RESUMO
OBJECTIVE: To assess the cost-effectiveness of neonatal screening on medium chain acyl-CoA dehydrogenase (MCAD) deficiency in a homogeneous population. STUDY DESIGN: For the scenario without neonatal screening, medical chart review and interviews were performed with physicians and families of 116 Dutch patients born between 1985 and July 2003 with clinically ascertained MCAD deficiency. For the scenario with neonatal screening, 66,205 unaffected and 11 affected newborns identified by prospective neonatal screening for MCAD deficiency in the northern part of the Netherlands were evaluated. The incremental cost-effectiveness ratio (ICER) used life years (LYs) as the outcome measure by combining both scenarios in a decision model with second-order Monte Carlo simulation. RESULTS: For the scenarios with and without neonatal screening for MCAD deficiency, costs were $6.10 and $4.22 per newborn, respectively. The main cost categories were institutionalization (64%), admissions (17%), special education (8%), laboratory testing (4%), and (para)medical contact (4%). The resulting ICER was $1653 per LY gained. Sensitivity analysis generated an ICER between $14,839 and $4345 per LY gained. CONCLUSIONS: Screening for MCAD deficiency in a well-defined population generates an ICER well within accepted boundaries for cost-effective interventions, even after sensitivity analysis.
Assuntos
Acil-CoA Desidrogenase/deficiência , Efeitos Psicossociais da Doença , Custos de Cuidados de Saúde , Erros Inatos do Metabolismo Lipídico/economia , Triagem Neonatal/economia , Estudos de Casos e Controles , Análise Custo-Benefício , Feminino , Humanos , Incidência , Recém-Nascido , Erros Inatos do Metabolismo Lipídico/diagnóstico , Erros Inatos do Metabolismo Lipídico/epidemiologia , Masculino , Triagem Neonatal/normas , Países Baixos , Valores de Referência , Estudos Retrospectivos , Medição de RiscoRESUMO
OBJECTIVE: To describe and analyze the use and costs of hospital services for children diagnosed with medium-chain acyl-CoA dehydrogenase (MCAD) deficiency either with newborn screening or clinical diagnosis in Australia between 1994 and 2002. MCAD deficiency is a potentially lethal disorder of fatty-acid oxidation. STUDY DESIGN: We conducted a retrospective audit of medical records supplemented by a parental survey. RESULTS: A total of 59 children with MCAD deficiency were identified, 24 by using newborn screening. In the first 4 years of life, screening children cost an average of A$1676 (US$1297) per year for inpatient, emergency department, and outpatient visits, compared with A$1796 (US$1390) for children in whom a clinical diagnosis was made. Forty-two percent of the children who underwent screening were admitted to the hospital, compared with 71% of children who did not undergo screening. Children who did not undergo screening used significantly more inpatient services and cost significantly more in emergency services. There were also some significant differences in use on a year-by-year basis. CONCLUSIONS: Children who do not undergo screening may be more likely to be admitted to the hospital and to incur higher emergency department costs than children who underwent screening, and children seem more likely to attend hospital outpatient clinics. Screening does not result in higher costs from a hospital perspective.