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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.


“Historically, orphan drugs have been very expensive, but that’s been perceived as okay by society, because such drugs have had a small impact on budgets while greatly helping patients. But with a growing number of orphan drugs, health care budgets are effected in a significant way,” said James Chambers, a study co-author and an associate professor in health economics at Tufts Medical Center.

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