Cost-effectiveness of a post-splenectomy registry for prevention of sepsis in the asplenic

BACKGROUND: Overwhelming, sometimes fatal infections represent a lifelong risk after surgical removal of the spleen, or in patients who develop hyposplenism as a consequence of illnesses. This risk may be reduced by all or a combination of vaccination, antibiotic prophylaxis and education. We aimed to determine if a registry approach to delivering these interventions would be cost effective using our own experience and published data. METHOD: The decision model compared a cohort of 1,000 people covered by a registry to a cohort of 1,000 people with no registry. The impact of the registry was assessed in terms of achieved rates of vaccination, chemoprophylaxis and education, consequent outcomes of overwhelming post-splenectomy infection (OPSI) and mortality (years of life lived). The cost-effectiveness of the registry compared with no registry was estimated in terms of additional cost per case of OPSI avoided and as additional cost per life year gained. RESULTS: In the first two years, the additional cost of the registry was dollar 152,611 per case of OPSI avoided or dollar 205,931 per life year gained. After this initial registration period the cost-effectiveness improves over time, such that over the cohort lifetime a post-splenectomy register is associated with an additional cost of dollar 105,159 per case of OPSI avoided or dollar 16,113 per life year gained. CONCLUSION: A registry-based approach is likely to prove cost effective in terms of mortality and rates of OPSI avoided.     

An empirical investigation of time‐varying cost‐effectiveness across the product life cycle

Cost‐effectiveness is traditionally treated as a static estimate driven by clinical trial efficacy and drug price at launch. Prior studies suggest that cost‐effectiveness varies over the drug's lifetime. We examined the impact of “learning by doing,” one of the least studied drivers of changes in cost‐effectiveness across the product life cycle. We combined time‐series trends in effectiveness over time by cancer regimen using the Surveillance, Epidemiology, and End Results‐Medicare database. We estimated the time‐varying effects of treatments in colorectal and pancreatic cancer over their life cycle, including FOLFOX (leucovorin, 5‐fluorouracil, and oxaliplatin) and gemcitabine, on survival of patients. Mean prices over time by strength and dosage form were calculated using historical wholesale acquisition costs. We found consistent downward trends in the mortality hazard ratios, which suggest that effectiveness improves over time. In the case of first‐line FOLFOX for colorectal cancer, the implied incremental cost‐effectiveness ratio based on the observational data fell from $610,000 per life year gained in 2004 to $27,000 per life year gained in 2011. Cost‐effectiveness estimated at launch is unlikely to be representative of cost‐effectiveness over the drug's lifetime. In the drugs studied, the impact of time‐varying clinical effectiveness dominated the impact of changing prices overtime.     

Prevention and infection in adults patients with hyposplenism

People with asplenia are at risk for infections due to many causative agents, mainly Streptococcus pneumoniae. Among adults, splenectomy is the most frequent etiology of hyposplenism followed with chronic hematological and connective diseases. Physiopathology of the immunologic impairment due to hyposplenia is multifactorial. Physicians and even patients must be aware of overwhelming sepsis occurring on these conditions. The prognosis of these life-threatening infections is related to the precocity of the treatment onset. These infections, mainly due to S. pneumoniae (50-90% of cases) could be prevented with appropriate precautions. Patients presenting with asplenia must be largely vaccinated against these infectious agents: S. pneumoniae, Haemophilus influenzae b, and possibly Neisseiria meningitidis. Oral phenoxymethylpenicillin seems to be the simplest chemoprophylaxis (despite the global increase of pneumococcal strains with reduced susceptibility). Duration of treatment following splenectomy is discussed: The French medicine agency (AFSSAPS) recommends a 2-year treatment after surgery and for patients having functional hyposplenism (persistency of Howell-Jolly bodies) and/or associated immunodeficiency. Despite these prevention policies, the patient must be informed of the risk of very severe infection.     

Economic evaluation of an extended acellular pertussis vaccine programme for adolescents in Ontario, Canada

PURPOSE: Pertussis is a frequent cause of cough illness in adolescents. In Canada, until recently immunization against pertussis in public programmes has been restricted to children under the age of 7. The purpose of this analysis was to estimate the health and economic impact of an additional booster dose of the acellular vaccine in adolescents in Ontario. METHODS: We performed a cost effectiveness analysis, based on a predictive spreadsheet dynamic model following a cohort of 144,000 adolescents in Ontario from the age of 12 years over a 10-year-period from the Ontario Ministry of Health (MoH) and societal perspectives. The model was used to compare costs and benefits of a combined vaccination programme (CVP) including tetanus, diphtheria, and acellular pertussis (dTacp) administered at age 12, compared to current practice. RESULTS: From the MoH perspective, booster vaccination of dacpT at 12 years via the CVP would produce a yearly additional expected cost of CAD $0.52 per adolescent in Ontario with an incremental cost-effectiveness ratio of CAD $168 per pertussis case avoided based on a 10-year-period. If outcomes are discounted at 3%, the incremental cost-effectiveness ratio rises to $188/discounted pertussis case avoided. From the societal perspective, the CVP would be cost saving CAD $858,106 at 10 years for the cohort. Over the 10-year-period, more than 4400 cases of pertussis would be prevented with approximately 50 hospital admissions averted. CONCLUSIONS: This study suggests that administering a booster dose of dTacp at 12 years of age to replace diphtheria and tetanus vaccination at 14 years may reduce the economic burden of pertussis treatment in the long term at a reasonable cost.     

Cost-effectiveness of drug-eluting coronary stents in Quebec, Canada

OBJECTIVES: The aim of this investigation was to assess the incremental cost-effectiveness of replacing bare metal coronary stents (BMS) with drug-eluting stents (DES) in the Province of Quebec, Canada. METHODS: The strategy used was a cost-effectiveness analysis from the perspective of the health-care provider, in the province of Quebec, Canada (population 7.5 million). The main outcome measure was the cost per avoided revascularization intervention. RESULTS: Based on the annual Quebec rate of 14,000 angioplasties with an average of 1.7 stents per procedure and a purchase cost of $2,600 Canadian dollar (CDN) for DES, 100 percent substitution of BMS with DES would require an additional $45.1 million CDN of funding. After the benefits of reduced repeat revascularization interventions are included, the incremental cost would be $35.2 million CDN. The cost per avoided revascularization intervention (18 percent coronary artery bypass graft, 82 percent percutaneous coronary intervention [PCI]) would be $23,067 CDN. If DES were offered selectively to higher risk populations, for example, a 20 percent subgroup with a relative restenosis risk of 2.5 times the current bare metal rate, the incremental cost of the program would be $4.9 million CDN at a cost of $7,800 per avoided revascularization procedure. Break-even costs for the program would occur at DES purchase cost of $1,161 for 100 percent DES use and $1,627 for selective 20 percent DES use for high-risk patients for restenosis (RR = 2.5). Univariate and Monte Carlo sensitivity analyses indicate that the parameters most affecting the analysis are the capacity to select patients at high risk of restenosis, the average number of stents used per PCI, baseline restenosis rates for BMS, the effectiveness ratio of restenosis prevention for DES versus BMS, the cost of DES, and the revascularization rate after initial PCI. Sensitivity analyses suggest little additional health benefits but escalating cost-effectiveness ratios once a DES penetration of 40 percent has been attained. CONCLUSIONS: Under current conditions in Quebec, Canada, selective use of DES in high-risk patients is the most acceptable strategy in terms of cost-effectiveness. Results of such an analysis would be expected to be similar in other countries with key model parameters similar to those used in this model. This model provides an example of how to evaluate the cost-effectiveness of selective use of a new technology in high-risk patients.     

PHB8 Cost-Effectiveness of Hormone Therapy Versus Calcium Therapy: An Osteoporosis Markov Model

Osteoporosis is a chronic degenerative disease with concentrated prevalence in the most rapidly growing segment of the population, the elderly. Its clinical and economic consequences are substantial. This study assesses the cost-effectiveness of two gold standard therapeutic approaches to reducing the impact of related fractures.
OBJECTIVES: To determine the cost-effectiveness ratios of the two therapeutic approaches. To model the cohort progression through the Markov states.
METHODS: Using published literature values, a Markov model was constructed. The model depicts a cohort of 1,000 women progressing from age 50 to 90 years or death, whichever comes first. The hormone therapy alternative includes calcium supplementation as part of the regimen. The model accounts for the following confounders: age dependent rate of death, predisposition to subsequent fracture, and the cardioprotective effect of hormone therapy. Sensitivity analysis was conducted on all relevant variables to assess the robustness of the findings. The primary outcome of interest was cost per fracture avoided. Additionally, Markov analysis of the model reports the distribution of women across each Markov state.
RESULTS: The study revealed that hormone therapy is more cost-effective than calcium therapy. Cost-effectiveness ratios for the two alternatives were $43,729.82 and $87,003.53 per fracture avoided for hormone therapy and calcium therapy, respectively. The incremental cost-effectiveness ratio was $32,828.60, indicating the cost of avoiding each additional fracture using the less desirable calcium therapy. The cardioprotective effect of hormone therapy accounted for nearly 3 additional years of life for each woman on therapy.
CONCLUSIONS: Hormone replacement therapy in conjunction with calcium supplementation is far more cost-effective than calcium supplementation alone.     

Cost effectiveness of an internet-delivered lifestyle intervention in primary care patients with high cardiovascular risk

ObjectiveTo assess the cost-effectiveness of an online adaptation of the diabetes prevention program (ODPP) lifestyle intervention.MethodsODPP was a before–after evaluation of a weight loss intervention comprising 16 weekly and 8 monthly lessons, incorporating behavioral tools and regular, brief, web-based individualized counseling in an overweight/obese cohort (mean age 52, 76% female, 92% white, 28% with diabetes). A Markov model was developed to estimate ODPP cost effectiveness compared with usual care (UC) to reduce metabolic risk over 10 years. Intervention costs and weight change outcomes were obtained from the study; other model parameters were based on published reports. In the model, diabetes risk was a function of weight change with and without the intervention.ResultsCompared to UC, the ODPP in our cohort cost $14,351 and $29,331 per quality-adjusted life-year (QALY) gained from the health care system and societal perspectives, respectively. In a hypothetical cohort without diabetes, the ODPP cost $7777 and $18,263 per QALY gained, respectively. Results were robust in sensitivity analyses, but enrolling cohorts with lower annual risk of developing diabetes (≤ 1.8%), enrolling fewer participants (≤ 15), or increasing the hourly cost (≥$91.20) or annual per-participant time (≥ 1.45 h) required for technical support could increase ODPP cost to >$20,000 per QALY gained. In probabilistic sensitivity analyses, ODPP was cost-effective in 20–58% of model iterations using an acceptability threshold of $20,000, 73–92% at $50,000, and 95–99% at $100,000 per QALY gained.ConclusionsThe ODPP may offer an economical approach to combating overweight and obesity.     

Cost-effectiveness of influenza vaccination of people aged 50-64 years in Australia: results are inconclusive

Objective: Influenza cost‐effectiveness studies use models for influenza clinical evolution based on a range of assumptions. We explore the importance of these assumptions and its implications in policy decisions. Methods: An influenza model was constructed to measure the cost‐effectiveness of universal influenza vaccination of people over 50 years compared to current policy to vaccinate people over 65 years in Australia using available epidemiological data. We explored two scenarios, one with an Australian estimate of influenza like illness incidence, and one with a European estimate. Further, we estimated uncertainty of model structure and various parameter assumptions, and compared with a previous study. Results: The scenario and sensitivity analysis has shown the incremental cost‐effectiveness ratio of the proposed compared to current policy varies from $112,000 to $6,000 per DALY. The model structure, parameter assumptions and limitations of existing epidemiological data lead to extensive unaccounted uncertainties in previous studies. Conclusion: The lack of influenza epidemiological data makes the influenza cost‐effectiveness studies that compare the universal influenza vaccinations of people over 50 years to current policy unreliable. Implications: It is imperative to appraise unreliability of influenza cost‐effectiveness studies in policy decisions. Research to acquire more data on influenza uncertainties in Australia should be funded.     

Cost-effectiveness of interferon beta-1b in slowing multiple sclerosis disability progression. First estimates

OBJECTIVE: To estimate the cost-effectiveness (CE) of interferon beta-1b (IFN beta-1b) in slowing disability progression in persons with relapsing-remitting multiple sclerosis (RRMS). METHODS: Treatment program costs and health outcomes are modeled for cohorts of 1,000 females and 1,000 males followed 40 years from onset. Fifteen scenarios model MS natural history progression, treatment efficacy, direct treatment costs, and MS healthcare costs. A single randomized placebo-controlled trial of IFN beta-1b found reduced disease activity by MRI, reduced frequency and severity of exacerbations, and a tendency toward slower disability progression. Disability years avoided are modeled as the primary health outcome analyzed. A ministry of health (MOH) perspective is adopted, using Nova Scotia population-based data. Annual IFN beta-1b direct treatment costs (Can $16,685) are high relative to both MOH healthcare costs per person with MS (Can $2,000) and estimated MOH costs avoided. RESULTS: Given "reference case" assumptions for women with RRMS, treatment reduces lifetime disability years by 10%. Cost per disability year avoided before discounting is Can $189,230 (US $124,892), and Can $274,842 (US $181,395) after discounting at 5%. Estimates for alternative scenarios vary greatly, leaving main findings unchanged. CONCLUSIONS: Using the Expanded Disability Status Scale, cost per disability year avoided due to interferon beta-1b treatment in RRMS is quite high. Comparable CE estimates, using MS-specific or generic health-related quality-of-life outcome measures, are even higher. Further research is required to better measure treatment effects, modification of MS natural history, and net societal costs of IFN beta-1b in RRMS.     

The Comparison of Trial Data–Based and Registry Data–Based Cost-Effectiveness of Infliximab Treatment for Rheumatoid Arthritis in Sweden Using a Modeling Approach

ObjectiveTo evaluate the precision of the predictive cost-effectiveness assessment based on a phase 3 clinical trial with infliximab for the treatment of rheumatoid arthritis in Swedish clinical practice.MethodsThree patient cohorts were identified: the patients included in the infliximab trial (ATTRACT), patients initially treated with infliximab from a Swedish registry (STURE), a subset of these registry patients meeting inclusion criteria for the ATTRACT trial was the third patient cohort; two sets of assumptions in relation to the efficacy data were evaluated: “ATTRACT” (efficacy data over the duration of the trial) and “STURE” (effectiveness data over 10 years). In addition, the impact of including the placebo effect for the comparator was evaluated as a basis for the calculation of cost-effectiveness by using a modeling approach. A health economic model was utilized to estimate the cost per quality-adjusted life-year (QALY) gained.ResultsThe results for the three patient cohorts ranged from cost saving to a cost per QALY gained of €2,400 and €24,900 to €26,000 when the ATTRACT and STURE assumptions were used, respectively. Sensitivity analyses indicated that the inclusion of placebo effect had the largest effect on the results, increasing the cost per QALY gained to approximately €50,000 for all patient cohorts.ConclusionsThe treatment effect of infliximab measured in clinical trials and clinical practice results in comparable cost-effectiveness ratios, as calculated by using a modeling approach, whereas the assumptions made in relation to the effectiveness data and the chosen comparator have a large impact on the results. This reinforces the value of early modeling studies based on randomized clinical trial data, but assumptions made need to be carefully assessed.     

Economic modeling of the combined effects of HIV-disease, cholesterol and lipoatrophy based on ACTG 5142 trial data

Background

Insomnia is perhaps the most common sleep disorder in the general population, and is characterised by a range of complaints around difficulties in initiating and maintaining sleep, together with impaired waking function. There is little quantitative information on treatment pathways, costs and outcomes. The aims of this New Zealand study were to determine from which healthcare practitioners patients with insomnia sought treatment, treatment pathways followed, the net costs of treatment and the quality of life improvements obtained.

Methods

The study was retrospective and prevalence based, and was both cost effectiveness (CEA) and a cost utility (CUA) analysis. Micro costing techniques were used and a societal analytic perspective was adopted. A deterministic decision tree model was used to estimate base case values, and a stochastic version, with Monte Carlo simulation, was used to perform sensitivity analysis. A probability and cost were attached to each event which enabled the costs for the treatment pathways and average treatment cost to be calculated. The inputs to the model were prevalence, event probabilities, resource utilisations, and unit costs. Direct costs and QALYs gained were evaluated.

Results

The total net benefit of treating a person with insomnia was $482 (the total base case cost of $145 less health costs avoided of $628). When these results were applied to the total at-risk population in New Zealand additional treatment costs incurred were $6.6 million, costs avoided $28.4 million and net benefits were $21.8 million. The incremental net benefit when insomnia was "successfully" treated was $3,072 per QALY gained.

Conclusions

The study has brought to light a number of problems relating to the treatment of insomnia in New Zealand. There is both inadequate access to publicly funded treatment and insufficient publicly available information from which a consumer is able to make an informed decision on the treatment and provider options. This study suggests that successful treatment of insomnia leads to direct cost savings and improved quality of life.     

An injury awareness education program on outcomes of juvenile justice offenders in Western Australia: an economic analysis

ABSTRACT: BACKGROUND: Injury is a major cause of mortality and morbidity of young people and the cost-effectiveness of many injury prevention programs remains uncertain. This study aimed to analyze the costs and benefits of an injury awareness education program, the P.A.R.T.Y. (Prevent Alcohol and Risk-related Trauma in Youth) program, for juvenile justice offenders in Western Australia. METHODS: Costs and benefits analysis based on effectiveness data from a linked-data cohort study on 225 juvenile justice offenders who were referred to the education program and 3434 who were not referred to the program between 2006 and 2011. RESULTS: During the study period, there were 8869 hospitalizations and 113 deaths due to violence or traffic-related injuries among those aged between 14 and 21 in Western Australia. The mean length of hospital stay was 4.6 days, a total of 320 patients (3.6%) needed an intensive care admission with an average length of stay of 6 days. The annual cost saved due to serious injury was $3,765 and the annual net cost of running this program was $33,735. The estimated cost per offence prevented, cost per serious injury avoided, and cost per undiscounted and discounted life year gained were $3,124, $42,169, $8,268 and $17,910, respectively. Increasing the frequency of the program from once per month to once per week would increase its cost-effectiveness substantially. CONCLUSIONS: The P.A.R.T.Y. injury education program involving real-life trauma scenarios was cost-effective in reducing subsequent risk of committing violence or traffic-related offences, injuries, and death for juvenile justice offenders in Western Australia.     

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 and Economic Burden Analyses on All First-Line Treatments of Chronic Lymphocytic Leukemia

ObjectivesSeveral chemoimmunotherapy and targeted treatment regimens are approved as front-line therapies in chronic lymphocytic leukemia. We estimated for the 10-year cost-effectiveness of these treatment regimens and the economic burden of following the estimated risk-stratified 21 040 patients with chronic lymphocytic leukemia diagnosed in 2020 for 10 years.MethodsA Markov model with 7 exclusive health states was specified over a 10-year time horizon. Treatment effectiveness inputs were obtained from a novel network meta-analysis on the progression-free survival, overall survival curves, and time to next treatment. Costs and utilities inputs were included for each health state for each treatment and discounted at 3.0%/year. Life-years (LYs) and quality-adjusted LYs (QALYs) for each treatment were determined. Using the lowest cost regimen as reference, the incremental cost-effectiveness ratio (ICER) and incremental cost-utility ratio (ICUR) were estimated. The 10-year per-patient cost was determined by risk status and by initial treatment.ResultsVenetoclax-plus-obinutuzumab was the lowest cost regimen, hence the reference. Superior in effectiveness to all chemoimmunotherapies, it was cost saving. With the highest effectiveness gains at 6.26 LYs and 5.01 QALYs and despite being the most expensive regimen ($1 298 638 per patient), acalabrutinib-plus-obinutuzumab yielded the best ICER ($409 343/LY gained) and ICUR ($501 236/QALY gained). The remaining ICERs of targeted therapies ranged from $512 101/LY gained to $793 236/LY gained and the ICURs from $579 737/QALY gained to $869 300/QALY gained. The 10-year postdiagnosis low/high (venetoclax-plus-obinutuzumab/acalabrutinib-plus-obinutuzumab) economic burden ranges were $42 690 to $98 665 for low-risk, $141 339 to $326 660 for intermediate-risk, and $273 650 to $632 453 for high-risk patients.ConclusionsCompared with venetoclax-plus-obinutuzumab, chemoimmunotherapies are associated with less health benefits at higher cost. The targeted therapies achieve greater benefits at higher cost.     

The impact of pharmaceutical innovation on premature cancer mortality in Switzerland, 1995–2012

The premature cancer mortality rate has been declining in Switzerland, but there has been considerable variation in the rate of decline across cancer sites (e.g., breast or digestive organs). I analyze the effect that pharmaceutical innovation had on premature cancer mortality in Switzerland during the period 1995–2012 by investigating whether the cancer sites that experienced more pharmaceutical innovation had larger declines in premature mortality, controlling for the number of people diagnosed and mean age at diagnosis. Premature cancer mortality before ages 75 and 65 is significantly inversely related to the cumulative number of drugs registered 5, 10, and 15 years earlier. The number of drugs registered during 1980–1997 explains 63 % of the variation across cancer sites in the 1995–2012 log change in the premature (before age 75) mortality rate. Controlling for the cumulative number of drugs, the cumulative number of chemical subgroups does not have a statistically significant effect on premature mortality. This suggests that drugs (chemical substances) within the same class (chemical subgroup) are not “therapeutically equivalent”. Over 17,000 life-years before age 75 were gained in 2012 due to drugs registered during 1990–2007. The number of life-years before age 75 gained in 2012 from drugs registered during two earlier periods (1985–2002 and 1980–1997) were more than twice as great. Since mean utilization of new drugs is much lower than mean utilization of older drugs, more recent drug registrations may have a smaller effect on premature mortality than earlier drug registrations even if the average quality of newer drugs is higher. Estimates of the cost per life-year gained before ages 75 and 65 in 2012 from drugs registered during 1990–2007 are $21,228 and $28,673, respectively. These figures are below even the lowest estimates from the value-of-life literature of the value of a quality-adjusted life-year. The estimates indicate that the cost per life-year before age 75 gained from drugs registered during earlier periods (1985–2002 and 1980–1997) were considerably lower: $5299 and $3218, respectively. The largest reductions in premature mortality occur at least a decade after drugs are registered, when their utilization increases significantly. This suggests that if Switzerland is to obtain substantial additional reductions in premature cancer mortality in the future (a decade or more from now) at a modest cost, pharmaceutical innovation (registration of new drugs) is needed today.     

The cost of antiretroviral therapy in Haiti

Background

Tobacco smoking is a risk factor for age-related macular degeneration, but studies of ex-smokers suggest quitting can reduce the risk.

Methods

We fitted a function predicting the decline in risk of macular degeneration after quitting to data from 7 studies involving 1,488 patients. We assessed the cost-effectiveness of smoking cessation in terms of its impact on macular degeneration-related outcomes for 1,000 randomly selected U.S. smokers. We used a computer simulation model to predict the incidence of macular degeneration and blindness, the number of quality-adjusted life-years (QALYs), and direct costs (in 2004 U.S. dollars) until age 85 years. Cost-effectiveness ratios were based on the cost of the Massachusetts Tobacco Control Program. Costs and QALYs were discounted at 3% per year.

Results

If 1,000 smokers quit, our model predicted 48 fewer cases of macular degeneration, 12 fewer cases of blindness, and a gain of 1,600 QALYs. Macular degeneration-related costs would decrease by $2.5 million if the costs of caregivers for people with vision loss were included, or by $1.1 million if caregiver costs were excluded. At a cost of $1,400 per quitter, smoking cessation was cost-saving when caregiver costs were included, and cost about $200 per QALY gained when caregiver costs were excluded. Sensitivity analyses had a negligible impact. The cost per quitter would have to exceed $77,000 for the cost per QALY for smoking cessation to reach $50,000, a threshold above which interventions are sometimes viewed as not cost-effective.

Conclusion

Smoking cessation is unequivocally cost-effective in terms of its impact on age-related macular degeneration outcomes alone.     

The Cost-Utility of Sequential Adjuvant Trastuzumab in Women with Her2/Neu-Positive Breast Cancer: An Analysis Based On Updated Results from the HERA Trial

Background:  The efficacy of sequential adjuvant trastuzumab (aTZ) after chemotherapy in women with early-stage human epidermal growth factor-2 (HER2/neu)-positive breast cancer reported by the updated Herceptin Adjuvant (HERA) trial appears less favorable than originally reported. Based on these updated results, we estimated the cost-utility (CU) of sequential aTZ relative to chemotherapy alone in terms of incremental cost per quality-adjusted life-year (QALY) gained.
Methods:  A Markov model estimated incremental costs and outcomes of 12 months of aTZ after adjuvant chemotherapy in women with HER2/neu-positive breast cancer over a 25-year horizon. The model incorporated four broad health states (disease-free, local recurrence [LCR], distant recurrence [DCR], death), stratified with or without symptomatic cardiotoxicity. Baseline event rates and 3-year relative risk (RR = 0.75) were derived from the HERA trial. As the duration of the benefit remains uncertain, the analysis considered 5-year and 3-year duration of benefit in two scenarios. Costs and utility weights were from the literature. The analysis took a direct payer perspective, with costs reported in 2007 Canadian dollars. Costs and QALYs were discounted by 3% annually.
Results:  The mean CU of sequential aTZ at a 25-year horizon was $72,292 per QALY gained in the 5-year scenario and $127,862 per QALY gained in the 3-year scenario. Results were particularly sensitive to the magnitude and duration of carryover benefit.
Conclusions:  The CU of sequential aTZ is primarily dependent on the magnitude and duration of benefit. Further clinical research is required to establish the optimum sequence and duration of aTZ therapy and clarify the magnitude and duration of treatment benefit.     

The benefits and costs of tamoxifen for breast cancer prevention

OBJECTIVE: To estimate the effects of key uncertainties on the effectiveness and cost-effectiveness of breast cancer prevention with tamoxifen. METHODS: The incremental cost-effectiveness ratio of tamoxifen therapy relative to placebo was estimated using decision analysis with Markov modelling of health states, outcomes and costs for a simulated cohort of women at high risk for breast cancer. Relative effects of tamoxifen's benefits and harms were estimated from meta-analyses of randomised controlled trials. Cost estimates were based on Australian treatment patterns and costs. The main outcome measure was cost per quality-adjusted life year (QALY) gained with costs and effects discounted at a 5% annual rate. RESULTS: Tamoxifen therapy over five years reduces the incidence of breast cancer by approximately 1.4%, which is offset by an increase in endometrial cancer of 0.7% and pulmonary embolism of 0.2%. If the reduction is permanent (preventing new breast cancers emerging over five years and no further treatment effect thereafter), the model estimates an increase in life expectancy of 0.057 QALYs and an extra cost of $2,193; or $38,271/QALY gained. A model assuming further treatment effects of tamoxifen preventing new breast cancers emerging for up to 10 years results in an incremental cost of $19,354/QALY. However, if five years of tamoxifen therapy merely delays when these breast cancers appear (such that by 10 years there is no longer a reduced incidence), the incremental cost per QALY saved is estimated to be $199,149. CONCLUSIONS: Tamoxifen is potentially cost-effective in preventing breast cancer in women at high risk. However, its cost-effectiveness as a preventive therapy is highly sensitive to whether these cancers are permanently prevented or their clinical presentation is only delayed. Long-term follow-up in randomised controlled trials is therefore crucial in forming health policy.     

Dynamic Medication Adherence Modeling in Primary Prevention of Cardiovascular Disease: A Markov Microsimulation Methods Application

BackgroundReal-world patients’ medication adherence is lower than that of clinical trial patients. Hence, the effectiveness of medications in routine practice may differ.ObjectivesThe study objective was to compare the outcomes of an adherence-naive versus a dynamic adherence modeling framework using the case of statins for the primary prevention of cardiovascular (CV) disease.MethodsStatin adherence was categorized into three state-transition groups on the basis of an epidemiological cohort study. Yearly adherence transitions were incorporated into a Markov microsimulation using TreeAge software. Tracker variables were used to store adherence transitions, which were used to adjust probabilities of CV events over the patient’s lifetime. Microsimulation loops “random walks” estimated the average accrued quality-adjusted life-years (QALYs) and CV events. For each 1,000-patient microsimulations, 10,000 outer loops were performed to reflect second-order uncertainty.ResultsThe adherence-naive model estimated 0.14 CV events avoided per person, whereas the dynamic adherence model estimated 0.08 CV events avoided per person. Using the adherence-naive model, we found that statin therapy resulted in 0.40 QALYs gained over the lifetime horizon on average per person while the dynamic adherence model estimated 0.22 incremental QALYs gained. Subgroup analysis revealed that maintaining high adherence in year 2 resulted in 0.23 incremental QALYs gained as compared with 0.16 incremental QALYs gained when adherence dropped to the lowest level.ConclusionsA dynamic adherence Markov microsimulation model reveals risk reduction and effectiveness that are lower than with an adherence-naive model, and reflective of real-world practice. Such a model may highlight the value of improving or maintaining good adherence.     

The Cost-Effectiveness of Expanding Newborn Screening for up to 21 Inherited Metabolic Disorders Using Tandem Mass Spectrometry: Results from a Decision-Analytic Model

OBJECTIVES: In 2005, in Ontario, Canada, newborns were only screened for phenylketonuria (PKU) and hypothyroidism. Tandem mass spectrometry (MS/MS) has since been implemented as a new screening technology because it can screen for PKU and many other diseases simultaneously. We estimated the cost-effectiveness of using this technology to expand the Ontario newborn screening program to screen for each disease independently and for hypothetical bundles of up to 21 metabolic diseases. METHODS: We constructed a decision-analytic model to estimate the incremental costs and life-years of survival that can be gained by screening or changing screening technologies. Costs and health benefits were estimated for a cohort of babies born in Ontario in 1 year. Secondary sources and expert opinion were used to estimate the test characteristics, disease prevalence, treatment effectiveness, disease progression rates, and mortality. The London Health Sciences Centre Case Costing Initiative, the Ontario Health Insurance Plan Schedule, and the Ontario Drug Benefits plan formulary were used to estimate costs. RESULTS: Changing screening technologies, from the Guthrie test to MS/MS, for PKU detection had an incremental cost of $5,500,000 per life-year (LY) gained. We identified no diseases for which the incremental cost of screening for just that disease was less than $100,000 per LY gained. The incremental costs of screening ranged from $222,000 (HMG-CoA lyase deficiency) to $142,500,000 (glutaric acidemia type II) per LY gained. Screening for a bundle of diseases including PKU and the 14 most cost-effective diseases to screen for cost less than $70,000 per LY gained, and the incremental cost-effectiveness of adding each of the 14 diseases to the bundle was less than $100,000 per LY gained. The incremental cost of adding the 15th most cost-effective disease was $309,400 per LY gained. CONCLUSIONS: Early diagnosis and treatment of metabolic disease is important to reduce disease severity and delay or prevent the onset of the disease. Screening at birth reduces the morbidity, mortality, and social burden associated with the irreversible effects of disease on the population. Our analysis suggests that the cost-efficiencies gained by using MS/MS to screen for bundles of diseases rather than just one disease are sufficient to warrant consideration of an expanded screening program. It is, however, not cost-effective to screen for all diseases that can be screened for using this technology.