The SMA Clinical Trial Readiness Program: Creation and Evaluation of a Program to Enhance SMA Trial Readiness in the United States

Senior managers Ilse Peterson and Ann Marie Stanley, with project coordinator Fatou Sarr, partnered with Cure SMA’s Rosangel Cruz and Jill Jarecki to establish a clinical trial readiness program for new and prospective spinal muscular atrophy (SMA) clinical trial sites. They published an article elaborating on the background of the program and its development, launch, and potential applicability to other regions and diseases in the Orphanet Journal of Rare Diseases.

Program development was informed by a review of the SMA clinical trial landscape, successful neuromuscular disease trial and care networks, and factors important to effective trial conduct in SMA. The program was piloted in 2018 with a virtual site readiness evaluation, a trial readiness toolkit, and a readiness program for physical therapists and clinical evaluators. Nine U.S. research hospitals participated in the pilot. Cure SMA evaluated the pilot program and resources through feedback surveys, which supported the program’s relevance and value. Since 2018, the program has been expanded with additional sites, new best practices toolkits, and workshops. In partnership with Cure SMA, SMA Europe is also extending programming to European countries. The program is significant as an example of a patient advocacy group working successfully with pharmaceutical companies, other patient advocacy organizations, and research hospitals to promote trial readiness, and may serve as a model for other organizations.