Abstract
ObjectivesTo 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.MethodsThe 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.ResultsApplying 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.ConclusionsThis 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.
Highlights
Healthcare decisionmaking is a complex process requiring simultaneous consideration of a number of elements including scientific judgment, economics and ethics
A highly synthesized version of the data presented to panelists is reported in Table 1
Growth hormone is the only available intervention indicated for the treatment of short stature in girls with Turner syndrome, a rare (1 in 2000) genetic disorder characterized by reduced life expectancy, absence of puberty, cardiovascular defects and short stature
Summary
Healthcare decisionmaking is a complex process requiring simultaneous consideration of a number of elements including scientific judgment, economics and ethics. A number of multicriteria models have emerged to support deliberation and assist consideration of the numerous factors implicated in healthcare decisionmaking [7,8,9,10,11,12,13,14,15]. Some elements of decisionmaking can be quantified, and multicriteria decision analysis (MCDA) provides a way to account for multiple streams of information [16]. Culyer [20] suggested a process that blends algorithmic (quantitative) and deliberative (non-quantitative) approaches. Such a comprehensive framework should allow explicit consideration of all elements of decision by a wide range of stakeholders [21] to provide accountability for reasonableness [22]
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