FDA on Rare Disease Day: New initiatives and an upcoming conference

Regulatory NewsRegulatory News | 01 March 2021 |  By 

Janet Woodcock, MD

On Monday, the US Food and Drug Administration (FDA) announced a series of efforts and a day-long conference centered on addressing the unmet needs of patients with rare diseases. FDA leaders announced the initiatives in recognition of Rare Disease Day, observed on February 28.
A new request for applications (RFA) issued by FDA augments the agency’s Orphan Products Grants Program. The RFA is seeking natural history studies to address unmet needs in rare diseases, looking for approaches that are both innovative and efficient.
“Both natural history studies and clinical trials for rare diseases are supported through the Orphan Products Grants Program with a goal to increase the number of treatments for rare diseases with an unmet medical need and exert a broad and positive impact on rare disease drug development,” according to the FDA Voices article penned by acting FDA commissioner Janet Woodcock, MD, and Janet Maynard, MD, director of the agency’s Office of Orphan Products Development.
The Orphan Products Grant Program is mandated by congress, and Woodcock and Maynard pointed to the fact that 2020 saw several product approvals associated with the program, including teprotumumab-trbw (Tepezza; Horizon Therapeutics) for thyroid eye disease and triheptanoin (Dojolvi; Ultragenyx) to address nutritional needs for individuals with long-chain fatty acid oxidation disorders. Overall, the program’s support has contributed to more than 70 product approvals since it began supporting rare disease clinical trial research in 1983.
In the same announcement, the FDA officials announced that the Center for Biologics Evaluation and Research (CBER) is working with two other federal agencies to develop a “Bespoke Gene Therapy Consortium.” CBER, together with the Foundation for the National Institutes of Health and the National Center for Advancing Translational Sciences, is building the consortium “to provide a standardized and efficient approach for development and delivery of bespoke [adeno-associated virus]-based gene therapies,” wrote Woodcock and Maynard. The effort is envisioned as a way to foster collaboration, and to harness regulatory innovation in the service of helping streamline a complex development process.
On 5 March, FDA will be holding a rare disease-focused virtual public meeting. At the meeting, FDA plans “to highlight strategies to support rare disease product development,” according to the announcement, by sharing examples of past rare disease product development programs, highlighting how natural history studies aid rare disease product development, and how patient engagement can inform the product development path.
FDA staff from the Office of Orphan Product Development and outside experts will be on hand for the morning’s presentations, while Woodcock and the directors of FDA’s centers will attend an afternoon discussion addressing rare disease product development in the individual medical product centers. Regulatory Focus will be reporting from the conference, providing regulator insight into rare disease product development.


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