RESUMO
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.
Clinical trials can be stressful experiences for patients and their caregivers, especially when participants are affected by serious diseases. By understanding trial participants' attitudes and experiences, researchers may be better able to accommodate their interests when designing and conducting research studies. This study sought insight into attitudes and experiences of spinal muscular atrophy (SMA) clinical trial participants by surveying people who participated in SMA clinical trials in the USA. The data used in analysis reflected 70 unique clinical trial experiences. Survey 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. Whether or not specific motivators, concerns, stressors, and benefits were important was predicted by participant gender, age, and race, as well as respondent type (participant or caregiver), knowledge about SMA, distance to the trial site, and treatment era. Top recommendations for improving study management all related to receiving more information. This research provides new perspective on patient experiences in SMA clinical trials, and may be used to inform future study design and interactions with research participants.
