Once upon a time, the pharmaceutical industry all but ignored potential treatments for rare diseases, which by virtue of their small patient numbers offered little promise of a good financial return. That changed with the 1983 passage of the Orphan Drug Act, which gave pharmaceutical companies financial incentives to develop drugs for rare, or “orphan,” diseases. The Act was, arguably, quite successful: more than 400 orphan drugs have been approved since, and hundreds of applications are submitted each year. But now, as the cost of those orphan drugs skyrockets, there’s a debate over whether the Act has backfired.

The booming orphan drug business also recently forced the FDA to increase its timeframe for reviewing orphan drug applications (their goal is now to review 75% of applications within 120 days, instead of 90 days). And orphan drugs also appear to be drawing increased FDA scrutiny: the agency recently revoked the orphan drug designation on four products.

This article examines the history of the orphan drug market, how it’s developed from a pariah to a cash cow for the pharmaceutical industry, and what that means for patients.

The Washington Post: High prices make once-neglected ‘orphan’ drugs a booming business.