FDA to expedite orphan drug processing queues

In a press release the U.S. Food and Drug Administration (FDA) released a plan for eliminating the backlog regarding orphan drug application status and also released plans for deadlines to clear the queues.

The new plan termed, the “Orphan Drug Modernization Plan” comes this week after FDA Commissioner Scott Gottlieb last week announced at a testimony before a Senate subcommittee that the backlogs would be cleared within 90 days and all new requests would be cleared within 90 days of receipt of application.

Orphan drugs thus are defined as drugs or biologics that are intended for use as safe and effective methods to treat, diagnose or prevent rare diseases. Rare diseases here are defined as diseases that affect less than 200,000 people in the United States.

This new program is authorized under the Orphan Drug Act and works to provide status of orphan drug. Drugs that receive this status provide several benefits to the sponsor at different stages of its development including tax credits on the costs incurred during clinical trials, relaxation in terms of prescription drug user fee if the disease for which it is prescribed is a rare disease or condition. The drug also enjoys and eligibility for marketing exclusivity of seven years after approval. This makes application for an orphan drug different from filing for marketing application with the FDA for any other drug.

At present the backlog of application for orphan drugs is around 200 strong. Over the past five years there has been a steady rise in number of orphan drug applications. For example in 2016 the FDA’s Office of Orphan Products Development saw 568 new orphan drug applications and this was more than double of what was received in 2012. Looking at past records, of the 4,174 orphan drug designation requests, only 15% or 625 have actually been approved as orphan drugs available to the public.

For the “Orphan Drug Modernization Plan” the FDA would have in place a Backlog SWAT team. This team would have senior and expert reviewers who are experts with orphan drugs. The team will begin with the oldest pending request and move to clear all the backlogs. The reviews would be reported in a new Designation Review Template that is meant to improve the reviews. Reviews will also work closely with the Office of Pediatric Therapeutics so that pediatric orphan drug requests are also taken care of. By mid September the backlog is likely to be cleared.

Orphan drug development has been a positive step especially for rare illnesses and its sufferers. According to FDA Commissioner Scott Gottlieb, the treatment options are little or absent for persons suffering from rare disease conditions. Since development of drugs for a small population is expensive, the treatment for these illnesses also comes dear to the sufferers. Congress allowed for orphan drug development by providing incentives he explained and the FDA continues to use its resources to the best of its ability to make the development of these innovative new drugs “as modern and efficient as possible.”

FDA has a long term plan called the “Medical Innovation Development Plan,” and this orphan drug program is a part of that whole program. The FDA with this plan attempts to sharpen its regulatory tools so that the rules and policies are efficient, based on risks with new agents and are modern in their approach. With effective and safer new drug development, believes the FDA, overall health care costs can also be reduced to a certain extent.

Dr. Ananya Mandal

Written by

Dr. Ananya Mandal

Dr. Ananya Mandal is a doctor by profession, lecturer by vocation and a medical writer by passion. She specialized in Clinical Pharmacology after her bachelor's (MBBS). For her, health communication is not just writing complicated reviews for professionals but making medical knowledge understandable and available to the general public as well.


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