title: An Economic Evaluation of Neonatal Screening for Inborn Errors of Metabolism Using Tandem Mass Spectrometry in Thailand.
abstract: BACKGROUND
  Inborn errors of metabolism (IEM) are a rare group of genetic diseases which can lead to several serious long-term complications in newborns. In order to address these issues as early as possible, a process called tandem mass spectrometry (MS/MS) can be used as it allows for rapid and simultaneous detection of the diseases. This analysis was performed to determine whether newborn screening by MS/MS is cost-effective in Thailand.
  
  
  METHODS
  A cost-utility analysis comprising a decision-tree and Markov model was used to estimate the cost in Thai baht (THB) and health outcomes in life-years (LYs) and quality-adjusted life year (QALYs) presented as an incremental cost-effectiveness ratio (ICER). The results were also adjusted to international dollars (I$) using purchasing power parities (PPP) (1 I$ = 17.79 THB for the year 2013). The comparisons were between 1) an expanded neonatal screening programme using MS/MS screening for six prioritised diseases: phenylketonuria (PKU); isovaleric acidemia (IVA); methylmalonic acidemia (MMA); propionic acidemia (PA); maple syrup urine disease (MSUD); and multiple carboxylase deficiency (MCD); and 2) the current practice that is existing PKU screening. A comparison of the outcome and cost of treatment before and after clinical presentations were also analysed to illustrate the potential benefit of early treatment for affected children. A budget impact analysis was conducted to illustrate the cost of implementing the programme for 10 years.
  
  
  RESULTS
  The ICER of neonatal screening using MS/MS amounted to 1,043,331 THB per QALY gained (58,647 I$ per QALY gained). The potential benefits of early detection compared with late detection yielded significant results for PKU, IVA, MSUD, and MCD patients. The budget impact analysis indicated that the implementation cost of the programme was expected at approximately 2,700 million THB (152 million I$) over 10 years.
  
  
  CONCLUSIONS
  At the current ceiling threshold, neonatal screening using MS/MS in the Thai context is not cost-effective. However, the treatment of patients who were detected early for PKU, IVA, MSUD, and MCD, are considered favourable. The budget impact analysis suggests that the implementation of the programme will incur considerable expenses under limited resources. A long-term epidemiological study on the incidence of IEM in Thailand is strongly recommended to ascertain the magnitude of problem.
authors.affiliation: Health Intervention and Technology Assessment Program (HITAP), Ministry of Public Health, Nonthaburi, Thailand.
authors.forename: Kittiphong
authors.initials: K
authors.lastname: Thiboonboon
authors.affiliation: Health Intervention and Technology Assessment Program (HITAP), Ministry of Public Health, Nonthaburi, Thailand.
authors.forename: Pattara
authors.initials: P
authors.lastname: Leelahavarong
authors.affiliation: Division of Medical Genetics, Department of Pediatrics, Faculty of Medicine Ramathibodi Hospital, Mahidol University, Bangkok, Thailand.
authors.forename: Duangrurdee
authors.initials: D
authors.lastname: Wattanasirichaigoon
authors.affiliation: Division of Medical Genetics, Department of Pediatrics, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand.
authors.forename: Nithiwat
authors.initials: N
authors.lastname: Vatanavicharn
authors.affiliation: Division of Medical Genetics, Department of Pediatrics, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand.
authors.forename: Pornswan
authors.initials: P
authors.lastname: Wasant
authors.affiliation: Center of Excellence for Medical Genetics, Department of Pediatrics, Faculty of Medicine, Chulalongkorn University, Bangkok, Thailand.
authors.forename: Vorasuk
authors.initials: V
authors.lastname: Shotelersuk
authors.affiliation: Genetic Unit, Department of Pediatrics, The Queen Sirikit National Institute of Child Health, Department of Medical Services, Ministry of Public Health, Bangkok, Thailand.
authors.forename: Suthipong
authors.initials: S
authors.lastname: Pangkanon
authors.affiliation: Genetic Unit, Department of Pediatrics, The Queen Sirikit National Institute of Child Health, Department of Medical Services, Ministry of Public Health, Bangkok, Thailand.
authors.forename: Chulaluck
authors.initials: C
authors.lastname: Kuptanon
authors.affiliation: Neonatal Screening Operation Centre, Department of Medical Science, Ministry of Public Health, Nonthaburi, Thailand.
authors.forename: Sumonta
authors.initials: S
authors.lastname: Chaisomchit
authors.affiliation: Health Intervention and Technology Assessment Program (HITAP), Ministry of Public Health, Nonthaburi, Thailand.
authors.forename: Yot
authors.initials: Y
authors.lastname: Teerawattananon
mesh_terms: D000592:Amino Acid Metabolism, Inborn Errors / Q000175:diagnosis / Q000191:economics; D003362:Cost-Benefit Analysis; D003663:Decision Trees; D006801:Humans; D007231:Infant, Newborn; D050769:Isovaleryl-CoA Dehydrogenase / Q000172:deficiency / Q000191:economics; D008375:Maple Syrup Urine Disease / Q000175:diagnosis / Q000191:economics; D008390:Markov Chains; D008661:Metabolism, Inborn Errors / Q000175:diagnosis* / Q000191:economics*; D018803:Models, Economic; D009100:Multiple Carboxylase Deficiency / Q000175:diagnosis / Q000191:economics; D015999:Multivariate Analysis; D015997:Neonatal Screening / Q000191:economics*; D010661:Phenylketonurias / Q000175:diagnosis / Q000191:economics; D011336:Probability; D056693:Propionic Acidemia / Q000175:diagnosis / Q000191:economics; D019057:Quality-Adjusted Life Years; D015203:Reproducibility of Results; D053719:Tandem Mass Spectrometry / Q000191:economics*; D013785:Thailand
chemical_list: D050769:Isovaleryl-CoA Dehydrogenase