Abstract

Introduction: The statistical quality of many trial-based economic evaluations is poor. When conducting trial-based economic evaluations, researchers often turn to national pharmacoeconomic guidelines for guidance. Therefore, this study reviewed which recommendations are currently given by national pharmacoeconomic guidelines on the statistical analysis of trial-based economic evaluations.Areas covered: 40 national pharmacoeconomic guidelines were identified. Data were extracted on the guidelines' recommendations on how to deal with baseline imbalances, skewed costs, correlated costs and effects, clustering of data, longitudinal data, and missing data in trail-based economic evaluations. Four guidelines (10%) were found to include recommendations on how to deal with baseline imbalances, five (13%) on how to deal with skewed costs, and seven (18%) on how to deal with missing data. Recommendations were very general in nature and recommendations on dealing with correlated costs and effects, clustering of data, and longitudinal data were lacking.Expert opinion: Current national pharmacoeconomic guidelines provide little to no guidance on how to deal with the statistical challenges to trial-based economic evaluations. Since use of suboptimal statistical methods may lead to biased results, and, therefore, possibly to a waste of scarce resources, national agencies are advised to include more statistical guidance in their pharmacoeconomic guidelines.

Original languageEnglish
JournalExpert review of pharmacoeconomics & outcomes research
DOIs
Publication statusE-pub ahead of print - 15 Nov 2019

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Pharmaceutical Economics
Cost-Benefit Analysis
Guidelines
Costs and Cost Analysis
Cluster Analysis
Expert Testimony
Research Personnel

Cite this

@article{2d11cd237aaa4ef092a48fe07ae96b01,
title = "What do national pharmacoeconomic guidelines recommend regarding the statistical analysis of trial-based economic evaluations?",
abstract = "Introduction: The statistical quality of many trial-based economic evaluations is poor. When conducting trial-based economic evaluations, researchers often turn to national pharmacoeconomic guidelines for guidance. Therefore, this study reviewed which recommendations are currently given by national pharmacoeconomic guidelines on the statistical analysis of trial-based economic evaluations.Areas covered: 40 national pharmacoeconomic guidelines were identified. Data were extracted on the guidelines' recommendations on how to deal with baseline imbalances, skewed costs, correlated costs and effects, clustering of data, longitudinal data, and missing data in trail-based economic evaluations. Four guidelines (10{\%}) were found to include recommendations on how to deal with baseline imbalances, five (13{\%}) on how to deal with skewed costs, and seven (18{\%}) on how to deal with missing data. Recommendations were very general in nature and recommendations on dealing with correlated costs and effects, clustering of data, and longitudinal data were lacking.Expert opinion: Current national pharmacoeconomic guidelines provide little to no guidance on how to deal with the statistical challenges to trial-based economic evaluations. Since use of suboptimal statistical methods may lead to biased results, and, therefore, possibly to a waste of scarce resources, national agencies are advised to include more statistical guidance in their pharmacoeconomic guidelines.",
author = "{van Dongen}, Hanneke and {El Alili}, Mohamed and Varga, {Anita N} and {Guevara Morel}, {Alejandra E} and {Jornada Ben}, Angela and Mojdeh Khorrami and {van Tulder}, {Maurits W} and Bosmans, {Judith E}",
year = "2019",
month = "11",
day = "15",
doi = "10.1080/14737167.2020.1694410",
language = "English",
journal = "Expert review of pharmacoeconomics & outcomes research",
issn = "1473-7167",
publisher = "Expert Reviews Ltd.",

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TY - JOUR

T1 - What do national pharmacoeconomic guidelines recommend regarding the statistical analysis of trial-based economic evaluations?

AU - van Dongen, Hanneke

AU - El Alili, Mohamed

AU - Varga, Anita N

AU - Guevara Morel, Alejandra E

AU - Jornada Ben, Angela

AU - Khorrami, Mojdeh

AU - van Tulder, Maurits W

AU - Bosmans, Judith E

PY - 2019/11/15

Y1 - 2019/11/15

N2 - Introduction: The statistical quality of many trial-based economic evaluations is poor. When conducting trial-based economic evaluations, researchers often turn to national pharmacoeconomic guidelines for guidance. Therefore, this study reviewed which recommendations are currently given by national pharmacoeconomic guidelines on the statistical analysis of trial-based economic evaluations.Areas covered: 40 national pharmacoeconomic guidelines were identified. Data were extracted on the guidelines' recommendations on how to deal with baseline imbalances, skewed costs, correlated costs and effects, clustering of data, longitudinal data, and missing data in trail-based economic evaluations. Four guidelines (10%) were found to include recommendations on how to deal with baseline imbalances, five (13%) on how to deal with skewed costs, and seven (18%) on how to deal with missing data. Recommendations were very general in nature and recommendations on dealing with correlated costs and effects, clustering of data, and longitudinal data were lacking.Expert opinion: Current national pharmacoeconomic guidelines provide little to no guidance on how to deal with the statistical challenges to trial-based economic evaluations. Since use of suboptimal statistical methods may lead to biased results, and, therefore, possibly to a waste of scarce resources, national agencies are advised to include more statistical guidance in their pharmacoeconomic guidelines.

AB - Introduction: The statistical quality of many trial-based economic evaluations is poor. When conducting trial-based economic evaluations, researchers often turn to national pharmacoeconomic guidelines for guidance. Therefore, this study reviewed which recommendations are currently given by national pharmacoeconomic guidelines on the statistical analysis of trial-based economic evaluations.Areas covered: 40 national pharmacoeconomic guidelines were identified. Data were extracted on the guidelines' recommendations on how to deal with baseline imbalances, skewed costs, correlated costs and effects, clustering of data, longitudinal data, and missing data in trail-based economic evaluations. Four guidelines (10%) were found to include recommendations on how to deal with baseline imbalances, five (13%) on how to deal with skewed costs, and seven (18%) on how to deal with missing data. Recommendations were very general in nature and recommendations on dealing with correlated costs and effects, clustering of data, and longitudinal data were lacking.Expert opinion: Current national pharmacoeconomic guidelines provide little to no guidance on how to deal with the statistical challenges to trial-based economic evaluations. Since use of suboptimal statistical methods may lead to biased results, and, therefore, possibly to a waste of scarce resources, national agencies are advised to include more statistical guidance in their pharmacoeconomic guidelines.

U2 - 10.1080/14737167.2020.1694410

DO - 10.1080/14737167.2020.1694410

M3 - Article

JO - Expert review of pharmacoeconomics & outcomes research

JF - Expert review of pharmacoeconomics & outcomes research

SN - 1473-7167

ER -