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.

在过去十年中，卫生技术评估机构在确定新药对个人和卫生保健系统的益处方面面临新的挑战。对肿瘤学领域越来越重要的一个主题是所谓的癌症靶向治疗（独立于肿瘤位置和组织学）的不可知监管批准，基于篮子试验。肿瘤学中的篮子试验提供了在单一临床试验中同时评估多种肿瘤甚至罕见癌症的治疗方法的优势。为了应对这些试验带来的新挑战，代表法国国家卫生局透明度委员会召集了一个跨学科小组，以阐明一种旨在保证透明、可重复、和公平评估法国国家健康保险基金报销的组织学不可知治疗。这种方法的要求包括需要随机化、临床相关终点、对多重显着性检验的适当校正、亚组异质性的表征以及基础生物标志物测定的验证。当直接比较的实施被认为不可行时，鼓励采用前瞻性指定的外部控制。我们还强调了在注册表中记录篮子试验结果以用作未来外部控制的重要性。亚组异质性的表征，以及潜在生物标志物测定的验证。当直接比较的实施被认为不可行时，鼓励采用前瞻性指定的外部控制。我们还强调了在注册表中记录篮子试验结果以用作未来外部控制的重要性。亚组异质性的表征，以及潜在生物标志物测定的验证。当直接比较的实施被认为不可行时，鼓励采用前瞻性指定的外部控制。我们还强调了在注册表中记录篮子试验结果以用作未来外部控制的重要性。