Preferences for Receiving Results from a Rare Disease Clinical Trial: A Survey of Subjects with Friedreich’s Ataxia and their Parents

Background Ethics committees today recommend making the overall results of clinical trials available to the participants of therapeutic trials, and many guidelines have been developed for both general and specific situations. People with rare diseases are known to have expectations of the medical follow-up that may differ from people with non-rare diseases. Objectives This study was designed to better understand how participants in a rare disease clinical trial and their parents expect to be informed of the results. Methods Patients with Friedreich’s ataxia, a rare neurodegenerative disease that affects gait and balance, who were involved in a previous randomized controlled trial (ACTFRIE) that took place between December 2008 and April 2013, and their parents, were contacted by email. They were asked to answer a survey questionnaire about their expectations concerning the announcement of results for the whole trial and for individual patients, their opinion about scientific publications, and their willingness to participate in another clinical trial in the future. Results In total, 28 patients and 35 parents of patients (100%) said they would like to be informed of the whole trial research results. Patients preferred to be informed via a personal email (65.4% “very favorable” and 15.4% “favorable”), and parents preferred via email and face-to-face interview (55.9% “very favorable” and 11.5% “favorable”). Most, but not all, respondents wanted to know their allocation group. A large proportion were interested in scientific articles. Conclusion Similar to findings with other diseases, respondents in this study preferred to be advised of results for the whole study and of their allocation group. Respondents considered scientific literature to be a source of information, which could be further investigated and addressed in future studies.