As Congress debates orphan reforms, new analysis finds 65% of orphan drugs approved for a single rare disease

As Con­gress con­sid­ers whether to shift away from or­phan drug in­cen­tives that have pro­vid­ed a wind­fall for in­dus­try in some cas­es, a new FDA and HHS analy­sis finds that the vast ma­jor­i­ty of such or­phan ap­provals are grant­ed for a sin­gle rare dis­ease, ac­cord­ing a three-decade re­view.

In a pa­per pub­lished in Health Af­fairs Mon­day, Kath­leen Miller, deputy di­rec­tor of the di­vi­sion of sci­ence and pub­lic health pol­i­cy at HHS, and the FDA’s Michael Lan­thi­er found that of the 491 or­phan drug ap­provals be­tween 1990 and 2022, 65% were ap­proved for a sin­gle rare dis­ease while 15% were ap­proved for mul­ti­ple rare dis­eases and 20% were ap­proved for both rare and com­mon dis­eases. Fur­ther, 10% of or­phan drugs sub­se­quent­ly re­ceived a pe­di­atric in­di­ca­tion for an or­phan dis­ease.

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