Economic evaluation of tandem mass spectrometry screening in California Feuchtbaum L, Cunningham G

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1 Economic evaluation of tandem mass spectrometry screening in California Feuchtbaum L, Cunningham G Record Status This is a critical abstract of an economic evaluation that meets the criteria for inclusion on NHS EED. Each abstract contains a brief summary of the methods, the results and conclusions followed by a detailed critical assessment on the reliability of the study and the conclusions drawn. Health technology The authors investigated the use of pilot tandem mass spectrometry (MS/MS) screening in combination with phenylketonuria screening. This screening intervention was compared with no MS/MS screening, in which only phenylketonuria screening was offered to newborn infants. Type of intervention Screening. Economic study type Cost-effectiveness analysis, cost-utility analysis and cost-benefit analysis. Study population The study population comprised a hypothetical cohort of 540,000 newborns in California. Setting The study setting was the community. The economic study was carried out in California, USA. Dates to which data relate The effectiveness data were derived from the experience of the California screening programme, which was conducted between 7 January 2002 and 13 June Effectiveness evidence was also derived from studies published between 2000 and The costs were adjusted to year Source of effectiveness data The effectiveness data were derived from the experience of the California screening programme. In cases where effectiveness data could not be obtained from the literature, expert opinion was used. Modelling The authors reported that a model was used. The clinical outcome categories used were death, severe neurological impairment, mild neurological impairment, acute complications only and asymptomatic. No further details of the model were reported. After assessing the effectiveness evidence, a base-case scenario of outcomes expected with screening was developed with input from a panel of metabolic specialists in California. A lifetime horizon was used. Outcomes assessed in the review The outcomes assessed in the review were the distribution of clinical outcome categories and the incidence of disorders detectable by MS/MS in the screened population. Page: 1 / 5

2 Study designs and other criteria for inclusion in the review Sources searched to identify primary studies Criteria used to ensure the validity of primary studies Methods used to judge relevance and validity, and for extracting data Number of primary studies included Two primary studies were included in the analysis. The authors did not cite a reference for the data they used from the California screening programme. Methods of combining primary studies Due to the limited number of studies with available data for each model probability, the authors did not combine the results of the individual primary studies. Investigation of differences between primary studies Not relevant. Results of the review The overall incidence of disorders detectable by MS/MS was 1 case per 6,700 newborns screened. The distribution of clinical outcome categories was not reported. Measure of benefits used in the economic analysis The measures of benefits used were monetary benefits, cases detected, life-years saved (LYS) and quality-adjusted lifeyears (QALYs). The total benefit to save one life was derived from a report by the US Environmental Protection Agency, which valued a life at $5.7 million in 2004 dollars. The utility values were derived from a review of published data. Direct costs The direct costs to the third-party payer were included in the analysis. The authors included the incremental costs associated with implementing MS/MS screening in the basic newborn screening programme. These covered personnel and administration, equipment, supplies, laboratory contacts and follow-up centres. The authors also included the total lifetime medical care costs for patients with neurological impairment or acute complications. The lifetime costs were derived from estimates published by the US Centers for Disease Control and Prevention for the lifetime costs for a person with mental retardation. The lifetime costs of moderate developmental delay, which were derived from a published study, were used as a proxy for the lifetime medical care costs for a moderately affected individual with an MS/MS detectable disorder. The authors did not report if these two studies had discounted future costs. The study reported the incremental costs. An inflation rate of 3% was applied to adjust the costs to year Page: 2 / 5

3 Statistical analysis of costs The costs were treated as point estimates (i.e. the data were deterministic). Indirect Costs The indirect costs were not included. Currency US dollars ($). Sensitivity analysis The authors conducted a series of one-way sensitivity analyses by varying the lifetime medical care costs avoided through screening, the lifetime medical care costs for an MS/MS detectable case, and the value per life saved. A second analysis was conducted in which the base-case clinical outcomes expected without screening were held constant and the expected costs were calculated using best- and worst-case scenarios. Estimated benefits used in the economic analysis With no MS/MS available, there would be a total of 10 deaths, 10 cases of severe neurological impairment, 10 cases of mild/moderate impairment, 25 cases of acute complications and 28 asymptomatic cases. With MS/MS screening, there would be a total of 2 deaths, 3 cases of severe neurological impairment, 3 cases of mild/moderate impairment, 47 cases of acute complications and 28 asymptomatic cases. The value of lives saved with MS/MS screening (i.e. 8 lives) was $45.6 million. The QALYs saved with MS/MS screening were 949. Cost results The incremental costs of the MS/MS screening programme were $5,664,500. The medical care costs avoided using MS/MS screening were $7,209,152. Synthesis of costs and benefits The costs and benefits were combined in three ways: using cost-effectiveness (i.e. the additional cost per case detected or per LYS); using an incremental cost-utility ratio (i.e. the additional cost per QALY gained); and using a cost-benefit analysis, in which the authors subtracted the net costs from the net benefits and also estimated the benefit-cost ratio. The incremental cost per case detected when MS/MS screening was compared with no MS/MS screening was $68,247. The incremental cost per LYS was $708,063. The additional savings per QALY gained when MS/MS screening was compared with no MS/MS screening were $1,628. The net incremental benefit of MS/MS screening over no MS/MS screening was $47,144,652, with a benefit-cost ratio of $9.32. Page: 3 / 5

4 The results of the sensitivity analysis showed that the benefit-cost ratio ranged from $11.67 with the best set of assumptions to $4.34 with the worst set of assumptions. In turn, under the worst-case scenario, the cost per QALY gained was $19,129. Authors' conclusions The benefits of tandem mass spectrometry (MS/MS) screening outweighed the costs. The net benefits produced were robust to various scenarios with conservative underlying assumptions. CRD COMMENTARY - Selection of comparators A justification was given for using a phenylketonuria screening programme with no MS/MS as the comparator, as this represented current practice in California. You should decide if the comparator used is current practice in your own setting. Validity of estimate of measure of effectiveness The authors did not report whether a systematic review of the literature was undertaken to identify relevant research and minimise biases. The authors used data from the experiences of the California MS/MS screening programme, but provided only brief details of this programme and did not report the methods used to derive the estimates of effectiveness. In cases where effectiveness data could not be obtained from the literature, such estimates were based on expert opinion. However, the authors did not carry out a sensitivity analysis on the effectiveness data. This may limit both the internal validity of the study and the generalisability of the results. Validity of estimate of measure of benefit The measures of benefits were derived by combining data from the California MS/MS screening programme and monetary and utility values derived from the literature. The use of LYS and QALYs gained permits comparison with the results of other studies. However, the method used to derive the utility values was not reported. Again, it would have been helpful had the authors provided more details of the California MS/MS programme, to aid an assessment of the validity of the results. Validity of estimate of costs All the cost categories relevant to the third-party payer perspective adopted were included in the analysis. No major relevant costs appear to have been omitted. The costs and the quantities were not reported separately, which will limit the generalisability of the results. The authors derived costs from a variety of sources. The screening programme costs were derived from cost data from the California screening programme, whereas the lifetime medical costs were derived from the literature. Appropriate one-way sensitivity analyses of the costs were performed. The authors did not report whether the studies used to derive the lifetime costs had discounted future costs. All costs were appropriately inflated to year The price year was reported, which will aid any future inflation exercises. Other issues The authors compared their results with those of the MS/MS screening programme in Wisconsin, in which MS/MS screening was also found to be cost-effective. The issue of generalisability to other settings was not addressed. The authors do not appear to have reported their results selectively and their conclusions reflected the scope of their analysis. The authors reported no further limitations to their study. Implications of the study Based on the results from their study, the authors would appear to recommend the implementation of an MS/MS screening programme in California. Page: 4 / 5

5 Powered by TCPDF ( Source of funding Funded by the Health Resources and Services Administration. Bibliographic details Feuchtbaum L, Cunningham G. Economic evaluation of tandem mass spectrometry screening in California. Pediatrics 2006; 117(5 Part 2): S280-S286 PubMedID DOI /peds G Other publications of related interest Because readers are likely to encounter and assess individual publications, NHS EED abstracts reflect the original publication as it is written, as a stand-alone paper. Where NHS EED abstractors are able to identify positively that a publication is significantly linked to or informed by other publications, these will be referenced in the text of the abstract and their bibliographic details recorded here for information. Insinga RP, Laessig RH, Hoffman GL. Newborn screening with tandem mass spectrometry: examining its costeffectiveness in the Wisconsin Newborn Screening Panel. J Pediatr 2002:141; Schulze A, Lindner M, Kohlmuller D, et al. Expanded newborn screening for inborn errors of metabolism by electrospray ionization-tandem mass spectrometry: results, outcome, and implications. Pediatrics 2003;111: Indexing Status Subject indexing assigned by NLM MeSH California; Cost-Benefit Analysis; Humans; Infant, Newborn; Mass Spectrometry /economics; Neonatal Screening /economics; Pilot Projects; Program Evaluation AccessionNumber Date bibliographic record published 28/02/2007 Date abstract record published 28/02/2007 Page: 5 / 5