PubMed · 15258007
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M M Brown, G C Brown. 2004. Value based medicine.. https://doi.org/10.1136/bjo.2003.040030
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The number of economic evaluations conducted on a multinational basis is increasing. Therefore, techniques are required to compare the results of such studies in a meaningful manner. This paper explores different approaches to comparing across country cost data applied to a European study of dialysis therapy for end-stage renal disease. A price and volume index is created at the level of the individual health care technology and compared to an exchange rate conversion and published purchasing power parities (PPPs). Both exchange rate and PPP conversions when published rates are used fail to accurately reflect the true resource use of the applied health care example. These differences can be related to specific issues of input mix and price variation. Alternatively, the use of technology specific PPPs provided a more robust approach for international comparisons and also have the potential for use in multi-centre economic evaluations within the same country.
Cost-effectiveness analysis (CEA) in health care is increasingly conducted alongside multicentre and multinational randomised controlled clinical trials (RCTs). The increased use of stochastic CEA is designed to account for between-patient sampling variability in cost-effectiveness data assuming that observations are independently distributed. However, between-location variability in cost-effectiveness may result if there is a hierarchical structure in the data; that is, if there is correlation in costs and outcomes between patients recruited in particular locations. This may be expected in multi-location trials given that centres and countries often differ in factors such as clinical practice, patient case-mix and the unit costs of delivering health care. A failure to acknowledge this feature may lead to misleading conclusions in a trial-based economic study. Multilevel modelling (MLM) is an analytical framework that can be used to handle hierarchical cost-effectiveness data. Using data from a recently conducted economic analysis, this paper shows how multilevel modelling can be used to obtain (a) more appropriate estimates of the population average incremental cost-effectiveness and associated standard errors compared to standard stochastic CEA; and (b) location-specific estimates of incremental cost-effectiveness which can be used to explore appropriately the variability between centres/countries of the cost-effectiveness results.
The availability of patient-level data from clinical trials has spurred a lot of interest in developing methods for quantifying and presenting uncertainty in cost-effectiveness analysis (CEA). Although the majority has focused on developing methods for using sample data to estimate a confidence interval for an incremental cost-effectiveness ratio (ICER), a small strand of the literature has emphasized the importance of incorporating risk preferences and the trade-off between the mean and the variance of returns to investment in health and medicine (mean-variance analysis). This paper shows how the exponential utility-moment-generating function approach is a natural extension to this branch of the literature for modelling choices from healthcare interventions with uncertain costs and effects. The paper assumes an exponential utility function, which implies constant absolute risk aversion, and is based on the fact that the expected value of this function results in a convenient expression that depends only on the moment-generating function of the random variables. The mean-variance approach is shown to be a special case of this more general framework. The paper characterizes the solution to the resource allocation problem using standard optimization techniques and derives the summary measure researchers need to estimate for each programme, when the assumption of risk neutrality does not hold, and compares it to the standard incremental cost-effectiveness ratio. The importance of choosing the correct distribution of costs and effects and the issues related to estimation of the parameters of the distribution are also discussed. An empirical example to illustrate the methods and concepts is provided.