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Lifetime Cost Effectiveness of Phenylketonuria Screening National Program in Iran

Alireza Heidari1, Mohammad Arab2, Behzad Damari3

  • 1Health Management and Social Development Research Center, Golestan University of Medical Sciences, Gorgan, Iran.

Iranian Journal of Public Health
|February 28, 2022
PubMed

Insights

Phenylketonuria (PKU) screening in Iran is highly cost-effective, saving the health system over $25 million. Early detection and treatment of this genetic disorder improve patient quality of life and reduce long-term healthcare costs.

Area of Science:

  • Medical Genetics
  • Public Health Economics
  • Newborn Screening Programs

Background:

  • Phenylketonuria (PKU) is an inherited metabolic disorder requiring early intervention.
  • Iran initiated a national PKU screening program in 2006 due to the disorder's high prevalence.
  • Timely diagnosis and management of PKU are crucial to prevent severe adverse health effects.

Purpose of the Study:

  • To evaluate the cost-effectiveness of the PKU screening program in Iran.
  • To compare the lifetime costs and health outcomes of screened versus unscreened populations.

Main Methods:

  • Economic evaluation conducted in 2013, analyzing data from 1,356,132 screened newborns.
  • Calculated lifetime costs, Quality-Adjusted Life Year (QALY) gains, and Incremental Cost-Effectiveness Ratio (ICER) from a governmental perspective.
  • Utilized a 3% discount rate for costs and QALYs, with sensitivity analysis to ensure result robustness.

Main Results:

  • The intervention (PKU screening and treatment) resulted in discounted lifetime cost savings of $25,766,547.84 compared to no screening.
  • The incremental cost-effectiveness ratio (ICER) was calculated at $1,844,420 per QALY gained.
  • Screening and treatment demonstrated significant economic benefits and improved health outcomes.

Conclusions:

  • PKU screening and early treatment represent a highly cost-effective public health strategy in Iran.
  • The program enhances patient quality of life and generates substantial financial savings for the healthcare system.
  • Continued investment in PKU screening is recommended for long-term public health benefits.
Abstract

Keywords:
Cost effectivenessEconomic evaluationNewborn screeningPhenylketonuria

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