Research priorities of patients with neuromuscular disease

Abstract

Purpose: This paper describes a study in which patients with neuromuscular disease (NMD) were engaged to list top-priorities for scientific research in order to complement the researchers’ agenda. Method: A dialogic model for research agenda setting was used. Interviews, focus groups and expert meetings with patients were held to identify research topics. Research topics were prioritized via a questionnaire. Agendas were integrated in a dialogue meeting with professionals and patients. Results: The research agenda of NMD patients is divided in four research domains, with a total of 24 research topics. These domains include (1) health; (2) quality of life; (3)quality of care and support, and (4) basic issues. Among the research domains highest priority was given to research on health, followed by research on quality of life. Conclusions: Both patients and professionals agreed a proper balance needs to be found between fundamental research and research on symptomatic treatment and quality of life. They concluded that more attention is required for research on the effective treatment of symptoms, quality of life and implementation of knowledge about NMD in regular care.

Implications for Rehabilitation

Neuromuscular disease

• Patients with neuromuscular disease (NMD) can effectively participate in a research agenda setting process, contributing with their own perspectives on research topics.

• Concerning rehabilitation research, patients give priority to research on movement and training (quality of care domain), and on partner support (quality of life domain).

• The patient-driven agenda reinforces the wide scope on rehabilitation encapsulated in the ICF model and both stipulates the need for more interdisciplinary research, as well as the implementation of research in rehabilitation practice.

Keywords::

• Neuromuscular disease
• patient involvement
• research agenda

Acknowledgements

We want to acknowledge the willingness of those who participated in this study, among them the members of the Dutch Association for NMD Patients (VSN) and the NMD researchers of the Dutch Neuromuscular Research Centre (ISNO).

Declaration of Interest: The authors received a grant from the Netherlands Organisation for Health Research and Development (ZonMw) to conduct this study.