Hematology Department, Saint-Louis Hospital, Paris, France.
Medical Oncology Department, Cancer Institute of the Hospices Civils of Lyon, Lyon, France.
Lancet Oncol. 2021 Oct;22(10):e430-e434. doi: 10.1016/S1470-2045(21)00337-5.
During the past decade, health technology assessment bodies have faced new challenges in establishing the benefits of new drugs for individuals and health-care systems. A topic of increasing importance to the field of oncology is the so-called agnostic regulatory approval of targeted therapies for cancer (independent of tumour location and histology) granted on the basis of basket trials. Basket trials in oncology offer the advantage of simultaneously evaluating treatments for multiple tumours, even rare cancers, in a single clinical trial. To address the novel challenges introduced by these trials, an interdisciplinary panel was convened on behalf of the Transparency Committee of the French National Authority for Health to clarify an approach designed to guarantee a transparent, reproducible, and fair assessment of histology-agnostic treatments for reimbursement by the French National Health Insurance Fund. The requirements of this approach include the need for randomisation, clinically relevant endpoints, appropriate correction for multiple significance testing, characterisation of subgroup heterogeneity, and validation of underlying biomarker assays. A prospectively designated external control is encouraged when the implementation of a direct comparison is deemed infeasible. We also underline the importance of recording outcomes from basket trials in a registry for use as future external controls.
在过去的十年中，卫生技术评估机构在确定新药对个人和医疗保健系统的益处方面面临着新的挑战。肿瘤学领域越来越重要的一个议题是，基于篮子试验，对癌症的靶向治疗（不考虑肿瘤位置和组织学）进行所谓的“不可知监管批准”。肿瘤学中的篮子试验具有同时评估多种肿瘤（甚至罕见癌症）的单一临床试验的优势。为了解决这些试验带来的新挑战，代表法国国家卫生管理局透明度委员会的一个跨学科小组召开会议，旨在阐明一种方法，以确保对法国国家健康保险基金报销的组织学不可知治疗方法进行透明、可重复和公平的评估。该方法的要求包括需要随机化、临床相关终点、适当校正多重显著性检验、亚组异质性的特征描述以及潜在生物标志物检测的验证。当直接比较被认为不可行时，鼓励使用前瞻性指定的外部对照。我们还强调了在登记处记录篮子试验结果以作为未来外部对照的重要性。
