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.

RARECast is a weekly series by Daniel S. Levine. Levine is an award-winning business journalist who has reported on the life sciences, economic development, and business policy issues throughout his 25-year career. He founded Levine Media Group in 2013, which produces The Bio Report and RARECast podcasts. Read his full bio here. 

1 thought on “RARECast: Why the OPEN ACT Could Greatly Expand Treatments for Rare Disease”

  1. I enjoyed this RARECast. The Rare Disease community desperately needs treatments. We will gladly stand behind the Open Act. I am the mother of a RARE child. Our son Hunter is three years old with Spinal Muscular Atrophy (SMA) Type 1. We learned of a study that found Celebrex caused increased suppression of SMN. SMA is caused by the absence of SMN. The less SMN the body produces, the more severe SMA is in the individual. We have been using Celebrex off label for more than two years now. The Open Act would benefit the SMA community. We informed our SMA support groups of our use of Celebrex. Many others started using it. However, many doctors are hesitant to prescribe Celebrex for this off label use. Thank you for your continued efforts!

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