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J Neuromuscul Dis. 2019;6(1):161-172. doi: 10.3233/JND-180368.

The Position of Neuromuscular Patients in Shared Decision Making. Report from the 235th ENMC Workshop: Milan, Italy, January 19-20, 2018.

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Children's Hospital of Eastern Ontario Research Institute, University of Ottawa, Ottawa, Canada and Division of Neurology, Department of Medicine, The Ottawa Hospital, Ottawa, Canada; Department of Neuropediatrics and Muscle Disorders, Medical Center - University of Freiburg, Faculty of Medicine, Freiburg, Germany.
Fondazione Telethon, Milan, Italy.
Department of Neurology, Radboud University Medical Center, Nijmegen, the Netherlands.
Uppsala University, Uppsala, Sweden.
Centre for Human and Clinical Genetics, Leiden University Medical Centre, Leiden, the Netherlands.
European Neuromuscular Centre, Baarn, the Netherlands.
Prinses Beatrix Spierfonds, the Hague, the Netherlands.
Spierziekten Nederland, Baarn, the Netherlands.
Centre for Science and Technology Studies (CWTS), University of Leiden, Leiden, the Netherlands and Spierziekten Nederland, Baarn, the Netherlands.
RTI International, Research Triangle Park, NC, USA.
AMO Pharma Ltd., Wonersh, UK.
Myotubular Trust, London, UK.
Department of Child Neurology, Catholic University, Rome, Italy.
Public Engagement, European Medicines Agency, London, UK.
SMA Europe, Freiburg, Germany.
Institute of Environmental Medicine, Karolinska Institutet, Stockholm, Sweden; and Icon plc, Stockholm, Sweden.
Swiss Foundation for Research on Muscle Diseases, Cortaillod, Switzerland.


In the era of patient-centered medicine, shared decision-making (SDM) - in which healthcare professionals and patients exchange information and preferences and jointly reach a decision - has emerged as the gold standard model for the provision of formal healthcare. Indeed, in many geographical settings, patients are frequently invited to participate in choices concerning the design and delivery of their medical management. From a clinical perspective, benefits of this type of patient involvement encompass, for example, enhanced treatment satisfaction, improved medical compliance, better health outcomes, and maintained or promoted quality of life. Yet, although the theory and enactment of SDM in healthcare are well-described in the literature [1-3], comparatively less attention has been devoted to contextualizing questions relating to if, when, and how to include patients in decisions within medical research. In this context, patient involvement would be expected to be potentially relevant for and applicable to a wide range of activities and processes, from the identification of research priorities and development of grant applications, to the design of patient information and consent procedures, formulation of interventions, identification and recruitment of study sample populations, feasibility of a clinical trial, identification, selection, and specification of endpoints and outcomes in clinical trials and observational studies, data collection and analysis, and dissemination of results. To this end, 45 clinicians, healthcare professionals, researchers, patients, caregivers, and representatives from regulatory authorities and pharmaceutical companies from 15 different countries met to discuss the level of involvement of patients with neuromuscular diseases, specifically in the following settings of medical research for neuromuscular diseases: i) registries and biobanks; ii) clinical trials; and iii) regulatory processes. In this report, we present summaries of the talks that were given during the workshop, as well as discussion outcomes from the three topic areas listed above.


Patient participation; biomedical research; consensus; quality of life


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