Exclusivity carrot to repurpose drugs for Orphan diseases

House Reps. Gus Bilirakis (R-Fla.) and G.K. Butterfield (D-N.C.) introduced a bill intended to spur companies with approved drugs to repurpose them to treat rare diseases. H.R. 5750, the Orphan Product Extensions Now Accelerating Cures and Treatments Act of 2014 (OPEN Act), would offer companies an additional six months of market exclusivity for adding a rare disease indication to the label of an approved drug. The bill was referred to the Committee on Energy & Commerce.

Julia Jenkins, executive director of the EveryLife Foundation for Rare Diseases, told BioCentury the extra six months of exclusivity would offer an economic incentive that could offset "perceived risks" associated with testing an approved drug in a "very sick population." She said companies fear that a serious adverse event might hurt the drug's larger market. ...