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…
