Combining multicriteria decision analysis, ethics and health technology assessment: Applying the EVIDEM decisionmaking framework to growth hormone for Turner syndrome patients

被引：64
作者：
Goetghebeur M.M. [1 ]
Wagner M. [1 ]
Khoury H. [1 ]
Rindress D. [1 ]
Grégoire J. [2 ]
Deal C. [3 ]
机构：
[1] BioMedCom Consultants inc, Dorval, Quebec
[2] Population Health Research Unit of the CHA, Faculty of Pharmacy, Laval University, Québec
[3] Endocrinology Service and Research Center of the CHU Sainte-Justine, Department of Pediatrics, University of Montréal, Montreal, Québec
来源：
Cost Effectiveness and Resource Allocation | / 8卷 / 1期
关键词：
Growth Hormone; Short Stature; Health Technology Assessment; Turner Syndrome; Growth Hormone Treatment;
D O I：
10.1186/1478-7547-8-4
中图分类号：
学科分类号：
摘要：
Objectives: 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.Methods: 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.Results: 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.Conclusions: 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. © 2010 Goetghebeur et al; licensee BioMed Central Ltd.
引用
收藏
相关论文
共 51 条
[1]  
Schlander M., The use of cost-effectiveness by the National Institute for Health and Clinical Excellence (NICE): no(t yet an) exemplar of a deliberative process, J Med Ethics, 34, pp. 534-539, (2008)
[2]  
Williams I., McIver S., Moore D., Bryan S., The use of economic evaluations in NHS decision-making: a review and empirical investigation, Health Technol Assess, 12, (2008)
[3]  
Nord E., Daniels N., Kamlet M., QALYs: some challenges, Value Health, 12, SUPPL. 1, (2009)
[4]  
Drummond M., Evans B., LeLorier J., Karakiewicz P., Martin D., Tugwell P., Et al., Evidence and values: requirements for public reimbursement of drugs for rare diseases--a case study in oncology, Can J Clin Pharmacol, 16, (2009)
[5]  
Drummond M.F., Schwartz J.S., Jonsson B., Luce B.R., Neumann P.J., Siebert U., Et al., Key principles for the improved conduct of health technology assessments for resource allocation decisions, Int J Technol Assess Health Care, 24, pp. 244-258, (2008)
[6]  
Jehu-Appiah C., Baltussen R., Acquah C., Aikins M., d'Almeida S.A., Bosu W.K., Et al., Balancing equity and efficiency in health priorities in Ghana: the use of multicriteria decision analysis, Value Health, 11, pp. 1081-1087, (2008)
[7]  
Browman G.P., Manns B., Hagen N., Chambers C.R., Simon A., Sinclair S., 6-STEPPPs: A modular tool to facilitate clinician participation in fair decisions for funding new cancer drugs, Journal of Oncology Practice, 4, pp. 2-7, (2008)
[8]  
Johnson A.P., Sikich N.J., Evans G., Evans W., Giacomini M., Glendining M., Et al., Health technology assessment: a comprehensive framework for evidence-based recommendations in Ontario, Int J Technol Assess Health Care, 25, pp. 141-150, (2009)
[9]  
Tierney M., Manns B., Optimizing the use of prescription drugs in Canada through the Common Drug Review, CMAJ, 178, pp. 432-435, (2008)
[10]  
Martin D.K., Pater J.L., Singer P.A., Priority-setting decisions for new cancer drugs: a qualitative case study, Lancet, 358, pp. 1676-1681, (2001)
123456