Orphan drugs and regulatory affairs
I watched the live broadcast of “Orphan Drug & Rare Disease Development: Understanding the European and US Regulatory Landscape” today, hosted by XTalks and presented by Premier Research.
Talking about the US regulatory scene, Nach Dave emphasised that orphan drugs spend less time in clinical trials, have a better chance of obtaining FDA approval, and spend nearly half the time obtaining that approval. They also have a potential for seven years’ exclusivity when developed. An approved product for a rare paediatric or tropical disease also earns a voucher for priority approval which can be transferred to another product or even another company. The American GAIN act, which promotes the development of antibacterial and antifungal drugs for serious infections, also offers exclusivity (5 years) and priority review with fast-tracking.
Carol Huntington covered the European market, which offers a number of similar incentives such as 10 years’ exclusivity and free or dramatically reduced charges for scientific advice relating to orphan medicine products. She also emphasised that many orphan diseases disproportionately affect children, and that an existing medicine may gain new approvals for orphan diseases (although it needs to go through the whole process).
There are solid measures in place to promote development for these neglected diseases, with significant benefits for pharmaceutical companies in terms of expedited review, official support and exclusivity until a drug becomes profitable, but there is still a huge gap to be closed between the number of diseases and the number of medicines currently available.