{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,21]],"date-time":"2026-05-21T00:37:27Z","timestamp":1779323847084,"version":"3.51.4"},"reference-count":40,"publisher":"BMJ","issue":"3","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J Epidemiol Community Health"],"published-print":{"date-parts":[[1999,3]]},"abstract":"<jats:p>STUDY OBJECTIVE: To estimate the net financial benefit of neonatal screening for phenylketonuria (PKU): by a simple pooling of cost data from the literature; and by a more complex modelling approach. DESIGN: A systematic literature review was conducted to identify papers containing data on the monetary costs and benefits of neonatal screening for PKU. The methodological quality of the studies was appraised, and data were extracted on resource use and expenditure. Monetary data were converted to common currency units, and standardised to UK incidence rates. Net benefits were calculated for median, best case and worst case scenarios, and the effect of excluding poor quality studies and data was tested. The net benefit was also estimated from a model based on data from the literature and assumptions appropriate for the current UK situation. Extensive sensitivity analysis was conducted. MAIN RESULTS: The direct net benefit of screening based on the median costs and benefits from the 13 studies identified was 143,400 Pounds per case detected and treated (39,000 Pounds and 241,800 Pounds for worst case and best case scenarios respectively). The direct net benefit obtained by the modelling approach was lower at 93,400 Pounds per case detected and treated. Screening remained cost saving under sensitivity analysis, except with low residential care costs (less than 12,300 Pounds per annum), or very low incidence rates (less than 1 in 27,000). CONCLUSIONS: The economic literature on PKU screening is of variable quality. The two methods of secondary analysis lead to the same conclusion: that neonatal PKU screening is worthwhile in financial terms alone in the UK, and that it justifies the infrastructure for collecting and testing neonatal blood samples. This result cannot necessarily be extrapolated to other countries.<\/jats:p>","DOI":"10.1136\/jech.53.3.179","type":"journal-article","created":{"date-parts":[[2009,1,5]],"date-time":"2009-01-05T14:15:53Z","timestamp":1231164953000},"page":"179-186","source":"Crossref","is-referenced-by-count":34,"title":["Secondary analysis of economic data: a review of cost-benefit studies of neonatal screening for phenylketonuria."],"prefix":"10.1136","volume":"53","author":[{"given":"J","family":"Lord","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"M J","family":"Thomason","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"P","family":"Littlejohns","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"R A","family":"Chalmers","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"M D","family":"Bain","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"G M","family":"Addison","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"A H","family":"Wilcox","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"C A","family":"Seymour","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"239","published-online":{"date-parts":[[1999,3,1]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"115","DOI":"10.1136\/bmj.306.6870.115","article-title":"Phenylketonuria due to phenylalanine hydroxylase deficiency: an unfolding story","volume":"306","author":"on Phenylketonuria, M.R.C.Working Party","year":"1993","unstructured":"MRC Working Party on Phenylketonuria. 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Med Care 1992;30:231\u201343.","journal-title":"Med Care"},{"key":"ref_6","article-title":"Methods for the economic evaluation of health care programmes","author":"Drummond, M.F.; Stoddart, G.L.; Torrance, G.W.","year":"1987","unstructured":"Drummond MF, Stoddart GL, Torrance GW. Methods for the economic evaluation of health care programmes. Oxford: Oxford University Press, 1987."},{"key":"ref_7","doi-asserted-by":"crossref","first-page":"155","DOI":"10.1002\/(SICI)1099-1050(199603)5:2<155::AID-HEC194>3.0.CO;2-O","article-title":"An exercise on the feasibility of carrying out secondary economic analysis","volume":"5","author":"JeVerson, T.; Mugford, M.; Gray, A.","year":"1996","unstructured":"JeVerson T, Mugford M, Gray A, et al. An exercise on the feasibility of carrying out secondary economic analysis. 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A case study: a CBA of the screening and treatment of PKU","author":"Wright, M.","year":"1990","unstructured":"Wright M. An evaluation of the cost benefit analysis approach. A case study: a CBA of the screening and treatment of PKU. [MSc Thesis]. Glasgow: University of Strathclyde, 1990."},{"key":"ref_11","first-page":"67","article-title":"Maternal phenylketonuria - diet, dangers and dilemmas","volume":"7","author":"Cockburn, F.; Clark, B.J.; Byrne, A.","year":"1992","unstructured":"Cockburn F, Clark BJ, Byrne A, et al. Maternal phenylketonuria - diet, dangers and dilemmas. International Paediatrics 1992;7:67\u201374.","journal-title":"International Paediatrics"},{"key":"ref_12","doi-asserted-by":"crossref","first-page":"95","DOI":"10.1002\/hec.4730030206","article-title":"Uncertainty in the economic evaluation of health care technologies: the role of sensitivity analysis","volume":"3","author":"Briggs, A.; Sculpher, M.J.; Buxton, M.J.","year":"1994","unstructured":"Briggs A, Sculpher MJ, Buxton MJ. Uncertainty in the economic evaluation of health care technologies: the role of sensitivity analysis. Health Econ 1994;3:95\u2013104.","journal-title":"Health Econ"},{"issue":"110","key":"ref_13","first-page":"11","article-title":"Two years of PKU testing in California. The role of the laboratory","volume":"1969","author":"Cunningham, G.C.","unstructured":"Cunningham GC. Two years of PKU testing in California. The role of the laboratory. 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New York: Springer-Verlag, 1980:7\u201318."},{"key":"ref_20","doi-asserted-by":"crossref","first-page":"33","DOI":"10.1177\/0272989X8200200107","article-title":"Health economic analysis of the Swedish neonatal metabolic screening programme: a method of optimizing routines","volume":"2","author":"Alm, J.; Larsson, A.; Rosenqvist, U.","year":"1982","unstructured":"Alm J, Larsson A, Rosenqvist U. Health economic analysis of the Swedish neonatal metabolic screening programme: a method of optimizing routines. Med Decis Making 1982;2: 33\u201345.","journal-title":"Med Decis Making"},{"key":"ref_21","first-page":"459","article-title":"Cost-benefit analysis of the detection and treatment of phenylketonuria (PKU) in newly-born babies, in Belgium","author":"Goss, S.","year":"1983","unstructured":"Goss S. Cost-benefit analysis of the detection and treatment of phenylketonuria (PKU) in newly-born babies, in Belgium. Cahiers Economique de Bruxelles 1983;459\u201368.","journal-title":"Cahiers Economique de Bruxelles"},{"key":"ref_22","first-page":"1","article-title":"The costs and benefits of screening for PKU in Wisconsin","volume":"31","author":"Barden, H.S.; Kessel, R.; Schuett, V.E.","year":"1984","unstructured":"Barden HS, Kessel R, Schuett VE. The costs and benefits of screening for PKU in Wisconsin. Soc Biol 1984;31:1\u201317.","journal-title":"Soc Biol"},{"key":"ref_23","doi-asserted-by":"crossref","first-page":"633","DOI":"10.1007\/BF01797933","article-title":"Economic evaluation of cost-benefit ratio of neonatal screening procedure for phenylketonuria and hypothyroidism","volume":"14","author":"Dhondt, J.-L.; Farriaux, J.-P.; Sailly, J.-C.","year":"1991","unstructured":"Dhondt J-L, Farriaux J-P, Sailly J-C, et al. Economic evaluation of cost-benefit ratio of neonatal screening procedure for phenylketonuria and hypothyroidism. J Inherit Metab Dis 1991;14:633\u20139.","journal-title":"J Inherit Metab Dis"},{"key":"ref_24","doi-asserted-by":"crossref","first-page":"382","DOI":"10.1017\/S0266462300006620","article-title":"Health economic analysis of the neonatal screening program in Japan","volume":"10","author":"Hisashige, A.","year":"1994","unstructured":"Hisashige A. Health economic analysis of the neonatal screening program in Japan. Int J Technol Assess Health Care 1994;10:382\u201391.","journal-title":"Int J Technol Assess Health Care"},{"key":"ref_25","doi-asserted-by":"crossref","first-page":"238","DOI":"10.7326\/0003-4819-116-3-238","article-title":"Cost-eVectiveness and cost-benefit analyses in the medical literature. Are the methods being used correctly?","volume":"116","author":"Udvarhelyi, I.S.; Colditz, G.A.; Rai, A.","year":"1992","unstructured":"Udvarhelyi IS, Colditz GA, Rai A, et al. Cost-eVectiveness and cost-benefit analyses in the medical literature. Are the methods being used correctly? Ann Intern Med 1992;116: 238\u201344.","journal-title":"Ann Intern Med"},{"key":"ref_26","doi-asserted-by":"crossref","first-page":"161","DOI":"10.1016\/0277-9536(92)90163-K","article-title":"Economic appraisal of asthma and COPD care: a literature review 1980\u201391","volume":"35","author":"MP, Rutten-van Molken; van Doorslaer EK; FF, Rutten","year":"1992","unstructured":"Rutten-van Molken MP, van Doorslaer EK, Rutten FF. Economic appraisal of asthma and COPD care: a literature review 1980\u201391. Soc Sci Med 1992;35:161\u201375.","journal-title":"Soc Sci Med"},{"key":"ref_27","doi-asserted-by":"crossref","first-page":"161","DOI":"10.1002\/(SICI)1099-1050(199703)6:2<161::AID-HEC258>3.0.CO;2-I","article-title":"Economic evaluation of mental health care interventions. A review","volume":"6","author":"Evers, S.M.A.A.; Van Wijk, A.S.; Ament, A.J.H.A.","year":"1997","unstructured":"Evers SMAA, Van Wijk AS, Ament AJHA. Economic evaluation of mental health care interventions. A review. Health Econ 1997;6:161\u201377.","journal-title":"Health Econ"},{"key":"ref_28","doi-asserted-by":"crossref","first-page":"25","DOI":"10.1002\/hec.4730030105","article-title":"Is vaccination against hepatitis B eYcient? A review of world literature","volume":"3","author":"JeVerson, T.; Demicheli, V.","year":"1994","unstructured":"JeVerson T, Demicheli V. Is vaccination against hepatitis B eYcient? A review of world literature. Health Econ 1994;3: 25\u201337.","journal-title":"Health Econ"},{"key":"ref_29","doi-asserted-by":"crossref","first-page":"85","DOI":"10.1002\/hec.4730040202","article-title":"Reporting guidelines for economic studies","volume":"4","author":"Mason, J.; Drummond, M.F.","year":"1995","unstructured":"Mason J, Drummond MF. Reporting guidelines for economic studies. Health Econ 1995;4:85\u201394.","journal-title":"Health Econ"},{"key":"ref_30","doi-asserted-by":"crossref","first-page":"355","DOI":"10.1002\/hec.4730040502","article-title":"Sensitivity analysis in economic evaluation: a review of published studies","volume":"4","author":"Briggs, A.; Sculpher, M.J.","year":"1995","unstructured":"Briggs A, Sculpher MJ. Sensitivity analysis in economic evaluation: a review of published studies. Health Econ 1995;4:355\u201371.","journal-title":"Health Econ"},{"key":"ref_31","doi-asserted-by":"crossref","first-page":"721","DOI":"10.1017\/S0266462300011004","article-title":"Costing methods in the Canadian literature on the economic evaluation of health care: A survey and assessment","volume":"12","author":"Jacobs, P.; Bachynsky, J.","year":"1996","unstructured":"Jacobs P, Bachynsky J. Costing methods in the Canadian literature on the economic evaluation of health care: A survey and assessment. Int J Technol Assess Health Care 1996;12: 721\u201334.","journal-title":"Int J Technol Assess Health Care"},{"key":"ref_32","doi-asserted-by":"crossref","first-page":"1172","DOI":"10.1001\/jama.1996.03540140060028","article-title":"The role of cost-eVectiveness analysis in health and medicine","volume":"276","author":"Russell, L.B.; Gold, M.R.; Siegel, J.E.","year":"1996","unstructured":"Russell LB, Gold MR, Siegel JE, et al. The role of cost-eVectiveness analysis in health and medicine. JAMA 1996;276:1172\u20137.","journal-title":"JAMA"},{"key":"ref_33","article-title":"Centre for Reviews and Dissemination. Making costeVectiveness information accessible: the NHS economic evaluation database project. CRD guidance for reporting critical summaries of economic evaluations. CRD Report Number 6","year":"1996","unstructured":"Centre for Reviews and Dissemination. Making costeVectiveness information accessible: the NHS economic evaluation database project. CRD guidance for reporting critical summaries of economic evaluations. CRD Report Number 6. York: NHS Centre for Reviews and Dissemination, University of York, 1996."},{"key":"ref_34","doi-asserted-by":"crossref","first-page":"619","DOI":"10.1111\/j.1651-2227.1986.tb10261.x","article-title":"Children with inborn errors of phenylalanine metabolism: prognosis and phenylalanine tolerance","volume":"75","author":"Alm, J.; Bodegard, G.; Larsson, A.","year":"1986","unstructured":"Alm J, Bodegard G, Larsson A, et al. Children with inborn errors of phenylalanine metabolism: prognosis and phenylalanine tolerance. Acta Paediatr Scand 1986;75:619\u201325.","journal-title":"Acta Paediatr Scand"},{"key":"ref_35","doi-asserted-by":"crossref","first-page":"345","DOI":"10.1093\/clinchem\/42.3.345","article-title":"Newborn screening by tandem mass spectrometry (MS-MS)","volume":"42","author":"Sweetman, L.","year":"1996","unstructured":"Sweetman L. Newborn screening by tandem mass spectrometry (MS-MS). Clin Chem 1996;42:345\u20136.","journal-title":"Clin Chem"},{"key":"ref_36","doi-asserted-by":"crossref","first-page":"66","DOI":"10.1093\/clinchem\/39.1.66","article-title":"Rapid diagnosis of phenylketonuria by quantitative analysis for phenylalanine and tyrosine in neonatal blood spots by tandem mass spectrometry","volume":"39","author":"Chace, D.H.; Millington, D.S.; Terada, N.","year":"1993","unstructured":"Chace DH, Millington DS, Terada N, et al. Rapid diagnosis of phenylketonuria by quantitative analysis for phenylalanine and tyrosine in neonatal blood spots by tandem mass spectrometry. Clin Chem 1993;39:66\u201371.","journal-title":"Clin Chem"},{"key":"ref_37","first-page":"525","article-title":"Phenylketonuria screening with a fluorometric microplate assay","volume":"32","author":"Lubenow, N.; Diepenbrock, F.; Schickling, H.","year":"1994","unstructured":"Lubenow N, Diepenbrock F, Schickling H, et al. Phenylketonuria screening with a fluorometric microplate assay. Eur J Clin Chem Clin Biochem 1994;32:525\u20138.","journal-title":"Eur J Clin Chem Clin Biochem"},{"key":"ref_38","first-page":"306","article-title":"Causes of death of institutionalized phenylketonuric (PKU) patients - a national survey","volume":"53","author":"Fisch, R.; Bilek, O.M.; Bruhl, H.H.","year":"1976","unstructured":"Fisch R, Bilek OM, Bruhl HH. Causes of death of institutionalized phenylketonuric (PKU) patients - a national survey. Minn Med 1976;53:306\u20139.","journal-title":"Minn Med"},{"key":"ref_39","article-title":"Unit costs of health and social care 1996","author":"Netten, A.; Dennett, J.","year":"1996","unstructured":"Netten A, Dennett J. Unit costs of health and social care 1996. Canterbury: PSSRU University of Kent, 1996."},{"key":"ref_40","article-title":"Living in Britain: Results from the 1994 General Household Survey. OYce of Population Censuses and Surveys, Social Survey Division","author":"Bennett, N.; Jarvis, L.; Rowlands, O.","year":"1996","unstructured":"Bennett N, Jarvis L, Rowlands O, et al. Living in Britain: Results from the 1994 General Household Survey. OYce of Population Censuses and Surveys, Social Survey Division. 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