While much progress has been made in approving treatments for rare diseases, more work is needed in this area, asserted Kerry Jo Lee, MD, associate director of rare diseases in the US Food and Drug Administration’s (FDA) Office of New Drugs (OND).
Lee addressed some of the agency’s work to spur the development of rare disease treatments and provided an update on the agency’s rare disease endpoint advancement (RDEA) pilot program, as well as recent approval trends at the agency’s annual Regulatory Education for Industry (REdI) conference on Tuesday.
The number of orphan drugs approved by the agency has dramatically increased over the past ten years.
Lee said the number of orphan drugs approved by the agency is now on par with other novel approvals. From 2015 to 2022, about half of new drugs and biologics were approved for rare disease indications. FDA defines rare diseases as ones that affect fewer than 200,000 people in the US.
She noted that the majority of orphan drugs are submitted through one or more of the agency’s expedited pathways, including priority review, fast-track, accelerated approval and breakthrough therapy designation.
Yet despite the “significant progress” being made in developing rare diseases, more work is needed, Lee said. “We are very, very cognizant of the fact that 30 million Americans have a rare disease and the vast majority of these patients do not have approved treatments,” she said.
ARC focusing on collaborating
Lee said that since CDER’s Accelerating Rare disease Cures (ARC) program launched in May 2022, much work has been done to strengthen internal partnerships at the agency.
She said that ARC serves as an “umbrella” organization and connects the activities of multiple offices to bridge the work being done in orphan drug development, including the Office of New Drugs, the Office of the Center Director and the Office of Translational Science.
Progress has also been made with engaging external experts on expediting the development of rare diseases. The ARC team has met with 23 rare disease patient groups over the past year and has participated in 19 patient-focused drug development meetings.
In other areas, ARC recently launched a quarterly newsletter that highlights the team’s activities; the launch was designed to heighten the transparency of the office.
ARC also just launched a metric onto the agency’s FDA-TRACK performance dashboard to compile the number of orphan drug approvals versus total approvals.
FDA to start accepting RDEA proposals
Lee said that FDA will start accepting applications on 1 July for RDEA, and that the agency will accept a single proposal for the pilot for Q4 FY 2023. For FY 2024-2027, FDA will accept up to one RDEA proposal per quarter, for a maximum of three proposals per year.
Sponsors seeking to participate should have an active pre-investigational new drug application (IND) or IND for a rare disease, and a novel efficacy endpoint intended to establish substantial evidence of effectiveness for a rare disease treatment.
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