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The Rise Of Orphan Drugs

posted by AHIP

on September 10, 2019


While drugs are being introduced at higher price points – orphan drugs are being introduced at price points that are many times that of other types of drugs. From 1998 to 2017, the average annual cost for orphan drugs increased 26-fold, while the cost for specialty and traditional drugs doubled.

They are being approved – and entering the market – at higher rates than ever before. Among newly launched drugs, the share of orphan drugs increased more than 4-fold, from 10% to 44%, over the same 20-year period.

They are also being prescribed in increasing numbers – often, for conditions that aren’t orphan conditions and can be treated with other therapies. In 2017, seven of the top 10 best-selling drugs were orphan drugs that were widely prescribed for non-orphan indications and off-label uses.

As a result, orphan drugs are one of the biggest factors driving out of control drug prices, which puts coverage and care out of reach for millions of hardworking American families.


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