癌症治疗的进步改善了患者的治疗效果，但提高治愈率是一个未满足的主要需求。虽然尽早测试新的抗癌药物可能很有吸引力，因为受益的机会可能最大，但研究人员也必须确保患者不会因治疗过度或治疗不足或拒绝及时的标准治疗而受到伤害。创新癌症疗法开发方法工作组 (MDICT) 在 ESMO 靶向抗癌疗法 (ESMO-TAT) 会议之前立即召开会议，该会议通常每年在法国巴黎举行，以解决被认为对早期学术临床试验重要的问题。 MDICT 2024 的重点是在新辅助 (NEO) 环境 (NEO-ECT) 中进行的新药早期信号寻求阶段临床试验，而不是关键验证性 NEO 试验 (NEO-CONF)，后者通常处于临床试验的 III 期。设计。建议包括四个关键概念：患者参与、审查风险效益比和临床/伦理平衡、要求随机化以减少偏倚并得出可靠的结论，以及选择适当的终点。 NEO-ECT 的精心设计将允许在早期疾病环境中测试新的抗癌疗法，希望这些活动能够带来更高的治愈率，同时还确保患者不会因治愈/最终治疗的延迟或长期治疗而受到伤害或迟发性毒性和发病率。需要进行额外的研究和调查，以进一步定义和完善在此环境中使用的可靠终点，包括基于成像、组织和血液的终点。版权所有 © 2024 作者。由爱思唯尔有限公司出版。保留所有权利。

Advances in the treatment of cancer have resulted in improved outcomes for patients, but improving the cure rate is a major unmet need. While testing new anticancer drugs in the earliest settings may be attractive as the chance of benefit may be greatest, it is also a setting where researchers must ensure patients are not harmed, by either over or undertreatment, or denial of timely standard curative treatments. The Methodology for the Development of Innovative Cancer Therapies Taskforce (MDICT) meets immediately before the ESMO-Targeted Anticancer Therapies (ESMO-TAT) meeting, usually held annually in Paris, France, to address questions that are considered important for early academic clinical trials. The focus of the MDICT 2024 was on early, signal-seeking phase clinical trials of new drugs conducted in the neoadjuvant (NEO) setting (NEO-ECTs) rather than pivotal confirmatory NEO trials (NEO-CONFs), which are typically phase III in design. Recommendations encompass four key concepts: patient engagement, reviewing risk-benefit ratio and clinical/ethical equipoise, the requirement for a randomization to reduce bias and allow robust conclusions to be drawn, and the selection of appropriate endpoints. The careful design of NEO-ECTs will allow the testing of new anticancer treatments in earlier disease settings where activity is hoped to result in higher cure rates, while also ensuring that patients are not harmed by delays to curative/definitive treatments nor by long-term or late-onset toxicity and morbidity. Additional research and investigation are required to further define and refine robust endpoints for use in this setting, including imaging, tissue and blood based endpoints.Copyright © 2024 The Author. Published by Elsevier Ltd.. All rights reserved.