Gottlieb plans to clear orphan drug backlog
- New Food and Drug Administration Commissioner Scott Gottlieb is making good on previously stated desire to streamline the regulatory review and approval process and make the agency more efficient.
- The FDA announced Thursday it will launch an Orphan Drug Modernization Plan to eliminate the backlog of orphan drug designation requests.
- The orphan drug designation qualifies the application sponsor for incentives like a tax credit for clinical trial costs, a discount on the prescription drug user fee and eligibility for seven years of marketing exclusivity upon approval.
New FDA Chief Gottlieb has been working quickly during his first few months in office to improve the efficiency of the agency. As promised during testimony to a Senate subcommittee last week, the Commissioner aims to clear the backlog of orphan designation requests within 90 days.
There has been a rise in the development of orphan drugs in recent years as pharmaceutical companies try to capitalize on the high price tags often attached to these drugs. It has meant a boost to profits for the industry, as well as an influx of new treatments for rare diseases.
The Orphan Drug Act was established in 1983 to incentivize development of treatments for rare diseases, which is categorized as having a patient population of less than 200,000 in the U.S.
According to the FDA, there are currently more than 200 orphan drug designation requests pending review. And the number of requests has been rising steadily. The agency approved 568 new designations in 2016, more than double the designations it approved in 2012.
"This is the first element of several efforts the FDA will undertake under its new ‘Medical Innovation Development Plan,' which is aimed at ensuring that the FDA’s regulatory tools and policies are modern, risk based, and efficient," the agency said in a statement.
As part of the Orphan Drug Modernization Plan, the agency will deploy a "Backlog SWAT team" with expertise in rare diseases to review backlogged requests, starting with the oldest ones.
A Designation Review Template will be initiated to improve consistency and efficiency and the agency will collaborate with the Office of Pediatric Therapeutics.
Moving forward, the FDA hopes to ensure any designation requests are reviewed within 90 days of receipt.
The granting of the designation does not guarantee future approval.
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