Combining multicriteria decision analysis, ethics and health technology assessment: applying the EVIDEM decisionmaking f

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RESEARCH

Open Access

Combining multicriteria decision analysis, ethics and health technology assessment: applying the EVIDEM decisionmaking framework to growth hormone for Turner syndrome patients Mireille M Goetghebeur1*, Monika Wagner1, Hanane Khoury1, Donna Rindress1, Jean-Pierre Grégoire2, Cheri Deal3

Abstract 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.

Background Healthcare decisionmaking is a complex process requiring simultaneous consideration of a number of elements including scientific judgment, economics and ethics. The cost-effectiveness (CE) model has become a prime model for healthcare resource allocation and decisionmaking globally. It was developed to support decisionmaking by integrating into unified metrics some of the key elements considered to be important. Although the * Correspondence: [email protected] 1 BioMedCom Consultants inc, Dorval, Quebec, Canada

methods developed in this field are valuable for examining the consequences of new healthcare interventions, the focus on CE ratios (e.g. cost per quality-adjusted life year [QALY]) has contributed to a “black box” syndrome, both at