RAREcast: How the Orphan Drug Act Reshaped the Rare Disease Landscape


The U.S. Food and Drug Administration approved more than 230 new drugs to treat rare diseases in the past decade and there are currently more than 450 orphan drugs in development, according to a new report from the Pharmaceutical Research and Manufacturers of America. Though developing drugs for rare diseases presents many challenges, policy changes and scientific breakthroughs have helped changed the landscape. We spoke to Gretta Stone, Deputy Vice President of Policy and Research at PhRMA, about the report, how the Orphan Drug Act encouraged investment in R&D for rare diseases, and what fuels her optimism for drug development efforts to combat this group of diseases in the years ahead. Also, we check in with Run4Rare’s Noah Coughlan as he continues his 3,100 mile run across the country to raise awareness for rare diseases.

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. 

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Global Genes Comments

  1. Are “Biosimilar” drug proposed legislation involved in any of the orphan drugs approved to date?

    Thank you – Annie

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