Grössmann N, Del Paggio J C, Wolf S, Sullivan R, Booth C M, Rosian K, Emprechtinger R, Wild C
Ludwig Boltzmann Institute for Health Technology Assessment (LBI-HTA), Vienna, Austria.
Department of Medicine, Division of Medical Oncology, University of Toronto, Toronto, Canada.
Eur J Cancer. 2017 Sep;82:66-71. doi: 10.1016/j.ejca.2017.05.029. Epub 2017 Jul 10.
Several societies have proposed frameworks to evaluate the benefit of oncology drugs; one prominent tool is the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS). Our objectives were to investigate the extent of European Medicines Agency (EMA)-approved cancer drugs that meet the threshold for 'meaningful clinical benefit' (MCB), defined by the framework, and determine the change in the distribution of grades when an adapted version that addresses the scale's limitations is applied.
We identified cancer drugs approved by the EMA (2011-2016). We previously proposed adaptations to the ESMO-MCBS addressing its main limitations, including the use of the lower limit of the 95% confidence interval in assessing the hazard ratio. To assess the MCB, both the original and adapted ESMO-MCBS were applied to the respective approval studies.
In total, we identified 70 approval studies for 38 solid cancer drugs. 21% of therapies met the MCB threshold by the original ESMO-MCBS criteria. In contrast, only 11% of therapies met the threshold for MCB when the adapted ESMO-MCBS was applied. Thus 89% and 79% of therapies did not meet the MCB threshold in the adapted and original ESMO-MCBS, respectively.
In most of the cancer drugs, the MCB threshold is not met at the time of approval when measured using both ESMO-MCBS scales. Since approval status does not translate into a MCB, stakeholders and decision makers should focus on the benefit/risk ratio of anticancer drugs to assure an appropriate allocation of resources in health care systems.
多个学会已提出评估肿瘤药物获益的框架；其中一个突出工具是欧洲医学肿瘤学会临床获益程度量表（ESMO-MCBS）。我们的目的是调查欧洲药品管理局（EMA）批准的癌症药物达到该框架所定义的“有意义的临床获益”（MCB）阈值的程度，并确定应用解决该量表局限性的改编版本时等级分布的变化。
我们确定了EMA在2011年至2016年期间批准的癌症药物。我们之前针对ESMO-MCBS的主要局限性提出了改编建议，包括在评估风险比时使用95%置信区间的下限。为评估MCB，将原始版和改编版ESMO-MCBS分别应用于各自的批准研究。
我们总共确定了38种实体癌药物的70项批准研究。按照原始ESMO-MCBS标准，21%的疗法达到了MCB阈值。相比之下，应用改编版ESMO-MCBS时，只有11%的疗法达到了MCB阈值。因此，在改编版和原始版ESMO-MCBS中，分别有89%和79%的疗法未达到MCB阈值。
在大多数癌症药物中，使用ESMO-MCBS的两个版本进行衡量时，在批准时均未达到MCB阈值。由于批准状态并不等同于MCB，利益相关者和决策者应关注抗癌药物的获益/风险比，以确保卫生保健系统中资源的合理分配。
