Gallmann, Elena
(2015)
A critical appraisal of coverage and resource allocation decisions through the use of health technology assessment: evidence on orphan drugs from four countries.
PhD thesis, London School of Economics and Political Science.
Abstract
Health Technology Assessment (HTA) relies on evidence-based medicine to inform drug coverage recommendations about the most efficient use of resources. Despite appraising the same evidence based on similar methodological approaches, HTA recommendations for the same drug differ across countries. This thesis aimed to understand the reasons for these differences, and based on cross-national comparisons, whether they are a consequence of methodological challenges in HTA. A mixed methods research design was used to develop a methodological framework that allows to breakdown these complex processes in a comparable and understandable manner, by considering: (a) the evidence appraised, (b) its interpretation, and (c) how this influenced the final decision. Ten orphan drug-indication pairs appraised in four countries (England, Scotland, Sweden and France (N=35)) were systematically analysed and compared on this basis. Results present the criteria accounted for at each stage of the process in the decisions, the reasons for differences across countries, and how HTA bodies are dealing with issues relating to orphan drugs. Quantitative analysis of these provided information about agency-specific risk and value preferences, and measured agreement in interpreting the same evidence. There was heterogeneity within and across countries in the criteria accounted for and reasons for cross-country differences. Interviews to competent authorities provided insights about these differences and implications for HTA. Although agreement was seen in the evidentiary requirements or preferences, there were subtle differences in the circumstances where uncertain evidence may be considered acceptable, partly explaining diverging HTA recommendations. The three main contributions of this thesis are: (1) the development of a methodological framework to understand what criteria feed into HTAs, which can be applied to other drugs and countries; (2) through its application, the identification of a full taxonomy of criteria considered in decisionmaking; and (3) the ability to understand the differences in HTA recommendations across countries. A better understanding of HTA in different settings may help advance these processes, and, ultimately, improve access to treatments.
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