Being Precise About Precision Medicine: What Should Value Frameworks Incorporate to Address Precision Medicine? A Report of the Personalized Precision Medicine Special Interest Group
Being Precise About Precision Medicine: What Should Value Frameworks Incorporate to Address Precision Medicine? A Report of the Personalized Precision Medicine Special Interest Group
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DOI：
10.1016/j.jval.2019.11.010
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发表时间：
2020-05-01
期刊：
影响因子：
4.5
通讯作者：
Payne, Katherine
中科院分区：
文献类型：
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作者：
Faulkner, Eric;Holtorf, Anke-Peggy;Payne, Katherine
精准医学是一个充满活力的领域，它包含多种多样且不断增加的方法，可以针对新药，筛查计划或预防性医疗保健策略，其中包括使用生物标记物或由算法驱动的复杂测试，也可能考虑到患者的偏好。国际药物经济学与成果研究学会（International Society for Pharmacoeconomics and Outcome Research）将其当前围绕精准医学的工作扩展到：（1）以当前和不断发展的应用为例描述精准医学的发展模式;（2）描述关键利益相关者对精准医学在各自领域的价值的看法;（3）定义精准医学价值评估框架中应考虑的核心因素。精准医疗的最终目标是改善明确定义的患者群体的健康，它将在多个层面影响医疗保健系统中的所有利益相关者，从个人角度到社会角度。为了建立一个高效、及时和实用的精准医疗价值评估框架，重要的是通过利益相关者之间就价值方面的稳健程序和措施建立共识，解决这些多个角度，包括精准机制的性能;精准机制和后续治疗的一致报销流程;对证据要求和研究设计的透明预期充分匹配精密机制的预期用途和较小的目标患者人群;认识到价值创造的潜在范围，如纳入和排除决定。
Precision medicine is a dynamic area embracing a diverse and increasing type of approaches that allow the targeting of new medicines, screening programs or preventive healthcare strategies, which include the use of biologic markers or complex tests driven by algorithms also potentially taking account of patient preferences. The International Society for Pharmacoeconomics and Outcome Research expanded its current work around precision medicine to (1) describe the evolving paradigm of precision medicine with examples of current and evolving applications, (2) describe key stakeholders perspectives on the value of precision medicine in their respective domains, and (3) define the core factors that should be considered in a value assessment framework for precision medicine. With the ultimate goal of improving health of well-defined patient groups, precision medicine will affect all stakeholders in the healthcare system at multiple levels spanning the individual perspective to the societal perspective. For an efficient, timely and practical precision medicine value assessment framework, it will be important to address these multiple perspectives through building consensus among the stakeholders for robust procedures and measures of value aspects, including performance of precision mechanism; aligned reimbursement processes of precision mechanism and subsequent treatment; transparent expectations for evidence requirements and study designs adequately matched to the intended use of the precision mechanism and to the smaller target patient populations; recognizing the potential range of value-generation such as ruling-in and ruling-out decisions.