Assessing Clinical Outcomes in a Data-Rich World—A Reality Check on Real-World Data
Menée à partir de données portant sur 1 161 patients atteints d'un cancer du poumon non à petites cellules et sur 1 150 patients atteints d'un cancer colorectal, cette étude évalue la corrélation entre des critères cliniques de jugement (survie sans progression basée sur l'imagerie, délai avant l'arrêt du traitement, délai avant le prochain traitement, ...) et la survie globale
Advancements in health information technology and data aggregation have led to the emergence of real-world data and real-world evidence (RWE). The 21st Century Cures Act broadly defined RWE as “data regarding the usage, or the potential benefits or risks, of a drug derived from sources other than randomized clinical trials” and required the US Food and Drug Administration (FDA) to create a program for RWE for expanding indications and postapproval study. In oncology, retrospective RWE generated from electronic health records has had demonstrable impacts in regulatory approval, leading to the FDA-accelerated approval of blinatumomab and expanded indication for palbociclib based on surrogate end points.
JAMA Network Open , éditorial en libre accès, 2020