Researchers Argue Orphan Drug Designation Has Been Overused

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.

© 2023 MMIT
Peter Johnson

Peter Johnson

Peter has been a reporter for nearly a decade. Before joining AIS Health, Peter covered a wide variety of topics in his hometown of Seattle, where he continues to live. Peter’s work has appeared in publications including The Atlantic and The Stranger. Peter attended Colby College.

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