TY - JOUR T1 - Preclinical efficacy studies in investigator brochures: Do they enable risk–benefit assessment? A1 - Wieschowski, Susanne A1 - Chin, William Wei Lim A1 - Federico, Carole A1 - Sievers, Sören A1 - Kimmelman, Jonathan A1 - Strech, Daniel Y1 - 2018/04/05 N2 - Author summary To make a clinical trial ethical, regulatory agencies and institutional review boards have to judge whether the trial-related benefits (the knowledge gain) outweigh the trial-inherent risks. For early-phase human research, these risk–benefit assessments are often based on evidence from preclinical animal studies reported in so-called “investigator brochures.” However, our analysis shows that the vast majority of such investigator brochures lack sufficient information to systematically appraise the strength of the supporting preclinical findings. Furthermore, the very rare reporting of preclinical efficacy studies that demonstrated no effect raises concerns about potential design and/or reporting biases. The poor preclinical study design and reporting thwarts risk–benefit evaluation during ethical review of early human research. Regulators should develop standards for the design and reporting of preclinical efficacy studies in order to support the conduct of ethical clinical trials. JF - PLOS Biology JA - PLOS Biology VL - 16 IS - 4 UR - https://doi.org/10.1371/journal.pbio.2004879 SP - e2004879 EP - PB - Public Library of Science M3 - doi:10.1371/journal.pbio.2004879 ER -