Font size

This article specifically aims at neuromuscular disorders (myopathies) but is of value to all patient communities.

In the field of neuromuscular disorders, engaging patients has since long been recognised as key issue. For patients and patient organisations, it is the time to discuss how and when they want to be engaged in research and health care and how patients want this co-creation process to further develop.
An effective example of the involvement of patient organisations in the neuromuscular research field is the European Neuromuscular Centre (ENMC) itself. The ENMC was co-founded by a group of neuromuscular patient organisations and clinicians to encourage and facilitate communication and collaboration in the field of neuromuscular research. Since then, a structural collaboration began between the ENMC Research Committee, consisting of researchers and clinicians working in the field, and the Executive Committee, consisting of patient organisation representatives. Moreover, ENMC encourages active participation of patients in each of its workshops.

A special workshop hosted by ENMC in Milan to celebrate its 25th anniversary aimed at discussing the level of patient participation in a set of domains considered relevant for the neuromuscular community with key stakeholders. The following topics were chosen for discussion:
1. psycho-social support of families going through the processes of screening and diagnosis
2. transition from child, to adolescent to adult patient
3. healthcare-related research that has major impact on daily life
4. registries and biobanks
5. clinical trial design
6. regulatory and consenting processes

[The concept of shared decision making was used throughout the workshop’s discussions to characterise the partnership-based identification of the wishes and needs of all stakeholders involved. Although the theory and enactment of shared decision making in healthcare are well-described in the literature, comparatively less attention has been devoted to contextualizing questions related to if, when, and how to include patients in decisions within medical research. At the meeting, the “ladder of participation” tool served as a model to evaluate the actual and the desired level of patients’ involvement in all topics addressed (Ambrosini et al, 2019; Lochmüller et al, 2019). Accordingly, patient’s role starts from providing “information” and advice (“consultation”), which are important steps towards the collection of patients’ knowledge and engagement if accompanied by an active listening of their voice by the other stakeholders. Higher levels of engagement are represented by the levels of “collaboration” (partnership in a project) and “control” (initiative of a project).

For the complete article go to ENMC White Paper: The position of neuromuscular patients in shared decision making. 2020

© 2020 Int. Mito Patients