Are Observational, Real-World Studies Suitable to Make Cancer Treatment Recommendations?
Menée aux Etats-Unis à l'aide des résultats de 141 essais randomisés incluant 85 118 patients atteints d'un cancer et à partir de données des registres des cancers portant sur 1 344 536 patients diagnostiqués entre 2004 et 2014, cette étude compare, du point de vue de la survie, les résultats des deux sources de données concernant l'efficacité de traitements anticancéreux
Kumar et al added a new chapter to a decades-long debate about whether observational studies—nonrandomized comparative effectiveness research (CER)—can replace randomized clinical trials (RCTs) to assess the efficacy of therapies. Their work is timely. The 21st Century Cures Act has empowered the US Food and Drug Administration to use real-world evidence beyond controlled trials to support drug approvals. Retrospective analyses of observational registries are used to justify a wider range of treatments, including the delivery of radiotherapy and surgery. However, a key question remains: when a physician relies on an observational study to make a therapeutic recommendation, how often is that recommendation correct?