The US Food and Drug Administration (FDA) has unveiled a 90-day plan to address the backlog of around 200 orphan drug designation requests that have been pending for more than 120 days. The regulatory body aims to complete reviews of all requests in this category by September 21st 2017 and plans to adhere to this 90-day response timeline with all future requests as part of it's Orphan Drug Modernization Plan.
Over the last five years, the number of orphan drug designation requests "increased steadily", according to the FDA. The Office of Orphan Product Developments (OOPD) received 568 requests in 2016, more than double the number received in 2012. Orphan drug designation is granted to medication developed to treat patients with rare diseases and conditions, which are usually defined as those affecting less than 200,000 patients in the US.
To encourage development of treatments for these conditions, pharmaceutical firms gaining orphan drug status for their products receive a number of benefits, including tax credits for clinical trial costs and relief from prescription drug user fee if the indication is for a rare disease or condition. If the drug gains FDA approval, the company is also eligible for seven years of marketing exclusivity.
FDA commissioner Scott Gottlieb, MD, highlighted the important role orphan drugs play in healthcare, stating: "People who suffer with rare diseases are too often faced with no, or limited, treatment options, and what treatment options they have may be quite expensive due in part to significant costs of developing therapies for smaller populations."
A SWAT Team of senior OOPD reviewers will be formed to focus on designation requests and to help the agency achieve its orphan drug goals. The FDA also announced plans to create a new streamlined Designation Review Template to improve the efficiency with which designation requests are reviewed.
Since the Orphan Drug Act was passed in 1984, more than 600 new orphan drugs have gained FDA approval, including HIV/AIDS drug Taxol (paclitaxel) and Apokyn (apomorphine HCL), which is used to treat Parkinson's disease. Recently, ProQR Therapeutics announced that the FDA had granted orphan drug designation to its Usher syndrome and retinitis pigments medication QRX-411.
