Researchers Say Reform of the Orphan Drug Act is Needed

The Orphan Drug Act has provided critical incentives that have helped fuel the development of scores of drugs to treat rare diseases. But health experts at Johns Hopkins Medicine are calling for reform of the act to stop potential abuses by drugmakers they say have gotten huge subsidies and tax breaks for drugs that have been used far more broadly than the law intended to reward. We spoke to Martin Makary, professor of surgery at Johns Hopkins and a coauthor of a recent commentary in the American Journal of Clinical Oncology, about the Oprhan Drug Act, the unintended role it is playing in the growing controversy over drug prices, and why he thinks reform that protects the original intention of the act is needed.