Coronary artery bypass surgery is still cost-effective: using calibrated models to update clinical trials.
Explore the source record for details and available documents.
Angioplasty, Balloon, Coronary↗
Biomedical subjects
Milton C Weinstein
Publications and source records attributed to Milton C Weinstein.
Cost-effectiveness of treatment for chronic hepatitis C infection in an evolving patient population.
CONTEXT: Approximately 2.7 million US individuals are chronically infected with the hepatitis C virus (HCV). As public health campaigns are pursued, a growing number of treatment candidates are likely to have minimal evidence of liver damage. OBJECTIVE: To examine the clinical benefits and cost-effectiveness of newer treatments for chronic hepatitis C infection in a population of asymptomatic, HCV sero-positive but otherwise healthy individuals. DESIGN AND SETTING: Cost-effectiveness analysis using a Markov model of the natural history of HCV infection and impact of treatment. We used an epidemiologic model to derive a range of natural history parameters that were empirically calibrated to provide a good fit to observed data on both prevalence of HCV seropositivity and time trends in outcomes related to HCV infection. PATIENTS: Cohorts of 40-year-old men and women with elevated levels of alanine aminotransferase, positive results on quantitative HCV RNA assays and serologic tests for antibody to HCV, and no histological evidence of fibrosis on liver biopsy. INTERVENTIONS: Monotherapy with standard or pegylated interferon alfa-2b; combination therapy with standard or pegylated interferon plus ribavirin. MAIN OUTCOME MEASURES: Lifetime costs, life expectancy, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios. RESULTS: The probability of patients with chronic HCV developing cirrhosis over a 30-year period ranged from 13% to 46% for men and from 1% to 29% for women. The incremental cost-effectiveness of combination therapy with pegylated interferon for men ranged from 26 000 dollars to 64 000 dollars per QALY for genotype 1 and from 10 000 dollars to 28 000 dollars per QALY for other genotypes; and for women ranged from 32 000 dollars to 90 000 dollars for genotype 1 and from 12 000 dollars to 42 000 dollars for other genotypes. Because the benefits of treatment were realized largely in the form of improvements in health-related quality of life, rather than prolonged survivorship, cost-effectiveness ratios expressed as dollars per year of life were substantially higher. Results were most sensitive to assumptions about the gains and decrements in health-related quality of life associated with treatment. CONCLUSIONS: While newer treatment options for hepatitis C appear to be reasonably cost-effective on average, these results vary widely across different patient subgroups and depend critically on quality-of-life assumptions. As the pool of persons eligible for treatment for HCV infection expands to the more general population, it will be imperative for patients and their physicians to consider these assumptions in making individual-level treatment decisions.
Adult↗
Daily cost prediction model in neonatal intensive care.
OBJECTIVES: One barrier to economic evaluation alongside neonatal randomized controlled trials is the expense of collecting detailed patient resource information. To reduce this data collection burden, we identified the key resource items that predict daily ancillary costs for extremely low birth weight infants. METHODS: Participants were 385 infants enrolled in the Trial of Indomethacin Prophylaxis for Preterms in nine tertiary level neonatal intensive care units in Canada. Information on eighty-nine nonpersonnel resource items was abstracted from the hospital chart from admission to tertiary hospital discharge. Unit costs were derived from a provincially standardized cost accounting system. Using stepwise linear regression, models correlating total daily ancillary costs with key resource items were constructed for each of five periods of admission. Models were derived in a randomly split half of the total sample of patient days and validated against the remainder. RESULTS: The 385 infants contributed resource information from 23,354 admission days. The regression model for weeks one to twelve included the covariates surfactant, chest radiograph, red blood cell transfusion, cranial ultrasound, abdominal radiograph, parenteral amino acid infusion, surgery, platelet transfusion, and echocardiogram and explained 91% of the variability in daily nonpersonnel costs (P<.0001). Models for other admission periods similarly included between four and eight covariates, were highly significant (P<.0001) and explained between 76% and 94% of daily ancillary cost variability. The regression equations showed excellent predictive power when applied to the second half of the patient data set. CONCLUSIONS: Daily nonpersonnel costs for extremely low birth weight infants are driven by a limited number of key resource variables. The ability to predict total ancillary costs with minimal data collection will facilitate inclusion of economic evaluations in neonatal trials.
Ancillary Services, Hospital↗
Cost-effectiveness of hepatic metastasectomy in patients with metastatic colorectal carcinoma: a state-transition Monte Carlo decision analysis.
OBJECTIVE: To evaluate the cost-effectiveness of hepatic resection ("metastasectomy") in patients with metachronous liver metastases from colorectal carcinoma (CRC), and to investigate the impact of operative and follow-up strategies on outcomes, cost, and cost-effectiveness. SUMMARY BACKGROUND DATA: There is substantial evidence that resection of CRC liver metastases can result in long-term survival in some patients. However, several unresolved issues are difficult to address using currently available clinical data. These include the appropriate threshold for resection, whether to perform repeat resection, and the relative cost-effectiveness of the procedure(s). METHODS: The authors developed a state-transition Monte Carlo decision model to evaluate the (societal) cost-effectiveness of hepatic metastasectomy in patients with metachronous CRC liver metastases. The model tracks the presence, number, size, location, growth, detection, and removal of up to 15 individual metastases in each patient. Survival, quality of life, and cost are predicted on the basis of disease extent. Imaging and surgery affect outcomes via detection and removal of individual metastases. Several patient management strategies were developed and compared with respect to cost, effectiveness, and incremental cost-effectiveness ($/quality-adjusted life year [QALY]). A reference strategy in which metastasectomy is not offered and imaging is not performed for the purpose of assessing resectability or operative planning ("no-surgery" strategy) was included for comparison. Extensive sensitivity analysis was performed to evaluate the impact of alternative model assumptions on results. RESULTS: A strategy permitting resection of up to six metastases and one repeat resection, with CT follow-up every 6 months, resulted in a gain of 2.63 QALYs relative to the no-test/no-treat strategy, at an incremental cost of 18,100 US dollars/QALY. When additional surgical strategies were considered, the incremental cost-effectiveness ratio (ICER; relative to the next least effective strategy) of the six metastases, one repeat, 6-month strategy was 31,700 US dollars/QALY. Across a range of model assumptions, more aggressive treatment strategies (i.e., resection of more metastases, resection of recurrent metastases) were superior to less aggressive strategies and had ICERs below 35,000 US dollars/QALY. Findings were insensitive to changes in most model parameters but somewhat sensitive to changes in surgery and treatment costs. CONCLUSIONS: Hepatic metastasectomy is a cost-effective option for selected patients with metachronous CRC metastases limited to the liver. When considering metastasectomy, more aggressive approaches are generally preferred to less aggressive approaches. Overall, surgeons should be encouraged to consider resection for all patients whose metastases can technically be removed.
Aged↗
Patient and community preferences for treatments and health states in multiple sclerosis.
OBJECTIVE: To examine preferences for treatments and health states for patients with relapsing-remitting MS and members of the community. METHODS: A survey was developed to evaluate health-related quality-of-life measures (utilities) for three treatments and six MS health states using a utility-elicitation software package, U-Titer II. Sixty-two MS patients at two large teaching hospitals in Boston, MA, and 67 members of the general community in San Diego, CA, completed the health-related quality-of-life survey using a computer. RESULTS: Assessment of quality of life decreased as disability level of MS health states increased for both respondent groups. Respondents rated less-disabled health states relatively highly (> 0.94 for patients and > 0.89 for community respondents). Quality-of-life measures for treatments in mean utilities ranged from 0.80 to 0.96. Patients assigned higher utilities for both MS health states and treatment states than community respondents; the ratings became more disparate as health states worsened. CONCLUSIONS: On average, respondents assigned utilities to currently available treatments for MS that are comparable to those of mild to moderate stages of the disease itself. These results underscore the importance of including preferences for health states and treatment alternatives in the decision to initiate treatment for individual patients or in the evaluation of effectiveness or cost-effectiveness of these treatments in patients with MS.
Adult↗
Cost-effectiveness analysis of predischarge monitoring for apnea of prematurity.
OBJECTIVE: It is standard practice to defer discharge of premature infants until they have achieved a set number of days without experiencing apnea. The duration of this period, however, is highly variable across institutions, and there is scant literature on its effectiveness or value-for-money. Our objective was to establish the economic impact of varying durations of predischarge observation for apnea of prematurity. METHODS: Using computer simulation, we compared the alternatives of hospital monitoring for 1 to 10 days, after apparent cessation of apnea, with no monitoring and with the next longest period of monitoring. The daily probability of apnea requiring stimulation after a given number of apnea-free days was obtained from chart review of 216 infants, beginning on the day they attained both full feeds and temperature stability in an open crib. Baseline rates of survival or impairment, utilities for calculation of quality-adjusted life years (QALYs), outcomes for respiratory arrest at home, and long-run costs for neurodevelopmental impairment were derived from the literature. Hospital expenditures were obtained from itemized billing records for infants on each of the final 10 days of hospitalization and converted to costs using Medicare cost-to-charge ratios. Costs are reported in 2000 US dollars. RESULTS: For infants born at 24 to 26 weeks' gestation, each additional day of monitoring cost from $41000 per QALY saved for the first day to >$130000 per additional QALY gained for the tenth day. Cost-effectiveness was poorer for infants who were born at gestational ages >30 weeks. Results were sensitive to the proportion of charted apneas requiring stimulation that would actually progress, without intervention, to respiratory arrest. CONCLUSIONS: In this model, the cost-effectiveness of predischarge monitoring for apnea of prematurity declined significantly as the duration of monitoring was increased. Consideration should be given to alternative uses for resources in formulating neonatal discharge guidelines.
Apnea↗
Cost-effectiveness implications of the timing of antiretroviral therapy in HIV-infected adults.
BACKGROUND: The appropriate time to initiate antiretroviral therapy is controversial for human immunodeficiency virus (HIV)-infected patients with CD4 cell counts between 200/microL and 350/microL and low levels of HIV RNA, potentially leading to barriers to treatment access. OBJECTIVE: To examine the effect of cholesterol changes and fat redistribution symptoms on the clinical benefits and cost-effectiveness of early antiretroviral therapy in these patients. METHODS: We used a state-transition model to compare initiating antiretroviral therapy at CD4 cell counts of 350/microL (early therapy) with initiating therapy at CD4 cell counts of 200/microL (deferred therapy) in patients with HIV RNA levels of 10,000 to 30,000 copies/mL. Data were from randomized clinical trials, cohort studies, and other published literature. RESULTS: If cholesterol changes associated with antiretroviral therapy resulted in a permanent increase in coronary heart disease risk, life expectancy with early therapy was 16.54 years (vs 16.66 years without this risk) and with deferred therapy was 13.73 years (vs 13.80 years without this risk). Early therapy was a more efficient use of resources (ie, dominated) compared with deferred therapy. Early therapy cost $13,000 per quality-adjusted life-year compared with no therapy with or without increased coronary heart disease risk, and $17,000 to $24,000 per quality-adjusted life-year taking into account the quality-of-life reduction in patients with fat distribution symptoms. Early therapy had a higher quality-adjusted life expectancy than deferred therapy as long as this quality-of-life reduction was 70% or less. CONCLUSIONS: Changes in cholesterol or quality of life associated with antiretroviral therapy do not justify limiting access to early HIV treatment. The effect of fat redistribution symptoms on quality of life will determine the optimal choice of early vs deferred therapy for an individual patient.
AIDS-Related Opportunistic Infections↗
Empirically calibrated model of hepatitis C virus infection in the United States.
This study presents a comprehensive epidemiologic model of hepatitis C in the United States. Through empirical calibration of model parameter values, the objectives were to gain insights into uncertain aspects of the natural history of hepatitis C and to improve the basis for projecting the future course of the epidemic. A systematic review of the published literature was conducted to define plausible ranges around model parameters, and multiple simulations of the model were undertaken using sampled values from these ranges. Model predictions produced by each set of sampled values were compared with available epidemiologic data on infection prevalence and mortality from liver cancer, and various goodness-of-fit criteria were used to identify the range of parameter values that were consistent with these data. The results of the study indicate that rates of progression to advanced liver disease may be lower than previously assumed. The authors also found that a wide range of plausible assumptions about heterogeneity in these rates, beyond that explained by age and sex, is consistent with observed epidemiologic trends. These findings have important implications both for individual clinical decisions and for broader public health policy.
Adult↗
Treatment for primary HIV infection: projecting outcomes of immediate, interrupted, or delayed therapy.
With limited data available on the optimal treatment of primary HIV infection, disease modeling can be used to project clinical outcomes and inform decision makers. The authors developed a simulation model to evaluate the clinical outcomes and life expectancy projections for three primary HIV infection treatment strategies: 1) continuous antiretroviral therapy (ART) initiated at CD4 count <350 cells/mm(3); 2) continuous ART initiated immediately on diagnosis of primary HIV infection; and 3) ART initiated on diagnosis followed by structured treatment interruption. Projected life expectancies for the three strategies were 23.92, 24.46, and 26.07 years, respectively. The impact of key variables was assessed in sensitivity analysis, with the structured treatment interruption strategy remaining the most effective over a broad range of inputs. The immunologic benefit associated with immediate therapy and the potential for antiretroviral resistance due to structured treatment interruption have the most important impact on the optimal strategy. Based on current data, immediate treatment on diagnosis of primary HIV infection followed by structured treatment interruption will likely yield the best outcome. These results can assist decision makers and those planning clinical trials in defining evidence-based performance measures for primary HIV infection treatment and future trials.
Anti-HIV Agents↗
Prophylaxis for human immunodeficiency virus-related Pneumocystis carinii pneumonia: using simulation modeling to inform clinical guidelines.
BACKGROUND: Human immunodeficiency virus (HIV)-infected patients receiving highly active antiretroviral therapy (HAART) have experienced a dramatic decrease in Pneumocystis carinii pneumonia (PCP), necessitating reassessment of clinical guidelines for prophylaxis. METHODS: A simulation model of HIV infection was used to estimate the lifetime costs and quality-adjusted life expectancy (QALE) for alternative CD4 cell count criteria for stopping primary PCP prophylaxis in patients with CD4 cell count increases receiving HAART and alternative agents for second-line PCP prophylaxis in those intolerant of trimethoprim-sulfamethoxazole (TMP/SMX). The target population was a cohort of HIV-infected patients in the United States with initial CD4 cell counts of 350/microL who began PCP prophylaxis after their first measured CD4 lymphocyte count less than 200/microL. Data were from randomized controlled trials and other published literature. RESULTS: For patients with CD4 cell count increases during HAART, waiting to stop prophylaxis until the first observed CD4 cell count was greater than 300/microL prevented 9 additional cases per 1000 patients and cost $9400 per quality-adjusted life year (QALY) gained compared with stopping prophylaxis at 200/microL. For patients intolerant of TMP/SMX, using dapsone increased QALE by 2.7 months and cost $4500 per QALY compared with no prophylaxis. Using atovaquone rather than dapsone provided only 3 days of additional QALE and cost more than $1.5 million per QALY. CONCLUSIONS: Delaying discontinuation of PCP prophylaxis until the first observed CD4 cell count greater than 300/microL is cost-effective and provides an explicit "PCP prophylaxis stopping criterion." In TMP/SMX-intolerant patients, dapsone is more cost-effective than atovaquone.
AIDS-Related Opportunistic Infections↗
Impact of referral patterns on the use of chemotherapy for lung cancer.
PURPOSE: To determine the extent to which unexplained variation in the use of chemotherapy for advanced lung cancer is due to access to oncologists' services as opposed to treatment decisions made after seeing an oncologist. METHODS: We performed a retrospective cohort study of 12,015 patients over age 65 diagnosed with metastatic lung cancer between 1991 and 1996 while living in one of 11 regions monitored by a Survival, Epidemiology, and End Results (SEER) tumor registry. Assessment by an oncologist and subsequent treatment with chemotherapy were determined by examining linked Medicare claims. RESULTS: Of patients who did not receive chemotherapy, 36% were never assessed by a physician who provides chemotherapy. Patients living in certain areas, those diagnosed in more recent years, and those who received care in a teaching hospital were all more likely to see a cancer specialist. These factors were unrelated to subsequent treatment decisions, however. Conversely, age and comorbidity did not have a significant effect on whether a patient was seen by an oncologist, but they were associated with the likelihood of subsequently receiving chemotherapy. Black race, probably acting as a proxy for lower socioeconomic status, was associated with both a diminished likelihood of seeing a cancer specialist and subsequently receiving chemotherapy. CONCLUSION: Nonmedical factors are important determinants of whether a lung cancer patient is seen by a physician who provides chemotherapy. After seeing such a physician, treatment decisions seem to be mostly explained by appropriate medical factors. Racial and socioeconomic disparities still exist at both steps, however. As therapeutic options expand, referring physicians must ensure that biases and barriers to care do not deprive patients of the opportunity to consider all of their treatment options.
Black or African American↗
Outcomes and costs of acute treatment of traumatic brain injury.
BACKGROUND: Although there are nearly a quarter of a million hospitalizations for traumatic brain injury (TBI) in the United States each year, data on the outcomes and costs of TBI treatment in the acute-care setting are limited. METHODS: Using a large, geographically diverse, multihospital database, we examined inpatient records for persons aged 16 years or older who were hospitalized for TBI between January 1, 1997, and June 30, 1999. Patients were stratified by TBI severity using an adaptation of the Abbreviated Injury Scale for administrative data (ICD/AIS), as follows: 2 = "moderate"; 3 = "serious"; 4 = "severe"; and 5 = "critical." Patient characteristics, patterns of treatment, and outcomes and costs were examined by injury severity and mechanism of injury. RESULTS: Of 8,717 study subjects identified, 12.5% had moderate, 44.8% had serious, 29.6% had severe, and 13.2% had critical TBI. Falls were the most common reported cause of injury (40.8%), followed by motor vehicle crashes (39.3%), blows to the head (11.3%), and gunshot wounds (2.4%). Average length of stay in hospital ranged from 6.7 days for moderate TBI to 17.5 days for critical TBI. The overall rate of death in hospital was relatively low among patients with moderate (1.3%), serious (5.7%), and severe (8.7%) TBIs, but much higher among the most critically injured patients (52.0%). Costs of hospitalization averaged 8,189 dollars for moderate, 14,603 dollars for serious, 16,788 dollars for severe, and 33,537 dollars for critical TBI. Costs also varied by injury type, averaging 20,084 dollars for gunshot wounds, 20,522 dollars for motor vehicle crashes, 15,860 dollars for falls, and 19,949 dollars for blows to the head. CONCLUSION: The economic burden of TBI in the acute-care setting is substantial; treatment outcomes and costs vary considerably by TBI severity and mechanism of injury.
Adolescent↗
Emerging issues in vaccine economics: perspectives from the USA.
Economic studies of vaccines, including vaccine development and delivery issues, are increasingly needed to inform policy recommendations and programmatic decisions in the USA. This need arises from the increasing costs of vaccines, the complexity of the US healthcare system and the limited number of vaccine manufacturers in the market. We have developed a national research agenda in domestic and global vaccine economics by conducting key informant interviews with 42 experts and inviting ideas from an additional 128 experts. To assess priorities among the 129 ideas that were generated, we asked 15 experts representing a broad range of perspectives to rank the ideas and we analyzed their votes. The highest-ranking domestic research ideas included evaluating: the costs of vaccine shortages, the cost-effectiveness of potential human papillomavirus vaccination and adult and adolescent pertussis vaccination programs and the cost-effectiveness of universal vaccine purchase programs for adults as well as children. The highest-ranking globally-oriented ideas included developing a resource allocation model to support the best vaccination program decisions with limited funds and assessing the cost-effectiveness of HIV, rotavirus, meningococcal and malaria vaccines in developing countries. To optimize the usefulness of vaccine economics research, conceptual issues, such as how to set values for the prevention of illness and how to maximize social equity through investments in vaccines, must be addressed.
Adult↗
Lifetime cost of HIV care in France during the era of highly active antiretroviral therapy.
OBJECTIVE: To estimate the treatment and health care costs of HIV infection or AIDS in France during the era of highly active antiretroviral therapy (HAART). DESIGN: We used a clinical database of HIV-infected patients to calculate the resource use and cost of care for different stages of HIV infection. Costs were incorporated into a computer-based, probabilistic simulation model of the natural history and treatment of HIV infection to estimate the lifetime cost of treating patients with HIV disease. SETTING: A northern France HIV clinical cohort. PARTICIPANTS: 1232 HIV-infected patients followed from January 1994 through July 1998. RESULTS: In the absence of an AIDS-defining event, the average total cost of care ranged from 670 euros (1 euro=US $1.19) per person-month in the highest CD4 stratum (>500/microl) to 1060 euros per person-month in the lowest CD4 stratum (< or = 50/microl). The mean cost of care was estimated at 3370 euros per person-month during the initial months around the occurrence of an AIDS-defining event; at 1750 euros per person-month during the period spanning from 2 months after the diagnosis of specific AIDS-defining event to 1 month prior to death; and at 13010 euros per person-month in the final month prior to death. If clinical management of HIV infection began at a CD4 cell count of 378/microl, as in this cohort, the discounted lifetime cost of treating an HIV-infected French patient was estimated at 214000 euros. The undiscounted costs were 309000 euros over a projected life expectancy of 16.4 years. CONCLUSION: The cost of HIV disease varies widely depending upon the stage of illness. These estimates of stage-specific and lifetime costs of HIV care will assist health policy planners in assessing the burden of disease in the era of HAART and projecting future resource requirements.
Adult↗
Long-term persistence in use of statin therapy in elderly patients.
CONTEXT: Knowledge of long-term persistence with 3-hydroxy-3-methylglutaryl coenzyme A reductase inhibitor (statin) therapy is limited because previous studies have observed patients for short periods of time, in closely monitored clinical trials, or in other unrepresentative settings. OBJECTIVE: To describe the patterns and predictors of long-term persistence with statin therapy in an elderly US population. DESIGN, SETTING, AND PATIENTS: Retrospective cohort study including 34 501 enrollees in the New Jersey Medicaid and Pharmaceutical Assistance to the Aged and Disabled programs who were 65 years of age and older, initiated statin treatment between 1990 and 1998, and who were followed up until death, disenrollment, or December 31, 1999. MAIN OUTCOME MEASURES: Proportion of days covered (PDC) by a statin in each quarter during the first year of therapy and every 6 months thereafter; predictors of suboptimal persistence during each interval (PDC <80%) were identified using generalized linear models for repeated measures. RESULTS: The mean PDC was 79% in the first 3 months of treatment, 56% in the second quarter, and 42% after 120 months. Only 1 patient in 4 maintained a PDC of at least 80% after 5 years. The proportion of patients with a PDC less than 80% increased in a log-linear manner, comprising 40%, 61%, and 68% of the cohort after 3, 12, and 120 months, respectively. Independent predictors of poor long-term persistence included nonwhite race, lower income, older age, less cardiovascular morbidity at initiation of therapy, depression, dementia, and occurrence of coronary heart disease events after starting treatment. Patients who initiated therapy between 1996-1998 were 21% to 25% more likely to have a PDC of at least 80% than those who started in 1990. CONCLUSIONS: Persistence with statin therapy in older patients declines substantially over time, with the greatest drop occurring in the first 6 months of treatment. Despite slightly better persistence among patients who began treatment in recent years, long-term use remains low. Interventions are needed early in treatment and among high-risk groups, including those who experience coronary heart disease events after initiating treatment.
Aged↗
Principles of good practice for decision analytic modeling in health-care evaluation: report of the ISPOR Task Force on Good Research Practices--Modeling Studies.
OBJECTIVES: Mathematical modeling is used widely in economic evaluations of pharmaceuticals and other health-care technologies. Users of models in government and the private sector need to be able to evaluate the quality of models according to scientific criteria of good practice. This report describes the consensus of a task force convened to provide modelers with guidelines for conducting and reporting modeling studies. METHODS: The task force was appointed with the advice and consent of the Board of Directors of ISPOR. Members were experienced developers or users of models, worked in academia and industry, and came from several countries in North America and Europe. The task force met on three occasions, conducted frequent correspondence and exchanges of drafts by electronic mail, and solicited comments on three drafts from a core group of external reviewers and more broadly from the membership of ISPOR. RESULTS: Criteria for assessing the quality of models fell into three areas: model structure, data used as inputs to models, and model validation. Several major themes cut across these areas. Models and their results should be represented as aids to decision making, not as statements of scientific fact; therefore, it is inappropriate to demand that models be validated prospectively before use. However, model assumptions regarding causal structure and parameter estimates should be continually assessed against data, and models should be revised accordingly. Structural assumptions and parameter estimates should be reported clearly and explicitly, and opportunities for users to appreciate the conditional relationship between inputs and outputs should be provided through sensitivity analyses. CONCLUSIONS: Model-based evaluations are a valuable resource for health-care decision makers. It is the responsibility of model developers to conduct modeling studies according to the best practicable standards of quality and to communicate results with adequate disclosure of assumptions and with the caveat that conclusions are conditional upon the assumptions and data on which the model is built.
Benchmarking↗
Cost-effectiveness of interferon beta-1a, interferon beta-1b, and glatiramer acetate in newly diagnosed non-primary progressive multiple sclerosis.
OBJECTIVE: To perform a cost-effectiveness analysis of three immunomodulatory treatments for newly diagnosed nonprimary progressive MS: interferon beta-1a, interferon beta-1b, and glatiramer acetate. METHODS: We developed a state-transition model to estimate the health effects and costs associated with interferon beta-1a, interferon beta-1b, glatiramer acetate, and no treatment for hypothetical cohorts of men and women with non-primary progressive MS. We used the Expanded Disability Status Scale as the measure of disability and included both relapses and disease progression in the model. We evaluated treatment strategies assuming a 10-year treatment duration using the societal perspective. We elicited preferences for disability and treatment states using standard-gamble questions and modeled the disutility associated with treatment administration and side effects explicitly. Main outcome measures were net gains in quality-adjusted life expectancy and incremental cost-effectiveness ratios in dollars per quality-adjusted life year (QALY) gained. RESULTS: For treatment duration of 10 years for newly diagnosed non-primary progressive MS, interferon beta-1a yielded the largest gain in quality-adjusted life expectancy with an incremental cost-effectiveness ratio of $2,200,000/QALY for women and $1,800,000/QALY for men, compared with no treatment. For a 5-year treatment duration, a "no treatment" strategy yielded more quality-adjusted life years than any of the treatment strategies. Cost-effectiveness ratios were similar for all three immunomodulatory treatments evaluated. CONCLUSIONS: Cost-effectiveness results for all three immunomodulatory treatments for MS were unfavorable in the simulated study population under a wide range of assumptions. For treatment duration less than or equal to 5 years, expected benefits of treatment may not outweigh disutility associated with side effects and treatment discomfort.
Adjuvants, Immunologic↗
Health economic evaluations: the special case of end-stage renal disease treatment.
This article synthesizes the evidence on the cost-effectiveness of renal replacement therapy and discusses the findings in light of the frequent practice of using the cost-effectiveness of hemodialysis as a benchmark of societal willingness to pay. The authors conducted a meta-analytic review of the medical and economic literature for economic evaluations of hemodialysis, peritoneal dialysis, and kidney transplantation. Cost-effectiveness ratios were translated into 2000 U.S. dollars per life-year (LY) saved. Thirteen studies published between 1968 and 1998 provided such information. The cost effectiveness of center hemodialysis remained within a narrow range of $55,000 to $80,000/LY in most studies despite considerable variation in methodology and imputed costs. The cost-effectiveness of home hemodialysis was found to be between $33,000 and $50,000/LY. Kidney transplantation, however, has become more cost-effective over time, approaching $10,000/LY. Estimates of the cost per life-year gained from hemodialysis have been remarkably stable over the past 3 decades, after adjusting for price levels. Uses of the cost-effectiveness ratio of $55,000/LY for center hemodialysis as a lower boundary of society's willingness to pay for an additional life-year can be supported under certain assumptions.
Attitude to Health↗