Researchers Argue Orphan Drug Designation Has Been Overused

March 11

A new study published in Health Affairs found that spending in the orphan drug category is overwhelmingly concentrated on so-called partial orphan drugs, which have both orphan and nonorphan indications. The study affirms growing concerns across the health care industry that drugmakers are misusing the orphan drug designation and introducing unwarranted cost into the drug channel.

“Our findings raise questions regarding whether the benefits of orphan drug status are always warranted for partial orphan drugs,” said Kao-Ping Chua, M.D., Ph.D., lead author of the new paper and a health services researcher at the University of Michigan, in a Feb. 26 press release.

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Radar on Drug Benefits

Peter Johnson

Peter has worked as a journalist since 2011 and has covered health care since 2020. At AIS Health, Peter covers trends in finance, business and policy that affect the health insurance and pharma sectors. For Health Plan Weekly, he covers all aspects of the U.S. health insurance sector, including employer-sponsored insurance, Medicaid managed care, Medicare Advantage and the Affordable Care Act individual marketplaces. In Radar on Drug Benefits, Peter covers the operations of (and conflicts between) pharmacy benefit managers and pharmaceutical manufacturers, with a particular focus on pricing dynamics and market access. Before joining AIS Health, Peter covered transportation, public safety and local government for various outlets in Seattle, his hometown and current place of residence. He graduated with a B.A. from Colby College.