FDA unveils rare disease innovation hub, plans public meeting this fall
The US Food and Drug Administration (US) announced the creation of a Rare Disease Innovation Hub to speed development of new treatments, integrate regulation of drugs and biologics, and engage patient advocacy communities.The Rare Disease Innovation Hub, or “the Hub”, will focus on diseases that are not fully understood and where development of treatments is “particularly challenging”, the agency explained. It will be co-led by Center for Biologics Evaluation and Research Director Peter Marks and Center for Drugs Evaluation and Research (CDER) Director Patrizia Cavazzoni.
The agency is also recruiting for a new senior leader – Associate Director for Rare Disease Strategy – who will serve as a single contact point of communication and engagement with rare diseases stakeholder groups. The leader will oversee a new joint, integrated CBER/CDER model “to accelerate development of treatments across modalities, including drugs, cell, gene therapies and other biologics”. As part of the job, they will create a rare disease strategic agenda action plan driven by the needs of patient communities.
“In developing such a plan, they will work closely with the CBER and CDER Center Directors, leadership of the CDER Accelerating Rare disease Cures (ARC) program, and comparable CBER programs initiatives to assure appropriate integration and alignment across each center,” FDA explained.
The agency plans to issue a public docket and hold an open meeting this fall to get feedback on the Hub plans and “help shape priorities and initiatives.”
Progress, but persistent unmet need
An estimated 30 million people in the US have rare diseases and over half the new drugs and biologics approved in 2023 were aimed at treatments for small populations, but more needs to be done, according to FDA. (RELATED: New Systems Needed for bringing rare disease treatments, at home devices to market, Regulatory Focus 15 February 2024).
“Many rare diseases lack treatment options and therefore many patients have high unmet medical needs,” FDA noted. “This requires new approaches to expedite development and approval of safe and effective drugs and biologics.”
The new integrated model has three main functions:
- “Serve as a single point of connection and engagement with the rare disease community, including patient and caregiver groups, trade organizations, and scientific/academic organizations, for matters that intersect CDER and CBER.”
- “Enhance intercenter collaboration to address common scientific, clinical and policy issues related to rare disease product development, including relevant cross-disciplinary approaches related to product review, and promote consistency across offices and Centers.”
- “Advance regulatory science with dedicated workstreams for consideration of novel endpoints, biomarker development and assays, innovative trial design, real world evidence, and statistical methods.”
FDA’s announcement did not come as a surprise to stakeholders, as FDA’s Marks had publicly discussed the idea for a rare disease hub at the Drug Information Association (DIA) meeting in June. The National Organization for Rare Disorders (NORD) and other stakeholders reacted positively to FDA’s formal announcement in July.
“BIG congratulations to @US_FDA on the #FDA Rare Disease Innovation Hub, an exciting milestone for millions of Americans in the #RareDisease community!’ NORD wrote on X. “NORD is thrilled to see the Hub take flight and is excited to support it.”
The nonprofit EveryLife Foundation for Rare Diseases had submitted a letter signed by more than 100 patient advocacy organizations to Congress asking for the creation of a Rare Disease Center of Excellence to integrate and streamline processes for treatment development in the 2025 Appropriations Bill. Scientific and policy leaders, with rare disease community members have had an impact, the EveryLife Foundation wrote in a July 17 statement about the Hub news.
“This is a positive and meaningful step forward in addressing the regulatory science challenges unique to rare disease therapy development and we look forward to working with the Agency and stakeholders to ensure that the Innovation Hub is sustained over time,” EveryLife wrote.
FDA
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