Medical Sciences, Dx-Rx Institute, Fredensborg, Denmark.
JCO Precis Oncol. 2022 Jun;6:e2200100. doi: 10.1200/PO.22.00100.
Within hematology and oncology, companion diagnostics (CDxs) play an increasing role in securing an optimal therapy for individual patients, and the US Food and Drug Administration (FDA) consider this type of assay essential for the safe and effective use of a corresponding therapeutic product. Most CDxs are developed prospectively using the drug-diagnostic codevelopment model, which normally secures the simultaneous approval of both drugs and diagnostics. A CDx assay is an important treatment decision tool that needs to be available simultaneously with the drug. However, within the past few years, several targeted drugs and new indications have been approved by the FDA without a CDx, despite the use of a predictive biomarker assay for patient selection during clinical development. A missing analytical and clinically validated CDx assay could affect the correct use of these drugs and ultimately patient safety. An alternative to FDA-approved or FDA-cleared CDxs could be to use a laboratory-developed test, which will normally miss documentation on the clinical validity. On the basis of the information available from different publicly available FDA databases, this article briefly discusses the issue of missing CDx assays in relation to the approval of hematological and oncological drugs and new indications.
在血液学和肿瘤学领域，伴随诊断（CDx）在为个体患者确保最佳治疗方面发挥着越来越重要的作用，美国食品和药物管理局（FDA）认为这种检测对于相应治疗产品的安全有效使用是必不可少的。大多数 CDx 都是使用药物-诊断共同开发模型前瞻性开发的，该模型通常可以同时确保药物和诊断的批准。CDx 检测是一种重要的治疗决策工具，需要与药物同时提供。然而，在过去几年中，尽管在临床开发期间使用预测生物标志物检测来选择患者，但仍有一些靶向药物和新适应症获得了 FDA 的批准，而没有 CDx。缺少分析和临床验证的 CDx 检测可能会影响这些药物的正确使用，并最终影响患者安全。替代 FDA 批准或批准的 CDx 的方法可以是使用实验室开发的检测，但通常会缺少关于临床有效性的文件。基于不同公开可用的 FDA 数据库中的可用信息，本文简要讨论了与血液学和肿瘤学药物及新适应症批准相关的缺少 CDx 检测的问题。
