Oosterhoff Marije, van der Maas Marloes E, Steuten Lotte M G
Panaxea b.v., Hengelosestraat 221, 7521 AC, Enschede, The Netherlands.
Fred Hutchinson Cancer Research Center, Seattle, USA.
Appl Health Econ Health Policy. 2016 Feb;14(1):51-65. doi: 10.1007/s40258-015-0198-x.
Diagnostic biomarkers have multiple applications along the care process and have a large potential in optimizing treatment decisions. However, many diagnostic biomarkers struggle to gain market access and obtain appropriate coverage because of a lack of evidence on their health economic impact.
The aim was to review the (methodological) characteristics of recent economic evaluations on diagnostic biomarkers and examine whether these studies dealt with specific issues such as different payer perspectives, preference heterogeneity, and multiple applications in subpopulations.
The PubMed database and the National Health Service Economic Evaluation Database were searched. Full economic evaluations published after 2009 assessing diagnostic biomarkers for the main non-communicable diseases in middle-income or high-income countries were considered eligible. Empirical and methodological study characteristics were summarized, as was the handling of specific issues related to the economic evaluation of personalized medicine.
Thirty-three economic evaluations were included, of which 25 were model-based analyses. The number of strategies compared ranged from two to 17 per study, and was especially large in studies assessing genetic testing in patients and their relatives. Cost-effectiveness results were most sensitive to test accuracy and costs of the biomarker (N = 7), the relative risk of an event (N = 4), and the proportion of people accepting genetic testing (N = 2). One study incorporated patient preferences, and none of the studies considered different payer perspectives, cost sharing arrangements or variable opportunity costs due to population density variability.
Published health economic evaluations of biomarkers used for diagnosing, staging diseases, and guiding treatment selection are characterized by a large number of comparators to model the potential clinical applications and to determine their value. Assessing outcomes beyond health as well as specific issues, such as different payer perspectives and patient preferences, is crucial to fully capture the potential health economic impact of diagnostic biomarkers and to inform value-based reimbursement.
诊断生物标志物在整个医疗过程中有多种应用，在优化治疗决策方面具有巨大潜力。然而，由于缺乏关于其健康经济影响的证据，许多诊断生物标志物难以进入市场并获得适当的覆盖范围。
旨在回顾近期诊断生物标志物经济评估的（方法学）特征，并检查这些研究是否处理了特定问题，如不同支付方的观点、偏好异质性以及亚人群中的多种应用。
检索了PubMed数据库和英国国家卫生服务经济评估数据库。2009年后发表的评估中高收入国家主要非传染性疾病诊断生物标志物的全面经济评估被视为符合条件。总结了实证和方法学研究特征，以及与个性化医疗经济评估相关的特定问题的处理情况。
纳入了33项经济评估，其中25项是基于模型的分析。每项研究比较的策略数量从2种到17种不等，在评估患者及其亲属基因检测的研究中尤为多。成本效益结果对生物标志物的检测准确性和成本（N = 7）、事件的相对风险（N = 4）以及接受基因检测的人群比例（N = 2）最为敏感。一项研究纳入了患者偏好，没有研究考虑不同支付方的观点、成本分摊安排或由于人口密度变化导致的可变机会成本。
已发表的用于诊断、疾病分期和指导治疗选择的生物标志物的健康经济评估的特点是有大量的比较对象，以模拟潜在的临床应用并确定其价值。评估健康以外的结果以及特定问题，如不同支付方的观点和患者偏好，对于充分捕捉诊断生物标志物的潜在健康经济影响并为基于价值的报销提供信息至关重要。
