College of Pharmacy, Ewha Womans University, Seoul 03760, Korea.
Division of Biology and Public Health, Mokwon University, Deajeon 35349, Korea.
Int J Environ Res Public Health. 2022 Apr 1;19(7):4204. doi: 10.3390/ijerph19074204.
The price of cancer drugs has skyrocketed, yet it is not clear whether their value is commensurate with their price. More cancer drugs are approved under expedited review, which considers less rigorous clinical evidence, yet only 20% of them show an overall survival gain in the confirmatory trial. Moreover, clinical data are often generated based on small, single-arm studies with surrogate outcomes, challenging economic evaluation. With their high price and uncertain (marginal) clinical value, cancer drugs are frequently rejected by health technology assessment (HTA) bodies. Therefore, agencies, including the UK's National Institute for Health and Care Excellence (NICE), have adopted cancer drug funds (CDF) or risk-sharing schemes to provide extra access for expensive cancer drugs which fail to meet NICE's cost effectiveness threshold. With rising pricing and fewer new cancer medications with novel mechanisms of action, it is unclear if newly marketed cancer therapies address unmet clinical needs or whether we are paying too much. Transparency, equity, innovativeness, and sustainability are all harmed by a "special" approach for cancer medications. If early access is allowed, confirmatory trials within a certain time frame and economic evaluation should be conducted, and label changes or disinvestment should be carried out based on those evaluations.
癌症药物的价格飞涨，但尚不清楚其价值是否与其价格相称。更多的癌症药物通过加速审查获得批准，该审查考虑的是不那么严格的临床证据，但其中只有 20%在确证性试验中显示出总体生存获益。此外，临床数据通常基于小型、单臂研究和替代终点生成，这对经济评估构成挑战。由于价格高昂且临床价值不确定（边际），癌症药物经常被健康技术评估（HTA）机构拒绝。因此，包括英国国家卫生与保健优化研究所（NICE）在内的机构已经采用了癌症药物基金（CDF）或风险分担计划，为未能达到 NICE 成本效益阈值的昂贵癌症药物提供额外的准入途径。随着价格的上涨和具有新型作用机制的新型癌症药物的减少，新上市的癌症疗法是否能满足未满足的临床需求，或者我们是否支付过多，尚不清楚。透明度、公平性、创新性和可持续性都因癌症药物的“特殊”方法而受到损害。如果允许早期准入，应在一定时间内进行确证性试验和经济评估，并根据这些评估进行标签更改或撤资。
