Immune-Checkpoint Inhibitors For The Treatment Of Non-Small Cell Lung Cancer: A Comparison Of The Regulatory Approvals In Europe And The United States

Abstract

The process of regulatory authorization of oncology drugs, including immune-checkpoint inhibitors, has been improved by the cooperation and coordination among the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Regulatory decisions of immune-checkpoint inhibitors by these agencies are often based on enhanced efficacy and acceptable toxicity profiles, investigated in randomized, open-label, clinical trials. In this study, we addressed the differences in regulatory approvals between the FDA and the EMA for immune-checkpoint inhibitors in the treatment of non-small cell lung cancer, from year 2015 until 2021; by focusing on: (1) time to approval of immune-checkpoint inhibitors, and (2) the considerations of patient-reported outcomes (PROs) by each agency. Both the FDA and the EMA recognize the value of PROs as important patient-centered endpoints when determining the efficacy of therapies, and considering drugs for approval. Despite similarities in the regulatory pathways and assessments used for immune-checkpoint inhibitor approvals, there are differences between the two agencies when time to approval or marketing authorization for these drugs were compared. Efforts to align regulatory practices, with high concordance approval outcomes, can lead to better use of resources associated with drug development. These efforts can also provide a more streamlined and predictable process for evaluation of the efficacy, safety and PRO measures for new immune-checkpoint inhibitors or disease indications. Further harmonization and collaboration between the EMA and the FDA on the PRO instrument development, measurement and validation are encouraged to increase the efficiency of the decision-making processes in the future.