Development of a Decision-Analytic Model for the Application of STR-Based Provenance Testing of Transrectal Prostate Biopsy Specimens

BackgroundThe diagnostic algorithm for most cancers includes the assessment of a tissue specimen by a surgical pathologist, but if specimen provenance is uncertain, the diagnostic and therapeutic process carries significant risk to the patient. Over the last decade, short tandem repeat (STR) analysis has emerged as a DNA-based method with clinical applicability for specimen identity testing (also known as specimen provenance testing). Although the clinical utility of identity testing using STR-based analysis has been demonstrated in many studies, its economic value has not been established.MethodsWe developed a decision-analytic model of the application of STR-based provenance testing of transrectal prostate biopsy specimens obtained as part of routine clinical care to rule out the presence of adenocarcinoma of the prostate, as compared with no STR-based testing. Using parameter values drawn from the published literature, the cost-effectiveness of STR-based testing was quantified by calculating the incremental cost-effectiveness ratio per quality-adjusted life-year gained.ResultsIn comparison to the current standard practice of no identity testing, identity testing by STR-based analysis has an incremental cost-effectiveness ratio of $65,570 per quality-adjusted life-year gained at a testing cost of $618 per person. At a cost of $515 per person, identity testing would meet the conservative standard of $50,000 per quality-adjusted life-year. At a test cost of $290 per person, identity testing would be cost saving.ConclusionGiven the rapidly declining pricing of STR-based identity testing, it is likely that testing to confirm the identity of positive prostate biopsy samples will be a cost-effective method for preventing treatment errors stemming from misidentification. Studies to formally establish the frequency of specimen provenance errors in routine clinical practice would therefore seem justified.     

Cost-Effectiveness of Telaprevir in Patients with Genotype 1 Hepatitis C in Australia

BackgroundProtease inhibitors such as telaprevir (Incivo) are reimbursed in Australia for the treatment of patients with genotype 1 hepatitis C virus (HCV) infection in combination with pegylated interferon and ribavirin (PR).ObjectivesTo assess the cost-effectiveness of telaprevir plus PR compared with PR alone in 1) previously untreated patients and 2) patients who had received treatment with PR earlier.MethodsSustained virological response rates and average treatment durations of telaprevir and PR (given with or without telaprevir) were taken from the telaprevir ADVANCE and REALIZE clinical trials but were modified to take account of differences in prescribing rules between the trials and Australian clinical practice. The probability of transitioning between Markov disease states was based on data from the Australian Kirby Institute where possible and supplemented using data from the published literature. Utility values obtained from the EuroQol five-dimensional questionnaire data collected in the ADVANCE and REALIZE trials were used to represent the utility during HCV treatment. Utility values for Markov health states were taken from the published literature. Unit costs (2014 AU $) were taken from Australian sources.ResultsIn treatment-naive patients, the discounted cost per life-years gained was AU $37,706 and the discounted cost per quality-adjusted life-year was AU $19,283. In treatment-experienced patients, the discounted cost per life-year gained was AU $23,855 and the discounted cost per quality-adjusted life-year was AU $14,948.ConclusionTelaprevir plus PR in the Australian setting is cost-effective when compared with PR alone in patients infected with genotype 1 HCV.     

The Influence of US Drug Price Dynamics on Cost-Effectiveness Analyses of Biologics

ObjectivesThis study aimed to evaluate the influence of drug price dynamics in cost-effectiveness analyses.MethodsWe evaluated scenarios involving typical US drug price increases during the exclusivity period and price decreases after the loss of exclusivity (LOE). Worked examples are presented using the Institute for Clinical and Economic Review’s assessments of tezepelumab for the treatment of severe asthma and targeted immune modulators for rheumatoid arthritis.ResultsTezepelumab case: yearly 2% price increases during the period of exclusivity and a post-LOE price decrease of 25% yielded an incremental cost per quality-adjusted life-year (QALY) gained that increased over the base case from $430 300 to $444 600 (+3.2%). Yearly 2% price increases followed by a steeper post-LOE price reduction of 40% resulted in a cost per QALY gained of $401 400 (6.8% reduction vs the base case). Rheumatoid arthritis case: incorporating post-LOE price reductions for etanercept (intervention) and adalimumab (comparator) ranging from 25% to 40% yielded an incremental cost per QALY of $121 000 and $122 300, respectively (< 3% increase from the base case of $119 200/QALY). Including a 2% yearly price increase during the projected exclusivity periods of both intervention and comparator increased the cost per QALY gained by > 60%.ConclusionTwo biologic treatment cases incorporating price dynamics in cost-effectiveness analyses had varied impacts on the cost-effectiveness ratio depending on the magnitude of pre-LOE price increase and post-LOE price decrease and whether the LOE also affected the comparator. Yearly price increase magnitude during the period of exclusivity, among other factors, may counterbalance the effects of lower post-LOE intervention prices.     

Value of Triage Treatment Strategies to Distribute Hepatitis C Direct-Acting Antiviral Agents in an Integrated Healthcare System: A Cost-Effectiveness Analysis

ObjectivesThis study aimed to assess the cost-effectiveness of fibrosis-based direct-acting antiviral treatment policies for patients with chronic hepatitis C virus at the Kaiser Permanente Mid-Atlantic States health system.MethodsWe used a Markov model to compare the lifetime costs and effects of treating patients with chronic hepatitis C virus at different stages of disease severity, or all stages simultaneously, based on a fibrosis score from the US healthcare sector perspective and societal perspective. The initial distribution of patients across fibrosis scores, the effectiveness of direct-acting antiviral therapy, and follow-up and monitoring protocols were specific to the Kaiser Permanente Mid-Atlantic States health system. Direct and indirect costs, transition probabilities, and utilities were derived from the literature. Deterministic and probabilistic sensitivity analyses were performed to assess the robustness of our results.ResultsThe “Treat All” option was dominant from both the societal and healthcare sector perspectives. The conclusion was robust in deterministic sensitivity analysis. The range of incremental costs between the less restrictive policies was small—the difference between the “Treat F1+” and the “Treat All” option was only $111 per person. Probabilistic sensitivity analyses showed, at both the $100 000/quality-adjusted life-year and $150 000/quality-adjusted life-year thresholds, there was a 70% chance that the “Treat All” option was more cost-effective than the “Treat F1+” option.ConclusionsWe found that expanded treatment access is cost-effective and, in many cases, cost saving. Although our results are primarily applicable to a regional integrated healthcare system, it offers some direction to any healthcare setting faced with resource constraints in the face of highly priced drugs.     

Economic Evaluation of Cinacalcet in the United States: The EVOLVE Trial

BackgroundPrevious economic evaluations of cinacalcet in patients with secondary hyperparathyroidism (sHPT) relied on the combination of surrogate end points in clinical trials and epidemiologic studies.ObjectivesThe objective was to conduct an economic evaluation of cinacalcet on the basis of the EValuation Of Cinacalcet HCl Therapy to Lower CardioVascular Events (EVOLVE) trial from a US payer perspective.MethodsWe developed a semi-Markov model to assess the cost-effectiveness of cinacalcet in addition to conventional therapy, compared with conventional therapy alone, in patients with moderate-to-severe sHPT receiving hemodialysis. We used treatment effect estimates from the unadjusted intent-to-treat (ITT) analysis and prespecified covariate-adjusted ITT analysis as our main analyses. We assessed model sensitivity to variations in individual inputs and overall decision uncertainty through probabilistic sensitivity analyses.ResultsThe incremental cost-effectiveness ratio (ICER) for cinacalcet was $61,705 per life-year and $79,562 per quality-adjusted life-year (QALY) gained using the covariate-adjusted ITT analysis. Probabilistic sensitivity analysis suggested a 73.2% chance of the ICER being below a willingness-to-pay threshold of $100,000. Treatment effects from unadjusted ITT analysis yielded an ICER of $115,876 per QALY. The model was most sensitive to the treatment effect on mortality.ConclusionsIn the unadjusted ITT analysis, cinacalcet does not represent a cost- effective use of health care resources when applying a willingness-to-pay threshold of $100,000 per QALY. When using the covariate-adjusted ITT treatment effect, which represents the least biased estimate, however, cinacalcet is a cost-effective therapy for patients with moderate-to-severe sHPT on hemodialysis.     

Linezolid Versus Vancomycin in the Empiric Treatment of Nosocomial Pneumonia: A Cost-Utility Analysis Incorporating Results from the ZEPHyR Trial

ObjectiveTo examine the cost-effectiveness of vancomycin versus linezolid in the empiric treatment of nosocomial pneumonias incorporating results from a recent prospective, double-blind, multicenter, controlled trial in adults with suspected methicillin-resistant Staphylococcus aureus (MRSA) nosocomial pneumonia.MethodsA decision-analytic model examining the cost-effectiveness of linezolid versus vancomycin for the empiric treatment of nosocomial pneumonia was created. Publicly available cost, efficacy, and utility data populated relevant model variables. A probabilistic sensitivity analysis varied parameters in 10,000 Monte-Carlo simulations, and univariate sensitivity analyses assessed the impact of model uncertainties and the robustness of our conclusions.ResultsResults indicated that the cost per quality-adjusted life-year (QALY) increased 6% ($22,594 vs. $23,860) by using linezolid versus vancomycin for nosocomial pneumonia. The incremental cost per QALY gained by using linezolid over vancomycin was $6,089, and the incremental cost per life saved was $68,615 with the use of linezolid. Vancomycin dominated linezolid in the subset of patients with documented MRSA. The incremental cost per QALY gained using linezolid if no mortality benefit exists between agents or a 60-day time horizon was analyzed was $19,608,688 and $443,662, respectively.ConclusionsLinezolid may be a cost-effective alternative to vancomycin in the empiric treatment of patients with suspected MRSA nosocomial pneumonia; however, results of our model were highly variable on a number of important variables and assumptions including mortality differences and time frame analyzed.     

Cost-Effectiveness of Gene-Expression Profiling for Tumor-Site Origin

ObjectivesGene-expression profiling (GEP) reliably supplements traditional clinicopathological information on the tissue of origin (TOO) in metastatic or poorly differentiated cancer. A cost-effectiveness analysis of GEP TOO testing versus usual care was conducted from a US third-party payer perspective.MethodsData on recommendation changes for chemotherapy, surgery, radiation therapy, blood tests, imaging investigations, and hospice care were obtained from a retrospective, observational study of patients whose physicians received GEP TOO test results. The effects of chemotherapy recommendation changes on survival were based on the results of trials cited in National Comprehensive Cancer Network and UpToDate guidelines. Drug and administration costs were based on average doses reported in National Comprehensive Cancer Network guidelines. Other unit costs came from Centers for Medicare & Medicaid Services fee schedules. Quality-of-life weights were obtained from literature. Bootstrap analysis estimated sample variability; probabilistic sensitivity analysis addressed parameter uncertainty.ResultsChemotherapy regimen recommendations consistent with guidelines for final tumor-site diagnoses increased significantly from 42% to 65% (net difference 23%; P<0.001). Projected overall survival increased from 15.9 to 19.5 months (mean difference 3.6 months; two-sided 95% confidence interval [CI] 3.2–3.9). The average increase in quality-adjusted life-months was 2.7 months (95% CI 1.5–4.3), and average third-party payer costs per patient increased by $10,360 (95% CI $2,982–$19,192). The cost per quality-adjusted life-year gained was $46,858 (95% CI $13,351–$104,269).ConclusionsGEP TOO testing significantly altered clinical practice patterns and is projected to increase overall survival, quality-adjusted life-years, and costs, resulting in an expected cost per quality-adjusted life-year of less than $50,000.     

An Empiric Estimate of the Value of Life: Updating the Renal Dialysis Cost-Effectiveness Standard

ObjectivesProposals to make decisions about coverage of new technology by comparing the technology's incremental cost-effectiveness with the traditional benchmark of dialysis imply that the incremental cost-effectiveness ratio of dialysis is seen a proxy for the value of a statistical year of life. The frequently used ratio for dialysis has, however, not been updated to reflect more recently available data on dialysis.MethodsWe developed a computer simulation model for the end-stage renal disease population and compared cost, life expectancy, and quality-adjusted life expectancy of current dialysis practice relative to three less costly alternatives and to no dialysis. We estimated incremental cost-effectiveness ratios for these alternatives relative to the next least costly alternative and no dialysis and analyzed the population distribution of the ratios. Model parameters and costs were estimated using data from the Medicare population and a large integrated health-care delivery system between 1996 and 2003. The sensitivity of results to model assumptions was tested using 38 scenarios of one-way sensitivity analysis, where parameters informing the cost, utility, mortality and morbidity, etc. components of the model were by perturbed +/?50%.ResultsThe incremental cost-effectiveness ratio of dialysis of current practice relative to the next least costly alternative is on average $129,090 per quality-adjusted life-year (QALY) ($61,294 per year), but its distribution within the population is wide; the interquartile range is $71,890 per QALY, while the 1st and 99th percentiles are $65,496 and $488,360 per QALY, respectively. Higher incremental cost-effectiveness ratios were associated with older age and more comorbid conditions. Sensitivity to model parameters was comparatively small, with most of the scenarios leading to a change of less than 10% in the ratio.ConclusionsThe value of a statistical year of life implied by dialysis practice currently averages $129,090 per QALY ($61,294 per year), but is distributed widely within the dialysis population. The spread suggests that coverage decisions using dialysis as the benchmark may need to incorporate percentile values (which are higher than the average) to be consistent with the Rawlsian principles of justice of preserving the rights and interests of society's most vulnerable patient groups.     

Adding a quadrivalent human papillomavirus vaccine to the UK cervical cancer screening programme: A cost-effectiveness analysis

Background

Intravitreal ranibizumab prevents vision loss and improves visual acuity in patients with neovascular age-related macular degeneration, but it is expensive, and efficacy beyond 2 years is uncertain.

Methods

We assessed the cost-effectiveness of ranibizumab compared with no ranibizumab over 10 years, using randomized trial efficacy data for the first 2 years, post-trial efficacy assumptions, and ranibizumab acquisition costs ranging from the wholesale price ($1,950 per dose) to the price of bevazicumab ($50), a similar molecule which may be equally efficacious. We used a computer simulation model to estimate the probability of blindness, the number of quality-adjusted life-years (QALYs), direct costs (in 2004 U.S. dollars), and cost-effectiveness ratios for a 67-year old woman. Costs and QALYs were discounted at 3% per year.

Results

The probability of blindness over 10 years was reduced from 56% to 34% if ranibizumab was efficacious for only 2 years, 27% if efficacy was maintained for a further 2 years only (base-case scenario), and 17% if visual acuity at 4 years was then sustained. It was cost-saving under all price assumptions, when caregiver costs were included. When caregiver costs were excluded, the cost per QALY for the base-case ranged from $5,600, assuming the bevazicumab price, to $91,900 assuming the wholesale ranibizumab price. The cost per QALY was < $50,000 when the cost of ranibizumab was less than $1000.

Conclusion

From a societal perspective, ranibizumab was cost-saving. From a health care funder's perspective, ranibizumab was an efficient treatment when it cost less than $1000 per dose.     

Cost-Effectiveness Analysis of Vaccination With Recombinant Zoster Vaccine Among Hematopoietic Cell Transplant Recipients and Persons With Other Immunocompromising Conditions Aged 19 to 49 Years

ObjectivesThis study aimed to estimate the cost-effectiveness of the use of recombinant zoster vaccine (RZV) (Shingrix), which protects against herpes zoster (HZ), among immunocompromised adults aged 19 to 49 years, as a contribution to deliberations of the Advisory Committee on Immunization Practices.MethodsHematopoietic cell transplant (HCT) recipients experience a high incidence of HZ, and the efficacy of RZV in preventing HZ has been studied in clinical trials. The cost-effectiveness model calculated incremental cost-effectiveness ratios that compared vaccination with RZV with a no vaccination strategy among adults aged 19 to 49 years. Costs and outcomes were calculated until age 50 years using the healthcare sector perspective and summarized as cost per quality-adjusted life-year (QALY) gained. The base case represents HCT recipients, with scenario analyses representing persons with other immunocompromising conditions, including hematologic malignancies, human immunodeficiency virus, and autoimmune and inflammatory conditions. Uncertainty was investigated using univariate, multivariate, and probabilistic sensitivity analyses.ResultsBase-case results indicated vaccination with RZV would avert approximately 35% of HZ episodes and complications, while saving approximately 11% of net costs. Compared with no vaccination, vaccination of HCT recipients with RZV generated cost-savings (ie, lower costs and improved health) in the base case and in 81% of simulations in the probabilistic analysis. In scenario analyses, vaccination cost US dollar ($) 9500/QALY among patients with hematologic malignancies, $79 000/QALY among persons living with human immunodeficiency virus, and $208 000/QALY among persons with selected autoimmune and inflammatory conditions.ConclusionsGenerally favorable economic estimates supported recommendations for vaccination of immunocompromised adults with RZV to prevent episodes of HZ and related complications.     

Cost-Effectiveness Analysis of Transcatheter Arterial Embolization Techniques for the Treatment of Gastrointestinal Bleeding in the United States

ObjectivesGastrointestinal (GI) bleeding is a common medical emergency associated with significant mortality. Transcatheter arterial embolization first was introduced by Rosch et al as an alternative to surgery for upper GI bleeding. The clinical success in patients with GI bleeding treated with transcatheter arterial embolization previously has been reported. However, there are no cost-effectiveness analyses reported to date. Here we report cost-effectiveness analysis of N-butyl 2-cyanoacrylate glue (NBCA) and ethylene-vinyl alcohol copolymer (Onyx) versus coil (gold standard) for treatment of GI bleeding from a healthcare payer perspective.MethodsFixed-effects modeling with a generalized linear mixed method was used in NBCA and coil intervention arms to determine the pooled probabilities of clinical success and mortality with complications with their confidence intervals, while the Clopper-Pearson model was used for Onyx to determine the same parameters. Models were provided by the “Meta-Analysis with R” software package. A decision tree was built for cost-effectiveness analysis, and Microsoft Excel was used for probabilistic sensitivity analysis. The cost-effective option was determined based on the incremental cost-effectiveness ratio and scatter plots of incremental cost versus incremental quality-adjusted life-years.ResultsComparing scatter plots and incremental cost-effectiveness ratio results, –$1024 and –$1349 per quality-adjusted life-year for Onyx and N-butyl 2-cyanoacrylate glue, respectively, Onyx was the least expensive and most effective intervention.ConclusionOnyx was the dominant strategy regardless of threshold values. Our analyses provide a framework for researchers to predict the target clinical effectiveness for early-stage TAE interventions and guide resource allocation decisions.     

Cost-utility analysis of hepatitis A prevention among health-care workers in Israel

This study was conducted to evaluate the efficiency of different strategies of preventing hepatitis A (HA) among physicians, nurses, and paramedical staff by means of cost-effectiveness analysis. The strategies compared were passive immunization during a hepatitis A outbreak, systematic mass vaccination of all workers, and screening for antibodies to HA virus followed by vaccination of nonimmune employees. To predict the prevented number of HA cases, an epidemiological model was incorporated based on our previous nationwide study on clinical HA cases, seroepidemiological survey, and other scientific medical literature. Under the model assumptions, the lowest cost per prevented HA case ($6240) was achieved by screening prior vaccination among 18- to 39-year-old physicians and paramedical workers, and highest ($61,858) by mass vaccination of nurses over age 39 years. Because the price per prevented case may show that mass vaccination is highly cost-effective, especially among 18- to 39-year-old physicians and paramedical workers ($6399 and $8196, respectively), the cost per gained quality-adjusted life-year seems less attractive ($56,532 and $61,350, respectively). This cost per saved quality-adjusted life-year is similar to many other medical interventions but is markedly higher than other primary prevention vaccines, which are mostly associated with a negative ratio. In view of the conducted study, and taking $60,000 as a limit cost per saved quality-adjusted life-year, we propose selective vaccination for physicians and for paramedical workers. Mass vaccination should be offered to all health care workers, aside from nurses 40+ years of age, once the active HA vaccine price is reduced to $23.     

International survey on willingness-to-pay (WTP) for one additional QALY gained: what is the threshold of cost effectiveness?

Although the threshold of cost effectiveness of medical interventions is thought to be £20 000–£30 000 in the UK, and $50 000–$100 000 in the US, it is well known that these values are unjustified, due to lack of explicit scientific evidence. We measured willingness-to-pay (WTP) for one additional quality-adjusted life-year gained to determine the threshold of the incremental cost-effectiveness ratio. Our study used the Internet to compare WTP for the additional year of survival in a perfect status of health in Japan, the Republic of Korea (ROK), Taiwan, Australia, the UK, and the US. The research utilized a double-bound dichotomous choice, and analysis by the nonparametric Turnbull method. WTP values were JPY 5 million (Japan), KWN 68 million (ROK), NT$ 2.1 million (Taiwan), £23 000 (UK), AU$ 64 000 (Australia), and US$ 62 000 (US). The discount rates of outcome were estimated at 6.8% (Japan), 3.7% (ROK), 1.6% (Taiwan), 2.8% (UK), 1.9% (Australia), and 3.2% (US). Based on the current study, we suggest new classification of cost-effectiveness plane and methodology for decision making. Copyright © 2009 John Wiley & Sons, Ltd.     

Cost-Effectiveness of a Program to Eliminate Disparities in Pneumococcal Vaccination Rates in Elderly Minority Populations: An Exploratory Analysis

ObjectiveInvasive pneumococcal disease is a major cause of preventable morbidity and mortality in the United States, particularly among the elderly (>65 years). There are large racial disparities in pneumococcal vaccination rates in this population. Here, we estimate the cost-effectiveness of a hypothetical national vaccination intervention program designed to eliminate racial disparities in pneumococcal vaccination in the elderly.MethodsIn an exploratory analysis, a Markov decision-analysis model was developed, taking a societal perspective and assuming a 1-year cycle length, 10-year vaccination program duration, and lifetime time horizon. In the base-case analysis, it was conservatively assumed that vaccination program promotion costs were $10 per targeted minority elder per year, regardless of prior vaccination status and resulted in the elderly African American and Hispanic pneumococcal vaccination rate matching the elderly Caucasian vaccination rate (65%) in year 10 of the program.ResultsThe incremental cost-effectiveness of the vaccination program relative to no program was $45,161 per quality-adjusted life-year gained in the base-case analysis. In probabilistic sensitivity analyses, the likelihood of the vaccination program being cost-effective at willingness-to-pay thresholds of $50,000 and $100,000 per quality-adjusted life-year gained was 64% and 100%, respectively.ConclusionsIn a conservative analysis biased against the vaccination program, a national vaccination intervention program to ameliorate racial disparities in pneumococcal vaccination would be cost-effective.     

玻璃体腔注射曲安奈德治疗黄斑水肿的临床观察

[目的]观察玻璃体腔注射曲安奈德治疗黄斑水肿的疗效。[方法]对72例(78只眼)黄斑水肿患者进行曲安奈德玻璃体腔注射治疗,对比分析术前及术后6个月不同时间点的最佳矫正视力、眼底及黄斑中心视网膜厚度、眼压等。[结果]术后最佳矫正视力有显著提高,黄斑水肿明显减轻,黄斑中心视网膜厚度显著降低,但也有少数患者发生高眼压、黄斑水肿复发等并发症。[结论]曲安奈德玻璃体腔注射是治疗黄斑水肿的有效方法,但也应注意并发症的发生。     

Cost-Effectiveness of the Addition of Rituximab to CHOP Chemotherapy in First-Line Treatment for Diffuse Large B-Cell Lymphoma in a Population-Based Observational Cohort in British Columbia,Canada

BackgroundDiffuse large B-cell lymphoma (DLBCL) has primarily been treated with cyclophosphamide, doxorubicin, vincristine, and predisone (CHOP) chemotherapy since the 1970s. Recently, the addition of rituximab to CHOP (CHOP-R) has been found to improve survival and trial-based results have suggested that it is a cost-effective alternative to CHOP.ObjectivesThe objective in this study was to evaluate the cost-effectiveness of CHOP-R relative to CHOP in first-line treatment of DLBCL in a population-based setting in British Columbia, Canada.MethodsWe created a patient-level simulation model describing potential pathways for DLBCL patients initiating treatment with either CHOP or CHOP-R. Model parameters were populated with statistical analyses of individual-level treatment and effectiveness data and published cost estimates. All results were stratified by age at treatment initiation (<60 years vs. ≥60 years). The base-case scenario was based on a 15-year time horizon and a 3% discount rate. Probabilistic sensitivity analysis was performed. All costs are reported as 2006 $CDN.ResultsFor the base-case scenario, incremental cost-effectiveness ratios (ICERs) for younger individuals ranged from $11,965 per disease-free life-year gained to $19,144 per quality-adjusted life-year gained. For older individuals, estimated ICERs for all health outcomes were below $10,000 per unit outcome gained for a 15-year time horizon.ConclusionsUsing population-based data, CHOP-R was found to be a cost-effective alternative to CHOP, particularly for individuals aged 60 years and older. Results from this Canadian observational data source were consistent with international clinical trial-based studies. The use of CHOP-R as a first-line treatment for DLBCL is recommended, with respect to both clinical and cost-effectiveness.     

A New Type 2 Diabetes Microsimulation Model to Estimate Long-Term Health Outcomes,Costs, and Cost-Effectiveness

ObjectivesThis study aimed to develop a microsimulation model to estimate the health effects, costs, and cost-effectiveness of public health and clinical interventions for preventing/managing type 2 diabetes.MethodsWe combined newly developed equations for complications, mortality, risk factor progression, patient utility, and cost—all based on US studies—in a microsimulation model. We performed internal and external validation of the model. To demonstrate the model’s utility, we predicted remaining life-years, quality-adjusted life-years (QALYs), and lifetime medical cost for a representative cohort of 10 000 US adults with type 2 diabetes. We then estimated the cost-effectiveness of reducing hemoglobin A1c from 9% to 7% among adults with type 2 diabetes, using low-cost, generic, oral medications.ResultsThe model performed well in internal validation; the average absolute difference between simulated and observed incidence for 17 complications was < 8%. In external validation, the model was better at predicting outcomes in clinical trials than in observational studies. The cohort of US adults with type 2 diabetes was projected to have an average of 19.95 remaining life-years (from mean age 61), incur $187 729 in discounted medical costs, and accrue 8.79 discounted QALYs. The intervention to reduce hemoglobin A1c increased medical costs by $1256 and QALYs by 0.39, yielding an incremental cost-effectiveness ratio of $9103 per QALY.ConclusionsUsing equations exclusively derived from US studies, this new microsimulation model achieves good prediction accuracy in US populations. The model can be used to estimate the long-term health impact, costs, and cost-effectiveness of interventions for type 2 diabetes in the United States.     

Public Health Impact and Cost-Effectiveness of Hepatitis A Vaccination in the United States: A Disease Transmission Dynamic Modeling Approach

ObjectiveTo assess the population-level impact and cost-effectiveness of hepatitis A vaccination programs in the United States.MethodsWe developed an age-structured population model of hepatitis A transmission dynamics to evaluate two policies of administering a two-dose hepatitis A vaccine to children aged 12 to 18 months: 1) universal routine vaccination as recommended by the Advisory Committee on Immunization Practices in 2006 and 2) Advisory Committee on Immunization Practices’s previous regional policy of routine vaccination of children living in states with high hepatitis A incidence. Inputs were obtained from the published literature, public sources, and clinical trial data. The model was fitted to hepatitis A seroprevalence (National Health and Nutrition Examination Survey II and III) and reported incidence from the National Notifiable Diseases Surveillance System (1980–1995). We used a societal perspective and projected costs (in 2013 US $), quality-adjusted life-years, incremental cost-effectiveness ratio, and other outcomes over the period 2006 to 2106.ResultsOn average, universal routine hepatitis A vaccination prevented 259,776 additional infections, 167,094 outpatient visits, 4781 hospitalizations, and 228 deaths annually. Compared with the regional vaccination policy, universal routine hepatitis A vaccination was cost saving. In scenario analysis, universal vaccination prevented 94,957 infections, 46,179 outpatient visits, 1286 hospitalizations, and 15 deaths annually and had an incremental cost-effectiveness ratio of $21,223/quality-adjusted life-year when herd protection was ignored.ConclusionsOur model predicted that universal childhood hepatitis A vaccination led to significant reductions in hepatitis A mortality and morbidity. Consequently, universal vaccination was cost saving compared with a regional vaccination policy. Herd protection effects of hepatitis A vaccination programs had a significant impact on hepatitis A mortality, morbidity, and cost-effectiveness ratios.     

Cost-Effectiveness of Systemic Treatments for Metastatic Castration-Sensitive Prostate Cancer: An Economic Evaluation Based on Network Meta-Analysis

ObjectivesTo assess the cost-effectiveness of systemic treatments for metastatic castration-sensitive prostate cancer from the US healthcare sector perspective with a lifetime horizon.MethodsWe built a partitioned survival model based on a network meta-analysis of 7 clinical trials with 7287 patients aged 36 to 94 years between 2004 and 2018 to predict patient health trajectories by treatment. We tested parameter uncertainties with probabilistic sensitivity analyses. We estimated drug acquisition costs using the Federal Supply Schedule and adopted generic drug prices when available. We measured cost-effectiveness by an incremental cost-effectiveness ratio (ICER).ResultsThe mean costs were approximately $392 000 with androgen deprivation therapy (ADT) alone and approximately $415 000, $464 000, $597 000, and $959 000 with docetaxel, abiraterone acetate, enzalutamide, and apalutamide, added to ADT, respectively. The mean quality-adjusted life-years (QALYs) were 3.38 with ADT alone and 3.92, 4.76, 3.92, and 5.01 with docetaxel, abiraterone acetate, enzalutamide, and apalutamide, added to ADT, respectively. As add-on therapy to ADT, docetaxel had an ICER of $42 069 per QALY over ADT alone; abiraterone acetate had an ICER of $58 814 per QALY over docetaxel; apalutamide had an ICER of $1 979 676 per QALY over abiraterone acetate; enzalutamide was dominated. At a willingness to pay below $50 000 per QALY, docetaxel plus ADT is likely the most cost-effective treatment; at any willingness to pay between $50 000 and $200 000 per QALY, abiraterone acetate plus ADT is likely the most cost-effective treatment.ConclusionsThese findings underscore the value of abiraterone acetate plus ADT given its relative cost-effectiveness to other systemic treatments for metastatic castration-sensitive prostate cancer.     

A Cost-Effectiveness Analysis of Remdesivir for the Treatment of Hospitalized Patients With COVID-19 in England and Wales

ObjectivesCOVID-19 is associated with significant morbidity and mortality. This study aims to synthesize evidence to assess the cost-effectiveness of remdesivir (RDV) for the treatment of hospitalized patients with COVID-19 in England and Wales.MethodsA probabilistic cost-effectiveness analysis was conducted informed by 2 large trials and uses a partitioned survival approach to assess short- and long-term clinical consequences and costs associated with COVID-19 in a hypothetical cohort of hospitalized patients requiring supplemental oxygen at the start of treatment. Given that it is uncertain whether RDV reduces death, 2 analyses are presented, assuming RDV either reduces death or does not. Published sources were used for long-term clinical, quality of life, and cost parameters.ResultsUnder the assumption that RDV reduces death, the incremental cost-effectiveness ratio for RDV is estimated at £11 881 per quality-adjusted life-year gained compared with standard of care (SoC) (probabilistic incremental cost-effectiveness ratio £12 400). The probability for RDV to be cost-effective is 74% at a willingness-to-pay threshold of £20 000 per quality-adjusted life-year gained. RDV was no longer cost-effective when the hazard ratio for overall survival compared with SoC was >0·915.ConclusionsResults from this study suggest that using RDV for the treatment of hospitalized patients with COVID-19 is likely to represent a cost-effective use of National Health Service resources at current willingness-to-pay threshold in England and Wales, only if it prevents death. Results needs to be interpreted caution as vaccination was introduced and the SoC and evidence available have also evolved considerably since the analysis is conducted.