The FDA has unveiled a plan to eliminate its backlog of requests for orphan drug designations by September 21, then respond to all new designation requests within 90 days of receipt—actions the agency said would bring more such treatments to market and begin rolling back their prices.
FDA Commissioner Scott Gottlieb, M.D., said the agency’s Orphan Drug Modernization Plan will begin by deploying a “Backlog SWAT team” to process the backlog of about 200 orphan drug designation requests, starting with the oldest requests. The team will consist of senior, experienced reviewers with “significant” expertise in orphan drug designation.
The number of designation requests handled through the Orphan Drug Designation Program has risen steadily over the past five years, the FDA said. Last year, the agency’s Office of Orphan Products Development received 568 new requests for designation—more than double the number of requests received in 2012.
“People who suffer with rare diseases are too often faced with no, or limited, treatment options, and what treatment options they have may be quite expensive due in part to significant costs of developing therapies for smaller populations,” Dr. Gottlieb, said in a statement.
“Congress gave us tools to incentivize the development of novel therapies for rare diseases and we intend to use these resources to their fullest extent in order to ensure Americans get the safe and effective medicines they need, and that the process for developing these innovations is as modern and efficient as possible,” he added.
Dr. Gottlieb first disclosed the new policy last week in testimony before a subcommittee of the U.S. Senate Committee on Appropriations, where he discussed the FDA’s proposed budget for FY 2018.
Abuses Under Investigation
The FDA’s action comes three months after the U.S. General Accountability Office said it will investigate potential abuses of the Orphan Drug Act at the request of U.S. Senators Orrin Hatch (R-UT) and Tom Cotton (R-AR). The investigation came weeks after NPR (National Public Radio) and the nonprofit healthcare policy and politics news service Kaiser Health News concluded that the system created to foster development of orphan drugs “is being manipulated by drug makers to maximize profits and to protect niche markets for medicines already being taken by millions.”
The news outlets acknowledged that such manipulation was legal—but added that as a result, drug makers had set sky-high prices for many “orphan” treatments for diseases affecting fewer than 200,000 people. Thirty-three orphan drugs were found to cost at least $28,000 for a 30-day supply, with four costing more than $70,000 per month.
The FDA said it also plans to roll out a new Designation Review Template designed to increase the consistency and efficiency of its reviews. The program will collaborate within the agency’s medical product centers to create greater efficiency—including reviewing rare pediatric disease designation requests by conducting joint reviews with the Office of Pediatric Therapeutics.
To ensure all future requests receive a response within 90 days of receipt, the agency said, it will undertake a series of actions that include reorganizing its review staff, tapping into expertise across the FDA’s medical product centers, and establishing a new FDA Orphan Products Council. The Council would be charged with helping address scientific and regulatory issues, to ensure agency consistency in regulating orphan drug products and reviewing designation requests.
The orphan drug measures are part of a broader “Medical Innovation Development Plan” designed to lower orphan drug costs. The agency said it “intends” to disclose its progress on successful elimination of the backlog by mid-September.