FDA Commissioner Vows to Close the “Orphan-Drug Loophole”

The Orphan Drug Act allows the U.S. Food and Drug Administration (FDA) to approve medicines intended to treat rare diseases affecting fewer than 200,000 people through an accelerated review process, and it qualifies manufacturers to receive various incentives for such products (tax credits for clinical trial costs, relief from prescription drug user fees, and potential marketing exclusivity for up to 7 years after a drug is approved).

Addressing concerns that pharmaceutical manufacturers are exploiting this “orphan-drug loophole,” FDA Commissioner Scott Gottlieb, MD, announced that the agency is implementing several new policies to improve the Orphan Drug Act, including a provision that lets pharmaceutical companies avoid evaluating a drug in pediatric clinical trials.

When a drug is granted orphan designation for a pediatric subtype of an otherwise common and “non-orphan” adult disease, the designation does not require the sponsor to study the drug in pediatric populations as required in the Pediatric Research Equity Act (PREA), Dr. Gottlieb explained in his FDA Voice blog. “By granting the drug a pediatric orphan designation, it means the drug never has to actually be studied for a pediatric use,” he continued. “It’s a loophole that is in direct opposition to what Congress intended [when it passed the Orphan Drug Act in 1983].”

The FDA plans to create guidance documents that will close this “inadvertent loophole” and ensure more pediatric research, he added.

These actions will continue the agency’s attempts to improve and modernize the review and approval of orphan drugs. Earlier this year, Dr. Gottlieb launched a plan to eliminate the agency’s backlog of orphan-drug designation requests within 90 days, as well as respond to any new requests for the designation within 90 days of receipt. In his recent post, he announced that the backlog of 200 orphan-drug designation requests has been eliminated as of August 28 – ahead of the original September 21 deadline.

To continue this momentum, the FDA is developing strategies to reduce delays and redundancies in the review process and plans to introduce a new process map this fall.

Sources: FDA Voice blog, September 12, 2017; STAT News, September 12, 2017.