February 28th marks Rare Disease Day. In keeping with the theme, EPM asked Dr. Asma Patel, vice president of integrated services for early development at Quotient Sciences, what barriers remain in rare disease drug development and the strategies that can bring the much-needed drugs to market faster.
Despite improvements in regulatory support and commercial rewards, difficulties remain for drug developers in bringing rare disease drugs to market. There are four main barriers impacting the development of orphan programs: (1) increasingly constrained R&D budgets, (2) challenging product development, which may need to be specialised on a per patient basis, (3) problematic clinical recruitment in rare patient populations and (4) regulatory hurdles in striving for fast track and accelerated approval.