Surrogate End Points and Their Validation in Oncology Clinical Trials
A partir d'une revue systématique de la littérature (52 essais identifiés entre 1974 et 2014, 9 762 patients inclus), cette étude évalue l'intérêt d'utiliser divers critères de jugement intermédiaire dans les essais randomisés de traitements chez des patients atteints d'un sarcome des tissus mous de stade avancé ou métastatique
A new drug must show effectiveness, usually derived from well-designed and sufficiently powered phase III clinical trials, to receive regulatory approval from the US Food and Drug Administration (FDA). The effectiveness of cancer drugs is demonstrated by prolonging survival and/or improving patient symptoms and quality of life. Overall survival (OS), defined as the time from random assignment to the date of death from any cause, is objective, precise, and easy to measure and interpret, and hence has been considered the most reliable and clinically meaningful end point for evaluating drug efficacy in oncology clinical trials...