On January 4th, 1983 President Ronald Reagan signed into effect the Orphan Drug Act. For over 30 years, orphan drug development has grown in volume, and has had a major impact on the biopharma and diagnostics industries. With advancement in technology including increased development of novel companion diagnostics as well as the mapping of the human genome, orphan drug approvals by FDA have continued to rise. New data has been released showing that 2013 was a big year for orphan drug approvals. In 2011, FDA approved 26 orphan drugs. The number dropped slightly in 2012 g to 25, but rebounded in 2013 to a record 31. Orphan drug designations were also high in 2013 reaching a record breaking 260.
It is estimated by PHRMA in a 2013 Medicines in Development report entitled “Rare Diseases: A Report on Orphan Drugs in the Pipeline,” that 30 million Americans have one of the 7000 diseases deemed rare. Rare disease is defined as one affecting under 200,000 people in the United States. Further, this report estimated that there were 452 medicines and vaccines in development for rare disease in 2013. To review this report, click here. To review a recent article in medcitynews.com covering the surge in orphan drug development and approvals, click here.
Pearl Pathways has the pleasure of working with several of our clients on developing the appropriate regulatory strategy, executing global regulatory filings, and assisting with clinical trials for orphan drugs. Contact us if you need assistance with your orphan drug or device.
We anticipate that this focus on orphan drug development will continue among both large pharm and small biotechs. Navigating these diseases in small patient populations is complex and can be daunting, but new technologies and the regulatory landscape have made it a bit easier to impact rare and debilitating diseases.