Expanding screening for rare metabolic disease in the newborn: an analysis of costs, effect and ethical consequences for decision-making in Finland

Acta Paediatr. 2005 Aug;94(8):1126-36. doi: 10.1111/j.1651-2227.2005.tb02056.x.

Abstract

Aim: Currently, the only metabolic disorder that newborns are screened for in Finland is congenital hypothyroidism. A proposal to start a pilot study on screening for other rare metabolic diseases using tandem mass spectrometry prompted a health technology assessment project on the effect and costs of expanded newborn screening programme options.

Method: A modelling study using data from current published studies, healthcare registers and expert opinion.

Results: The annual running cost of screening 56,000 newborns for the chosen five disorders (congenital adrenal hyperplasia, medium-chain acyl-CoA dehydrogenase deficiency [MCADD], long chain 3-hydroxyacyl-CoA dehydrogenase deficiency [LCHADD], phenylketonuria [PKU] and glutaric aciduria type 1 [GA 1]) was estimated to be euros 2.5 million or euros 45 per newborn when starting costs were included. The costs per quality-adjusted life year (QALY) gained are a maximum of euros 25,500. Prevention of severe handicap in one newborn would reduce the costs to a maximum of euros 18,000 per QALY gained.

Conclusions: Expanding the Finnish neonatal screening programme would require a new organization. The cost-effectiveness, resources, ethics and equity need to be considered when deciding in favour of or against starting a new screening programme.

Publication types

  • Comparative Study

MeSH terms

  • Cost-Benefit Analysis
  • Decision Making
  • Female
  • Finland / epidemiology
  • Health Care Costs*
  • Health Care Surveys
  • Humans
  • Infant, Newborn
  • Male
  • Mass Screening / economics
  • Mass Screening / ethics
  • Metabolism, Inborn Errors / diagnosis*
  • Metabolism, Inborn Errors / economics
  • Metabolism, Inborn Errors / epidemiology
  • Neonatal Screening / economics*
  • Neonatal Screening / ethics*
  • Quality-Adjusted Life Years*
  • Rare Diseases
  • Registries
  • Risk Assessment
  • Sensitivity and Specificity
  • Severity of Illness Index