Orphan drugs accounted for only 7.9% of total drug sales in the U.S. in 2016, according to a recent QuintilesIMS study, but have accounted for more than 40% of new molecular entity approvals over the past three years. The FDA has recently moved to improve the orphan-drug approval process. So what is in store? 

Orphan drugs are defined as drugs that treat diseases that affect 200,000 or fewer people, according to the FDA, which provides incentives, including market exclusivity benefits, for developing orphan drugs. Genzyme, which was acquired by Sanofi in 2011 for $20 billion, optimized the rare-disease drug model with several blockbuster drugs. With increased interest in orphan drugs, the FDA is seeking to reduce orphan-drug backlogs and improve review times. So what is the agency doing?

Inside the orphan drugs market

Orphan drugs accounted for only 7.9% of total drug sales in the US in 2016, according to a study issued by the QuintilesIMS Institute. The study, commissioned by the National Organization for Rare Disorders (NORD), analyzed the role of the Orphan Drug Act and orphan-drug usage and costs, to address a number of public policy and healthcare financing issues in the rare-disease community. In 2016, the Office of Orphan Products Development (OOPD) received 568 new requests for orphan designation, more than double the number of requests received in 2012. In 2016, 9 of the 22 the new molecular entities approved by the FDA were orphan drugs. Of these 22 new molecular entity approvals, two were diagnostic agents, so 9 or 45% of the new drugs approved in 2016 were orphan drugs. In 2015, 21 or 47% of the 45 new molecular entities approved by the FDA’s Center for Research and Evaluation were orphan drugs and in 2014, 17 of the 41 new molecular entities approved or 41% were orphan drugs.

Since the enactment of the Orphan Drug Act in 1983, the US Food and Drug Administration (FDA) has approved almost 600 orphan drugs. The Orphan Drug Act (ODA) provides for granting special status to a drug or biological product to treat a rare disease or condition, which is defined by the FDA as a disease that affects fewer than 200,000 people in the US. Orphan designation qualifies the sponsor of the drug for various development incentives of the ODA, including tax credits for qualified clinical testing, certain exemptions from prescription-drug user fees, and incentives for market exclusivity.  

FDA moves to improve orphan-drug review process

In June 2017, the FDA unveiled a strategic plan to completely eliminate the agency’s existing orphan designation backlog and ensure continued timely response to all new requests for designation with firm deadlines. The agency’s Orphan Drug Modernization Plan is part of a commitment by the FDA Commissioner Scott Gottlieb to eliminate the backlog within 90 days and respond to all new applications within 90 days of receipt. As part of this new plan, by September 21, 2017, FDA was scheduled to have completed reviews of all orphan-drug designations that were older than 120 days. Following that 90-day period, the agency has committed to respond to 100% of all new orphan drug designation requests within 90 days of their receipt by the FDA.

To achieve these goals, among other steps, the FDA said it will create a SWAT Team of senior, experienced, and proficient reviewers to focus on designation requests in the order that the agency receives these requests. To reduce administrative burden on the agency’s experts, the FDA will create and implement a new streamlined “Designation Review Template.” The goal of this new template is to facilitate consistent and efficient reviews of new designation requests. The Center for Drug Evaluation and Research and the Center for Biologics Evaluation and Research will engage in this cross-functional strategy that will also include the participation of the Offices of Pediatric Therapeutics to jointly review Rare Pediatric Disease Designation requests, in order to review orphan drug designation requests in a timely manner, according to the FDA. The FDA said it will also establish an “FDA Orphan Products Council” to address scientific and regulatory issues related to orphan products to ensure a consistent approach to regulating these products.