New report shows price increases for orphan medications correlated with non-orphan use

Trend could jeopardize the affordability of life-saving orphan drugs for patients with rare diseases and conditions.

The analysis looked at a sample of 46 orphan drugs available from 2012 to 2014 and found only a handful were used exclusively for the rare orphan disease they were developed to treat. As these medications are increasingly prescribed for conditions beyond their original orphan indication, on-label or off-label, their already high prices increased by upwards of 37% on average while generating blockbuster profits for drug companies.

This report looked at only 46 orphan medications. With 500 orphan treatments approved by the FDA, the widespread economic impact of these price hikes pose a serious financial impact on the health system.

“A proper balance has to be struck between ensuring that the incentives remain for those firms focused on developing these very important, and much-needed orphan disease therapies, while not allowing for the [Orphan Drug] Act to be exploited purely for financial gain,” says report author Craig F. Burns, Vice President of Research, The Center for Policy & Research, at AHIP.

Key findings include