In 2018, the European Neuromuscular Centre (ENMC) held a special meeting dedicated to the position of neuromuscular patients in Shared-Decisino-making (SDM)1,2 Today, the white paper is published.
In the field of neuromuscular disorders, engaging patients has since long been recognized as key issue. One of the most effective examples of patient involvement in the neuromuscular research field is the ENMC itself! The ENMC was co-founded by a group of patient organisations and clinicians to encourage and facilitate communication and collaboration in the field of neuromuscular research and health care. Since then a structural collaboration began between the ENMC Research Committee, consisting of researchers and clinicians working in the neuromuscular field, and the Executive Committee consisting of 8 European neuromuscular patient associations. ENMC has proactively encouraged participation of patient themselves to workshops in the past 10 years. With this SDM meeting, ENMC aimed to further strengthen patient participation for a set of important domains:
- Genetic Diagnosis, Screening and Predictive Testing
- Transition from Child- to Adulthood
- Health Research
- Registries and Biobanks
- Clinical Trial Design
- Regulatory Issues and Health Technology Assessment
The meeting was attended by 45 participants from 15 different countries, who represented a wide range of stakeholders: 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.
It became clear during the discussions that education about the involvement of patients up to the level of decision-making requires training and coaching of all stakeholders.“It is important to ensure proficient communication about the wishes and challenges we face in implementing patients’ participation at the co-creation level in the various domains addressed. This effort requires changing attitudes, and therefore, ambassadors are needed who inspire and empower others about the benefits of patients’ participation and perhaps at the local level it may need individual coaching.”
More specific goals for each of the six topics and required actions for relevant stakeholders are described in the White Paper attached, which is based on two scientific reports published in 20191,2. The White Paper forms a very important contribution to recognize the necessary collaboration between all neuromuscular stakeholders and to promote even more lively and dynamic discussions between them at the local and international level.
1Lochmüller et al., Journal of Neuromuscular Diseases, 2019, 6:161–172
2Ambrosini et al., Orphanet Journal of Rare Diseases 2019, 14:126