Combining multicriteria decision analysis, ethics and health technology assessment: applying the EVIDEM decision-making framework to growth hormone for Turner syndrome patients.
Combining multicriteria decision analysis, ethics and health technology assessment: applying the EVIDEM decision-making framework to growth hormone for Turner syndrome patients.
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DOI：
10.1186/1478-7547-8-4
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发表时间：
2010-04-08
期刊：
影响因子：
--
通讯作者：
Deal C
中科院分区：
文献类型：
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作者：
Goetghebeur MM;Wagner M;Khoury H;Rindress D;Grégoire JP;Deal C
测试和进一步发展的医疗保健政策和临床决策支持框架使用生长激素（GH）特纳综合征（TS）作为一个复杂的案例研究。EVIDEM框架得到了进一步发展，以补充多标准决策分析价值矩阵，该矩阵包括15个可量化的决策组成部分，分为四个领域（证据质量、疾病、干预和经济学），定性工具包括六个与伦理和卫生系统有关的决策组成部分。对文献进行了广泛的审查，以制定一个卫生技术评估报告（HTA），适合于每个组成部分的决定，内容由专家进行了验证。然后，一个由利益攸关方代表组成的小组通过对决定中的每个MCDA组成部分进行加权和评分，估计了加拿大TS的生长激素MCDA值，然后考虑了决定中不可量化组成部分的影响。应用该框架揭示了重大的数据差距，以及将研究问题与数据需求相结合以真正为决策提供信息的重要性。专家组成员估计TS的GH值为MCDA量表上最大值的41%，在个体水平上具有良好的一致性（复检值40%; ICC：0.687），并且专家组成员之间的差异较大。该小组特定值的主要贡献者是“疗效改善”、“疾病严重程度”和“证据质量”。关于效用、效率和公平性的伦理考虑以及GH的潜在滥用对治疗的感知价值产生了混合影响。提出这一框架是为了超越目前的成本效益模式，将卫生技术评估、军事和民防资源评估、价值观和道德规范结合起来。它支持系统地考虑决定的所有组成部分和现有证据，以提高透明度。需要进一步的测试和验证，以建立MCDA方法结合务实的卫生技术评估在医疗保健决策。
To test and further develop a healthcare policy and clinical decision support framework using growth hormone (GH) for Turner syndrome (TS) as a complex case study. The EVIDEM framework was further developed to complement the multicriteria decision analysis (MCDA) Value Matrix, that includes 15 quantifiable components of decision clustered in four domains (quality of evidence, disease, intervention and economics), with a qualitative tool including six ethical and health system-related components of decision. An extensive review of the literature was performed to develop a health technology assessment report (HTA) tailored to each component of decision, and content was validated by experts. A panel of representative stakeholders then estimated the MCDA value of GH for TS in Canada by assigning weights and scores to each MCDA component of decision and then considered the impact of non-quantifiable components of decision. Applying the framework revealed significant data gaps and the importance of aligning research questions with data needs to truly inform decision. Panelists estimated the value of GH for TS at 41% of maximum value on the MCDA scale, with good agreement at the individual level (retest value 40%; ICC: 0.687) and large variation across panelists. Main contributors to this panel specific value were "Improvement of efficacy", "Disease severity" and "Quality of evidence". Ethical considerations on utility, efficiency and fairness as well as potential misuse of GH had mixed effects on the perceived value of the treatment. This framework is proposed as a pragmatic step beyond the current cost-effectiveness model, combining HTA, MCDA, values and ethics. It supports systematic consideration of all components of decision and available evidence for greater transparency. Further testing and validation is needed to build up MCDA approaches combined with pragmatic HTA in healthcare decisionmaking.