The Cure SMA Clinical Trial Experience Survey: A Study of Trial Participant Perspectives on Clinical Trial Management and Patient-Centric Management Practices

Abstract

Introduction Understanding clinical trial experiences can illuminate opportunities to optimize trial design and management, with potential benefits for recruitment and retention. This study sought to better understand clinical trial participant experiences and attitudes within spinal muscular atrophy (SMA), and how the evolving treatment landscape and participant characteristics may predict attitudes. Methods A survey was developed following a review of published literature and discussions with caregivers of SMA trial participants. This was distributed via email to known trial participants in Cure SMA’s database, announcements in Cure SMA’s newsletter, and emails to SMA clinical trial principal investigators. Results Seventy complete surveys reflecting unique clinical trial experiences were included in analysis. Responses revealed positive attitudes about clinical trial management overall. Top motivators for trial participation included clinical benefit, investigational drug access, and the opportunity to help others. Top concerns were safety, whether benefits would justify risks, and concerns about pain accompanying tests. The greatest stressors were fear of pain, adverse event concerns, and challenges managing medical complications of SMA. Top benefits of trial participation were hope for a better future, helping others, and relationships with the study team. In regression analysis, participant gender, age, and race all emerged as significant predictors ( p < 0.05) of motivators, concerns, stressors, and benefits, as did respondent type, knowledge about SMA, distance to the trial site, and treatment era. Top recommendations for improving study management all related to receiving more information. Conclusion This research provides new perspective on patient experiences in SMA clinical trials. It underscores the importance of information and efforts to anticipate and accommodate participant needs. These findings may inform study design and interactions with research participants. They may become especially important in supporting recruitment and retention as more treatment options become available. Supplementary Information The online version contains supplementary material available at 10.1007/s40120-022-00360-w.

Facioscapulohumeral Muscular Dystrophy European Patient Survey: Assessing Patient Reported Disease Burden and Preferences in Clinical Trial Participation

“…Verhaart et al (2019) found that communication of results was considered important, but only received by 26% of participants [45]. The desire for better access to information is not unique to FSHD, and was noted in a recent study regarding patient preferences in spinal muscular atrophy [46]. Whilst educational sources are available, patients are not sufficiently informed of where to access this.…”