The Orphan Drug Act of 1983 (ODA) opened the doors, business-wise, for pharma companies to pursue drug development with some assurance that the business could be profitable. Activity percolated in the following years, but has significantly ramped up in the past few years. An eye-catching 17 of the 39 drug approvals granted by FDA in 2013 were for orphan drugs exclusively (generally defined as treatments for diseases with fewer than 200,000 patients in the US). Thanks to fast-track review protocols and reduced development timelines, a variety of tax breaks, marketing exclusivity (seven years for the first drug approved for a given orphan designation), and other financial incentives, and the opportunity for label extension later, many biopharma companies are now viewing these small market opportunities as big business.
Click here to read the article in Pharmaceutical Commerce.