Authors' Affiliations: New Enterprise Associates, Inc., Menlo Park; CCS Associates, Mountain View; Myraqa, Redwood Shores, California; Johnson & Johnson/Veridex, LLC, Raritan, New Jersey; University of Texas, MD Anderson Cancer Center, Houston, Texas; Center for Diagnostics and Radiologic Health, Office of In Vitro Diagnostics, Personalized Medicine Program, Silver Spring; Center for Medical Technology Policy, Baltimore; Opus Three LLC; National Cancer Institute, Division of Cancer Treatment and Diagnosis, Rockville, Maryland; and Kaiser Permanente Research Affiliates Evidence-Based Practice Center, Kaiser Permanente Center for Health Research, Portland, Oregon.
Clin Cancer Res. 2014 Mar 15;20(6):1428-44. doi: 10.1158/1078-0432.CCR-13-2961.
This article defines and describes best practices for the academic and business community to generate evidence of clinical utility for cancer molecular diagnostic assays. Beyond analytical and clinical validation, successful demonstration of clinical utility involves developing sufficient evidence to demonstrate that a diagnostic test results in an improvement in patient outcomes. This discussion is complementary to theoretical frameworks described in previously published guidance and literature reports by the U.S. Food and Drug Administration, Centers for Disease Control and Prevention, Institute of Medicine, and Center for Medical Technology Policy, among others. These reports are comprehensive and specifically clarify appropriate clinical use, adoption, and payer reimbursement for assay manufacturers, as well as Clinical Laboratory Improvement Amendments-certified laboratories, including those that develop assays (laboratory developed tests). Practical criteria and steps for establishing clinical utility are crucial to subsequent decisions for reimbursement without which high-performing molecular diagnostics will have limited availability to patients with cancer and fail to translate scientific advances into high-quality and cost-effective cancer care. See all articles in this CCR Focus section, "The Precision Medicine Conundrum: Approaches to Companion Diagnostic Co-development."
本文为学术和商业界定义并描述了生成癌症分子诊断检测临床实用性证据的最佳实践。除了分析和临床验证外，成功证明临床实用性还需要收集足够的证据，以证明诊断测试可改善患者的治疗效果。本讨论与美国食品和药物管理局、疾病控制和预防中心、美国国家医学研究院和医疗技术政策中心等之前发布的指南和文献报告中描述的理论框架相辅相成。这些报告全面且明确地阐明了制造商、临床实验室改进修正案认证实验室（包括开发检测的实验室）的适当临床用途、采用和报销，这些实验室包括那些开发检测的实验室（实验室开发的检测）。建立临床实用性的实用标准和步骤对于后续的报销决策至关重要，否则高性能的分子诊断将无法广泛用于癌症患者，也无法将科学进步转化为高质量和具有成本效益的癌症护理。请参阅 CCR 重点部分“精准医疗的难题：伴随诊断共同开发的方法”中的所有文章。
