Scientific Rigor Recommendations for Optimizing the Clinical Applicability of Translational Research.

Abstract

The approval of new therapies to treat neurodegenerative disease conditions by the Food and Drug administration (FDA) has been hindered by many failed clinical trials, which were based upon "significant" efficacy in preclinical or translational studies. Additional problems during drug development related to significant adverse events and unforeseen toxicity have also hampered drug development. Recent reviews of preclinical data suggests that many studies have over-estimated efficacy due to poor or inadequate study design, exclusion of important data (negative or neutral) and lack of study randomization and blinding. This article describes in detail a set of recommendations to improve the quality of science being conducted in laboratories worldwide, with the goal of documenting in the peer-reviewed literature, including Journal of Neurology and Neurophysiology, the scientific basis for the continued development of specific strategies to treat neurodegenerative diseases such as Stroke, Alzheimer's disease, Huntington's disease, Parkinson's disease, Spinal cord injury, and Amyotrophic lateral sclerosis. The minimum recommendations for effective translational research include the need for model justification, study group randomization and blinding, power analysis calculations, appropriate statistical analysis of all data sets, and a conflict of interest statement by investigators. It will also be beneficial to demonstrate reproducible efficacy in multiple species and in studies done by independent laboratories.