More than 70 percent of U.S. spending on top-selling “partial orphan drugs”—drugs for rare diseases that also are approved to treat common diseases—is for non-orphan indications, a study in latest edition of Health Affairs concludes.
Makers of partial orphan drugs could be earning substantial sums, in part, because 340B drug discounts on orphan drugs are not owed on sales to rural and free-standing cancer hospitals, the study by Kao-Ping Chua and Lauren E. Kimmel of the University of Michigan and Rena Conti of Boston University notes. Policymakers who “wish to decrease the rewards for repurposing a common disease drug to treat rare disease” might consider limiting the 340B discount exemption “to instances in which the drug is used for the rare disease for which it has orphan drug designation,” the researchers said. U.S. Reps. Peter Welch (D-Vt.) and David McKinley (R-W.Va.) have introduced such legislation, they pointed out.
More than 70 percent of U.S. spending on top-selling “partial orphan drugs”—drugs for rare diseases that also are approved to treat common diseases—is for non-orphan indications, a study in latest edition of Health Affairs concludes. Policymakers who “wish to decrease the rewards for repurposing a common disease drug to treat rare disease” might consider limiting the 340B discount exemption “to instances in which the drug is used for the rare disease for which it has orphan drug designation,” the researchers said.
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