CLAREMONT, Calif.—Staff members at the Food and Drug Administration are doing something unusual. They are leaving Washington to help drug makers take a crucial step in developing drugs for rare diseases.
Hempel FamilyTwins Addison, left, and Cassidy Hempel, who have a fatal cholesterol metabolism disorder, get cyclodextrin infusions April 13 in Reno, Nev.
The staffers help administer the Orphan Drug Act, which provides incentives to create therapies for so-called orphan diseases—those that affect fewer than 200,000 Americans. There are about 7,000 such maladies, most of them serious, that have few or no drugs to treat them, from adenoid cystic carcinoma, a rare head and neck cancer, to Zollinger-Ellison syndrome, which is associated with a tumor that causes the production of high levels of stomach acid.
As a result, doctors may end up prescribing drugs developed for other diseases off-label, but not all insurers will cover this kind of use.
Getting an orphan-drug designation opens the door to incentives once the FDA approves a medicine for sale in the U.S., including seven years’ marketing exclusivity and tax breaks. Last year, just 250 requests for orphan-drug designation were filed, and 160 received it.
“We’re barely scratching the surface,” says Timothy Coté, director of the FDA’s Office of Orphan Products Development, the workshop’s sponsor. He says there are roughly 350 orphan drugs approved, covering about 150 rare diseases.
Tim Cunniff, vice president of global regulatory affairs at Lundbeck Inc., which has a number of approved orphan drugs, says most companies developing orphan drugs are small.
Big companies are starting to get more interested in rare diseases, but the key issue is the high cost of developing a drug and the typically long time it takes to move it from a lab into a clinic as a treatment that gets prescribed. Before starting down this arduous path, a company needs to feel there is a reasonable chance of making a profit.
A mother of twin daughters who have a rare disease gets guidance and files an application at an FDA workshop on orphan drug designations.
To help get more applications, Dr. Coté’s office put out the word: Help is available, in two workshops with on-the-spot regulatory advice. The first workshop, held last month at the Keck Graduate Institute here, drew 29 potential sponsors, from major drug companies to academic centers, small biotechs and even some patient advocates. In a follow-up survey, 74% said they had never before filed an application for orphan drug designation.