Abstract

BackgroundInformation and theory beyond copula concepts are essential to understand the dependence relationship between several marginal covariates distributions. In a therapeutic trial data scheme, most of the time, censoring occurs. That could lead to a biased interpretation of the dependence relationship between marginal distributions. Furthermore, it could result in a biased inference of the joint probability distribution function. A particular case is the cost-effectiveness analysis (CEA), which has shown its utility in many medico-economic studies and where censoring often occurs.MethodsThis paper discusses a copula-based modeling of the joint density and an estimation method of the costs, and quality adjusted life years (QALY) in a cost-effectiveness analysis in case of censoring. This method is not based on any linearity assumption on the inferred variables, but on a punctual estimation obtained from the marginal distributions together with their dependence link.ResultsOur results show that the proposed methodology keeps only the bias resulting statistical inference and don’t have anymore a bias based on a unverified linearity assumption. An acupuncture study for chronic headache in primary care was used to show the applicability of the method and the obtained ICER keeps in the confidence interval of the standard regression methodology.ConclusionFor the cost-effectiveness literature, such a technique without any linearity assumption is a progress since it does not need the specification of a global linear regression model. Hence, the estimation of the a marginal distributions for each therapeutic arm, the concordance measures between these populations and the right copulas families is now sufficient to process to the whole CEA.

Highlights

  • Information and theory beyond copula concepts are essential to understand the dependence relationship between several marginal covariates distributions

  • There is Zhao and Zhou who work with copula models using medical costs, but in a stochastic process context, which is not adapted to quality adjusted life years (QALY) and costs data when the information is limited

  • For all data generating processes (DGP), the Kendall’s tau was identical and represented an intermediate level of dependence between marginal distributions to be fair with the reality: τK = 0.60

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Summary

Introduction

Information and theory beyond copula concepts are essential to understand the dependence relationship between several marginal covariates distributions. Due to the variety of treatments for a specific health problem and in conjunction with their increasing costs, cost-effectiveness studies of new therapies is challenging These studies could achieve to a statistical analysis since that the common practice in laboratories is to collect individual patient cost data in randomized studies. Fontaine et al BMC Medical Research Methodology (2017) 17:27 where C1 is the cost of the tested therapeutic, C0 is the cost of the control group which is usually measured in term of a given monetary unit, T1 is the effectiveness of the tested therapeutic which is usually measured in term of survival life years, T0 is the effectiveness of the control group and, E() is the expectation function It is an indicator of the monetary cost of using a new therapy in terms of survival time.

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