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Orphan Drug Changes Brewing at the FDA

Initiatives to De-Risk Drug Development Could Include Redesigning Clinical Trial Requirement

• Ritu S. Baral

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FDA officials have pointed out that orphan drugs must prove probable benefit and safety to the same degree as all other drugs. The complexity of these diseases added to FDA’s stance on approval translates into the fact that drug development for orphan diseases can be risky and unclear. [Shutterstock images/Tom McNemar] Click Image To Enlarge +

FDA officials have pointed out that orphan drugs must prove probable benefit and safety to the same degree as all other drugs. The complexity of these diseases added to FDA’s stance on approval translates into the fact that drug development for orphan diseases can be risky and unclear. [Shutterstock images/Tom McNemar]

There is an initiative under way to revamp the way FDA approaches orphan drug development that goes above and beyond the 1983 Orphan Drug Act. The 2010 Brownback/Brown amendment was created as a market-based approach to address rare and neglected diseases by incentivizing biotech and pharmaceutical companies to invest in drugs for these conditions.

The first push to incentivize drug companies to develop therapies for orphan diseases, the 1983 U.S. Orphan Drug Act, was focused more on the economic incentives for drug development as market size was inherently limited. Companies were given seven years of data exclusivity, approval application fee waivers, and tax credits for up to 50% of R&D costs.

The Brownback/Brown amendment requires formation of an FDA team that will submit a report to Congress by March 11, 2011. The document is supposed to cover current issues regarding development as well as approval of therapies for rare and neglected diseases. As per the Brownback/Brown amendment, FDA held an open public hearing to gather suggestions.