[Cost-effectiveness methods of newborn screening assessment.]

Area of Science:

• Public Health
• Health Economics
• Pediatrics

Background:

• Newborn screening programs are crucial for secondary prevention and pre-symptomatic detection of various health conditions.
• Effective implementation necessitates rigorous evaluation of a program's effectiveness, safety, cost-effectiveness, feasibility, and budget impact.
• Economic evaluations are vital for the sustainability of health systems, guiding public funding decisions for health interventions based on robust evidence.

Purpose of the Study:

• To highlight the challenges in evaluating the cost-effectiveness of newborn screening programs.
• To underscore the importance of robust evidence for justifying public funding of these programs.
• To discuss the limitations and strategies for economic analysis in the context of newborn screening.

Main Methods:

• The study reviews the methodologies and challenges associated with economic evaluations of newborn screening programs.
• It addresses the scarcity of scientific evidence and the reliance on expert opinion.
• Methodological challenges in measuring quality-adjusted life years (QALYs) in pediatric populations are discussed.

Main Results:

• A significant limitation in evaluating the cost-effectiveness of newborn screening for hereditary disorders and metabolic errors is the scarcity of scientific evidence.
• Expert opinion is often necessary to supplement limited data, but introduces challenges related to biases and heterogeneity.
• Measuring QALYs in children presents unique methodological difficulties.

Conclusions:

• Despite challenges, establishing standardized newborn screening policies and programs is essential.
• Guidelines are being developed in Spain to homogenize newborn screening policies based on available scientific evidence.
• Addressing data scarcity and methodological issues is key to improving the economic evaluation of newborn screening programs.