Angie Drakulich, in PharmaTech Talk, writes about new FDA policies to further increase its support of Orphan Drugs.
There are more than 6000 rare diseases - ones with patient populations below 200,000 in the US. But, over 30 million Americans have one of those diseases. Since the inception of the Orphan Drug Act, 357 drugs and biologics have come to market.
Given these numbers, the FDA has increased its powers (or had its powers increased) to further help the situation. Since 2007, the FDA has more authority to fast track an Orphan drug to approval. It has been able to work with other regulatory agencies, like EMEA, to allow a single joint orphan-drug filing with both agencies. It's added a new Associate Director for Rare Diseases to its CDER branch that is in addition to its Office of Orphan Products Development (OOPD).
This month the FDA launched its new database to aid rare disease drug development. This was described recently on the FDA transparency blog and posted here. The database contains information on compounds that are already approved and could be applied to other diseases. As noted in the FDA's video, the agency believes it can play a major role in insuring Orphan Drug information is disseminated and shared. That includes a training course to be held in October, and a two committee reports to Congress on rare diseases expected next year.
Frost & Sullivan earlier this year reported that rare and orphan diseases would be an increasing focus of the pharma industry in the next 5-10 years as a hedge against their flagging pipelines. It is also reflective of the opportunity that personalized medicine may represent. The FDA seems to be plugging into the trend to help in any way it can which is good news for patients and their families.
Posted by Bruce Lehr July 2nd 2010.