Banca de DEFESA: NEY CRISTIAN AMARAL BOA SORTE

Uma banca de DEFESA de MESTRADO foi cadastrada pelo programa.
DISCENTE : NEY CRISTIAN AMARAL BOA SORTE
DATA : 03/09/2021
HORA: 14:00
LOCAL: Sessão Remota
TÍTULO:

ANALYSIS OF NEONATAL SCREENING FOR GALACTOSEMIA IN STATE OF BAHIA: COST-EFFECTIVENESS STUDY

PALAVRAS-CHAVES:

Galactosemia; Newborn Screening; Cost-effectiveness; Incidence.

PÁGINAS: 80
GRANDE ÁREA: Ciências da Saúde
ÁREA: Saúde Coletiva
RESUMO:

BACKGROUND: Galactosemia, an inborn error of galactose metabolism, results on
sepsis and acute liver failure in the neonatal period, with a high risk of death during this
period. Given this, several European countries, Canadian provinces and American states
(USA) perform universal neonatal screening (NS) for this pathology. In 2018, CONITEC
considered that, for clinical diagnostic strategy, the existing evidence was insufficient to
establish the appropriateness of newborn screening for galactosemia. However, in May
2021, an expanded list of diseases that will be compound of the National Newborn
Screening Program (NNSP) was presented to include galactosemia. Health Technology
Assessment procedures require real-life data and economic analyses. AIMS: To describe
data from the pilot project of neonatal screening for galactosemia conducted in Bahia and
to evaluate the cost-effectiveness of neonatal screening compared with a clinical
diagnostic strategy. METHODS: A population screening study was carried out, followed
by a follow-up cohort of patients, coupled with a cost-effectiveness study. Operational
parameters of the newborn screening process were calculated. An analytical model was
developed to estimate the incremental cost-effectiveness ratio (ICER) between neonatal
screening strategies and clinical diagnosis (standard) from the National Health System
perspective. The health outcomes evaluated were death averted and potential life-years
gained. A 5% discount rate was applied. Costs were estimated from the SIGTAP table
and government procurement data. Univariate deterministic and probabilistic sensitivity
analyses were performed. Value of one Gross Domestic Product (GDP) per capita was
used as a threshold of willingness-to-pay. RESULTS: Between November 2015 and
March 2016, 77,148 newborns sequentially had galactose-1-phosphate (Gal-1-P) dosage
in dried blood on filter paper in the state of Bahia. Of these, 184 (0.23%) were positive in
the first screening and, 7.6% (14/184) remained with high Gal-1-P, and 5 cases were
confirmed, with a birth prevalence of 1:15,429 live births. The incremental cost per death
averted was estimated at R$259,052.37 and, R$4,143.31 for the potential gain of one year
of life. The birth prevalence of galactosemia and the probability of making a clinical
diagnosis were the variables with the greatest impact in the univariate deterministic
analysis. For the outcome of potential years of life gained, the acceptability curve showed
that for ICER values starting at R$7,034.40, the diagnostic strategy with neonatal
screening becomes more likely to be cost-effective than the clinical diagnostic strategy.
CONCLUSION: Among the population-based data available in Brazil, the state of Bahia
has the highest birth prevalence of galactosemia. Still, given the low frequency of the
disease, the neonatal screening strategy is not cost-effective at the willingness-to-pay
threshold of 1 GDP per capita to avoid one death. However, considering potential years
of life gained, at a WTP of R$10,561.60, neonatal screening was 100% cost-effective

MEMBROS DA BANCA:
Externo à Instituição - JOSÉ SIMON CAMELO JUNIOR - USP
Presidente - 701.890.055-72 - FABIANA RAYNAL FLORIANO - MS
Externo à Instituição - IVAN RICARDO ZIMMERMANN