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Handling Missing Data in Within-Trial Cost-Effectiveness Analysis: A Review with Future Recommendations.

Andrea Gabrio1, Alexina J Mason2, Gianluca Baio3

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Summary

Missing data in cost-effectiveness analyses (CEAs) alongside randomized controlled trials (RCTs) are often inadequately handled. Improved reporting and methods are needed to ensure reliable healthcare technology assessments.

Keywords:
Miss Data AnalysisMiss Data MechanismMiss Data MethodMissingness MechanismRobustness Analysis

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Area of Science:

  • Health economics
  • Clinical trial methodology
  • Biostatistics

Background:

  • Cost-effectiveness analyses (CEAs) integrated with randomized controlled trials (RCTs) are crucial for healthcare technology assessment.
  • Missing data in resource use and health status measures can compromise the validity of CEAs.
  • Previous reviews highlighted issues in handling missing data in CEAs.

Purpose of the Study:

  • To systematically review the handling of missing cost and effect data in RCT-based CEAs from 2009-2015.
  • To propose guidelines for improved reporting and methods for addressing missing data.
  • To assess the quality of methods used and the justification provided for their selection.

Main Methods:

  • Systematic literature review of 81 RCT-based CEAs published between 2009 and 2015.
  • Analysis focused on the description of missing data, statistical methods employed, and the rationale for method choice.
  • Development of a quality evaluation scheme for guiding missing data handling.

Main Results:

  • Missing data in RCT-based CEAs continue to be frequently handled inadequately.
  • The reporting of missing data and the justification for chosen statistical methods are often insufficient.
  • Overall quality of information supporting missing data handling remains unsatisfactory.

Conclusions:

  • There is a persistent need for better practices in managing missing data within RCT-based CEAs.
  • Implementing structured quality evaluation schemes can improve the transparency and rigor of missing data handling.
  • Enhanced reporting and methodological choices are essential for reliable healthcare economic evaluations.