FDA Database Aims to Spark Orphan-Disease Drug Development for Rare Diseases
For months now, the Food and Drug Administration has been trying novel ways of encouraging drug makers to develop drugs for rare diseases.
Today the agency’s Office of Orphan Products Development is launching a new gambit—a database of FDA-approved compounds and products that show promise in rare diseases too.
The concept is called “repurposing” — finding new ways to use products that have already been okayed by the FDA for some other use.
The already-approved products in the Rare Disease Repurposing Database are unique in that they’ve also previously received orphan-drug designation, meaning they’ve shown potential to treat one or more of the diseases affecting 200,000 or fewer Americans. (While orphan-drug status doesn’t guarantee FDA approval as a treatment for the rare disease, it’s a prerequisite for getting incentives such as tax breaks and marketing exclusivity if the agency does okay the drug for that purpose.)
Timothy Coté, director of the orphan products development office, says that testing an already-approved drug as a treatment for a rare disease has significant advantages — for one, it’s already been found safe by the FDA. And running trials on an existing drug is much cheaper than trying to develop a totally new compound.
Despite the incentives in the Orphan Drug Act, there are only about 350 such drugs that have been approved — and there are some 7,000 rare diseases.
Information in the database is already publicly available through the FDA but until now has been scattered. Coté says officials went through more than 2,000 orphan drug designations to compile the new 235-drug database, which he says is likely to be used not only by drug companies but also by venture capitalists looking for promising investments and patient advocacy groups eager to drive drug development for their rare condition.
Coté says he hopes the new database will spark drug development efforts. But he plans to do some repurposing of his own. He says the next step after the database is released is for his office to carefully study the list, identify some particularly promising products, and urge the companies to move forward.
June 23, 2010