START Pilot Program Updates

In our 4Q2023 newsletter, we reported on FDA’s Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program.  This program is designed to help facilitate more efficient development of potentially life-saving therapies for rare diseases and to help Sponsors generate high-quality data to support future NDA/BLA applications.  FDA recently published the list of Sponsors who were selected to participate, and the vast majority of Sponsors selected were small organizations!  It is easy to see how the increased engagement with FDA and the potential of more rapid achievement of regulatory milestones would be desirable for small organizations who are trying to do more with less.  However, in a survey reported in RAPS regulatory news, summary results from 13 companies that participated in various FDA pilot programs suggest that while pilot programs are on the whole highly beneficial, there is a significant amount of additional work needed to participate.  These results mirror our experience at Facet.  We have been involved in several successful pilots and we understand the pros and cons of participating in pilot programs.  We encourage our small organizations to weigh the benefits and drawbacks (e.g., time, money, process gaps) before throwing your hat into the pilot ring. If you think the benefits of participation outweigh the possible challenge and are interested in applying for the START pilot program, contact Facet today!