Use and Outcomes of Cost-Effectiveness Analyses and Additional Considerations in Pharmaceutical Reimbursement in the Netherlands.

BACKGROUND AND

objectiveIn the Netherlands, the National Health Care Institute (ZIN) assesses pharmaceuticals and advises the Minister of Health, Welfare, and Sport (MoH) on their reimbursement. Since 2015, two policy reforms-the introduction of willingness-to-pay reference values and the lock procedure, which temporarily withholds high-budget hospital pharmaceuticals from reimbursement pending assessment and price negotiations-have changed the decision-making process and expanded the role of cost-effectiveness analysis (CEA). This study provides a comprehensive overview of the use and outcomes of CEAs in Dutch pharmaceutical reimbursement over the past decade. Additionally, we identify how additional considerations beyond cost-effectiveness were used in reimbursement recommendations.

methodsWe analysed reimbursement dossiers and recommendations published by ZIN between 2015 and 2024. Descriptive methods were used to provide insight into characteristics of assessments, CEA outcomes, and recommendations. For a subset of pharmaceutical indications with a CEA and a recommendation to negotiate, we performed a content analysis of ZIN's appraisal and recommendation letters to identify how additional considerations influenced the reimbursement recommendation alongside cost-effectiveness.

resultsZIN advised the MoH on 330 unique reimbursement dossiers covering a total of 350 pharmaceutical indications in the timeframe. CEAs were conducted for 86 indications, primarily for hospital pharmaceuticals (n = 49), for which assessment became mandatory following the introduction of the lock procedure. Of the 86 indications with CEAs, 27 (31%) were deemed cost-effective and 42 (49%) were not, and cost-effectiveness was classified as unknown for 17 (20%), mostly because these CEAs were judged methodologically insufficient by ZIN. Many cost-ineffective pharmaceutical indications exceeded their reference value substantially. Mean incremental cost-effectiveness ratios (ICERs) reported by ZIN for pharmaceutical indications associated with reference values of €20,000, €50,000, and €80,000 per quality-adjusted life-year (QALY) were €97,300, €81,400, and €278,900 per QALY, respectively. CEA results informed proposed price reductions, by relating the ICER to the relevant reference value. ZIN also frequently based its proposed discount on a more plausible scenario analysis or invoked additional arguments, such as uncertainty in costs or effects, anticipated indication expansions, or ample revenue from earlier indications, to determine a societally acceptable price.

conclusionsIn Dutch pharmaceutical reimbursement decision-making, CEA outcomes directly inform price negotiations, and explicit reference values function primarily as anchors for negotiations rather than as strict cut-off points for reimbursement. Additional considerations, such as uncertainty about long-term effects and anticipated changes in the treatment landscape, were frequently invoked alongside cost-effectiveness to argue for further price reductions.