Cure SMA examined patient and caregiver experiences in SMA clinical trials to understand motivations, expectations, and experiences surrounding trial participation and provide insights to support patient-centered management for future trials.

Little has been published on factors informing patient experiences in SMA trials, which can be particularly intensive. Understanding experiences will enable identification of opportunities to optimize experiences.

This survey was developed following a review of published literature and discussions with families of SMA trial participants. The survey was open from November 15^th 2019 through January 10^th 2020 and distributed via email to known trial participants in Cure SMA’s database; it was also promoted in Cure SMA’s newsletter and sent to SMA clinical trial principal investigators to share with trial participants. SMA trial participants and caregivers of majority age were eligible to complete the survey.

Eighty-nine eligible individuals completed surveys reflecting 79 unique experiences. The top three motivators for trial participation included clinical benefit (WA=2.92 of 3, where 1=not significant and 3=very significant), investigational drug access (WA=2.56), and the opportunity to help others with SMA (WA=2.55). The top three concerns were about safety (WA=2.0), whether benefits would justify risks (WA=1.96), and concerns about pain accompanying tests (WA=1.92). Finally, the highest ranked stressors were fear of pain accompanying tests (WA=1.94), adverse event concerns (WA=1.90), and challenges managing medical complications of SMA (WA=1.83). Reported stress buffers included trust in the study team (WA=2.73), understanding expressed by the study team (WA=2.7), and witnessing improvements (WA=2.67); in regression analysis, information about SMA and clinical trials also emerged as a buffer.

Patient-focused clinical trial management may improve trial participants’ experiences. Actions that may improve experiences include increasing access to information; building trusting relationships between study staff and participants; and proactively accommodating participant needs. These findings have applicability for other disease areas as well.