Efficiency of alternative policy options for screening for developmental dysplasia of the hip in the United Kingdom

Aims: To assess, using a decision model, the efficiency of ultrasound based and clinical screening strategies for developmental dysplasia of the hip. Methods: The additional cost per additional favourable outcome was compared for the following strategies: clinical screening alone using the Ortolani and Barlow tests; addition of static and dynamic ultrasound examination of the hips of all infants (universal ultrasound) or restricted to infants with defined risk factors (selective ultrasound); "no screening" (that is, clinical diagnosis only). Results: Ultrasound based screening strategies are predicted to be more effective but more costly than clinical screening or no screening. Estimated total costs per 100 000 live births are approximately £4 million for universal ultrasound, £3 million for selective ultrasound, £1 million for clinical screening alone, and £0.4 million for no screening. The relative efficiency of selective ultrasound and clinical screening is poorly differentiated, and depends on how infants are selected for ultrasound as well as the expertise of clinical screening examiners. If training costs less than £20 per child screened, clinical screening alone would be more efficient than selective ultrasound. Relative to no screening, each of the 16 additional favourable outcomes achieved as a result of selective ultrasound costs approximately £0.2 million, while each of the five favourable outcomes achieved through universal ultrasound screening, over and above selective ultrasound, costs approximately £0.3 million. Conclusions: Policy choice depends on values attached to the different outcomes, willingness to pay to achieve these and total budget.     

Performance, treatment pathways, and effects of alternative policy options for screening for developmental dysplasia of the hip in the United Kingdom.

AIMS: To compare, using a decision model, performance, treatment pathways and effects of different newborn screening strategies for developmental hip dysplasia with no screening. METHODS: Detection rate, radiological absence of subluxation at skeletal maturity and avascular necrosis of the femoral head, as favourable and unfavourable treatment outcomes respectively, were compared for the following strategies: clinical screening alone using the Ortolani and Barlow tests; the addition of static and dynamic ultrasound examination of the hips of all infants (universal ultrasound) or restricted to infants with defined risk factors (selective ultrasound); "no screening" (that is, clinical diagnosis only). RESULTS: Universal or selective ultrasound detects more more affected children (76% and 60% respectively) than clinical screening alone (35%), results in a higher proportion of affected children with favourable treatment outcomes (92% and 88% respectively) than clinical screening alone (78%) or no screening (75%), and the highest proportion of these achieved without recourse to surgery (64% and 79% respectively) compared with clinical screening alone (18%). However, ultrasound based strategies are also associated with the highest number of unfavourable treatment outcomes arising in unaffected children treated following a false positive screening result. The detection rate of clinical screening alone becomes similar to that reported for universal ultrasound when based on studies using experienced examiners (80%) rather than junior medical staff (35%). CONCLUSION: From the largely observational data available, ultrasound based screening strategies appear to be most sensitive and effective but are associated with the greatest risk of potential adverse iatrogenic effects arising in unaffected children.     

Performance,treatment pathways,and effects of alternative policy options for screening for developmental dysplasia of the hip in the United Kingdom

Aims: To compare, using a decision model, performance, treatment pathways and effects of different newborn screening strategies for developmental hip dysplasia with no screening. Methods: Detection rate, radiological absence of subluxation at skeletal maturity and avascular necrosis of the femoral head, as favourable and unfavourable treatment outcomes respectively, were compared for the following strategies: clinical screening alone using the Ortolani and Barlow tests; the addition of static and dynamic ultrasound examination of the hips of all infants (universal ultrasound) or restricted to infants with defined risk factors (selective ultrasound); "no screening" (that is, clinical diagnosis only). Results: Universal or selective ultrasound detects more more affected children (76% and 60% respectively) than clinical screening alone (35%), results in a higher proportion of affected children with favourable treatment outcomes (92% and 88% respectively) than clinical screening alone (78%) or no screening (75%), and the highest proportion of these achieved without recourse to surgery (64% and 79% respectively) compared with clinical screening alone (18%). However, ultrasound based strategies are also associated with the highest number of unfavourable treatment outcomes arising in unaffected children treated following a false positive screening result. The detection rate of clinical screening alone becomes similar to that reported for universal ultrasound when based on studies using experienced examiners (80%) rather than junior medical staff (35%). Conclusion: From the largely observational data available, ultrasound based screening strategies appear to be most sensitive and effective but are associated with the greatest risk of potential adverse iatrogenic effects arising in unaffected children.     

Universal versus targeted screening of infants for sickle cell disease: a cost-effectiveness analysis

OBJECTIVE: To compare the health outcomes, costs, and incremental cost-effectiveness of universal neonatal screening for sickle cell disease (SCD) with screening targeted to African Americans. STUDY DESIGN: A cost-effectiveness analysis was done by using a Markov simulation model that considered the costs and outcomes associated with the prevention and treatment of sepsis in those with sickle cell anemia and sickle beta(0)-thalassemia. Three strategies were compared: (1) no screening, (2) targeted screening of African Americans, and (3) universal screening for SCD. RESULTS: In the base case analysis, targeted screening of African Americans compared with no screening cost $6709 per additional year of life saved, and universal screening compared with targeted screening cost $30,760 per additional year of life saved. In a sensitivity analysis, the cost per additional year of life saved with universal screening compared with targeted screening was positively correlated with the delivery rate of targeted screening and was inversely related to the proportion of African Americans in the population. CONCLUSIONS: Targeted screening of African American newborns for SCD compared with no screening is always cost-effective. Universal screening compared with targeted screening always identifies more infants with disease, prevents more deaths, and is cost-effective given certain delivery rates for targeted screening and proportions of African Americans in the population.     

A cost-effectiveness evaluation of newborn hemoglobinopathy screening from the perspective of state health care systems

Objective: To determine the most cost-effective strategy for newborn hemoglobinopathy screening from the perspective of state health care systems. Study design: Using Alaska as an example, we used decision analysis to compare a policy of no screening to universal or targeted screening with selective follow-up only of infants who are homozygous or compound heterozygous for an abnormal hemoglobin variant and to universal or targeted screening with complete follow-up, including follow-up of infants with clinically insignificant traits. Probabilities and costs were varied over values that might be expected for other states. Results: Among the selective follow-up options, targeted screening would be the most cost-effective strategy for Alaska at a cost of $206 192 per death averted; by contrast, universal screening would prevent 50% more deaths at an incremental cost of $2 040 000 per death averted. Universal would be more cost-effective than targeted screening for several scenarios expected to occur in other states, including a high sickle cell disease prevalence, a low screening test cost, and a high cost per screen associated with racial targeting. Among the complete follow-up options, both targeted and universal screening would cost at least $200 000 per death averted over the range of all variables tested during sensitivity analysis; the incremental cost of universal versus targeted screening would be at least $600 000 per death averted. Conclusions: Our data suggest each state should determine the most cost-effective option based on state-specific values for sickle cell disease prevalence, test costs and racial targeting costs.     

The potential impact on costs and staffing of introducing clinical networks and British Association of Perinatal Medicine standards to the delivery of neonatal care

OBJECTIVE: To produce models to estimate the impact of introducing clinical networks and the 2001 BAPM standards to the delivery of neonatal care. DESIGN: Prospective observational study using a geographically defined population and data collected by questionnaire on staffing levels and cot availability. SETTING: Trent Health Region UK. SUBJECTS: All infants born to Trent resident mothers at or before 32 weeks gestation between 1 January 1998 and 31 December 1999. Staffing numbers and cot availability for neonatal care in 2001. METHODS: A modelling exercise was carried out using information for all neonatal admissions for Trent resident infants. Three models were investigated: (a). the current care provision; (b). a network where three lead centres provided the intensive care for the region and the remaining units provided either high dependency or special care alone; (c). a network where six lead centres provided the intensive care for the region and the remaining units provided either high dependency or special care alone. Overall costings, staffing levels, and cot requirements were calculated for each model. Data on staffing levels and cot availability were used to calculate current care provision costings. RESULTS: The current cost of running the service is approximately pound 33.35 million, although a proportion of nursing posts are currently unfilled. Estimates for the introduction of a three centre model meeting BAPM 2001 standards range from pound 37.31 to pound 43.40 million. Equivalent figures for the six centre model were: pound 36.32 to pound 42.62 million. Approximately 370 and 230 babies a year would be involved in transfer in the three and six centre models respectively. This is in contrast with 374 and 368 urgent transfers that actually took place in 1998 and 1999 respectively. CONCLUSION: The costs associated with the introduction of managed clinical networks and meeting BAPM standards of care are not excessive, especially when considered against the likely implementation timetable of perhaps 7-10 years. Attracting and retaining sufficient staff will pose the major challenge.     

Cost-effectiveness and implications of newborn screening for prolongation of QT interval for the prevention of sudden infant death syndrome

OBJECTIVE: To determine the cost-effectiveness of universal and high-risk neonatal electrocardiographic (ECG) screening for QT prolongation as a predictor of sudden infant death syndrome (SIDS) risk in a theoretical group of neonates.Study design: Incremental cost-effectiveness analysis with decision analytic modeling. A hypothetical cohort of healthy, term infants was modeled, comparing options of no screening, high-risk neonate screening, and universal screening. The high-risk strategy is speculative, because no currently accepted methodology is known for identifying infants at high risk for SIDS. Given the uncertain mechanisms of association between prolonged corrected QT interval (QTc) and SIDS, analyses were repeated under different assumptions. Sensitivity analyses were also performed on all input variables for both costs and effectiveness. RESULTS: Under the assumption that neonatal electrocardiographic screening detects long QT syndrome responsive to conventional therapy, the cost-effectiveness of high-risk screening was $3403 per life year gained, whereas universal screening cost $18,465 per additional life year gained. However, if the effectiveness of SIDS therapy falls below 10%, the cost-effectiveness deteriorates to $28,376 per life year saved for the high-risk strategy and $118,900 for universal screening. The analyses were robust to a broad array of sensitivity analyses. CONCLUSIONS: The acceptability of the cost-effectiveness of neonatal electrocardiographic screening is heavily dependent on the pathophysiologic mechanism of SIDS and on the efficacy of monitoring and antiarrhythmic treatment. The nature of this association must be elucidated before routine neonatal electrocardiographic screening is warranted.     

A cost-effectiveness analysis of newborn hearing screening strategies

CONTEXT: Congenital hearing loss affects between 1 and 3 out of every 1,000 children. Screening of all neonates has been made possible by the development of portable automated devices. Universal screening is a 2-stage screening process using automated transient-evoked otoacoustic emissions, followed when indicated by automated auditory brain response testing. Targeted screening reserves the 2-stage screening process for those infants at risk for congenital hearing loss. OBJECTIVE: To compare the expected costs and benefits of targeted screening with universal screening for the detection of significant bilateral congenital hearing loss. DESIGN: Cost-effectiveness analysis from the health care system perspective. including costs directly related to screening and initial follow-up evaluation. MAIN OUTCOME MEASURES: Number of cases identified, number of false positives, and cost per case. RESULTS: For every 100,000 newborns screened, universal screening detects 86 of 110 cases of congenital hearing loss, at a cost of $11,650 per case identified. Targeted screening identifies 51 of 110 cases, at $3,120 per case identified. Universal screening produces 320 false-positive results, 304 more than targeted screening. Switching to universal screening from targeted screening would cost an additional $23, 930 for each extra case detected. CONCLUSIONS: Universal screening detects more cases of congenital hearing loss, at the expense of both greater cost and more false-positive screening results. Little is known about the negative impact of false-positive screening and about the benefits of early intervention for congenital hearing loss. Those who advocate adoption of universal screening should be aware not only of the direct costs of universal screening, but of the indirect costs and strategies to increase the benefits of screening.     

Potential costs and benefits of newborn screening for severe combined immunodeficiency

OBJECTIVE: Severe combined immunodeficiency (SCID) is a rare, treatable disorder of the immune system. The incidence is unknown but may be more common than published estimates because infants frequently die of infection before diagnosis. SCID is a candidate for universal newborn screening, so there is a need to determine under which circumstances screening would be cost-effective. STUDY DESIGN: We assumed a screening program for SCID would use T-cell lymphopenia as the screening criterion and performed a cost-utility analysis comparing universal screening with screening only those with a family history of SCID. RESULTS: Assuming society is willing to pay $50,000 for every quality-adjusted life-year saved, a SCID screening test that cost less than $5 with a false-negative rate of 0.9% and a false-positive rate of 0.4% would be considered cost-effective. A nationwide screening program would cost an additional $23.9 million per year for screening costs but would result in 760 years of life saved per year of screening. The cost to detect 1 case of SCID would be $485,000. CONCLUSION: SCID screening could result in a large benefit to detected individuals, making screening relatively cost-effective in spite of the low incidence of the disease. However, an adequate test is critical to cost-effectiveness.     

Evidence for changing guidelines for routine screening for retinopathy of prematurity

CONTEXT: Existing guidelines recommended by the Canadian Pediatric Society (CPS) and American Academy of Pediatrics (AAP) for routine screening for retinopathy of prematurity (ROP) remain controversial. OBJECTIVE: To determine whether current guidelines for routine screening for ROP should be changed. DESIGN: We examined data that were collected as part of a larger study of 14 neonatal intensive care units (NICUs) in Canada. We examined the effect of strategies using different birth weight (BW) and gestational age (GA) criteria for routine ROP screening, and performed a cost-effectiveness analysis. SETTING: The 14 NICUs (except one) are regional tertiary level referral centres serving geographic regions of Canada, and include approximately 60% of all tertiary-level NICU beds in Canada. PATIENTS: This large cohort included all 16 424 infants admitted to 14 Canadian NICUs from January 8, 1996, to October 31, 1997. INTERVENTIONS: None. MAIN OUTCOME MEASURE: Treatment for ROP. RESULTS: The most cost-effective strategy was to routinely screen only infants having a BW of 1200 g or less. This included all infants treated for ROP (except 1 outlier at 32 weeks GA and 1785 g BW), at a marginal cost per additional person with improved vision of $513 081 for screening patients between 28 weeks GA and 1200 g BW, compared with $1 800 039 and $2 075 874 for using the current AAP and CPS guidelines, respectively (cryotherapy outcomes). Results for laser therapy were similar, but costs were slightly lower. This strategy reduced the number of infants screened under the current CPS guidelines by 46%. CONCLUSION: Screening only infants having a BW of 1200 g or less is the most cost-effective strategy for routine ROP screening.     

Respiratory syncytial virus infection in high risk infants and the potential impact of prophylaxis in a United Kingdom cohort.

BACKGROUND: Bronchiolitis caused by respiratory syncytial virus (RSV) is an important cause of morbidity in ex-premature infants. In a randomised placebo controlled trial monoclonal antibody prophylaxis showed a 55% reduction in relative risk of hospital admission for these high risk infants, against a background incidence of 10.6 admissions per 100 high risk infants. AIMS: To follow a cohort of high risk infants in order to assess hospitalisation rate from RSV and the potential impact of prophylaxis for these patients in a UK local health authority. METHODS: A cohort of high risk infants from a local health authority were followed over the 1998/99 and 1999/2000 RSV seasons. The high risk population was defined as infants who, at the beginning of the seasons studied, were: (1) under 6 months old and born prior to 36 weeks gestation with no domiciliary oxygen requirement; or (2) under 24 months of age and discharged home in supplemental oxygen. All admissions with bronchiolitis during the season were identified. RESULTS: A total of 370 high risk infants were identified for the 1998/99 season and 286 for the following year. Over the two years there were 68 admissions. Significantly more admissions occurred from group 2 infants. RSV was identified in 27 cases (four admissions per hundred high risk infants). Prophylaxis may have saved up to pound 195,134 in hospital costs over the two years, but would have cost pound 1.1 million in drug acquisition costs. CONCLUSIONS: Careful consideration of risk factors is needed when selecting infants for RSV prophylaxis.     

Cost effectiveness analysis of neonatal extracorporeal membrane oxygenation based on four year results from the UK Collaborative ECMO Trial

OBJECTIVE: To assess the cost effectiveness of extracorporeal membrane oxygenation (ECMO) for mature newborn infants with severe respiratory failure over a four year time span. DESIGN: Cost effectiveness analysis based on a randomised controlled trial in which infants were individually allocated to ECMO (intervention) or conventional management (control) and then followed up to 4 years of age. SETTING: Infants were recruited from 55 approved recruiting hospitals throughout the United Kingdom. Infants allocated to ECMO were transferred to one of five specialist regional centres. Follow up of surviving infants was performed in the community. SUBJECTS: A total of 185 mature (gestational age at birth >or= 35 weeks, birth weight >or= 2000 g) newborn infants with severe respiratory failure (oxygenation index >or= 40). MAIN OUTCOME MEASURES: Incremental cost per additional life year gained; incremental cost per additional disability-free life year gained. RESULTS: Over four years, the policy of neonatal ECMO was effective at reducing known death or severe disability (relative risk = 0.64; 95% confidence interval 0.47 to 0.86; p = 0.004). After adjustment for censoring and discounting at 6%, the mean additional health service cost of neonatal ECMO was pound 17367 (95% confidence interval pound 12072 to pound 22224) per infant ( pound UK, 2001 prices). Over four years, the incremental cost of neonatal ECMO was pound 16707 ( pound 9828 to pound 37924) per life year gained and pound 24775 ( pound 13106 to pound 69690) per disability-free life year gained. These results remained robust after variations in the values of key variables performed as part of a sensitivity analysis. CONCLUSIONS: The study provides rigorous evidence of the cost effectiveness of ECMO at four years for mature infants with severe respiratory failure.     

Selective screening for neonatal galactosemia: an alternative approach

No universal consensus exists for population-based neonatal screening for galactosemia. In our institution, selective screening for classical galactosemia is carried out on infants under 2 wk of age and those with symptoms suggestive of this disorder. Eighteen cases were diagnosed from 25,099 tests done; 17 were symptomatic at the time of diagnosis. CONCLUSION: We suggest that improved clinical vigilance and selective screening would identify most infants with severe galactosemia as early as a population-based program.     

Economic evaluation of strategies for managing crying and sleeping problems.

AIMS: To estimate the financial cost to the NHS of infant crying and sleeping problems in the first 12 weeks of age and to assess the cost effectiveness of behavioural and educational interventions aimed at reducing infant crying and sleeping problems relative to usual services. METHODS: A cost burden analysis and cost effectiveness analysis were conducted using data from the Crying Or Sleeping Infants (COSI) Study, a three armed prospective randomised controlled trial that randomly allocated 610 mothers to a behavioural intervention (n = 205), an educational intervention (n = 202), or existing services (control, n = 203). Main outcome measures were annual total cost to the NHS of infant crying and sleeping problems in the first 12 weeks, and incremental cost per interruption free night gained for behavioural and educational interventions relative to control. RESULTS: The annual total cost to the NHS of infant crying and sleeping problems in the first 12 weeks was 65 pound sterling million (US$104 million). Incremental costs per interruption free night gained for the behavioural intervention relative to control were 0.56 pound sterling (US$0.92). For the educational intervention relative to control they were 4.13 pound sterling (US$6.80). CONCLUSIONS: The annual total cost to the NHS of infant crying and sleeping problems is substantial. In the cost effectiveness analysis, the behavioural intervention incurred a small additional cost and produced a small significant benefit at 11 and 12 weeks of age. The educational intervention incurred a small additional cost without producing a significant benefit.     

Hepatitis A vaccination options for Australia

The epidemiology of hepatitis A is changing, with an increasing proportion of the population becoming susceptible to infection. The burden of hepatitis A is comparable to that of other vaccine-preventable diseases for which new vaccines are available. Options for vaccination include selective programmes for high-risk groups, which could involve screening prior to vaccination, or universal programmes for infants and/or adolescents. Selective programmes have been shown to be highly cost-effective if well implemented, but there is evidence that they might be poorly implemented. If a universal vaccination programme were considered for Australia, an infant programme, with doses at 18 months and 2 years, possibly with an additional adolescent programme, would be the recommended option. Universal hepatitis A vaccination for infants and/or adolescents is of comparable cost-effectiveness compared with other preventive strategies, but needs to be considered in the context of competing vaccination options.     

State programs for universal newborn hearing screening

Published reports of several statewide and hospital-based systems for universal newborn hearing screening demonstrate that successful large-scale programs that appropriately identify infants with hearing loss in the earliest months of life can be developed. These programs are characterized by nursery-based screening rates of 95% or higher, referral rates of 6% or less, and reasonable per-infant costs. Less data are available regarding the outcome of these screening programs in ensuring confirmation of hearing loss by 3 months of age and initiation of intervention by 6 months of age. The results of the MDNC survey provide important information on the status of newborn hearing screening, audiologic assessment, and intervention services in 16 states. The survey reveals that hospitals have initiated universal newborn hearing screening programs using appropriate technology but that confirmation of hearing loss, fitting of amplification, and enrollment in early intervention are often delayed beyond the JCIH recommendations. Several factors might contribute to late confirmation of hearing loss and delayed amplification and intervention. First, as shown in the Colorado report, lack of a mandatory statewide system for tracking and reporting may delay transition of infants and families from screening to diagnosis, and diagnosis to intervention. In addition, many states lack a centralized system for reporting confirmed hearing loss. Successful statewide programs for universal newborn hearing screening, audiologic diagnosis, and early intervention depend on data-reporting strategies that facilitate transition of infants and families through a system of care. Second, lack of understanding about the urgent need for intervention in the earliest months of life may hinder referral to early intervention programs. Recent data from Colorado's universal newborn hearing screening program reveals that infants who are deaf or who have hearing losses achieve significantly better language development outcomes if intervention begins before age 6 months than infants whose intervention begins after 6 months of age. Hopefully, as these data become more widely available, the compelling need for early intervention will facilitate transition into these services. Although universal newborn hearing screening programs are increasing rapidly, states have not yet developed the coordinated systems for linking universal newborn hearing screening programs to audiologic diagnostic services and audiologic diagnostic services to early intervention programs. Key issues impeding development of these systems may be lack of tracking and reporting systems, lack of standardized guidelines for screening, diagnostic audiologic assessment, hearing aid fitting for very young infants, and lack of understanding about the compelling need for intervention in the earliest months of life. Development of complete systems of care must become a priority for universal newborn hearing screening to provide its ultimate benefit.     

NICU‐only versus universal screening for newborn hearing loss: Population audit

Aim: Targeted newborn hearing screening for infants in neonatal intensive care units (NICUs) may be considered when resources preclude universal newborn hearing screening (UNHS). However, process outcomes have not been compared between stand‐alone NICU hearing screening programs and NICU screening within a full UNHS program. Methods: Comparison of two consecutive hearing screening programs delivered under similar conditions in the four NICUs in Victoria, Australia. All NICU infants were eligible for pre‐discharge automated auditory brainstem response (AABR) hearing screening. Capture, referral and diagnostic data were collected for all NICU infants during the NICU‐only (April 2003–February 2005) and subsequent UNHS (April 2005–June 2006) programs. Results: 4704 eligible infants were admitted during the 23‐month NICU‐only period, and 3160 during the 15‐month UNHS period. Double AABR using ALGO 3i equipment was planned for both programs but, due to clinician concern about this high‐risk clinical population, the NICU‐only protocol was amended to single AABR using AccuScreen equipment. Capture rates were 71.1% (NICU‐only) vs. 95.4% (UNHS) (P < 0.001), successful follow‐up rates were 85.8% vs. 96% (P= 0.004), and mean corrected age at the first audiology appointment was 51.5 vs. 40.2 days (P= 0.05). Conclusions: NICU screening offered within a larger UNHS program outperformed the stand‐alone NICU hearing screening program on all measured parameters. Greater resourcing might address shortcomings of the stand‐alone program but would also reduce its potential savings. The high loss to follow‐up also argues against the often‐advocated approach of referring all NICU infants for diagnostic audiologic testing, bypassing hearing screening altogether.     

Costing model for neonatal screening and diagnosis of haemoglobinopathies

AIM: To compare the costs and cost effectiveness of universal and targeted screening for the haemoglobinopathies; to compare the cost of two laboratory methods; and to estimate the cost effectiveness of programmes at different levels of prevalence and mix of haemoglobinopathy traits. METHODS: A retrospective review of laboratory and follow up records to establish workload and costs, and estimation of costs in a range of circumstances was made in a haematology department and sickle cell and thalassaemia centre, providing antenatal and neonatal screening programmes in Inner London. The costs for 47,948 babies, screened during 1994, of whom 25 had clinically significant haemoglobinopathies and 704 had haemoglobinopathy traits, were retrospectively assessed. RESULTS: The average cost per baby tested (isoelectric focusing and high power liquid chromatography) was 3.51 Pounds /3.83 Pounds respectively; the cost per case of sickle cell disease identified (IEF/HPLC) was 6738 Pounds /7355 Pounds; the cost per trait identified (IEF/HPLC) was 234 Pounds /255 Pounds; the cost per extra case of SCD and trait identified by universal programme varied. CONCLUSIONS: IEF and HPLC are very similar in terms of average cost per test. At 16 traits/1000 and 0.5 SCD/1000 there was no significant identification cost difference between universal and targeted programmes. Below this prevalence, a targeted programme is cheaper but likely to miss cases of SCD. If targeted programmes were 90-99% effective, universal programmes would cease to be good value except at very high prevalence. Greater use of prenatal diagnosis, resulting in termination, and therefore fewer affected births, reduces the cost effectiveness of universal screening. Screening services should aim to cover a screened population which will generate a workload over 25,000 births a year, and preferably over 40,000.     

Newborn screening with tandem mass spectrometry: examining its cost-effectiveness in the Wisconsin Newborn Screening Panel

OBJECTIVE: To examine the cost-effectiveness of tandem mass spectrometry (MS/MS) in a neonatal screening panel for 14 fatty acid oxidation and organic acidemia disorders in the Wisconsin Newborn Screening Program. STUDY DESIGN: An incremental cost-effectiveness analysis with a hypothetical cohort of 100,000 infants was performed. A threshold of $50,000/QALY (quality-adjusted life-year) was used to determine whether screening for medium-chain acyl-CoA dehydrogenase deficiency (MCAD) alone is cost-effective or whether additional disorders would need to be incorporated into the analysis to arrive at a conclusion regarding the overall cost-effectiveness of MS/MS. RESULTS: Under conservative assumptions, screening for MCAD alone yields an incremental cost-effectiveness ratio of $41,862/QALY. With the use of more realistic assumptions, screening becomes more cost-effective ($6008/QALY) and remains cost-effective so long as the incremental cost of screening remains under $13.05 per test. Adding the incremental costs of detecting the 13 other disorders on the screening panel still yields a result well within accepted norms for cost-effectiveness ($15,252/QALY). CONCLUSIONS: In Wisconsin, MS/MS screening for MCAD alone appears to be cost-effective. Future analyses should examine the cost-effectiveness of alternative follow-up and treatment regimens for MCAD and other panel disorders.     

Economic outcome for intensive care of infants of birthweight 500–999 g born in Victoria in the post surfactant era

Objective: To determine the incremental cost of improving the outcome for extremely low birthweight (ELBW, birthweight 500–999 g) infants born in Victoria after the introduction of exogenous surfactant (the post surfactant era). Methodology: This was a geographically determined cohort study of ELBW children in Victoria, Australia of consecutive livebirths born in three distinct eras: (i) 1979–80 (n= 351); (ii) 1985–87 (n= 560); and (iii) 1991–92 (n= 429). Exogenous surfactant was first used in Victoria in March, 1991. The consumption of nursery resources per livebirth, and the survival and sensorineural disability rates at 2 years of age for each era were investigated. Utilities were assigned as follows: 0 for dead, 0.4 for severe disability, 0.6 for moderate disability, 0.8 for mild disability, and 1 for no disability. Utilities were multiplied for more than one disability. Dollar costs were assumed to be $1470 ($A 1992) per day of assisted ventilation, and one dose of exogenous surfactant was assumed to be equivalent to one third of a day of assisted ventilation. Cost-effectiveness (additional costs per additional survivor or life-year gained) and cost-utility (additional costs per additional quality-adjusted survivor or life-year gained) ratios were calculated for the pre-surfactant era (1985–87 vs 1979–80), and for the post surfactant era (1991–92 vs 1985–87). Results: Considering only the costs incurred during the primary hospitalization, cost-effectiveness and cost-utility ratios were lower (i.e. economically better) in the post surfactant era than in the pre-surfactant era (pre-surfactant vs post surfactant; S7040 vs$4040 per life year gained; $6700 vs$5360 per quality-adjusted life year gained). Both ratios fell with increasing birthweight. In contrast with the pre-surfactant era, cost-utility ratios were less favourable than cost-effectiveness ratios in the post surfactant era. With costs for long-term care of severely disabled children added, both cost ratios were higher in the post surfactant era. Conclusion: The incremental cost during the primary hospitalization of improving the outcome for ELBW infants has fallen in the post surfactant era.