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Decision analysis, economic evaluation, and newborn screening: challenges and opportunities
Lisa A Prosser1, Scott D Grosse, Alex R Kemper
1Child Health Evaluation and Research Unit, Division of General Pediatrics, University of Michigan, Ann Arbor, Michigan, USA.
Insights
Evaluating new newborn screening tests requires better data on costs and outcomes. Decision analyses and economic models can guide policy for expanding newborn screening programs effectively.
Area of Science:
- Public Health
- Genetics
- Health Economics
Background:
- Newborn screening panels in the US have expanded rapidly.
- Evaluating rare candidate conditions for screening is challenging due to limited data.
Purpose of the Study:
- To outline data requirements for decision analysis and cost-effectiveness models in newborn screening.
- To discuss challenges in applying these methods for policy decisions.
Main Methods:
- Review of data needs for decision analysis and economic evaluations.
- Discussion of challenges in applying modeling techniques to newborn screening.
Main Results:
- Scarcity of data on screening, follow-up, treatment, and disability costs hinders evaluation.
- Decision analyses and economic evaluations offer systematic approaches to synthesize evidence.
Conclusions:
- Improved data collection is crucial for evaluating new newborn screening conditions.
- Modeling approaches can project long-term outcomes and inform policy for screening programs.
Abstract:
The number of conditions included in newborn screening panels has increased rapidly in the United States during the past decade, and many more conditions are under consideration for addition to state panels. The rare nature of candidate conditions for newborn screening makes their evaluation challenging. The scarcity of data on the costs of screening, follow-up, treatment, and long-term disability must be addressed to improve the evaluation process for nominated conditions. Decision analyses and economic evaluations can help inform policy decisions for newborn screening programs by providing a systematic approach to synthesizing available evidence and providing projected estimates of long-term clinical and economic outcomes when long-term data are not available. In this review, we outline the types of data required for the development of decision analysis and cost-effectiveness models for newborn screening programs and discuss the challenges faced when applying these methods in the arena of newborn screening to help inform policy decisions.Genet Med advance online publication 5 April 2012.
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