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 (http://onphr.ma/1EsGJw6) 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.