A scoping review of pediatric economic evaluation 1980-2014: do trends over time reflect changing priorities in evaluation methods and childhood disease?

ABSTRACT

Introduction: Economic evaluations conducted in children have unique features compared to adults. Important developments in pediatric economic evaluation in recent years include new options for valuing health states for cost-utility analysis (CUA) and shifting child health priorities. The Pediatric Economic Database Evaluation (PEDE) project includes a comprehensive database of pediatric health economic evaluations published since 1980. The objective of this scoping review was to identify trends over time in the use of CUA and other analytic techniques, and the therapeutic areas chosen for study.

Areas covered: Medical and grey literature were searched, key characteristics were extracted, frequencies were tabulated and cross-tabulations were performed. Differences between early (1980 and 1999) and late (2000 and 2014) periods were assessed using a chi-squared statistic. Of the 2,776 pediatric economic evaluations published between 1980 and 2014, substantially more were cost-effectiveness analyses (CEAs) and CUAs than cost benefit analyses and cost minimization analyses (63.9 and 24.9% versus 7.6 and 3.6%, respectively). This pattern was consistent regardless of the type of intervention, disease or age group studied. A trend toward higher proportions of CUAs and CEAs was evident in the later period (X² p < 0.0001). Other significant trends included a higher proportion of studies of preventive interventions (X² p < 0.0001), and more studies in children aged 1 to 12 years and fewer in perinates in the later period (X² p < 0.0001). Overall the most common disease class studied was infectious diseases (29.2%).

Expert commentary: Pediatric economic evaluation continues to grow in volume and methodologic complexity. While CUAs have increased, whether their quality has improved remains unknown. Although most studies are in infectious disease, the volume of publications may not align with emerging child health priorities such as adolescent health, injury, developmental disabilities, mental health, and the use of personalized medicine. Increasing economic evaluations in these areas will enhance pediatric decision-making.

KEYWORDS:

• Economic evaluation
• cost-utility analysis
• cost-effectiveness analysis
• child health
• temporal trends
• child health priorities

Acknowledgments

The following individuals are acknowledged with gratitude for their substantial contribution to the Pediatric Economic Database Evaluation (PEDE) project: Sarah Costa, Seija Kromm, Shayanthan Parameswaran, Richard Zur, Heather Burnett and Elizabeth Uleyryk. We thank Ms. Jeri Danyelko and Christine Millan for administrative and technical support.

Declaration of interest

The sponsor had no role in the study design, collection of data, analysis, interpretation of data, writing of the report or the decision to submit the article for publication. The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

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Funding

This research was supported with funding from the Hospital for Sick Children Research Institute.