FDA, kickin’ butt and taking orphan names

A recent blog by FDA commissioner Scott Gottlieb outlines the progress FDA has made eliminating the backlog of ~200 orphan drug designation requests. Recall, an orphan or rare disease is one that “generally affects fewer than 200,000 people in the United States.” But these orphans aren’t cute. They are big business. According to EvaluatePharma “worldwide orphan drug sales are forecasted to total $209bn in 2022.” According to Gottlieb, the number of orphan designation requests more than doubled between 2012 and 2016, rising to 568 new requests. FDA plans to “close a loophole that allows sponsors to avoid an obligation to study drugs in pediatric indications.” The Pediatric Research Equity Act (PREA) was enacted to promote more pediatric studies. Hasn’t happened but Dr. G. plans to change that. Bravo.