Dinan Michaela A, Lyman Gary H, Schilsky Richard L, Hayes Daniel F
Duke University Medical Center, Durham, NC.
Fred Hutchinson Cancer Research Center and the University of Washington, Seattle, WA.
JCO Precis Oncol. 2019 Dec;3:1-10. doi: 10.1200/PO.19.00210.
Cancer precision medicine depends on high-quality tumor biomarker tests (TBTs) for treatment selection. TBT reimbursement within the United States in the current regulatory environment is not tied to premarket evidence of clinical utility, resulting in a vicious cycle wherein low-level evidence of utility leads to poor reimbursement, thereby impeding investment in developing new, clinically valuable TBTs supported by high-level evidence. Rational, value-based TBT pricing presents many practical challenges. Precise one-to-one mapping of reimbursement to cost savings or cost effectiveness is precluded by an absence of formal cost-effectiveness analyses for many emerging TBTs, and for more established TBTs, it has become clear that such analyses may yield wildly variable, subjective estimates. To address these challenges, we propose a system of tiered reimbursement that rewards development of high-quality TBTs within specific use contexts, supported by strong evidence of analytic validity and clinical utility. We propose three use contexts of TBTs, each defined by its influence on treatment decisions relative to the current standard of care-Opt-Out, Opt-In, and the use of appropriate, alternative, effective therapies (Opt-Alt). By ensuring minimum levels of reimbursement, this system provides a return on investment to encourage and support the research and development needed to generate high levels of evidence for claims of clinical utility for TBTs by using a robust, objective, and value-based system. We believe our proposed evaluation system will serve as a practical starting point to raise the bar for TBT quality and utility, which has the potential to redirect health care dollars from futile or ineffective treatment to investment in the development of high-quality TBTs needed for safe and effective precision cancer care.
癌症精准医疗依赖高质量的肿瘤生物标志物检测（TBT）来进行治疗选择。在美国当前的监管环境下，TBT的报销与上市前临床效用证据无关，这导致了一个恶性循环，即低水平的效用证据导致报销不佳，从而阻碍了对开发有高水平证据支持的、具有临床价值的新TBT的投资。合理的、基于价值的TBT定价面临诸多实际挑战。由于许多新兴TBT缺乏正式的成本效益分析，无法将报销与成本节约或成本效益进行精确的一对一映射，而对于更成熟的TBT，很明显此类分析可能会产生差异极大的主观估计。为应对这些挑战，我们提出了一种分层报销系统，该系统在特定使用背景下奖励高质量TBT的开发，并辅以分析有效性和临床效用的有力证据。我们提出了TBT的三种使用背景，每种背景根据其相对于当前护理标准对治疗决策的影响来定义——退出（Opt-Out）、加入（Opt-In）以及使用适当的替代有效疗法（Opt-Alt）。通过确保最低报销水平，该系统提供投资回报，以鼓励和支持开展所需的研发工作，从而通过使用一个强大、客观且基于价值的系统，为TBT的临床效用声明生成高水平证据。我们相信，我们提出的评估系统将成为提高TBT质量和效用标准的一个切实可行的起点，这有可能将医疗保健资金从无效或低效的治疗转向投资开发安全有效的精准癌症治疗所需的高质量TBT。
