Developing, Implementing, and Evaluating a Care Management Model for Patients With Neuromuscular Diseases: Protocol for a Participatory Mixed Methods Study

TL;DR

This study aims to develop and evaluate a family-centered care management model for neuromuscular disease patients in Switzerland to improve care and reduce caregiver burden.

Contribution

A novel participatory mixed methods study protocol to develop and evaluate a care management model for neuromuscular diseases in Switzerland.

Findings

Identified gaps in current care practices for neuromuscular disease patients and their families.

Developed a structured care management model and training program based on stakeholder insights.

Evaluated the model's impact on care coordination, quality of life, and caregiver burden.

Abstract

Neuromuscular diseases (NMDs) are often progressive conditions that can reduce quality of life and life expectancy and require long-term caregiving. As patients age, caregivers face increasing demands, often resulting in significant caregiver burden. Treatment and care are complex, and a coordinated, family-centered approach is indicated. The current care situation for individuals with NMDs and their families in Switzerland is poorly understood, and there is no standardized NMD care management available.

The proposed study aims to assess the current care practices for individuals with NMDs in Switzerland, identifying unmet needs and challenges faced by patients, families, and health care providers. On the basis of these findings, a care management model will be developed, implemented, and evaluated to enhance support structures, strengthen specialized neuromuscular centers, and improve overall health care outcomes for affected individuals and their families.

We planned a 3-phase participatory mixed methods study (the Care-NMD-CH study). First, qualitative descriptive data and quantitative survey data were used to assess the current state of care practices. Then, a care management model and training program were codeveloped based on these insights. Finally, the care management service was implemented in pilot specialized neuromuscular centers and evaluated using quantitative, qualitative, and integrated methods. In addition to interview data and patient records, we measured patient-reported outcomes (quality of life and self-efficacy), family-reported outcomes (family functioning and caregiver burden), and stakeholder-reported outcomes (quality of care and interprofessional collaboration). The study population comprises individuals with NMDs and their families living in Switzerland.

Ethics approval was obtained in 2020. Baseline data (T0) were collected and analyzed in 2021 (phase A). The NMD care management model (phase B) was developed based on these data and finalized in 2022. Following the start of implementation in 8 centers in 2023, data collection for process and outcome evaluation (T1-T4) commenced and was completed in May 2025. Data analysis is currently ongoing, and results will be published separately. This paper reports only the study protocol.

This study represents an important step toward implementing and evaluating an evidence-based, family-centered care management service for individuals with NMDs and their families in specialized neuromuscular centers. Building on a 3-phase approach, the project identified gaps in current care; developed a structured care management model, including training; and evaluated its impact on patients, family members, and interprofessional care teams. Expected outcomes include possible improvements in the areas of care coordination, quality of life, and self-efficacy and family functioning. In addition, potential alleviation of disease and caregiver burden, as well as reduction in health care costs for those affected and the health system, will be investigated.

DERR1-10.2196/82833

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