Gene Therapy Versus Common Care for Eligible Individuals With Sickle Cell Disease in the United States : A Cost-Effectiveness Analysis.

BACKGROUND: Sickle cell disease (SCD) and its complications contribute to high rates of morbidity and early mortality and high cost in the United States and African heritage community. OBJECTIVE: To evaluate the cost-effectiveness of gene therapy for SCD and its value-based prices (VBPs). DESIGN: Comparative modeling analysis across 2 independently developed simulation models (University of Washington Model for Economic Analysis of Sickle Cell Cure [UW-MEASURE] and Fred Hutchinson Institute Sickle Cell Disease Outcomes Research and Economics Model [FH-HISCORE]) using the same databases. DATA SOURCES: Centers for Medicare & Medicaid Services claims data, 2008 to 2016; published literature. TARGET POPULATION: Persons eligible for gene therapy. TIME HORIZON: Lifetime. PERSPECTIVE: U.S. health care sector and societal. INTERVENTION: Gene therapy versus common care. OUTCOME MEASURES: Incremental cost-effectiveness ratios (ICERs), equity-informed VBPs, and price acceptability curves. RESULTS OF BASE-CASE ANALYSIS: At an assumed $2 million price for gene therapy, UW-MEASURE and FH-HISCORE estimated ICERs of $193 000 per QALY and $427 000 per QALY, respectively, under the health care sector perspective. Corresponding estimates from the societal perspective were $126 000 per QALY and $281 000 per QALY. The difference in results between models stemmed primarily from considering a slightly different target population and incorporating the quality-of-life (QOL) effects of splenic sequestration, priapism, and acute chest syndrome in the UW model. From a societal perspective, acceptable (>90% confidence) VBPs ranged from $1 million to $2.5 million depending on the use of alternative effective metrics or equity-informed threshold values. RESULTS OF SENSITIVITY ANALYSIS: Results were sensitive to the costs of myeloablative conditioning before gene therapy, effect on caregiver QOL, and effect of gene therapy on long-term survival. LIMITATION: The short-term effects of gene therapy on vaso-occlusive events were extrapolated from 1 study. CONCLUSION: Gene therapy for SCD below a $2 million price tag is likely to be cost-effective when applying a societal perspective at an equity-informed threshold for cost-effectiveness analysis. PRIMARY FUNDING SOURCE: National Heart, Lung, and Blood Institute.

Lifetime Health and Economic Outcomes of Biparametric Magnetic Resonance Imaging as First-Line Screening for Prostate Cancer : A Decision Model Analysis.

BACKGROUND: Contemporary prostate cancer (PCa) screening uses first-line prostate-specific antigen (PSA) testing, possibly followed by multiparametric magnetic resonance imaging (mpMRI) for men with elevated PSA levels. First-line biparametric MRI (bpMRI) screening has been proposed as an alternative. OBJECTIVE: To evaluate the comparative effectiveness and cost-effectiveness of first-line bpMRI versus PSA-based screening. DESIGN: Decision analysis using a microsimulation model. DATA SOURCES: Surveillance, Epidemiology, and End Results database; randomized trials. TARGET POPULATION: U.S. men aged 55 years with no prior screening or PCa diagnosis. TIME HORIZON: Lifetime. PERSPECTIVE: U.S. health care system. INTERVENTION: Biennial screening to age 69 years using first-line PSA testing (test-positive threshold, 4 µg/L) with or without second-line mpMRI or first-line bpMRI (test-positive threshold, PI-RADS [Prostate Imaging Reporting and Data System] 3 to 5 or 4 to 5), followed by biopsy guided by MRI or MRI plus transrectal ultrasonography. OUTCOME MEASURES: Screening tests, biopsies, diagnoses, overdiagnoses, treatments, PCa deaths, quality-adjusted and unadjusted life-years saved, and costs. RESULTS OF BASE-CASE ANALYSIS: For 1000 men, first-line bpMRI versus first-line PSA testing prevented 2 to 3 PCa deaths and added 10 to 30 life-years (4 to 11 days per person) but increased the number of biopsies by 1506 to 4174 and the number of overdiagnoses by 38 to 124 depending on the biopsy imaging scheme. At conventional cost-effectiveness thresholds, first-line PSA testing with mpMRI followed by either biopsy approach for PI-RADS 4 to 5 produced the greatest net monetary benefits. RESULTS OF SENSITIVITY ANALYSIS: First-line PSA testing remained more cost-effective even if bpMRI was free, all men with low-risk PCa underwent surveillance, or screening was quadrennial. LIMITATION: Performance of first-line bpMRI was based on second-line mpMRI data. CONCLUSION: Decision analysis suggests that comparative effectiveness and cost-effectiveness of PCa screening are driven by false-positive results and overdiagnoses, favoring first-line PSA testing with mpMRI over first-line bpMRI. PRIMARY FUNDING SOURCE: National Cancer Institute.

Association between achieving adequate antenatal care and health-seeking behaviors: A study of Demographic and Health Surveys in 47 low- and middle-income countries.

BACKGROUND: Antenatal care (ANC) is essential for ensuring the well-being of pregnant women and their fetuses. This study models the association between achieving adequate ANC and various health and health-seeking indicators across wealth quintiles in low- and middle-income countries (LMICs). METHODS AND FINDINGS: We analyzed data from 638,265 women across 47 LMICs using available Demographic and Health Surveys from 2010 to 2022. Via multilevel logistic regression analyses adjusted for a series of confounding variables and country and wealth quintile fixed effects, we estimated the projected impact of achieving adequate ANC utilization and quality on a series of health and health care indicators: facility birth, postnatal care, childhood immunizations, and childhood stunting and wasting. Achieving adequate levels of ANC utilization and quality (defined as at least 4 visits, blood pressure monitoring, and blood and urine testing) was positively associated with health-seeking behavior across the majority of countries. The strongest association was observed for facility birth, followed by postnatal care and child immunization. The strength of the associations varied across countries and wealth quintiles, with more significant ones observed in countries with lower baseline ANC utilization levels and among the lower wealth quintiles. The associations of ANC with childhood stunting and wasting were notably less statistically significant compared to other indicators. Despite rigorous adjustments for potential confounders, a limitation to the methodology is that it is possible that unobserved variables may still impact outcomes. CONCLUSIONS: Strengthening ANC is associated with improved use of other health care in LMICs. ANC could serve as a critical platform for improving health outcomes for mothers and their children, emphasizing its importance beyond direct impact on maternal and neonatal mortality.

Evaluating Policies of Expanding Versus Restricting First-Line Treatment Choices: A Cost-Effectiveness Analysis Framework.

OBJECTIVES: Healthcare payers often implement coverage policies that restrict the utilization of costly new first-line treatments. Cost-effectiveness analysis can be conducted to inform these decisions by comparing the new treatment with an existing one. However, this approach may overlook important factors such as treatment effect heterogeneity and endogenous treatment selection, policy implementation costs, and diverse patient preferences across multiple treatment options. We aimed to develop a cost-effectiveness analysis framework that considers these real-world factors, facilitating the evaluation of alternative policies related to expanding or restricting first-line treatment choices. METHODS: We introduced a metric of incremental cost-effectiveness ratio (ICER) that compares an expanded choice set (CS) including the new first-line treatment with a restricted CS excluding the new treatment. ICER(CS) accounts for treatment selection influenced by heterogeneous treatment effects and policy implementation costs. We examined a basic scenario with 2 standard first-line treatment choices and a more realistic scenario involving diverse preferences toward multiple choices. To illustrate the framework, we conducted a retrospective evaluation of including versus excluding abiraterone acetate plus prednisone (AAP) (androgen deprivation therapy [ADT] + AAP) as a first-line treatment for metastatic hormone-sensitive prostate cancer. RESULTS: The traditional ICERs for ADT + AAP versus ADT alone and ADT+ docetaxel were $104 269 and $206 324/quality-adjusted life-year, respectively. The ICER(CS) for comparing an expanded CS with ADT + AAP with a restricted CS without ADT + AAP was $123 179/quality-adjusted life-year. CONCLUSIONS: The proposed framework provides decision makers with policy-relevant tools, enabling them to assess the cost-effectiveness of alternative policies of expanding versus restricting patients' and physicians' first-line treatment choices.

Estimating the Allocation of the Economic Value Generated by Utilization of All-Oral Direct-Acting Antivirals for Hepatitis C in the United States, 2015 to 2019.

OBJECTIVES: Between 2013 to 2019, several all-oral direct-acting antivirals (DAAs) were launched with the potential to cure patients with hepatitis C virus (HCV). They generated economic value in terms of the health gains for patients and cost-savings for the US healthcare system. We estimated the share of this value allocated to 4 manufacturers vs society. METHODS: For 2015 to 2019, we estimated the incremental impact of DAAs on HCV health outcomes and costs. We used the Center for Disease Analysis Foundation Polaris Observatory database to estimate utilization. Per-patient projections of lifetime quality-adjusted life-years (QALYs) gained and medical costs avoided were based on a standard 9-state HCV disease-progression model for DAA treatment vs alternatives. Annual QALY gains were valued at $114 000 per QALY. Outcomes and costs were discounted at 3%. Estimated revenues were based on reported sales. RESULTS: An estimated 1 080 000 patients received DAAs: 81.5% would not have received the pre-DAA standard of care. On average, these patients were projected to gain 4.4 QALYs and save $104 400 in lifetime healthcare costs, generating $531.8 billion in value. Those who would have received treatment gained 1.7 QALYs and saved $41 500 in lifetime costs, generating $47.4 billion in economic value. As treatment costs fell nearly 75%, the 4 manufacturers reported $37.4 billion from DAA sales-an allocation of 6.5% of the total value. CONCLUSIONS: The significant majority (∼90%) of the economic value of curing HCV with DAAs were health benefits to patients and net cost-savings to society. DAA manufacturers received a minority share (6.5%) of the aggregate economic value generated.

Cost-effectiveness of TB diagnostic technologies in Ethiopia: a modelling study.

BACKGROUND: Tuberculosis (TB) is a major threat to public health, particularly in countries where the disease is highly prevalent, such as Ethiopia. Early diagnosis and treatment are the main components of TB prevention and control. Although the national TB guideline recommends the primary use of rapid TB diagnostics whenever feasible, there is limited evidence available that assess the efficiency of deploying various diagnostic tools in the country. Hence, this study aims to evaluate the cost-effectiveness of rapid TB/MDR-TB diagnostic tools in Ethiopia. METHODS: A hybrid Markov model for a hypothetical adult cohort of presumptive TB cases was constructed. The following TB diagnostic tools were evaluated: X-pert MTB/RIF, Truenat, chest X-ray screening followed by an X-pert MTB/RIF, TB-LAMP, and smear microscopy. Cost-effectiveness was determined based on incremental costs ($) per Disability-adjusted Life Years (DALY) averted, using a threshold of one times Gross Domestic Product (GDP) per capita ($856). Data on starting and transition probabilities, costs, and health state utilities were derived from secondary sources. The analysis is conducted from the health system perspective, and a probabilistic sensitivity analysis is performed. RESULT: The incremental cost-effectiveness ratio for X-pert MTB/RIF, compared to the next best alternative, is $276 per DALY averted, making it a highly cost-effective diagnostic tool. Additionally, chest X-ray screening followed an X-pert MTB/RIF test is less cost-effective, with an ICER of $1666 per DALY averted. Introducing X-pert MTB/RIF testing would enhance TB detection and prevent 9600 DALYs in a cohort of 10,000 TB patients, with a total cost of $3,816,000. CONCLUSION: The X-pert MTB/RIF test is the most cost-effective diagnostic tool compared to other alternatives. The use of this diagnostic tool improves the early detection and treatment of TB cases. Increased funding for this diagnostic tool will enhance access, reduce the TB detection gaps, and improve treatment outcomes.

Economic Evaluation of a Point-of-Care Test for Bacterial Vaginosis Among Women With Vaginal Symptoms.

BACKGROUND: There is an unmet need for a clinical diagnostic technology to detect bacterial vaginosis (BV) rapidly and accurately. Novel point-of-care (POC) tests have the potential to fulfill this gap. Our objective was to determine the cost-effectiveness of a hypothetical clinician-administered POC test for diagnosing BV in the United States. METHODS: We developed a state-transition microsimulation model to evaluate the cost-effectiveness of using the POC test versus usual care among women of reproductive age with vaginal symptoms. We adopted a healthcare sector perspective that included relevant healthcare costs and a societal perspective that further incorporated productivity costs. Model parameters were empirically estimated based on commercial insurance claims data or derived from published literature. The primary model outcome was incremental cost-effectiveness ratio. We started with analyzing a hypothetical POC test with a sensitivity and specificity of 0.9 and a cost of $40, followed by extensive sensitivity analyses. RESULTS: Using the hypothetical POC test to diagnose BV increased costs by $16 and quality-adjusted life-years by 0.0005 per person compared with the usual care, leading to an incremental cost-effectiveness ratio of $31,108 per quality-adjusted life-year gained. When also capturing the productivity costs, the POC test resulted in an average cost savings of $57. The sensitivity analyses showed that the POC test's sensitivity was more influential on its cost-effectiveness than specificity. CONCLUSIONS: Using the POC test to diagnose BV is likely to be cost-effective relative to usual care, especially with a high sensitivity or a substantial positive effect on productivity.

Value-Based Pricing for Patent-Protected Medicines Over the Product Life Cycle: Pricing Anomalies in the "Age of Cures" and Their Implications for Dynamic Efficiency.

OBJECTIVES: Conventional cost-effectiveness analysis (CEA) for the value-based pricing of new medicines largely ignores the implications of limited market exclusivity (ie, patent-protection periods plus any exclusivity granted by regulators). This paper explores the implications of this methodological shortcoming, which produces several pricing anomalies with potentially unintended effects on research and development (R&D) incentives. METHODS: We illustrate these implications by comparing 4 stylized examples of increasing complexity, from short-term cures for acute conditions to long-term cures for rare, health-catastrophic conditions. RESULTS: (1) Conventional-CEA will project a different result than an adjusted CEA that considers generic or biosimilar entry; (2) free and flexible pricing of long-term treatments (eg, statins for hypercholesterolemia) or repeated-dose cures (eg, insulin for type 1 diabetes) for chronic conditions will likely result in predictable price increases at the end of the exclusivity period that may be perceived as unjustified or unsupported; and (3) one-time administration "cures" (eg, gene therapy for spinal muscular atrophy) have the potential to allocate a large share of the social surplus to the manufacturer over the product lifetime, which may or may not be dynamically efficient per se, but may also inadvertently disadvantage the development of valuable long-term treatments or repeated-dose cures for chronic conditions. CONCLUSIONS: We highlight the need for additional research on long-term solutions to these issues that would aim to promote dynamically efficient global R&D. More work is needed on the following: (1) relationships between social surplus allocation and the amount and composition of global R&D, as we may be as likely to be encouraging excessive R&D in some areas as to be undersupplying it in others; and (2) relating the size of the surplus reward to R&D cost and, thus, the return on investment.

Does Clinical Evidence of Heterogeneity Impact Treatment Selection? A Case Study of Abiraterone for Metastatic Prostate Cancer.

BACKGROUND: Two pivotal randomized controlled trials (RCTs) demonstrate that abiraterone acetate + prednisone (AAP) combined with androgen deprivation therapy (ADT) significantly extends the survival of men with metastatic hormone-sensitive prostate cancer (mHSPC) compared with ADT alone. Their subgroup analyses indicate that the survival benefit is significant for younger men but not older men. We aimed to assess whether publication of the RCTs was associated with differential real-world AAP utilization by age groups. METHODS: Using TriNetX electronic medical records data collected from 43 healthcare organizations across the United States, we performed a difference-in-differences event study among men with newly diagnosed mHSPC observed from June 2014 to June 2019. Eligible subjects were identified based on a comprehensive published algorithm. We analyzed the change in utilization rate of AAP before versus after publication of the RCTs among men aged <70 years versus ≥70 years, adjusting for demographic factors and clinical conditions. RESULTS: Our study included 6,888 men with newly diagnosed mHSPC with 12,738 observations, of whom 46% were aged <70 years. The prepublication trends of AAP utilization were similar between the age groups, whereas publication of the RCTs was associated with a 3.5% higher adjusted uptake rate of AAP among younger men (95% CI, 1.2%-5.8%) relative to older men. This estimate reflects an uptake rate nearly 3 times higher than would have been expected had younger men followed the same utilization trends as older men. The estimates remained consistent throughout the postpublication period. CONCLUSIONS: Our study suggests that publication of the RCTs was associated with faster uptake of AAP among younger versus older men with newly diagnosed mHSPC, despite the absence of clinical guidance for differential treatment selection. This finding highlights the importance of confirmatory studies among older men, considering the uncertainties of subgroup analyses in RCTs.

Health State Utilities for Sickle Cell Disease: A Catalog Prepared From a Systematic Review.

OBJECTIVES: Sickle cell disease (SCD) is a complex, chronic condition that impairs health-related quality of life of affected individuals and their caregivers. As curative therapies emerge, comprehensive cost-effectiveness models will inform their value. These models will require descriptions of health states and their corresponding utility values that accurately reflect health-related quality of life over the disease trajectory. The objectives of this systematic review were to develop a catalog of health state utility (HSU) values for SCD, identify research gaps, and provide future directions for preference elicitation. METHODS: Records were identified through searches of PubMed and Embase, Tufts Medical Center Cost-Effectiveness Analysis Registry, reference lists of relevant articles, and consultation with SCD experts (2008-2020). We removed duplicate records and excluded ineligible studies. For included studies, we summarized the study characteristics, methods used for eliciting HSUs, and HSU values. RESULTS: Five studies empirically elicited utilities using indirect methods (EQ-5D) (n = 3) and Short Form-6 Dimension (n = 2); these represent health states associated with general SCD (n = 1), SCD complications (n = 2), and SCD treatments (n = 3). Additionally, we extracted HSUs from 7 quality-adjusted life-years-based outcome research studies. The HSU among patients with general SCD without specifying complications ranged from 0.64 to 0.887. Only 36% of the HSUs used in the quality-adjusted life-year-based outcomes research studies were derived from individuals with SCD. No study estimated HSUs in caregivers. CONCLUSIONS: There is a dearth of literature of HSUs for use in SCD models. Future empirical studies should elicit a comprehensive set of HSUs from individuals with SCD and their caregivers.

Application of validated mapping algorithms between generic PedsQL scores and utility values to individuals with sickle cell disease.

PURPOSE: There is a paucity of empirically estimated health state utility (HSU) values to estimate health-related quality of life among individuals with sickle cell disease (SCD). This study aims to map the Pediatric Quality of Life Inventory generic core scales (PedsQL GCS) to HSUs for children and adolescents with SCD in the United States, using published algorithms, and to assess the construct validity of these HSUs against SCD-specific PedsQL scores. METHODS: We used the published mapping algorithms identified in four published articles, in which the PedsQL GCS was mapped to either the EuroQol-5 Dimension 3-Level, Youth Version or the Child Health Utility 9-Dimension to obtain HSUs. We employed the algorithms to calculate HSUs for a sample of children and adolescents from the Sickle Cell Clinical Research and Intervention Program. To assess the construct validity of the mapped HSUs in SCD patients, we computed Spearman's correlation coefficient comparing the HSUs with the PedsQL SCD total score and separately with each PedsQL SCD dimension-specific score. RESULTS: The mean mapped HSU across published algorithms was 0.792 (95% CI: 0.782-0.801). It was significantly higher among children aged 5-12 years than children aged 13-17 years. The Spearman's correlation coefficient for HSUs versus PedsQL SCD total scores was 0.64 (95% CI: 0.57-0.71). Correlations ranged from 0.40 (95% CI: 0.32-0.48) to 0.60 (95% CI: 0.54-0.66) for HSUs versus PedsQL SCD dimension-specific scores. CONCLUSIONS: The existing mapping algorithms show acceptable construct validity in children and adolescents with SCD. Additional algorithms are needed for adults and for specific SCD comorbidities.

Is the Direct Anterior Approach to THA Cost-effective? A Markov Analysis.

BACKGROUND: The use of the direct anterior approach, a muscle-sparing technique for THA, has increased over the years; however, this approach is associated with longer procedure times and a more expensive direct cost. Furthermore, studies have shown a higher revision rate in the early stages of the learning curve. Whether the clinical advantages of the direct anterior compared with the posterior approach-such as less soft tissue damage, decreased short-term postoperative pain, a lower dislocation rate, decreased length of stay in the hospital, and higher likelihood of being discharged home-outweigh the higher cost is still debatable. Determining the cost-effectiveness of the approach may inform its utility and justify its use at various stages of the learning curve. QUESTIONS/PURPOSES: We used a Markov modeling approach to ask: (1) Is the direct anterior approach more likely to be a cost-effective approach than the posterior approach over the long-term for more experienced or higher volume hip surgeons? (2) How many procedures does a surgeon need to perform for the direct anterior approach to be a cost-effective choice? METHODS: A Markov model was created with three health states (well-functioning THA, revision THA, and death) to compare the cost-effectiveness of the direct anterior approach with that of the posterior approach in five scenarios: surgeons who performed one to 15, 16 to 30, 31 to 50, 51 to 100, and more than 100 direct anterior THAs during a 6-year span. Procedure costs (not charges), dislocation costs, and fracture costs were derived from published reports, and model was run using two different cost differentials between the direct anterior and posterior approaches (USD 219 and USD 1800, respectively). The lower cost was calculated as the total cost differential minus pharmaceutical and implant costs to account for differences in implant use and physician preference regarding postoperative pain management. The USD 1800 cost differential incorporated pharmaceutical and implant costs. Probabilities were derived from systematic review of the evidence as well as from the Australian Orthopaedic Association National Joint Replacement Registry. Utilities were estimated from best available literature and disutilities associated with dislocation and fracture were incorporated into the model. Quality of life was expressed in quality-adjusted life years (QALYs), which are calculated by multiplying the utility of a health state (ranging from 0 to 1) by the duration of time in that health state. The primary outcome measure was the incremental cost-effectiveness ratio, or the change in costs divided by the change in QALYs when the direct anterior approach was used for THA. USD 100,000 per quality-adjusted life years was used as a threshold for willingness to pay. One-way and probabilistic sensitivity analyses were performed for the scenario in which the direct anterior approach is cost-effective to further account for uncertainty in model inputs. RESULTS: At a cost differential of USD 219 (95% CI 175 to 263), the direct anterior approach was associated with lower cost and higher effectiveness compared with the posterior approach for surgeons with an experience level of more than 100 operations during a 6-year span. At a cost differential of USD 1800 (95% CI 1440 to 2160), the direct anterior approach remained a cost-effective strategy for surgeons who performed more than 100 operations. At both cost differentials, the direct anterior approach was not cost-effective for surgeons who performed fewer than 100 operations. One-way sensitivity analyses revealed the model to be the most sensitive to fluctuations in the utility of revision THA, probability of revision after the posterior approach THA, probability of dislocation after the posterior approach THA, fluctuations in the probability of dislocation after direct anterior THA, cost of direct anterior THA, and probability of intraoperative fracture with the direct anterior approach. At the cost differential of USD 219 and for surgeons with a surgical experience level of more than 100 direct anterior operations, the direct anterior approach was still the cost-effective strategy for the entire range of values. CONCLUSION: For high-volume hip surgeons, defined here as surgeons who perform more than 100 procedures during a 6-year span, the direct anterior approach may be a cost-effective strategy within the limitations imposed by our analysis. For lower volume hip surgeons, performing a more familiar approach appears to be more cost-effective.

Clarifying the Trade-Offs of Risk-Stratified Screening for Prostate Cancer: A Cost-Effectiveness Study.

Cancer risk prediction is necessary for precision early detection, which matches screening intensity to risk. However, practical steps for translating risk predictions to risk-stratified screening policies are not well established. We used a validated population prostate-cancer model to simulate the outcomes of strategies that increase intensity for men at high risk and reduce intensity for men at low risk. We defined risk by the Prompt Prostate Genetic Score (PGS) (Stratify Genomics, San Diego, California), a germline genetic test. We first recalibrated the model to reflect the disease incidence observed within risk strata using data from a large prevention trial where some participants were tested with Prompt PGS. We then simulated risk-stratified strategies in a population with the same risk distribution as the trial and evaluated the cost-effectiveness of risk-stratified screening versus universal (risk-agnostic) screening. Prompt PGS risk-adapted screening was more cost-effective when universal screening was conservative. Risk-stratified strategies improved outcomes at a cost of less than $100,000 per quality-adjusted life year compared with biennial screening starting at age 55 years, but risk stratification was not cost-effective compared with biennial screening starting at age 45. Heterogeneity of risk and fraction of the population within each stratum were also important determinants of cost-effectiveness.

Catalog of Age- and Medical Condition-Specific Healthcare Costs in the United States to Inform Future Costs Calculations in Cost-Effectiveness Analysis.

OBJECTIVES: This study aims to develop a catalog of annual age- and medical condition-specific healthcare costs per capita among those who are living at a certain age (survivors) and the costs attributable to death itself for those who die at that age (decedents) in the United States. These estimates can be used to inform future cost calculations in cost-effectiveness analysis (CEA). METHODS: We discussed a theoretical framework to incorporate futures costs in CEA. We used the nationally representative Medical Expenditure Panel Survey data to estimate costs among survivors and death costs. For survivors, we obtained cost estimates nonparametrically using kernel-based regression and locally weighted scatterplot smoothing. We estimated costs attributable to death using inverse probability weights comparing decedents with appropriately weighted survivors at a given age after controlling for more than 270 clinical condition classifications, demographics, and interactions. Cost estimates were expressed in 2019 US dollar and also separately by sex and specific clinical conditions. RESULTS: Average healthcare costs per capita among survivors, expectedly, rose over age from $2062 (95% confidence interval [CI] $1553-$2478) during the first year of life to $14 307 (95% CI $13 706-$14 956) at 85 years or older. Average costs of death were $44 569 (95% CI $14 304-$67 369) during the first year of life and declined by -$321 (95% CI -$620 to -$22) per 1 year older. CONCLUSIONS: The US catalog of healthcare costs among survivors and decedents can facilitate calculations of future costs in CEA as recommended by the Second Panel on Cost-Effectiveness in Health and Medicine.

Economic Evaluation of Urine-Based or Magnetic Resonance Imaging Reflex Tests in Men With Intermediate Prostate-Specific Antigen Levels in the United States.

OBJECTIVES: For men with intermediate prostate-specific antigen (PSA) levels (4-10 ng/mL), urine-based biomarkers and multiparametric magnetic resonance imaging (MRI) are increasingly used as reflex tests before prostate biopsy. We assessed the cost effectiveness of these reflex tests in the United States. METHODS: We used an existing microsimulation model of prostate cancer (PCa) progression and survival to predict lifetime outcomes for a hypothetical cohort of 55-year-old men with intermediate PSA levels. Urine-based biomarkers-PCa antigen (PCA3), TMPRSS2:ERG gene fusion (T2:ERG), and the MyProstateScore (MPS) for any PCa and for high-grade (Gleason score ≥7) PCa (MPShg)-were generated using biomarker data from 1112 men presenting for biopsy at 10 United States institutions. MRI results were based on published sensitivity and specificity for high-grade PCa. Costs and utilities were sourced from literature and Medicare reimbursement schedules. Outcome measures included life years, quality-adjusted life years (QALYs), and lifetime medical costs per patient. Incremental cost-effectiveness ratios were empirically calculated on the basis of simulated life histories under different reflex testing strategies. RESULTS: Biopsying all men provided the most life years and QALYs, followed by reflex testing using MPShg, MPS, MRI, T2:ERG, PCA3, and biopsying no men (QALY range across strategies 15.98-16.09). Accounting for costs, MRI and MPShg were dominated by other strategies. PCA3, T2:ERG, and MPS were likely to be the most cost-effective strategy at willingness-to-pay thresholds of $100 000/QALY, $125 000/QALY, and $150 000/QALY, respectively. CONCLUSIONS: Using PCA3, T2:ERG, or MPS as reflex tests has greater economic value than MRI, biopsying all men, or biopsying no men with intermediate PSA levels.

Decision Analysis Model for Prehospital Triage of Patients With Acute Stroke.

Background and Purpose- We used a decision analysis approach to analyze triage strategies for patients with acute stroke symptoms while accounting for prehospital large vessel occlusion (LVO) screening methods and key time metrics. Methods- Our decision analysis compared anticipated functional outcomes for patients within the IV-tPA (intravenous tissue-type plasminogen activator) treatment window in the mothership and drip-and-ship frameworks. Key branches of the model included IV-tPA eligibility, presence of an LVO, and endovascular therapy eligibility. Our decision analysis evaluated 2 prehospital LVO screening approaches: (1) no formal screening and (2) the use of clinical LVO screening scales. An excellent outcome was defined as modified Rankin Scale scores 0-1. Probabilities and workflow times were guideline-based or imputed from published studies. In sensitivity analyses, we individually and jointly varied transport time to the nearest primary stroke center, additional time required to transport directly to a comprehensive stroke center, and LVO screening scale predictive probabilities. We evaluated 2 separate scenarios: one in which ideal time metrics were achieved and one under current real-world metrics. Results- In the ideal metrics scenario, the drip-and-ship strategy was almost always favored in the absence of formal LVO screening. For patients screened positive for an LVO, mothership was favored if the additional transport time to the comprehensive stroke center was <3 to 23 minutes. Under real-world conditions, in which primary stroke center workflow is slower than ideal, the mothership strategy was favored in more scenarios, regardless of formal LVO screening. For example, mothership was favored with an additional transport time to the comprehensive stroke center of <32 to 99 minutes for patients screened positive for an LVO and <28 to 39 minutes in the absence of screening. Conclusions- Joint consideration of LVO probability, screening, workflow times, and transport times may improve prehospital stroke triage. Drip-and-ship was more favorable when more ideal primary stroke center workflow times were modeled.

Cost-effectiveness of neighbourhood slow zones in New York City.

BACKGROUND: Neighbourhood slow zones (NSZs) are areas that attempt to slow traffic via speed limits coupled with other measures (eg, speed humps). They appear to reduce traffic crashes and encourage active transportation. We evaluate the cost-effectiveness of NSZs in New York City (NYC), which implemented them in 2011. METHODS: We examined the effectiveness of NSZs in NYC using data from the city's Department of Transportation in an interrupted time series analysis. We then conducted a cost-effectiveness analysis using a Markov model. One-way sensitivity analyses and Monte Carlo analyses were conducted to test error in the model. RESULTS: After 2011, road casualties in NYC fell by 8.74% (95% CI 1.02% to 16.47%) in the NSZs but increased by 0.31% (95% CI -3.64% to 4.27%) in the control neighbourhoods. Because injury costs outweigh intervention costs, NSZs resulted in a net savings of US$15 (95% credible interval: US$2 to US$43) and a gain of 0.002 of a quality-adjusted life year (QALY, 95% credible interval: 0.001 to 0.006) over the lifetime of the average NSZ resident relative to no intervention. Based on the results of Monte Carlo analyses, there was a 97.7% chance that the NSZs fall under US$50 000 per QALY gained. CONCLUSION: While additional causal models are needed, NSZs appeared to be an effective and cost-effective means of reducing road casualties. Our models also suggest that NSZs may save more money than they cost.

Optimising the cost-effectiveness of speed limit enforcement cameras.

BACKGROUND: Using the 140 speed cameras in New York City (NYC) as a case study, we explore how to optimise the number of cameras such that the most lives can be saved at the lowest cost. METHODS: A Markov model was built to explore the economic and health impacts of speed camera installations in NYC as well as the optimal number and placement. Both direct and indirect medical savings associated with speed cameras are weighed against their cost. Health outcomes are measured in terms of quality-adjusted life years (QALYs). RESULTS: Over the lifetime of an average NYC resident, the existing 140 speed cameras increase QALYs by 0.00044 units (95% credible interval (CrI) 0.00027 to 0.00073) and reduce costs by US$70 (95% CrI US$21 to US$131) compared with no speed cameras. The return on investment would be maximised where the number of cameras more than doubled to 300. This would further increase QALY gains per resident by 0.00083 units (95% CrI 0.00072 to 0.00096) while reducing medical costs by US$147 (95% CrI US$70 to US$221) compared with existing speed cameras. Overall, this increase in cameras would save 7000 QALYs and US$1.2 billion over the lifetime of the current cohort of New Yorkers. CONCLUSION: Speed cameras rank among the most cost-effective social policies, saving both money and lives.

Depressive Symptoms and Sexually Transmitted Disease: Evidence from a Low-Income Neighborhood of New York City.

We examined the association between sexually transmitted disease (STD) and depressive symptoms. Our analysis utilized the 2015 cross-sectional Washington Heights Community Survey. Multivariable binary logistic regression analysis was used to examine the primary association between having a history of STD and patient health questionnaire-9 (PHQ-9) score while adjusting for potential confounders. Then in separate models, we adjusted for the interaction of social factors with PHQ-9 score to test for modification effect on the primary association. In this low-income neighborhood, STD history was not significantly associated with PHQ-9 score in the overall logistic regression model for the primary association. However, in interaction models, STD and depressive symptoms were associated in sub-groups defined by social factors, namely being Hispanic [odds ratio (OR) 1.08; 95% confidence interval (CI) 1.02-1.15], foreign-born (OR 1.08; 95% CI 1.02-1.15), and having low to moderate social support (OR 1.09; 95% CI 1.02-1.15). Our results demonstrate a need for targeted interventions to be applied to vulnerable subgroups identified.

The Cost-Effectiveness of Bike Share Expansion to Low-Income Communities in New York City.

The "Citi Bike" bike share program in New York City is the largest bike share program in the USA. We ask whether expanding this program to lower-income communities is cost-effective means of encouraging exercise and reducing pollution in New York City. We built a stochastic Markov model to evaluate the cost-effectiveness of the Citi Bike expansion program, an effort to extend bike share to areas with higher costs and risks over a 10-year time horizon. We used one-way sensitivity analyses and Monte Carlo simulation to test the model uncertainty. The incremental cost-effectiveness ratio of the Citi Bike expansion program relative to the current program (status quo) was $7869/quality-adjusted life year gained. The Citi Bike expansion program in New York City offers good value relative to most health interventions.