Newborn Screening and Treatment of Phenylketonuria: Projected Health Outcomes and Cost-Effectiveness.

The objective of this study was to evaluate the cost-effectiveness of newborn screening and treatment for phenylketonuria (PKU) in the context of new data on adherence to recommended diet treatment and a newly available drug treatment (sapropterin dihydrochloride). A computer simulation model was developed to project outcomes for a hypothetical cohort of newborns with PKU. Four strategies were compared: (1) clinical identification (CI) with diet treatment; (2) newborn screening (NBS) with diet treatment; (3) CI with diet and medication (sapropterin dihydrochloride); and (4) NBS with diet and medication. Data sources included published literature, primary data, and expert opinion. From a societal perspective, newborn screening with diet treatment had an incremental cost-effectiveness ratio of $6400/QALY compared to clinical identification with diet treatment. Adding medication to NBS with diet treatment resulted in an incremental cost-effectiveness ratio of more than $16,000,000/QALY. Uncertainty analyses did not substantially alter the cost-effectiveness results. Newborn screening for PKU with diet treatment yields a cost-effectiveness ratio lower than many other recommended childhood prevention programs even if adherence is lower than previously assumed. Adding medication yields cost-effectiveness results unlikely to be considered favorable. Future research should consider conditions under which sapropterin dihydrochloride would be more economically attractive.

Health and economic outcomes of newborn screening for infantile-onset Pompe disease.

PURPOSE: To estimate health and economic outcomes associated with newborn screening (NBS) for infantile-onset Pompe disease in the United States. METHODS: A decision analytic microsimulation model simulated health and economic outcomes of a birth cohort of 4 million children in the United States. Universal NBS and treatment was compared with clinical identification and treatment of infantile-onset Pompe disease. Main outcomes were projected cases identified, costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs) over the life course. RESULTS: Universal NBS for Pompe disease and confirmatory testing was estimated to cost an additional $26 million annually. Additional medication costs associated with earlier treatment initiation were $181 million; however, $8 million in medical care costs for other services were averted due to delayed disease progression. Infants with screened and treated infantile-onset Pompe disease experienced an average lifetime increase of 11.66 QALYs compared with clinical detection. The ICER was $379,000/QALY from a societal perspective and $408,000/QALY from the health-care perspective. Results were sensitive to the cost of enzyme replacement therapy. CONCLUSION: Newborn screening for Pompe disease results in substantial health gains for individuals with infantile-onset Pompe disease, but with additional costs.

Knowledge, Attitudes, and Practices Regarding Dietary Sodium in College Students.

OBJECTIVE: Test a dietary sodium survey in a US adult population of college students using a survey previously validated in a non-US adult population. METHODS: Cross-sectional study of a convenience sample of college students from a Midwest (n = 168) and Pacific Island (n = 152) university. Main outcome measures were knowledge, attitudes, and practices regarding dietary sodium (38 items). Sum scores and percentages for constructs were calculated. A score <75% was considered unfavorable; t test or ANOVA were used to examine group differences. RESULTS: Midwest students were primarily non-Hispanic White individuals (81%) and 65% female. Pacific Island students were predominantly Asian (51%) and 66% female. Mean ± SD construct scores (percentage) for knowledge, attitudes, and practices were 58.69 ± 10.62, 63.96 ± 16.18, 66.00 ± 12.34 (Midwest) and 57.54 ± 10.93, 64.84 ± 14.96, 64.94 ± 13.18 (Pacific Island), respectively; there were no significant differences between schools or race. CONCLUSIONS AND IMPLICATIONS: College students scored low in knowledge, attitudes, and practices regarding sodium. Results from this formative study may inform assessment strategies in future dietary sodium interventions.

Aggregate health and economic burden of herpes zoster in the United States: illustrative example of a pain condition.

Our objective was to develop comprehensive national estimates of the total burden of herpes zoster (HZ) among U.S. adults, including direct (ie, medical costs) and indirect (ie, productivity losses) costs, as well as its psychosocial impact (ie, quality of life losses). Using a patient-level microsimulation model, we projected health and economic outcomes among U.S. adults aged 18 years and older using a 10-year time horizon. We conducted a comprehensive systematic literature review to generate parameter values and conducted simulation modeling to generate our outcomes, including numbers of cases of uncomplicated HZ, postherpetic neuralgia (PHN), and ocular complications, productivity losses, and losses in quality-adjusted life years (QALYs). We used a societal perspective for outcomes; the costing year was 2015. Projected outcomes for an unvaccinated population included 1.1 million HZ cases, 114,000 PHN cases, and 43,000 ocular complications annually, resulting in approximately 67,000 QALYs lost. HZ and its complications would incur costs of $2.4 billion in direct medical costs and productivity losses annually. Projected QALY losses were most sensitive to HZ and PHN health utility values in the model. Cost estimates were most sensitive to the probability of HZ and to the costs per episode of PHN. The national burden of direct, indirect, and psychosocial HZ costs is substantial. Our results can inform economic analyses for HZ vaccines. Comprehensive, national assessments of the total burden of other painful conditions would be very informative.

The financial and time burden associated with phenylketonuria treatment in the United States.

BACKGROUND: Phenylketonuria (PKU) imposes a substantial burden on people living with the condition and their families. However, little is known about the time cost and financial burden of having PKU or caring for a child with the condition. METHODS AND FINDINGS: Primary data were collected with a detailed cost and utilization survey. Primary outcomes included utilization and out-of-pocket costs of medical services, medical formula, and prescribed low-protein food consumption, as well as the time and perceived effort involved in following the PKU diet. Respondents were people living with PKU or parents of children with PKU identified through a state newborn screening program database. Secondary administrative claims data were also used to calculate mean total, insurer, and out-of-pocket payments in inpatient, outpatient (office visits, emergency room, and laboratory tests), and pharmacy settings for privately insured persons with PKU. Payments were calculated for sapropterin and for PKU formula.In primary data analysis (children n = 32, adults n = 52), annual out-of-pocket costs were highest for low-protein foods (child = $1651; adult = $967) compared with other categories of care. The time burden of PKU care was high; families reported spending more than 300 h per year shopping for and preparing special diet foods.In secondary data analysis, children 12-17 years old had the highest average medical expenditures ($54,147; n = 140) compared to children 0-11 years old ($19,057; n = 396) and adults 18 years and older ($40,705; n = 454). Medication costs were the largest contributor to medical costs, accounting for 61-81% of total costs across age groups. Sapropterin was the largest driver of medication costs, accounting for 85% of child medication costs and 92% of adult medication costs. CONCLUSION: Treatment for PKU incurs a substantial time and cost burden on persons with PKU and their families. Estimated medical expenditures using claims data varied by age group, but sapropterin represented the largest cost for PKU treatment from a payer perspective across age groups.

A Cost-Effectiveness Analysis of Vaccination for Prevention of Herpes Zoster and Related Complications: Input for National Recommendations.

Background: The U.S. Advisory Committee on Immunization Practices recently developed recommendations for use of a new recombinant zoster vaccine (RZV). Objective: To evaluate the cost-effectiveness of vaccination with RZV compared with zoster vaccine live (ZVL) and no vaccination, the cost-effectiveness of vaccination with RZV for persons who have previously received ZVL, and the cost-effectiveness of preferential vaccination with RZV over ZVL. Design: Simulation (state-transition) model using U.S. epidemiologic, clinical, and cost data. Data Sources: Published data. Target Population: Hypothetical cohort of immunocompetent U.S. adults aged 50 years or older. Time Horizon: Lifetime. Perspective: Societal and health care sector. Intervention: Vaccination with RZV (recommended 2-dose regimen), vaccination with ZVL, and no vaccination. Outcome Measures: The primary outcome measure was the incremental cost-effectiveness ratio (ICER). Results of Base-Case Analysis: For vaccination with RZV compared with no vaccination, ICERs ranged by age from $10 000 to $47 000 per quality-adjusted life-year (QALY), using a societal perspective and assuming 100% completion of the 2-dose RZV regimen. For persons aged 60 years or older, ICERs were less than $60 000 per QALY. Vaccination with ZVL was dominated by vaccination with RZV for all age groups 60 years or older. Results of Sensitivity Analysis: Results were most sensitive to changes in vaccine effectiveness, duration of protection, herpes zoster incidence, and probability of postherpetic neuralgia. Vaccination with RZV after previous administration of ZVL yielded an ICER of less than $60 000 per QALY for persons aged 60 years or older. In probabilistic sensitivity analyses, RZV remained the preferred strategy in at least 95% of simulations, including those with 50% completion of the second dose. Limitation: Few data were available on risk for serious adverse events, adherence to the recommended 2-dose regimen, and probability of recurrent zoster. Conclusion: Vaccination with RZV yields cost-effectiveness ratios lower than those for many recommended adult vaccines, including ZVL. Results are robust over a wide range of plausible values. Primary Funding Source: Centers for Disease Control and Prevention.

Are there gender differences in acute management and secondary prevention of acute coronary syndromes in Barbados? A cohort study.

OBJECTIVES: In Barbados, high case fatality rates have been reported after myocardial infarction (MI) with higher rates in women than men. To explore this inequality, we examined documented pharmacological interventions for ST-segment elevated myocardial infarction (STEMI), non-STEMI (NSTEMI) and unstable and chronic angina in women and men. DESIGN: Prospective cohort registry data for STEMI and NSTEMI and retrospective chart review for unstable and chronic angina. SETTING: Tertiary care (acute coronary syndromes) and primary care (chronic angina) centres in Barbados. PARTICIPANTS: For the years 2009-2016, a total of 1018 patients with STEMI or NSTEMI were identified via the prospective study. For unstable and chronic angina, 136 and 272 notes were reviewed respectively for the years 2010-2014. OUTCOME MEASURES: The proportions of patients prescribed recommended medication during the first 24 hours after an acute event, at discharge and for chronic care were calculated. Prescribed proportions were analysed by gender after adjustment for age. RESULTS: Between 2009 and 2016, for the acute management of patients with NSTEMI and STEMI, only two (aspirin and clopidogrel) of six drugs had documented prescription rates of 80% or more. Patients with STEMI (n=552) had higher prescription rates than NSTEMI (n=466), with gender differences being more pronounced in the former. Among patients with STEMI, after adjustment for age, diabetes, hypertension and smoking, men were more likely to receive fibrinolytics acutely, OR 2.28 (95% CI 1.24 to 4.21). Compared with men, a higher proportion of women were discharged on all recommended treatments; this was only statistically significant for beta-blockers: age-adjusted OR 1.87 (95% CI 1.16 to 3.00). There were no statistically significant differences in documented prescription of drugs for chronic angina. CONCLUSION: Following acute MI in Barbados, the proportion of patients with documented recommended treatment is relatively low. Although women were less likely to receive appropriate acute care than men, by discharge gender differences were reversed.

Economic assessment of integrated cancer and cardiovascular registries: The Barbados experience.

BACKGROUND: This report describes the resources required to support the integrated approach of the Barbados National Registry for Chronic Non-communicable Diseases (BNR) to non-communicable disease (NCD) case registration, and to identify differences in cost for collecting and maintaining information on cancer and cardiovascular disease (CVD) case registration. METHODS: We used the modified Centers for Disease Control and Prevention's International Registry Costing Tool to collect data from the CVD and cancer registries. We used cancer and CVD cost data for the annual period April 2014 through March 2015 to estimate the total cost and cost per case. We used prospectively collected average annual CVD cases, and for cancer cases we assumed 2 or 3 years are needed for retrospective data collection. RESULTS: The Ministry of Health provided 56% of the resources for the registries. Labor accounted for over 70% of both registries' budgets, while management and administration, along with data collection and analysis, incurred the highest costs per case. Total variable cost activities related to data collection and analysis were higher for the CVD component (US$131,297) than the cancer component (US$58,917). The CVD cost per case (US$489) was in between the cancer 2-year (US$382) and the cancer 3-year (US$573) cost-per-case estimates. CONCLUSION: These findings indicate that there are substantial fixed costs related to management and administration of NCD registries. All registries need management and administration support. When registries are combined, management and administration costs can be shared. We project that registries that can share fixed-cost infrastructure are likely to incur a lower total cost per case.

Predicting the burden of acute myocardial infarction in a country with limited resources: combining data from routine sources.

BACKGROUND: Prior to implementation of a national surveillance system for cardiovascular disease in Barbados, a small island state with limited health resources, the national burden of acute myocardial infarction (MI) was unknown. METHODS: We retrospectively estimated national acute MI incidence rates (IRs) per 100,000 during the decade before registry implementation (1999-2008), using easily accessible routine data from different sources, assessing changes over time through Poisson regression. Future events (2009-2013) were estimated using simple sensitivity analysis to incorporate prediction uncertainty. Model predictions were compared with actual IRs from initial years of the registry. RESULTS: In 2000, crude IR was 85.5 (95% CI: 74.9-97.2), rising to 92.1 (81.2-103.9) in 2008. Accounting for population ageing, the model anticipated IR of 115.9 in 2010 (99.7-132.1), vs actual crude IR 129.7 (115.9-144.6). CONCLUSIONS: Despite no electronic medical record system in Barbados, data were simple to collect, and provided a rough baseline for acute MI burden. We show that, in countries with small populations, limited resources and in absence of surveillance, national mortality statistics and routine hospital data can be combined to adequately model national estimates of acute MI incidence. This cheap and simple, yet fairly accurate method could be a key tool for other low-resource countries with ageing populations and increasing cardiovascular disease levels.

Biological diagnosis of meningococcal meningitis in the African meningitis belt: current epidemic strategy and new perspectives.

Laboratory diagnosis is an essential component in surveillance of meningococcal epidemics, as it can inform decision-makers of the Neisseria meningitidis serogroup(s) involved and the most appropriate vaccine to be selected for mass vaccination. However, countries most affected face real limitations in laboratory diagnostics, due to lack of resources. We describe current diagnostic tools and examine their cost-effectiveness for use in an epidemic context. The conclusion is that current WHO recommendations to use only the latex agglutination assay (Pastorex) at epidemic onset is cost-effective, but recently developed rapid diagnostic tests for the major epidemic-causing meningococcal serogroups may prove a breakthrough for the future.