Fig. 2

Factors important to successful clinical trial management in SMA. Results of a 2017 survey of US-based SMA clinical trial sites played a critical role in informing Cure SMA’s approach to creating the Clinical Trial Readiness Program. Weighted averages of responses (n = 19) to the question “How important have the following factors been in enabling you to successfully run clinical trials in SMA?” revealed that, overall, sites viewed staff bandwidth and a high-performing clinical trial coordinator as most important to successful trial management, closely followed by strong staff coordination. The rating scale for this question included the options: not important, somewhat important, important, very important, and extremely important. These findings contributed to Cure SMA’s decisions to create resources on trial coordination and SMA as well as materials specifically for clinical research coordinators. They also informed recommendations made during site interviews in 2018