Implementation of a neuromuscular clinical trial network: a rare disease model for enhancing clinical trial readiness, capacity, and access in Canada

Abstract

Abstract Background Rare diseases affect over 300 million people globally, yet clinical trial conduct in rare disease populations remains complex due to small patient numbers, geographic dispersion, heterogeneous phenotypes, and limited trial infrastructure. Neuromuscular diseases (NMDs) exemplify these challenges. Coordinated trial networks have emerged as a strategy to improve feasibility, access, and trial performance. Methods Guided by the Consolidated Framework for Implementation Research (CFIR), we conducted a multi-phase qualitative needs assessment involving neuromuscular investigators and clinical trial personnel across Canada (n = 34) and clinical trial personnel (n = 16) across Canada. Data were analyzed using conventional qualitative content analysis to identify barriers, facilitators, and capacity-building needs informing the early implementation of a Canadian Neuromuscular Clinical Trial Network (CTN). Results Key barriers included burdensome feasibility processes, limited workforce capacity, lack of centralized trial visibility, and fragmented coordination across stakeholders. Trial personnel highlighted unmet needs in disease-specific training, mentorship, peer networking, and workforce sustainability. Early Neuromuscular CTN strategies, including centralized feasibility triage, a national Community of Practice, one-on-one operational monitoring, mentorship, and alignment with patient and registry infrastructures directly addressed these challenges. Conclusion The Neuromuscular CTN describes a pragmatic and potentially transferable model for strengthening rare disease clinical trial ecosystems. By ensuring trials not only occur but are optimally supported and performed, coordinated networks can enhance trial readiness, equity of access, and long-term sustainability in rare disease research. Clinical trial number Not applicable.