Facing the Challenges of Clinical Trials

Facing the Challenges of Clinical Trials

Two especially interesting and topical articles which review the current practice, challenges and some potential solutions for clinical trials have been published in the academic press in recent months. Writing in Muscle and Nerve, Robin Conwit and colleagues (Conwit et al., 2011) discuss problems facing clinical trials for neuromuscular diseases – such as the rarity and wide geographical distribution of patients.

Adding more muscle and nerve to clinical trials

Robin A. Conwit, MD, Minal J. Bhanushali, MD, John D. Porter, PhD, Petra Kaufmann, MD, MSc and Laurie Gutmann, MD

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.

Muscle Nerve 44: 695–702 (2011), doi: 10.1002/mus.22130

This material is reproduced with permission of John Wiley & Sons, Inc.

© 2009 Wiley Periodicals, Inc.

Meanwhile, Nature features a news article from Heidi Ledford (Ledford, 2011) which also discusses some of the challenges faced. She presents four particular strategies which some researchers, ‘have come up with to give clinical trials a better success rate.’

Translational research: 4 ways to fix the clinical trial

Heidi Ledford

Published online 28 September 2011, Nature 477, 526-528 (2011), doi:10.1038/477526a

12 Apr 2017