Biologics Evaluation and Research (CBER), provided an update on the agency’s Rare Disease Innovation Hub (the Hub) in a Nov. 1, 2024 FDA Voices blog post (1). The Hub was created in July 2024 (2) as a cross-center program that operates as a single point of engagement with stakeholders in the development of treatments for rare diseases. The Hub is the agency’s way of creating a community that can share new approaches for drug development for rare diseases.

In establishing the Hub, FDA gathered feedback from stakeholders through a public meeting, held on Oct. 16, 2024 in collaboration with the Reagan-Udall Foundation. The meeting included advocates from rare disease patient groups, academics, regulators, and other stakeholders who discussed how the Hub can work with the community to prioritize work.

A Hub director was also appointed. Amy Comstock Rick will be the Director of Strategic Coalitions for the Hub and be accountable to both Cavazzoni and Marks. Previously, Ms. Rick worked as a consultant at Leavitt Partners and was president and CEO of the Food & Drug Law Institute.

“Ms. Rick brings to this new role more than 20 years of experience as a leader and trusted ally in advocating for policies throughout healthcare, biomedical research, and nonprofit communities, which includes extensive engagement with patient communities and government agencies on issues related to rare diseases. We are excited to have her join and begin creating the important partnerships needed to strengthen the Hub’s engagement throughout the rare disease community,” stated Cavazzoni and Marks in the blog post.

As part of their work with the Hub, Marks and Cavazzoni plan on collaborating with other FDA centers that work with rare diseases, such as the Center for Devices and Radiological Health, the Oncology Center of Excellence, and the Office of Orphan Products Development. In addition, a Rare Disease Innovation Hub Steering Committee has been established to bring the groups together, and a new CBER-CDER Rare Disease Policy and Portfolio Council has been created. The council will bring experts from the different FDA centers together to discuss scientific, regulatory, and policy issues.

“The collaborative model of the Hub will build upon the knowledge and skills of staff within their respective centers, promote adoption of best practices, and avoid confusion over decision-making authorities within those organizations. Meetings between the FDA and sponsors relating to specific drug or biological products will remain the same, as will the way decisions are made about specific programs and how advisory committee meetings are structured and run,” Cavazzoni and Marks stated. “By working together, we believe that we can further facilitate the development, evaluation, and availability of safe and effective therapies. We are committed to ongoing and open communication and look forward to continuing this dialogue with the rare disease community.”

References

1. Cavazzoni, P. and Marks, P.FDA Takes Exciting Steps Toward Establishing the Rare Disease Innovation Hub. FDA Voices. FDA.gov. Nov. 1, 2024. https://www.fda.gov/news-events/fda-voices/fda-takes-exciting-steps-toward-establishing-rare-disease-innovation-hub
2. FDA. FDA Rare Disease Innovation Hub to Enhance and Advance Outcomes for Patients. Press Release. July 17, 2024.