Pharmaceutical Medicine

, Volume 31, Issue 5, pp 329–337 | Cite as

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

  • Vincent AmelotEmail author
  • Catherine Bungener
  • Sophie Guilmin-Crepon
  • Julien Schroedt
  • Corinne Alberti
  • Isabelle Husson
Original Research Article



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.


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.


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.


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.


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.



The authors thank the AFAF (French Friedreich’s Ataxia Association) for their support. The AFAF contributed financially to this research.

Compliance with Ethical Standards

This research is considered an ancillary study of ACTFRIE and was approved by the ethics committee of the Robert Debré hospital [CEERB-RD advice n°2013/96(2)]. All participants (patients and parents) provided informed consent (signed by the patient themselves or by the parents).


This research was funded by the AFAF.

Conflicts of interest

VA, CB, SG-C, JS, CA, and IH have no conflicts of interest.

Supplementary material

40290_2017_201_MOESM1_ESM.pdf (939 kb)
Supplementary material 1 (PDF 938 kb)


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Copyright information

© Springer International Publishing AG 2017

Authors and Affiliations

  1. 1.Laboratory of Psychopathology and Health Processes-EA 4057University Paris Descartes, Sorbonne Paris CitéParisFrance
  2. 2.Pediatric Neurology DepartmentRobert Debré Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP)ParisFrance
  3. 3.INSERM UMR-S1123 and CIC-EC1426, ECEVE, Sorbonne Paris CitéParisFrance
  4. 4.Unit of Clinical EpidemiologyRobert-Debré Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP), Univ. Paris DiderotParisFrance

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