Why the OPEN ACT Could Greatly Expand Treatments for Rare Disease

Though there are almost 7,000 rare diseases, less than 500 drugs have been approved to treat any one of them. What that means for millions of people with a rare disease is that there’s no approved treatment they can use. There is, however, a growing effort to look at drugs approved for other uses and repurpose them to treat rare diseases in which they might be effective. We spoke to Julia Jenkins, executive director of the EveryLife Foundation for Rare Diseases about why repurposing approved drugs could greatly increase the available treatments for rare diseases, what draft legislation would do to encourage drugmakers to pursue these opportunities, and why experiences from past legislation suggest the approach could be quite effective.