Specific guidelines for assessing and improving the methodological quality of economic evaluations of newborn screening.

Astrid Langer,Rolf Holle,Jürgen John

doi:10.1186/1472-6963-12-300

Abstract

BackgroundEconomic evaluation of newborn screening poses specific methodological challenges. Amongst others, these challenges refer to the use of quality adjusted life years (QALYs) in newborns, and which costs and outcomes need to be considered in a full evaluation of newborn screening programmes. Because of the increasing scale and scope of such programmes, a better understanding of the methods of high-quality economic evaluations may be crucial for both producers/authors and consumers/reviewers of newborn screening-related economic evaluations. The aim of this study was therefore to develop specific guidelines designed to assess and improve the methodological quality of economic evaluations in newborn screening.MethodsTo develop the guidelines, existing guidelines for assessing the quality of economic evaluations were identified through a literature search, and were reviewed and consolidated using a deductive iterative approach. In a subsequent test phase, these guidelines were applied to various economic evaluations which acted as case studies.ResultsThe guidelines for assessing and improving the methodological quality of economic evaluations in newborn screening are organized into 11 categories: “bibliographic details”, “study question and design”, “modelling”, “health outcomes”, “costs”, “discounting”, “presentation of results”, “sensitivity analyses”, “discussion”, “conclusions”, and “commentary”.ConclusionsThe application of the guidelines highlights important issues regarding newborn screening-related economic evaluations, and underscores the need for such issues to be afforded greater consideration in future economic evaluations. The variety in methodological quality detected by this study reveals the need for specific guidelines on the appropriate methods for conducting sound economic evaluations in newborn screening.

Highlights

Economic evaluation of newborn screening poses specific methodological challenges
Besides the dearth of long-term follow-up studies of newborn screening programmes and the associated extrapolation of short-term data to capture a lifetime time horizon, other challenges pertain to the problem of appropriate discounting, the use of quality adjusted life years (QALYs) in newborns, and which costs and outcomes need to be considered in a full evaluation of newborn screening programmes
Using a deductive iterative process, data from textbooks on health economic evaluation [1,14,15], principles of good practice published by different ISPOR Task Forces [16,17], and from several guidelines, checklists and criteria lists [7,8,18,19,20,21,22] were extracted qualitatively based on general categories identified in previous work [19]

Summary

Introduction

Economic evaluation of newborn screening poses specific methodological challenges. Amongst others, these challenges refer to the use of quality adjusted life years (QALYs) in newborns, and which costs and outcomes need to be considered in a full evaluation of newborn screening programmes. Because of the increasing scale and scope of such programmes, a better understanding of the methods of high-quality economic evaluations may be crucial for both producers/authors and consumers/reviewers of newborn screening-related economic evaluations. Besides the dearth of long-term follow-up studies of newborn screening programmes and the associated extrapolation of short-term data to capture a lifetime time horizon, other challenges pertain to the problem of appropriate discounting, the use of quality adjusted life years (QALYs) in newborns, and which costs and outcomes need to be considered in a full evaluation of newborn screening programmes. Because of the increasing scale and scope of such programmes, a better understanding of the methods of high-quality economic evaluations may be crucial for both producers/authors and consumers/reviewers of newborn screening-related economic evaluations in order to assure high quality allocation decisions in newborn screening. The guidelines were tested with respect to various economic evaluations in the areas of newborn hearing screening and newborn screening for medium-chain acyl–CoA dehydrogenase deficiency (MCADD) using tandem mass spectrometry (MS/MS)

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