title: Adding more muscle and nerve to clinical trials.
abstract: In this review we illustrate both the fundamentals and challenges of randomized clinical trials in neuromuscular disorders and suggest directions for prospective efforts to improve the design, conduct, rigor, and objectivity of these trials. Current research in clinical trials for neuromuscular disorders and key issues affecting these trials are reviewed. This perspective addresses the planning of clinical research, level of preclinical data needed to justify trials, patient recruitment and retention, and opportunities to access federal funding and infrastructure in support of clinical trials. The need for innovation in trial design and conduct, rigorous standards for the preclinical efficacy and safety data that support trial rationale, novel collaborative paradigms, objective interpretations of outcomes, and sharing of the lessons learned from trials in any one disorder among all neuromuscular trialists are imperative to improving the heretofore limited success in delivering novel, safe, and effective therapies to patients burdened by neuromuscular disorders.
authors: Office of Clinical Research, National Institute of Neurological Disorders and Stroke, National Institutes of Health, 6001 Executive Boulevard, Suite 2-216, Rockville, Maryland 20892, USA.
authors: Robin A
authors: RA
authors: Conwit
authors: Minal J
authors: MJ
authors: Bhanushali
authors: John D
authors: JD
authors: Porter
authors: Petra
authors: P
authors: Kaufmann
authors: Laurie
authors: L
authors: Gutmann
mesh_terms: D002985:Clinical Protocols / Q000592:standards; D006801:Humans; D009468:Neuromuscular Diseases / Q000453:epidemiology / Q000628:therapy*; D018579:Patient Selection; D011446:Prospective Studies; D016032:Randomized Controlled Trials as Topic / Q000379:methods* / Q000592:standards; D012107:Research Design / Q000592:standards