Magnitude of clinical benefit of cancer drugs approved by the US Food and Drug Administration based on single-arm trials
A partir des données de 138 essais cliniques ayant permis entre 2006 et 2016 la mise sur le marché américain d'anticancéreux, cette étude analyse, selon les critères de l'"European Society for Medical Oncology Magnitude of Clinical Benefit Scale", les bénéfices cliniquement significatifs des médicaments autorisés par la "US Food and Drug Administration" sur la base d'essais à simple bras
For regulatory approval, the US Food and Drug Administration (FDA) requires evidence of safety and efficacy of new drugs from adequate and well-controlled trials.1 Improved understanding of the molecular basis of cancer, has led to rapid development of new drugs. In combination with regulatory changes providing rapid review and approval of drugs for diseases with unmet need,2 this acceleration has led the FDA to use overall response rates (ORRs) assessed in single-arm trials as the basis for approval.
JAMA Oncology , résumé, 2017