Patient Survey Findings Show Positive Attitudes Toward SMA Trial Experiences


The results, say the study researchers, offer new insights into patient experiences during spinal muscular atrophy (SMA) clinical trials

New survey results reveal that patients who participated in spinal muscular atrophy (SMA) clinical trials generally have favorable attitudes toward their trial.

Researchers disseminated a survey among patients who had participated in SMA trials throughout the United States—for 70 unique clinical trial experiences—and found that patients had positive opinions regarding overall management of their clinical trial.

Their findings published recently in Neurology and Therapy.

“While certain aspects of patient experiences in clinical trials such as informed consent have been studied at length, few studies have looked as holistically at clinical trial experiences, and no known research has focused specifically on clinical trial experiences in SMA,” explained the researchers. “This study provides new perspective on attitudes surrounding trial participation as well as preferences surrounding logistics and communication.”

Notably, respondents felt their needs were considered in scheduling and that communication with the study team was done in a way that aligned with their preferences. Over half of participants (55.7%) said they had very positive experiences communicating with the team, and 35.7% said they had a positive experience.

In exploring ways that clinical trials could improve, participants cited several factors related to receiving more information.

The biggest motivator for participating in a trial identified by the patients was the desire for a clinical benefit and improved quality of life, with 92.8% considering it a significant motivator and the remaining 7.2% identifying it as moderately motivating. More than half of patients also identified access to an investigational drug (71.4%), an opportunity to help other patients with SMA (62.3%), and positive interactions with the study team (50.7%) as significant motivators. Other motivators included access to medical experts, opportunity to contribute to science, and support from their doctor.

Concerns identified by the study participants included safety of the treatment, whether benefits of treatment would outweigh the risks, and trepidations of the physical/mental pain of accompanying tests, all 3 of which were identified by approximately 25% of patients as being a significant concern.

The top 3 stressors during the trials were fear of physical/mental pain of accompanying tests, concerns about adverse events, and challenges with managing SMA-related complications. These factors were considered a significant stressor by 26.1%, 17.4%, and 18.8% of participants, respectively.

“Insights from this research may facilitate more personalized approaches to interaction with trial participants. The findings illustrate the many motivators and concerns beyond clinical benefit and risk that inform decisions about trial participation and their relative importance to the SMA community,” wrote the group. “Together, they show how—perhaps not surprisingly— motivators are weighted more heavily than concerns. This research also provides new, nuanced perspective on predictors of specific motivators, concerns, stressors, and benefits, such as gender, age, race, and distance to the trial site.”

Performing a regression analysis, the researchers found that gender, age, race, respondent type, knowledge about SMA, distance to trial site, and treatment era all were significant predictors of survey responses. Compared with White participants, participants of color were significantly more likely to express concerns about loss of control in their clinical trial.

When asked about the top benefits of participating in their clinical trial, study respondents cited hope for a better future, helping others with SMA, and relationships with the study team.


Peterson IS, Mazzella AJ, Belter LT, Curry MA, Cruz RE, Jarecki J. The cure SMA clinical trial experience survey: A study of trial participant perspectives on clinical trial management and patient-centric management practices. Neurol Ther. Published online May 30, 2022. doi:10.1007/s40120-022-00360-w

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