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Table 1 Common elements of successful NMD research and care networks

From: The SMA Clinical Trial Readiness Program: creation and evaluation of a program to enhance SMA trial readiness in the United States

Through benchmarking with leaders of established neuromuscular disease research and care networks–including NeuroNEXT, the Parent Project Muscular Dystrophy (PPMD) Certified Duchenne Care Center Program (CDCCP), and the Cooperative International Neuromuscular Research Group (CINRG)–Cure SMA identified several common elements of successful programs. These elements–and how they informed Cure SMA’s approach to creating the Clinical Trial Readiness Program–are described below.
Recommendations from Established Programs & Actions Taken by Cure SMA
Consider human and financial resources needed to make programs sustainable
 ▪ Proactive and detailed approach to project planning and budgeting at outset, followed by annual strategic planning and budgeting
 ▪ Virtual activities enable greater sustainability
Understand established sites’ needs and research infrastructure, staffing, capabilities, and ability to coordinate care
 ▪ 2017 survey of SMA clinical trial sites provided insight on established sites
 ▪ Similar information addressed in site evaluation survey
Develop baseline criteria for participation
 ▪ Baseline criteria identified based on consensus readiness checklist and sponsor input
Ensure evaluations are sensitive to site workloads
 ▪ Virtual site evaluation focused on elements identified as important by sites and sponsors
Establish site “champions” to spearhead readiness efforts and manage trials
 ▪ Direct engagement of principal investigators (PIs) who can drive internal decision-making
Consider timelines and enrollment targets for future trials
 ▪ 2017 recruitment model projected future needs
 ▪ Ongoing review of planned trials and discussion with sponsors about anticipated needs