Lifetime Cost Effectiveness of Phenylketonuria Screening National Program in Iran
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Abstract
Background: Phenylketonuria (PKU) is an autosomal recessive disorder that screening and timely control of this disorder can prevent the adverse effects. Regarding the high prevalence of PKU in Iran, the PKU screening program was started in Iran in 2006. This study was conducted to determine the cost-effectiveness of PKU screening in Iran.
Methods: An economic evaluation was performed among screened and treated individuals compared to no screening in 2013. The study population included 1356132 newborns that screened for PKU diagnosis. Lifetime costs, quality-adjusted life year (QALY) gains and incremental cost-effectiveness ratio (ICER) were calculated from the perspective of government. A discount rate of 3% was considered for both QALYs and costs. A one-way sensitivity analysis was used for assessing the robustness of the results.
Results: The discounted lifetime cost of intervention and non-intervention were $59528953.8 and $85295501.6 respectively. Therefore, the total estimated cost saving was $25766547.84. PKU screening produces an ICER of $1844420 per QALY gained.
Conclusion: Screening and early treatment for PKU is highly cost-effective. Therefore, the screening can improve quality of life of the patients and increase financial saving in health system.
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2. Gunduz M, Arslan N, Unal O, et al (2015). Depres-sion and anxiety among parents of phenylke-tonuria children. Neurosciences (Riyadh), 20(4): 350–356.
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5. Berry SA, Brown C, Grant M, et al (2013). Newborn screening 50 years later: access issues faced by adults with PKU. Genet Med, 15(8):591-9.
6. Bosch AM, Burlina A, Cunningham A, et al (2015). Assessment of the impact of phenylketonuria and its treatment on quality of life of patients and parents from seven European countries. Orphanet J Rare Dis, 10:80.
7. Soleymani Z, Keramati N, Rohani F, et al (2015). Fac-tors Influencing Verbal Intelligence and Spoken Language in Children with Phenylketonuria. Indian Pediatr, 52(5): 397-401.
8. Karamifar H, Ordoei M, Karamizadeh Z, et al (2010). Incidence of neonatal hyper phenylalaninemia in Fars province, South Iran. Iran J Pediatr, 20(2): 216-220.
9. Habib A, Fallahzadeh MH, Kazeroni HR, et al (2010). Incidence of phenylketonuria in southern Iran. Iran J Basic Med Sci, 35(2): 137-139.
10. Heidari A, Arab M, Etemad K, et al (2016). Challeng-es of Implementation of the National Phenylke-tonuria Screening Program in Iran: A Qualitative Study. Electron Physician, 8(10): 3048–3056.
11. Hatam N, Shirvani S, Javanbakht M, et al (2013). Cost-Utility Analysis of Neonatal Screening Pro-gram, Shiraz University of Medical Sciences, Shi-raz, Iran, 2010. Iran J Pediatr, 23(5): 493-500.
12. Ramalho AR, Ramalho RJ, Oliveira CR, et al (2014). Evaluation of effectiveness and outcome of PKU screening and management in the State of Sergi-pe, Brazil. Arq Bras Endocrinol Metabol, 58(1): 62-7.
13. Kabir MJ, Vatankhah S, Delgoshaei B, et al (2013). Determinant criteria for designing Health benefit package in selected countries. Life Sci J, 10(3):1392-1403.
14. Thiboonboon K, Leelahavarong P, Wattanasirichai-goon D, et al (2015). An Economic Evaluation of Neonatal Screening for Inborn Errors of Metab-olism Using Tandem Mass Spectrometry in Thailand. PLoS One, 10(8): e0134782.
15. Giżewska M, MacDonald A, Bélanger-Quintana A, et al (2016). Diagnostic and management practices for phenylketonuria in 19 countries of the South and Eastern European Region: survey results. Eur J Pediatr, 175(2): 261-72.
16. Drummond MF, Stoddart GL, Torrance. GW (1988). Methods for the economic evaluation of health care programs. 4th ed. Oxford: Oxford University Press.
17. Ryder HF, McDonough C, Tosteson AN, et al (2009). Decision Analysis and Cost-effectiveness Analysis. Semin Spine Surg, 21(4): 216–222.
18. Shiva S, Barzgar M, Serajian E, et al (2011). Seizure Frequency and Electroencephalographic Findings in Children with Phenylketonuria. Med J Tabriz Uni Med Sciences, 33(4):37-41.
19. Martynyuk AE, Ucar DA, Yang DD, et al (2007). Epilepsy in phenylketonuria: a complex depend-ence on serum phenylalanine levels. Epilepsia, 48(6): 1143-50.
20. Jancar J (1998). Increased life expectancy in people with untreated phenylketonuria. J Intellect Disabil Res, 42 (Pt 1):97-9.
21. Dhondt JL, Farriaux JP, Sailly JC, et al (1991). Eco-nomic evaluation of cost-benefit ratio of neonatal screening procedure for phenylketonuria and hy-pothyroidism. J Inherit Metab Dis, 14(4): 633-9.
22. Hatam N, Shirvani S, Javanbakht M, et al (2013). Cost-utility analysis of neonatal screening pro-gram, Shiraz University of Medical Sciences, Shi-raz, Iran, 2010. Iran J Pediatr, 23(5): 493-500.
23. Hamers FF, Rumeau-Pichon C (2012). Cost-effectiveness analysis of universal newborn screening for medium chain acyl-CoA dehydro-genase deficiency in France. BMC Pediatr, 12:60.
24. Ng HL, Mak CM, Johnston JM (2016). Cost-Effectiveness Analysis of Newborn Screening for Organic Acidemias in Hong Kong. SM J Clin Pathol, 1(1):1001.
25. Tiwana SK., Rascati KL, Park H (2012). Cost-Effectiveness of Expanded Newborn Screening in Texas. Value Health, 15(5):613-21.
26. Carroll AE, Downs SM (2006). Comprehensive cost-utility analysis of newborn screening strate-gies. Pediatrics, 117(5 Pt 2):S287-95.
27. Bosch AM, Tybout W, van Spronsen FJ, et al (2007). The course of life and quality of life of early and continuously treated Dutch patients with phenyl-ketonuria. J Inherit Metab Dis., 30(1): 29–34.
28. Gassio R, Campistol J, Vilaseca MA, et al (2003). Do adult patients with phenylketonuria improve their quality of life after introduction/resumption of a phenylalanine-restricted diet? Acta Paediatr, 92(12):1474-8.
29. Sladkevicius E, Pollitt RJ, Mgadmi A, et al (2010). Cost effectiveness of establishing a neonatal screening programme for phenylketonuria in Libya. Appl Health Econ Health Policy, 8(6): 407-20.
30. Geelhoed EA, Lewis B, Hounsome D, et al (2005). Economic evaluation of neonatal screening for phenylketonuria and congenital hypothyroidism. J Paediatr Child Health, 41(11): 575-9.
31. Lord J, Thomason MJ, Littlejohns P, et al (1999). Secondary analysis of economic data: a review of cost-benefit studies of neonatal screening for phenylketonuria. J Epidemiol Community Health, 53(3):179-86.
2. Gunduz M, Arslan N, Unal O, et al (2015). Depres-sion and anxiety among parents of phenylke-tonuria children. Neurosciences (Riyadh), 20(4): 350–356.
3. Purevsuren J, Bolormaa B, Narantsetseg C, et al (2016). The first Mongolian cases of phenylke-tonuria in selective screening of inborn errors of metabolism. Mol Genet Metab Rep, 9:71-74.
4. Grosse S D (2015). Showing Value in Newborn Screening: Challenges in Quantifying the Effec-tiveness and Cost-Effectiveness of Early Detec-tion of Phenylketonuria and Cystic Fibrosis. Healthcare (Basel), 3(4): 1133-57.
5. Berry SA, Brown C, Grant M, et al (2013). Newborn screening 50 years later: access issues faced by adults with PKU. Genet Med, 15(8):591-9.
6. Bosch AM, Burlina A, Cunningham A, et al (2015). Assessment of the impact of phenylketonuria and its treatment on quality of life of patients and parents from seven European countries. Orphanet J Rare Dis, 10:80.
7. Soleymani Z, Keramati N, Rohani F, et al (2015). Fac-tors Influencing Verbal Intelligence and Spoken Language in Children with Phenylketonuria. Indian Pediatr, 52(5): 397-401.
8. Karamifar H, Ordoei M, Karamizadeh Z, et al (2010). Incidence of neonatal hyper phenylalaninemia in Fars province, South Iran. Iran J Pediatr, 20(2): 216-220.
9. Habib A, Fallahzadeh MH, Kazeroni HR, et al (2010). Incidence of phenylketonuria in southern Iran. Iran J Basic Med Sci, 35(2): 137-139.
10. Heidari A, Arab M, Etemad K, et al (2016). Challeng-es of Implementation of the National Phenylke-tonuria Screening Program in Iran: A Qualitative Study. Electron Physician, 8(10): 3048–3056.
11. Hatam N, Shirvani S, Javanbakht M, et al (2013). Cost-Utility Analysis of Neonatal Screening Pro-gram, Shiraz University of Medical Sciences, Shi-raz, Iran, 2010. Iran J Pediatr, 23(5): 493-500.
12. Ramalho AR, Ramalho RJ, Oliveira CR, et al (2014). Evaluation of effectiveness and outcome of PKU screening and management in the State of Sergi-pe, Brazil. Arq Bras Endocrinol Metabol, 58(1): 62-7.
13. Kabir MJ, Vatankhah S, Delgoshaei B, et al (2013). Determinant criteria for designing Health benefit package in selected countries. Life Sci J, 10(3):1392-1403.
14. Thiboonboon K, Leelahavarong P, Wattanasirichai-goon D, et al (2015). An Economic Evaluation of Neonatal Screening for Inborn Errors of Metab-olism Using Tandem Mass Spectrometry in Thailand. PLoS One, 10(8): e0134782.
15. Giżewska M, MacDonald A, Bélanger-Quintana A, et al (2016). Diagnostic and management practices for phenylketonuria in 19 countries of the South and Eastern European Region: survey results. Eur J Pediatr, 175(2): 261-72.
16. Drummond MF, Stoddart GL, Torrance. GW (1988). Methods for the economic evaluation of health care programs. 4th ed. Oxford: Oxford University Press.
17. Ryder HF, McDonough C, Tosteson AN, et al (2009). Decision Analysis and Cost-effectiveness Analysis. Semin Spine Surg, 21(4): 216–222.
18. Shiva S, Barzgar M, Serajian E, et al (2011). Seizure Frequency and Electroencephalographic Findings in Children with Phenylketonuria. Med J Tabriz Uni Med Sciences, 33(4):37-41.
19. Martynyuk AE, Ucar DA, Yang DD, et al (2007). Epilepsy in phenylketonuria: a complex depend-ence on serum phenylalanine levels. Epilepsia, 48(6): 1143-50.
20. Jancar J (1998). Increased life expectancy in people with untreated phenylketonuria. J Intellect Disabil Res, 42 (Pt 1):97-9.
21. Dhondt JL, Farriaux JP, Sailly JC, et al (1991). Eco-nomic evaluation of cost-benefit ratio of neonatal screening procedure for phenylketonuria and hy-pothyroidism. J Inherit Metab Dis, 14(4): 633-9.
22. Hatam N, Shirvani S, Javanbakht M, et al (2013). Cost-utility analysis of neonatal screening pro-gram, Shiraz University of Medical Sciences, Shi-raz, Iran, 2010. Iran J Pediatr, 23(5): 493-500.
23. Hamers FF, Rumeau-Pichon C (2012). Cost-effectiveness analysis of universal newborn screening for medium chain acyl-CoA dehydro-genase deficiency in France. BMC Pediatr, 12:60.
24. Ng HL, Mak CM, Johnston JM (2016). Cost-Effectiveness Analysis of Newborn Screening for Organic Acidemias in Hong Kong. SM J Clin Pathol, 1(1):1001.
25. Tiwana SK., Rascati KL, Park H (2012). Cost-Effectiveness of Expanded Newborn Screening in Texas. Value Health, 15(5):613-21.
26. Carroll AE, Downs SM (2006). Comprehensive cost-utility analysis of newborn screening strate-gies. Pediatrics, 117(5 Pt 2):S287-95.
27. Bosch AM, Tybout W, van Spronsen FJ, et al (2007). The course of life and quality of life of early and continuously treated Dutch patients with phenyl-ketonuria. J Inherit Metab Dis., 30(1): 29–34.
28. Gassio R, Campistol J, Vilaseca MA, et al (2003). Do adult patients with phenylketonuria improve their quality of life after introduction/resumption of a phenylalanine-restricted diet? Acta Paediatr, 92(12):1474-8.
29. Sladkevicius E, Pollitt RJ, Mgadmi A, et al (2010). Cost effectiveness of establishing a neonatal screening programme for phenylketonuria in Libya. Appl Health Econ Health Policy, 8(6): 407-20.
30. Geelhoed EA, Lewis B, Hounsome D, et al (2005). Economic evaluation of neonatal screening for phenylketonuria and congenital hypothyroidism. J Paediatr Child Health, 41(11): 575-9.
31. Lord J, Thomason MJ, Littlejohns P, et al (1999). Secondary analysis of economic data: a review of cost-benefit studies of neonatal screening for phenylketonuria. J Epidemiol Community Health, 53(3):179-86.
| Files | ||
| Issue | Vol 50 No 10 (2021) | |
| Section | Original Article(s) | |
| DOI | https://doi.org/10.18502/ijph.v50i10.7512 | |
| Keywords | ||
| Cost effectiveness Economic evaluation Phenylketonuria Newborn screening | ||
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How to Cite
1.
Heidari A, Arab M, Damari B. Lifetime Cost Effectiveness of Phenylketonuria Screening National Program in Iran. Iran J Public Health. 2021;50(10):2105-2110.
