OBJECTIVES: Neuromuscular scoliosis (NMS) can result in severe disability. Nonoperative management minimally slows scoliosis progression, but operative management with posterior spinal fusion (PSF) carries high risks of morbidity and mortality. In this study, we compare health and economic outcomes of PSF to nonoperative management for children with NMS to identify opportunities to improve care. METHODS: We performed a cost-effectiveness analysis. Our decision analytic model included patients aged 5 to 20 years with NMS and a Cobb angle ≥50°, with a base case of 15-year-old patients. We estimated costs, life expectancy, quality-adjusted life-years (QALYs), and incremental cost-effectiveness from published literature and conducted sensitivity analyses on all model inputs. RESULTS: We estimated that PSF resulted in modestly decreased discounted life expectancy (10.8 years) but longer quality-adjusted life expectancy (4.84 QALYs) than nonoperative management (11.2 years; 3.21 QALYs). PSF costs $75 400 per patient. Under base-case assumptions, PSF costs $50 100 per QALY gained. Our findings were sensitive to quality of life (QoL) and life expectancy, with PSF favored if it significantly increased QoL. CONCLUSIONS: In patients with NMS, whether PSF is cost-effective depends strongly on the degree to which QoL improved, with larger improvements when NMS is the primary cause of debility, but limited data on QoL and life expectancy preclude a definitive assessment. Improved patient-centered outcome assessments are essential to understanding the effectiveness of NMS treatment alternatives. Because the degree to which PSF influences QoL substantially impacts health outcomes and varies by patient, clinicians should consider shared decision-making during PSF-related consultations.
Background Pre-exposure prophylaxis with oral antiretroviral treatment (oral PrEP) for HIV-uninfected injection drug users (IDUs) is potentially useful in controlling HIV epidemics with a significant injection drug use component. We estimated the effectiveness and cost effectiveness of strategies for using oral PrEP in various combinations with methadone maintenance treatment (MMT) and antiretroviral treatment (ART) in Ukraine, a representative case for mixed HIV epidemics. Methods and Findings We developed a dynamic compartmental model of the HIV epidemic in a population of non-IDUs, IDUs who inject opiates, and IDUs in MMT, adding an oral PrEP program (tenofovir/emtricitabine, 49% susceptibility reduction) for uninfected IDUs. We analyzed intervention portfolios consisting of oral PrEP (25% or 50% of uninfected IDUs), MMT (25% of IDUs), and ART (80% of all eligible patients). We measured health care costs, quality-adjusted life years (QALYs), HIV prevalence, HIV infections averted, and incremental cost effectiveness. A combination of PrEP for 50% of IDUs and MMT lowered HIV prevalence the most in both IDUs and the general population. ART combined with MMT and PrEP (50% access) averted the most infections (14,267). For a PrEP cost of $950, the most cost-effective strategy was MMT, at $520/QALY gained versus no intervention. The next most cost-effective strategy consisted of MMT and ART, costing $1,000/QALY gained compared to MMT alone. Further adding PrEP (25% access) was also cost effective by World Health Organization standards, at $1,700/QALY gained. PrEP alone became as cost effective as MMT at a cost of $650, and cost saving at $370 or less. Conclusions Oral PrEP for IDUs can be part of an effective and cost-effective strategy to control HIV in regions where injection drug use is a significant driver of the epidemic. Where budgets are limited, focusing on MMT and ART access should be the priority, unless PrEP has low cost.
Chronic hepatitis C viral (HCV) infection affects millions of Americans. Healthcare systems face complex choices between multiple highly efficacious, costly treatments. This study assessed the cost-effectiveness of HCV treatments for chronic, genotype 1 HCV monoinfected, treatment-naïve individuals in the Department of Veterans Affairs (VA) and general U.S. healthcare systems.We conducted a decision-analytic Markov model-based cost-effectiveness analysis, employing appropriate payer perspectives and time horizons, and discounting benefits and costs at 3% annually. Interventions included: Sofosbuvir/ledipasvir (SOF-LDV); ombitasvir/paritaprevir/ritonavir/dasabuvir (3D); sofosbuvir/simeprevir (SOF-SMV); sofosbuvir/pegylated interferon/ribavirin (SOF-RBV-PEG); boceprevir/pegylated interferon/ribavirin (BOC-RBV-PEG); and pegylated interferon/ribavirin (PEG-RBV). Outcomes were sustained virologic response (SVR), advanced liver disease, costs, quality adjusted life years (QALYs), and incremental cost-effectiveness.SOF-LDV and 3D achieve higher SVR rates compared to older regimens and reduce advanced liver disease (>20% relative to no treatment), increasing QALYs by over 2 years per person. For the non-VA population, at current prices ($5,040 per week for SOF-LDV and $4,796 per week for 3D), SOF-LDV's lifetime cost ($293,370) is $18,000 lower than 3D's because of its shorter treatment duration in subgroups. SOF-LDV costs $17,100 per QALY gained relative to no treatment. 3D costs $208,000 per QALY gained relative to SOF-LDV. Both dominate other treatments and are even more cost-effective for the VA, though VA aggregate treatment costs still exceed $4 billion at SOF-LDV prices of $3,308 per week. Drug prices strongly determine relative cost-effectiveness for SOF-LDV and 3D; With sufficient price reductions (approximately 20-30% depending on the health system), 3D could be cost-effective relative to SOF-LDV. Limitations include the lack of long-term head-to-head regimen effectiveness trials.New HCV treatments are cost-effective in multiple healthcare systems if trial-estimated efficacy is achieved in practice, though, at current prices, total expenditures could present substantial challenges.
Vaccination for the 2009 pandemic did not occur until late in the outbreak, which limited its benefits. Influenza A (H7N9) is causing increasing morbidity and mortality in China, and researchers have modified the A (H5N1) virus to transmit via aerosol, which again heightens concerns about pandemic influenza preparedness.To determine how quickly vaccination should be completed to reduce infections, deaths, and health care costs in a pandemic with characteristics similar to influenza A (H7N9) and A (H5N1).Dynamic transmission model to estimate health and economic consequences of a severe influenza pandemic in a large metropolitan city.Literature and expert opinion.Residents of a U.S. metropolitan city with characteristics similar to New York City.Lifetime.Societal.Vaccination of 30% of the population at 4 or 6 months.Infections and deaths averted and cost-effectiveness.In 12 months, 48 254 persons would die. Vaccinating at 9 months would avert 2365 of these deaths. Vaccinating at 6 months would save 5775 additional lives and $51 million at a city level. Accelerating delivery to 4 months would save an additional 5633 lives and $50 million.If vaccination were delayed for 9 months, reducing contacts by 8% through nonpharmaceutical interventions would yield a similar reduction in infections and deaths as vaccination at 4 months.The model is not designed to evaluate programs targeting specific populations, such as children or persons with comorbid conditions.Vaccination in an influenza A (H7N9) pandemic would need to be completed much faster than in 2009 to substantially reduce morbidity, mortality, and health care costs. Maximizing non-pharmaceutical interventions can substantially mitigate the pandemic until a matched vaccine becomes available.Agency for Healthcare Research and Quality, National Institutes of Health, and Department of Veterans Affairs.
This chapter provides a logical, patient-centered approach to selecting and interpreting diagnostic tests. These two ideas, selection and interpretation, are linked to a third idea, trying to predict the effect of test results on patient care, something to do before ordering a test but also after one knows the results. The first part of the chapter talks about taking action when the consequences are uncertain. The second and third parts discuss the treatment-threshold probability and the decision to obtain a diagnostic test, respectively. The fourth and fifth part talk about choosing between diagnostic tests and choosing the best combination of diagnostic tests, respectively. The remaining four parts talk about setting the treatment-threshold probability, taking account of the utility of experiencing a test, a clinical case: test selection for suspected brain tumor, and sensitivity analysis.
Objective: To evaluate the cost effectiveness of voluntary prenatal and routine postnatal HIV screening in the cohort of pregnant women and newborns in the United States. Design: Cost-effectiveness analysis. We developed a decision model to analyze the cost effectiveness of enhanced prenatal screening and routine newborn screening for HIV. We also analyzed the incremental cost effectiveness of routine newborn screening when improved voluntary prenatal screening is already in place. Participants: Analysis of the cohort of pregnant women and newborns in the United States. Interventions: Enhanced prenatal screening, or routine newborn screening for HIV. Main Outcome Measures: Infections averted, life expectancy, costs, and incremental cost effectiveness. Results: Improved participation in voluntary prenatal HIV screening would result in an additional 1.1 million women being screened annually, would identify an additional 527 HIV-infected mothers annually, would avert 150 infections in newborns, and would cost $8,900 U.S. per life-year gained. Routine newborn HIV screening would test 3.9 million infants annually, would identify 1061 HIV-infected mothers, would avert 266 infections in newborns, and would cost $7,000 U.S. per life-year gained. If improved voluntary prenatal screening is already in place, routine newborn screening would avert an additional 135 infections in newborns, at an incremental cost of $10,600 U.S. per life-year gained. The screening programs are likely to be cost effective over a wide range of assumptions regarding key factors in the analysis. Conclusions: Improved voluntary prenatal HIV screening of women and routine screening of newborns are cost effective. Routine newborn screening becomes less attractive as the rate of voluntary prenatal screening increases. Improved participation in voluntary prenatal screening has the added benefit that mothers maintain their right to determine whether they are tested for HIV.
