Trial-related informational needs and decision-making in the SMA community after broad treatment access: results of the 2nd Cure SMA clinical trial experience survey

Abstract

Abstract Background When clinical trials were the only avenue for people with spinal muscular atrophy (SMA) to access potentially effective disease-modifying therapies (DMTs), people often traveled long distances for trials and were willing to tolerate significant treatment-related risks. Today’s widespread availability of DMTs and newborn screening, however, may affect tolerance for inconvenience and risks. In 2023, Cure SMA surveyed the US SMA community to understand experiences with, and views on, trials. This survey included a combination of closed- and open-ended questions and was distributed to adults with SMA and caregivers of children in Cure SMA’s database in spring 2023. Descriptive analyses and hybrid coding were used to analyze quantitative and qualitative data, respectively. Data were stratified based on trial participation status and further by respondent type (adult or caregiver), for visual comparison of subgroup results. This manuscript relates key findings on community information-seeking, motivators for and concerns about participation, essential information for trial decision-making, and ways to improve recruitment and enrollment. Results 270 responses were included in the analysis: 167 (62%) from adults and 103 (38%) from caregivers. 50 (19%) were trial participants, 98 (36%) reported having considered participation, and 122 (45%) had not considered trial participation. Trial participants most frequently consulted neurologists for trial information. Neurologists were also viewed as the most helpful resource by caregivers, on average, while adults identified principal investigators as most helpful. Ratings of motivators and concerns varied by trial participation status and respondent type. Top motivators included improved quality of life, hope for a better future, and treating or preventing SMA symptoms. Top concerns included drug safety, benefit/risk, and travel. Understanding adverse events and potential benefits of the trial drug were most often identified as essential information for trial decision-making. More easy-to-understand materials was the most highly rated avenue for improving recruitment and enrollment. Conclusions These findings provide a roadmap for enhancing the delivery of information on clinical trials to the SMA community. They indicate desire for easier-to-understand resources, identify topics that matter in trial decision-making, and identify the parties to which the SMA community is likely to turn for information.

The authors' abstract, as published at the source. Orphanet Journal of Rare Diseases, 2026 · DOI ↗