In the past decade, there have been a record number of oncology therapy approvals by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Besides the EMA's conditional marketing authorisation programme and the FDA's Accelerated Approval Program, we observe a tendency towards fast approval for exploratory studies with non-randomised, uncontrolled designs and surrogate endpoints. This issue raises concerns about the robustness and effectiveness of accepted treatments, leaving patients and health-care professionals in a state of uncertainty. A substantial number of accelerated approvals have recently been withdrawn in the USA, with some still authorised in Europe, emphasising discrepancies in regulatory standards that affect both patients and society as a whole. We highlight examples of drugs, authorised on the basis of surrogate endpoints, that were later withdrawn due to an absence of overall survival benefit. Our findings address the challenges and consequences of accelerated approval pathways in oncology. In conclusion, this Policy Review calls for regulatory bodies to better align their procedures and insist on robust evidence, preferably through unbiased randomised controlled trials. Drug approval processes should prioritise patient benefit, overall survival, and quality of life to minimise risks and uncertainties for patients.

中文翻译：

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从肿瘤药物适应症快速批准上市后撤回中汲取的经验教训

在过去的十年中，美国食品和药物管理局 (FDA) 和欧洲药品管理局 (EMA) 批准的肿瘤治疗药物数量创历史新高。除了 EMA 的有条件营销授权计划和 FDA 的加速审批计划外，我们还观察到快速批准非随机、非受控设计和替代终点的探索性研究的趋势。这个问题引起了人们对已接受治疗的稳健性和有效性的担忧，使患者和医疗保健专业人员处于不确定状态。美国最近撤回了大量加速审批，其中一些在欧洲仍获得授权，这凸显了影响患者和整个社会的监管标准的差异。我们重点介绍了一些药物的例子，这些药物是根据替代终点授权的，但后来由于缺乏总体生存获益而被撤回。我们的研究结果解决了肿瘤学加速审批途径的挑战和后果。总之，本政策审查呼吁监管机构更好地调整其程序并坚持提供强有力的证据，最好是通过无偏见的随机对照试验。药物审批流程应优先考虑患者利益、总体生存率和生活质量，以尽量减少患者的风险和不确定性。