Reports over the last decade have challenged the quality and strength of preclinical evidence, the scientific rationale that’s behind the launch of clinical trials. The U.S. National Institutes of Health (NIH) now requires applicants to address scientific rationale, rigor, biological variables, and the authenticity of biological and chemical reagents. Applying such considerations in developing the case for clinical testing of an experimental therapeutic can help ensure that trials do not fail for avoidable reasons.

The European Neuromuscular Consortium (ENMC) recently held a workshop entitled "Finalizing a Plan to Guarantee Quality in Translational Research for Neuromuscular Diseases." MDF staff participated in the workshop that emphasized “choke points” in ensuring rigorous research—the funders and publishers. The proceedings of the workshop are to be published in Neuromuscular Disorders.

All stakeholders are responsible for adequacy of the rationale for clinical trials. DM investigators are encouraged to observe established standards for preclinical efficacy studies, such as the National Institute of Neurological Disorders and Stroke (NINDS) rigor guidelines (pdf). The path to an approved therapy for DM is an arduous one and should not be complicated by clinical trials of candidate therapeutics that do not have a solid scientific basis.