Orphan drugs provide unique opportunity for manufacturers

Among the special designations the Food and Drug Administration can ascribe to treatments, orphan drugs have seen steady growth recently — growth that, because of these drugs’ ability to help patients with few or no options and the market exclusivity that the designation promises, is likely to continue.

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In the last two years, the FDA has broken its record for the approval of these drugs to treat diseases that affect 200,000 or fewer people. In 2014, of the 41 novel new drugs approved, 17 were orphan drugs for rare diseases, and in 2015, that number grew to almost half of the total novel new drug approvals — 21 of 45 were orphan drugs.



But why are manufacturers making such a heavy push among drugs meant to treat tiny patient populations? As Diplomat Pharmacy emerging therapeutic analyst Ryan Chandanais told Drug Store News, “rare diseases aren’t really that rare if you look at them as a whole.” Indeed, the 17 orphan drugs among novel new drug approvals in 2014 were only a fraction of the 49 approved orphan drugs that year — a large increase from the 32 that were approved in 2013. For manufacturers, orphan drugs represent a unique opportunity, in part because of the speed with which they can be approved.



Rare diseases also present a unique business opportunity for drug makers, according to Chandanais.



“I think manufacturers are seeing opportunities in the rare disease states,” he said. “In many cases there are no available treatments for some of these rare diseases or very few available, and if they get a drug approved then they can have the complete market or a significant portion of the market available to them with little or no competition.”