Skip to Main Content

As regulators approve a growing number of orphan drugs, prices for these medicines, which treat small groups of patients with rare diseases, have been climbing ever higher. And a new study finds that, while the drugs may offer larger health gains than other medications, they are not nearly as cost-effective.

To wit, the researchers found that orphan drugs were five times more likely to offer a health benefit than other medicines approved by the Food and Drug Administration. But the median costs were substantially higher — $47,650 versus $2,870. Moreover, payers would have to spend 2.7 times more money on an orphan drug than another medicine in order for a patient to gain an extra year of perfect health.

Unlock this article by subscribing to STAT+ and enjoy your first 30 days free!