FDA Acceptance of Surrogate End Points for Cancer Drug Approval: 1992-2019
Menée notamment à partir de documents et de données de la "US Food and Drug Administration" (FDA) et à partir de la littérature scientifique, cette étude détermine le nombre d'anticancéreux autorisés entre janvier 1992 et juillet 2019 par la FDA sur la base de critères de jugement de substitution, puis analyse le niveau de corrélation entre ces critères de jugement et la survie globale
The US Food and Drug Administration (FDA) approves cancer drugs based on direct measures of patient benefit—such as overall survival (OS) or quality of life—or surrogate measures, such as change in biomarker level or tumor size on imaging studies. Surrogate end points often have weak or unknown correlations with OS, and postmarketing studies are limited. A surrogate end point can be used repeatedly in a particular cancer setting, such as response rate (tumor shrinkage) in mantle cell lymphoma in 2013 (with ibrutinib and lenalidomide) and 2017 (with acalabrutinib) after it was accepted for the first time in 2006 (with bortezomib).
JAMA Internal Medicine , résumé, 2019