Abstract
BACKGROUND: The high costs of novel anticancer drugs have caused concern among healthcare stakeholders. To address the knowledge gap on the proportion of survival benefit with the related economic expenditure, we aimed to assess the correlation between the costs and value of innovative drugs targeted to specific tumours, before and after price negotiation policy implementation. METHODS: We identified new drugs for lung and breast cancer that entered the National Reimbursement Drug List (NRDL) through price negotiation from 2016 to 2023. Therapeutic value consisted of traditional clinical endpoints, like the percentage improvement of overall survival (ΔOS%) and progression-free survival (ΔPFS%), and the quantified gains of the American Society of Clinical Oncology Value Framework (ASCO-VF) and the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS). We calculated monthly drug costs and used Spearman's correlation coefficient and Cohen's kappa statistics for statistical analysis. RESULTS: Twenty-nine innovative price-negotiated drugs were collected between 2016 and 2023. The median monthly costs were US$3381.31 out of NRDL and US$1095.88 within NRDL, with an ΔOS% of 22.24% (IQR = 6.45-29.48) and a ΔPFS% of 83.82% (IQR = 50.41-104.05). The median ASCO-VF score was 40.98, and 17 drugs scored the meaningful benefit of ESMO-MCBS. We found no association between clinical benefits and their costs before and after NRDL, either overall or for specific cancers. The agreement between the two frameworks was stable. CONCLUSIONS: The negotiation policy decreased medication costs, but did not generate the expected correlation between the value and costs of anticancer drugs. Comprehensive value assessments need to be performed in the future to explore more in-depth findings and promote the affordability and availability of effective anticancer drugs.