Characteristics of professional society oncological drug evaluation in the Netherlands from 2016 to 2020: A retrospective analysis

Abstract

Background

In the Netherlands oncological drug approvals by the European Medicines Agency (EMA) are evaluated for added clinical benefit by the cieBOM (commission ‘Beoordeling Oncologische Middelen’). A positive evaluation (further depicted as ‘approval’) by the cieBOM is of value in drug reimbursement decision making. In this study we explore characteristics of drug evaluations by the cieBOM.

Methods

We identified new drugs and drug indications for malignant solid tumours approved by the American Food and Drug Administration (FDA) and/or EMA from January 2016 to December 2020 and compared these to assessments by the cieBOM.

Results

A total of 136 new drug indications were identified of which 133 were evaluated by the FDA, 111 were evaluated by EMA and 87 were evaluated by cieBOM. The cieBOM initially approved 76 of 104 (73 %) EMA-approved indications, and 76 of 124 (61 %) of all FDA-approved indications. cieBOM approvals were more often based on phase III trials. Neither the percentage of approvals with an OS benefit, nor the magnitude of benefit, nor the hazard ratio for death differed significantly between agencies. PFS and QoL gains for approvals were also similar between agencies.

Discussion

The cieBOM evaluated less new drug indications and subsequently approved less often as compared to the EMA and the FDA, with approvals more frequently based on phase III trials. The gain in clinical or surrogate endpoints did not differ between cieBOM and FDA or EMA approvals. Globally, stricter criteria for both selection of studies to be assessed by advisory commissions such as cieBOM and drug approval agencies are needed in order to limit the advent of new drugs and drug indications to only those of high value.