Hopes, Fears for Orphan Drug Act

Passed 40 years ago, it may be time for ODA 2.0

By: Steve Usdin, Washington Editor, BioCentury

As the Orphan Drug Act slides into middle age, patient advocates and biopharma companies are applauding its accomplishments, lamenting legislative setbacks that have limited its utility, and developing proposals for expanding its reach.

In the 40 years since the ODA was signed into law, FDA has approved more than 600 drugs for orphan conditions, up from 10 in the prior decade. The success has been tangible, but new policies will be needed to increase the pace of drug development if society is to come close to meeting the needs of the millions of patients living with thousands of rare conditions that have no treatments.