本文由小咖机器人翻译整理

期刊来源：BMJ

原文链接：https://doi.org/10.1136/bmj-2023-077391

摘要内容如下：

目标

评估肿瘤药物的附加效益和收入，探索其相关性，并调查欧洲药品管理局（EMA）不同审批途径的附加效益和收入之间的潜在差异。

设计

回顾性队列研究。

设置

1995年至2020年期间EMA批准的肿瘤药物及其适应症。

主要结果指标

使用七个组织（美国、法国、德国和意大利的卫生技术评估机构、两个医学肿瘤学会和一个药物公报）发布的评级来评估附加效益。所有检索到的评级都使用四点等级量表进行重新分类，以表示负面或不可量化的、次要的、实质性的或主要的附加效益。收入数据是从公开的财务报告中提取的，并与公布的研发（R&D）成本估计数进行比较。最后，评估了附加效益与收入之间的关系。所有分析均在整个研究队列中进行，并在基于EMA批准途径的亚组中进行：标准上市授权、有条件上市授权和特殊情况下的授权。

结果

在规定的时间内，至少有一个组织评估了131种肿瘤药物（166个适应症）的附加效益，共产生458个附加效益评级。189例（41%）为阴性或无法量化。抵消研发成本中位数（6.84亿美元，5.35亿英镑，6.02亿欧元，调整至2020年数值）的中值时间为三年；55种药物中有50种（91%）在8年内收回了这些成本。附加效益评级较高的药物通常收入较高。与标准上市授权相比，有条件上市授权和特殊情况下授权的负面或不可量化的附加效益评级更为常见（相对风险1.53，95%置信区间1.23至1.89）。与标准上市许可相比，有条件上市许可产生的收入较低，抵消研发成本所需的时间较长（标准上市许可为四年，标准上市许可为三年）。

结论

虽然收入似乎与附加效益一致，但大多数肿瘤药物在几年内就能收回研发成本，尽管提供的附加效益很少。通过有条件上市许可批准的药物尤其如此，这些药物本身似乎缺乏全面的证据。政策制定者应评估当前的监管和报销激励措施是否能有效促进为需求最大的患者开发最有效的药物。

英文原文如下：

Abstracts

OBJECTIVES  To evaluate the added benefit and revenues of oncology drugs, explore their association, and investigate potential discrepancies between added benefit and revenues across different approval pathways of the European Medicines Agency (EMA).

DESIGN  Retrospective cohort study.

SETTING  Oncology drugs and their indications approved by the EMA between 1995 and 2020.

MAIN OUTCOME MEASURES  Added benefit was evaluated using ratings published by seven organisations: health technology assessment agencies from the United States, France, Germany, and Italy, two medical oncology societies, and a drug bulletin. All retrieved ratings were recategorised using a four point ranking scale to indicate negative or non-quantifiable, minor, substantial, or major added benefit. Revenue data were extracted from publicly available financial reports and compared with published estimates of research and development (R&D) costs. Finally, the association between added benefit and revenue was evaluated. All analyses were performed within the overall study cohort, and within subgroups based on the EMA approval pathway: standard marketing authorisation, conditional marketing authorisation, and authorisation under exceptional circumstances.

RESULTS  131 oncology drugs with 166 indications were evaluated for their added benefit by at least one organisation within the required timeframe, yielding a total of 458 added benefit ratings; 189 (41%) were negative or non-quantifiable. The median time to offset the median R&D costs ($684m, £535m, €602m, adjusted to 2020 values) was three years; 50 of 55 (91%) drugs recovered these costs within eight years. Drugs with higher added benefit ratings generally had greater revenues. Negative or non-quantifiable added benefit ratings were more frequent for conditional marketing authorisations and authorisations under exceptional circumstances than for standard marketing authorisations (relative risk 1.53, 95% confidence interval 1.23 to 1.89). Conditional marketing authorisations generated lower revenues and took longer to offset R&D costs than standard marketing authorisations (four years compared with three years).

CONCLUSIONS  While revenues seem to align with added benefit, most oncology drugs recover R&D costs within a few years despite providing little added benefit. This is particularly true for drugs approved through conditional marketing authorisations, which inherently appear to lack comprehensive evidence. Policy makers should evaluate whether current regulatory and reimbursement incentives effectively promote development of the most effective drugs for patients with the greatest needs.

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