ENMC Papers on Patient Participation in NMD research and health care decisions

White Paper

On Rare Disease Day 2020, the outcomes and consensus from the ENMC workshop on “The position of the neuromuscular patient in shared decision making” were published in a WhitePaper. The workshop participants represented a wide range of stakeholders from 15 different countries: patients and parents, representatives from neuromuscular diseases organisations, clinicians, health care professionals, researchers, societal and policy researchers, psychologists, ethicists, representatives from regulatory authorities and pharmaceutical companies. The White Paper is based on the outcome of the workgroup discussions (published by Lochmüller et al, 2019 and Ambrosini et al, 2019) and, in particular, it reports the consensus reached on specific recommendations for patient organizations and health care professionals, together with key examples analysed during the meeting.

The workshop took place in Milan, Italy in 2018 and was attended by 45 participants from 15 different countries.

The role of the NMD patient in Shared-Decision-Making by Ambrosini et al, Orphanet Journal of Rare Diseases (2019) 14:126

“It is a call to action to all stakeholders to become ambassadors and promote the direct involvement of people living with a neuromuscular disease in all aspects of health care and medicine development related to their disease”

Fig. 1 Levels of patient involvement along the participation ladder
(A. Ambrosini; modified with permission from De Wit et al, 2015)

The role of patients in neuromuscular research by Lochmüller et al, Journal of Neuromuscular Diseases 6 (2019): 161-172

“Expand the role of patients, the true experts, in neuromuscular disease research, is the conclusion of this international ENMC workshop!”