FDA Plans to Speed Rare Disease Drug Designation

The agency plans to deploy a team of senior reviewers with expertise in drugs to treat diseases with 200,000 patients or fewer, known as orphan drugs.

The goal will be to eliminate a backlog of 200 orphan drug designation requests, starting with the oldest. The agency aims to clear the backlog by mid-September.

Pharmaceutical companies have become increasingly interested in developing orphan drugs since they can command prices in the hundreds of thousands of dollars.

Soliris, for example, a drug made by Alexion Pharmaceuticals Inc to treat paroxysmal nocturnal hemoglobinuria, a rare disease that destroys red blood cells, can cost up to $440,000 a year.

In 2016 the FDA received 568 new requests for orphan drug designation, more than double the number received in 2012.

"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," FDA Commissioner Scott Gottlieb said in a statement.

The agency is in a broad push to speed new drugs to the market, a mandate expressed in legislation passed last year known as the 21st Century Cures Act.

On Tuesday the FDA released a list of roughly 180 drugs that have lost patent protection but have no generic rivals, and said it will prioritize applications of generic competition for these drugs.

The goal is to prevent the kind of price hikes seen when Martin Shkreli, formerly chief executive officer of Turing Pharmaceuticals, acquired and then raised the price of an old anti-parasitic drug called Daraprim to $750 a pill from $13.50.

The agency plans to respond to orphan drug applicants within 90 days of receiving an application and establish an Orphan Products Council to help ensure the FDA is applying a consistent approach to regulating and reviewing these products.

Drugs that win orphan drug status are given a variety of incentives, including tax credits and eligibility for seven years of marketing exclusivity.