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Alternative decision analysis modeling in the economic evaluation of tumor necrosis factor inhibitors for rheumatoid arthritis.

OBJECTIVES: To provide a review of the studies that use decision models in the economic evaluation of tumor necrosis factor (TNF) inhibitors in rheumatoid arthritis (RA) and to address some important issues surrounding the choice of such modeling techniques in these economic evaluations. METHODS: A systematic literature search was conducted by 1 author from the literature published from January 1996 to March 2005 through Medline, Embase, and Cochrane library databases. RESULTS: The review yielded 29 studies that used decision models. Only 10 studies used a decision model in the economic analysis of the TNF inhibitors and were included in the final review. Decision model types included the following in the review articles: decision tree (2), Markov model (7), and discrete event simulation (1). These models vary in complexity and their choice depends on the course of disease, the impact of treatment, and the available data. CONCLUSIONS: Based on the results derived from alternative modeling techniques, it is safe to say that all methods can provide useful information with regard to economic evaluations of TNF inhibitors. Even though different modeling techniques provide an appropriate representation of available data, their results should be interpreted contingent on the input data, assumptions, sensitivity analyses, and other alternative scenario analyses. RELEVANCE: The transparency in the models will encourage end users such as policymakers and prescribers to make informed judgments regarding the appropriateness of the methods and the validity of the results.

Antirheumatic Agents↗

Assessing the economics of vaccination for Neisseria meningitidis in industrialized nations: a review and recommendations for further research.

OBJECTIVES: To review the existing health economic literature on meningococcal disease vaccination. METHODS: A Medline search for economic evaluations of vaccination programs for meningococcal disease in developed countries was conducted. All identified studies were reviewed. RESULTS: Nine published studies were identified examining either mass vaccination during outbreaks or routine vaccination. Although net expenses were estimated in almost all studies, the resulting cost-effectiveness ratios varied widely. Vaccination of college-age students was found to be potentially cost-effective in Australia but not in the United States. With one exception, routine vaccination of children and adolescents in Europe was predicted to be cost-effective. Many simplifying assumptions were made, and important elements were often left out, in particular the potential for reduced transmission of disease. CONCLUSIONS: The methods used and the vaccination strategies vary widely, and results do not provide strong grounds for making conclusions as to whether vaccination is cost-effective. Furthermore, in all instances, transmission of disease, changes in population carriage rates, and outbreaks are either ignored, dealt with using very broad simplifying assumptions, or are not necessarily generalizable to other settings. The analyses provide some insight into the potential cost-effectiveness of vaccination, but more importantly, they highlight areas requiring further study. Economic evaluations based on observed outcomes from recently implemented strategies would be helpful, as would more sophisticated health economic models. The choice of vaccination strategies cannot be based on the results of existing economic analyses.

Developed Countries↗

The clinical and cost-effectiveness of pulsatile machine perfusion versus cold storage of kidneys for transplantation retrieved from heart-beating and non-heart-beating donors.

OBJECTIVES: To evaluate the clinical and cost-effectiveness of machine perfusion (MP) compared to cold storage (CS), as a means of preserving kidneys prior to transplantation. Transplantation of kidneys from both heart-beating donors (HBDs) and non-heart-beating donors (NHBDs) is considered. Finally to review whether the use of MP can allow valid testing of kidney viability prior to transplantation. DATA SOURCES: Fifteen electronic bibliographic databases were searched. The reference lists of relevant articles and sponsor submissions were hand searched and various health service research-related resources were consulted via the Internet. REVIEW METHODS: A literature search was undertaken to identify relevant studies and a meta-analysis performed on the studies that had appropriate comparator groups and reported sufficient data. A structured review examined tests of viability of kidneys on MP. Economic modelling was used to determine the cost-effectiveness and cost-utility of MP. RESULTS: The meta-analysis suggested that the use of MP, as compared with CS, is associated with a relative risk of delayed graft function (DGF) of 0.804 (95% confidence limits 0.672 to 0.961). There was no evidence to suggest that this effect is different in kidneys taken from HBDs as opposed to NHBDs. Meta-analysis of 1-year graft survival data showed no significant effect, but the studies, even when aggregated, were severely underpowered with respect to the likely impact on graft survival. The size of effects demonstrated were in line with those predicted by an indirect model of graft survival based on the association of DGF with graft loss. The economic assessment indicated that it is unlikely that in the UK health setting complete cost recovery will be obtained from a reduction in the incidence of DGF. The probability that MP is cheaper and more effective than CS in the long term was estimated at around 80% for NHBD recipients and 50-60% for HBD recipients. Flow characteristics of the perfusate of kidneys undergoing MP may be an indicator of kidney viability, but data were inadequate to calculate the sensitivity and specificity of any test based on this. The concentration of alpha-glutathione-S-transferase (a marker of cell damage) in the perfusate may be the basis of a valid test. A threshold of 2800 micrograms/100 g gave a sensitivity of 93% and specificity of 33% (and hence a likelihood ratio of 1.41). CONCLUSIONS: The baseline analysis indicated that in the long-term MP would be expected to be cheaper and more effective than CS for both HBD and NHBD recipients. A definitive study of the clinical benefit of MP in order to establish its effect on DGF and longer term graft survival would be valuable, together with an economic evaluation of the benefits. While direct evidence relating to improvements in graft survival would be preferable, the small predicted improvement indicates that a very large sample size would be required. In addition to seeking direct evidence of the impact on DGF, research quantifying the impact of DGF on graft survival in this technology is required. Research is also needed to establish whether a valid test (or combination of tests) of kidney viability can be developed.

Cell Survival↗

Screening for diabetic retinopathy in James Bay, Ontario: a cost-effectiveness analysis.

BACKGROUND: Retinopathy is a common complication of diabetes mellitus that if detected early by regular retinal examinations can be treated; thus, blindness can be delayed or prevented. Providing high-quality retinal screening is difficult, especially in rural and remote areas. Canada's First Nations population has a higher prevalence of diabetes and is, in general, more geographically isolated than the broader Canadian population. We modelled the cost-effectiveness of retinopathy screening by travelling retina specialists versus retinal photography with a portable digital camera in an isolated First Nations cohort with diabetes. METHODS: The 2 screening programs were modelled to run concurrently for 5 years, with outcomes evaluated over 10 years. To construct economic models for the population of Cree individuals living in the western James Bay area of northern Ontario, we used available data on the prevalence of diabetes in the area and estimates of the incidence of retinopathy derived from the published literature. We compared the screening models and calculated total costs, visual outcome, costs per sight-year saved and costs per quality-adjusted life year (QALY). We also estimated the costs of implementing a screening program for all First Nations individuals in Ontario with diabetes and no access to an ophthalmologist. RESULTS: From the perspective of the health care system the camera program was preferable to the specialist-based program. Over 10 years, 67 v. 56 sight years were saved, compared with no screening, at costs of 3900 Canadian dollars v. 9800 Canadian dollars per sight year and 15,000 Canadian dollars v. 37,000 Canadian dollars per QALY. Generalizing these results to the province of Ontario, the camera system could allow most isolated First Nations people with diabetes to be screened for 5 years for approximately 1.2 million Canadian dollars. INTERPRETATION: A portable retinal camera is a cost-effective means of screening for diabetic retinopathy in isolated communities of at-risk individuals.

Cost-Benefit Analysis↗

Effect of the tobacco price support program on cigarette consumption in the United States: an updated model.

OBJECTIVES: This study evaluated the direct effect of the tobacco price support program on domestic cigarette consumption. METHODS: We developed an economic model of demand and supply of US tobacco to estimate how much the price support program increases the price of tobacco. We calculated the resultant increase in cigarette prices from the change in the tobacco price and the quantity of domestic tobacco contained in US cigarettes. We then assessed the reduction in cigarette consumption attributable to the price support program by applying the estimated increase in the cigarette price to assumed price elasticities of demand for cigarettes. RESULTS: We estimated that the tobacco price support program increased the price of tobacco leaf by $0.36 per pound. This higher tobacco price translates to a $0.01 increase in the price of a pack of cigarettes and an estimated 0.21% reduction in cigarette consumption. CONCLUSION: Because the tobacco price support program increases the price of cigarettes minimally, its potential health benefit is likely to be small. The adverse political effect of the tobacco program might substantially outweigh the potential direct benefit of the program on cigarette consumption.

Humans↗

Brazil: the health care model of the military modernizers and technocrats.

Post-1964 political changes in Brazil had dire implications for the health sector as successive governments pursued economic and social policies which aggravated inequities and benefitted narrow elites. The "military modernizers" embraced a concept of development inimical to basic human needs, an economic model favoring growth over distribution and development over social welfare, and budget priorities favoring vocal, urban middle sectors at the expense of marginal populations. The result was deteriorating health and social conditions among the majority of the population that did not share in the benefits of the "economic miracle."

Brazil↗

A Markov model of treatment of newly diagnosed epilepsy in the UK. An initial assessment of cost-effectiveness of topiramate.

Long-term comparative trials among newer antiepileptic drugs are lacking; therefore decision models are needed to guide treatment decisions. The goal of this study was to develop an economic model of newly diagnosed epilepsy in the UK and to provide the first assessment of topiramate. A Markov model was developed combining data from clinical trials, cost-of illness, mortality, and utility studies. Expected costs and utilities associated with treatment strategies (first- and second-line treatments) were compared to find the cost-effectiveness frontier. First- and second-line monotherapy with topiramate or carbamazepine in partial seizures was less costly and more effective than other scenarios. In generalised seizures first-line topiramate was cost-effective with valproate or lamotrigine as second-line treatments depending on the set of utilities used. Models provide a relevant framework within which costs and health gains of antiepileptic drugs treatment options can be studied. Our findings are further evidence of the promising role of topiramate for patients with newly diagnosed epilepsy.

Journal Article↗

Disability studies and health care curriculum: the great divide.

Models or paradigms of disability are used to guide health care professionals' perceptions so that they can serve people with disabilities, enhance their futures, and facilitate the resources they need. Health care curricula, which in essence train students to make such decisions, are influenced by these models. The medical model, which locates disability within the individual, assumes the individual with a disability is a victim who must be cured or made more normal. The functional-limitation paradigm expands on the medical model, focusing on the interaction of physical or mental limitations with social and environmental factors. The economic model, based on the concept of employability, emphasizes a health-related inability (or limited ability) to work rather than physical functioning of the individual. The sociopolitical model views disability as a policy and civil rights issue. Health care professionals face a dilemma as the disability rights movement demands a shift in social power from the paternalistic view of the medical model to the autonomist view of the sociopolitical model. The question is asked if curricula are preparing our future health care professionals to distinguish how to view each situation and each individual through the lens of the appropriate model.

Activities of Daily Living↗

Diabetes in Canada: direct medical costs of major macrovascular complications.

OBJECTIVES: To estimate direct medical costs of managing major macrovascular complications in diabetic patients. METHODS: Costs were estimated for acute myocardial infarction (AMI) and ischemic stroke by applying unit costs to typical resource use profiles. Data were obtained from many Canadian sources, including the Ontario Case Cost Project, provincial physician and laboratory fee schedules, provincial formularies, government reports, and peer-reviewed literature. For each complication, the event costs per patient are those associated with resource use specific to the acute episode and any subsequent care occurring in the first year. State costs are the annual costs per patient of continued management. All costs are expressed in 1996 Canadian dollars. RESULTS: Acute hospital care accounts for approximately half of the first year management costs ($15,125) of AMI. Given the greater need for postacute care, acute hospital care has less impact (28%) on event costs for stroke ($31,076). The state costs for AMI and stroke are $1544 and $8141 per patient, respectively. CONCLUSIONS: Macrovascular complications of diabetes potentially represent a substantial burden to Canada's health care system. As new therapies emerge that may reduce the incidence of some diabetic complications, decision makers will need information to make critical decisions regarding how to spend limited health care dollars. Published literature lacks Canadian-specific cost estimates that may be readily translated into patient-level cost inputs for an economic model. This paper provides two key pieces of the many needed to understand the scope of the economic burden of diabetes and its complications for Canada.

Adult↗

Direct medical costs of complications resulting from type 2 diabetes in the U.S.

OBJECTIVE: To estimate direct medical costs of managing the complications of type 2 diabetes. RESEARCH DESIGN AND METHODS: Costs were estimated for 15 diabetic complications by applying unit costs to typical resource-use profiles. Resource used and unit costs were estimated from many sources, including acute care discharge databases, clinical guidelines, government reports, fee schedules, and peer-reviewed literature. For each complication, the event costs are those associated with resource use that is specific to the acute episode and any subsequent care occurring in the 1st year. State costs are the annual costs of continued management. All costs are expressed in 1996 U.S. dollars. RESULTS: As expected, the more severe or debilitating events, such as acute myocardial infarction ($27,630 event cost; $2,185 state cost), generate a greater financial burden than do early-stage complications, such as microalbuminuria ($62 event cost; $14 state cost). Yet, complications that are initially relatively low in cost (e.g., microalbuminuria) can progress to more costly advanced stages (e.g., end-stage renal disease, $53,659 state cost); therefore, minor complications should also be considered in any economic analysis of diabetes. CONCLUSIONS: The recent literature has lacked cost estimates that may be readily translated into patient-level cost inputs for an economic model. Emerging therapies that may reduce the incidence of some diabetic complications will need to be scrutinized economically in today's cost-conscious environment. The cost estimates from this study provide one piece of the economic analysis needed to evaluate these new interventional therapies.

Animals↗

Modelling in health economic evaluation. What is its place? What is its value?

This paper itemizes the current and developing roles of modelling in health economic evaluation and discusses its value in each role. We begin by noting the emptiness of the dichotomy that some commentators have sought to create between modelling and controlled trials as mechanisms for informing decisions. Both are necessary parts of the armoury. Recent literature discussions are examined and the accelerating prevalence of modelling is reported. The identified roles include: extrapolating outcomes to the longer term; adjusting for prognostic factors in trials; translating from intermediate to final outcomes; extending analysis to the relevant comparators; generalizing from specific trial populations to the full target group for an intervention and to other settings and countries; systematic sensitivity analyses; and the use of modelling for the design and prioritization of future trials. Roles are illustrated with 20 recent examples, mostly from within our own work analysing new or contentious interventions for the Trent Development and Evaluation Committee, which is planned to be incorporated into the UK National Institute for Clinical Excellence (NICE). Each role discussed has been essential at some point in this policy-making forum. Finally, the importance of quality assurance, critical review and validity testing is reiterated and there are some observations on processes to ensure probity and quality.

Biomedical Technology↗

Design of a climate-dependent water reuse project.

Reclaimed water storage is imperative in water reuse management. Climate is a primary factor controlling reclaimed water storage design by its significant influence on irrigation water needs as well as on stored water quality. This study presents a modelling approach that has been applied to assist the design of a climate-dependent water reuse project on an Atlantic island. Models for predicting irrigation water needs and water quality in tertiary lagoons were coupled with a technical-economic model to design reclaimed water storage facilities. Three scenarios corresponding to different augmentation of current reclaimed water reuse were investigated. According to the modelling, the storage sizes to meet the water quantity required for irrigation increased with water deficit--the difference between evapotranspiration and precipitation. The size of tertiary lagoons to meet required water quality was found to be larger than the size to meet required water quantity. To meet both quantitative irrigation needs and <1,000 FC/100 ml irrigation and disposal regulation, extending the tertiary lagoon system would be more cost-effective than storage calculated to meet only quantitative irrigation needs supplemented with UV disinfection. The reliability of reclaimed water storage design was estimated with 40 years historic climatic records.

Climate↗

Pharmacoeconomics of angiotensin II antagonists in type 2 diabetic patients with nephropathy: implications for decision making.

Angiotensin II receptor antagonists (angiotensin II receptor blockers; ARBs) are a class of antihypertensive drugs that are generally considered comparable to ACE inhibitors in the prevention of heart and kidney failure. However, these two classes of agents do interfere in different stages of the renin-angiotensin system. In patients with type 2 diabetes mellitus, advantages for ARBs over conventional (non-ACE inhibitor) therapy on progression from micro- to macroalbuminuria and overt nephropathy and end-stage renal disease have been shown in clinical trials. In patients with type 2 diabetes and end-stage renal disease, the need for dialysis and/or transplantation results in the use of major healthcare resources. This paper reviews the available economic evidence on treatment with ARBs in type 2 diabetic patients with advanced renal disease.Within-trial analytic and Markov model economic evaluations of the RENAAL (Reduction of Endpoint in Non-insulin dependent diabetes mellitus with Angiotensin II Antagonist Losartan), IDNT (Irbesartan Diabetic Nephropathy Trial) and IRMA (IRbesartan in type 2 diabetes with MicroAlbuminuria)-2 studies suggest that treatment with ARBs in patients with type 2 diabetes with overt or incipient nephropathy confers health gains and net cost savings compared with conventional (non-ACE inhibitor) therapy. For reimbursement and reference pricing decisions, there is a need for a head-to-head comparison of an ACE inhibitor with ARBs to model all possible costs and effects of ACE inhibitors and ARBs. This will result in a proper pharmacoeconomic outcome, where both types of drugs can be compared for healthcare decisions.

Angiotensin II Type 1 Receptor Blockers↗

The pervasiveness of pharmaceutical expenditure inertia in the OECD countries.

This paper constructs and estimates an economic model for testing statistically the strength of possible 'expenditure inertia' as a plausible reason for rising drug expenditures of the Organization for Economic Cooperation and Development (OECD) countries. The ethical drugs sector in the OECD health care systems is increasingly targeted as the major culprit in the rising cost. Using multiple regression analysis, and the maximum likelihood estimation method, the data of each country (taken from OECD Health Data, 1997) were first tested for functional form optimality with the Box-Cox power family transformations model. Drug expenditure elasticities, at data means, were computed using each country's optimal regression model estimates. The results indicate that the traditionally fitted a priori limited functional form models (e.g., linear, log-log) are not globally consistent with data across countries. The effect of a one-period lagged real per-capita drug expenditure (capturing inertia or habit persistence) on current period real per-capita prescription expenditure is statistically significant in most countries. Pharmaceutical demands are inelastic, and tend to behave like a necessity, as expected. Since the significant effects of economic, demographic, and other drivers of high drug spending differ across countries, country-specific implications and policy suggestions for cost controls ought to differ.

Aged↗

The current and future burden and cost of overactive bladder in five European countries.

OBJECTIVE: To estimate and compare the current and future direct cost of overactive bladder (OAB) to the health care systems of five European countries. METHOD: A health economic model was created to estimate the number of people currently affected by OAB symptoms, the expected number to be affected in the future, and the resultant economic burden on health care systems in Germany, Italy, Spain, Sweden, and the United Kingdom. RESULTS: The model estimated that in 2000, 20.2 million people over age 40 in the five countries experienced the symptoms of OAB; 7 million of these had urgency with urge incontinence. This figure is expected to rise to 25.5 million by 2020, including 9 million who will have urgency with urge incontinence. Average annual direct costs of OAB management ranged from euro269 to euro706 per patient per year. The largest cost was the use of incontinence pads, accounting for an average of 63% of the annual per patient cost of OAB management. Total cost to health care systems across all five countries was estimated at euro4.2 billion in 2000, and by 2020, the expected total cost was estimated to be euro5.2 billion, an increase of euro1 billion (26%). CONCLUSION: OAB is prevalent, with a substantial direct cost that is anticipated to increase in the future in line with aging populations. The overall burden, including indirect costs, may be considerably larger, and will fall predominantly on the elderly OAB population with urge incontinence. Recommended medical treatments could help manage those costs and should be evaluated.

Adult↗

Health outcomes in multiple sclerosis.

PURPOSE OF REVIEW: Economic considerations are increasingly important in the evaluation of innovative medical technologies. In the past few years, evaluations of cost and cost-effectiveness analysis became a popular topic for multiple sclerosis research. Here, we review cost-of-illness and cost-utility studies in multiple sclerosis published during the past 2 years. RECENT FINDINGS: Despite differences in methodology, several cost-of-illness studies unequivocally demonstrated that indirect costs as a result of sick leave, premature retirement or loss of income made up almost half of the overall costs, and that total costs were higher in the more advanced stages of the disease. Cost-effectiveness studies of recombinant IFN-beta preparations demonstrated a marked variability in the incremental cost per quality-adjusted life-year, with amounts ranging from Euro 28 432 to US$338 738. For glatiramer acetate and mitoxantrone, only limited data are available, but even these few studies differed in their results. SUMMARY: Health outcome studies constitute a new and emerging field of multiple sclerosis research. All studies performed so far underscore the importance of indirect cost in multiple sclerosis. However, the marked differences in cost-effectiveness studies illustrate that the method of economic modeling has considerable impact on the results of these studies, which need standardization in order to evaluate properly the economic consequences of new and expensive therapies in multiple sclerosis.

Cost-Benefit Analysis↗

Strengths and limitations of competitive versus non-competitive models of integrated capitated fundholding.

Integrated budget-holding (fundholding) based on risk-adjusted capitation is commonly proposed as a central element of health system reform. Two contrasting models have been developed: the competitive model where fundholders or health plans compete for enrollees; and the non-competitive model, where plan membership is determined according to an objective attribute such as place of residence. Under the competitive model, efficiency is sought through consumer choice of plan. A range of regulatory elements may also be introduced to moderate undesirable elements of competition. Under the non-competitive model, efficiency is achieved through government regulation and the fact that the fundholder has continuing responsibility for the health of a defined population, supported by micro-management tools (such as quality assurance and selective payment arrangements). In theory, the non-competitive model encourages population-based health services planning. While both models assume risk-adjusted capitated funding, the requirements of any formula are more stringent under the competitive model. Economic theory, as well as documented health system experience, can help identify the relative strengths and limitations of each model. Concerns with the competitive model relate primarily to the capacity to develop robust risk adjusters for capitation sufficient to reduce the incentives for patient risk selection. Possible reductions in the quality of care are also a concern, compounded by difficulties for consumers in discriminating between plans. Efficiency under the non-competitive model requires a strong and appropriate regulatory/policy framework and effective use of micro-management tools. Funding equity objectives can be met through either model by the adoption of income-related contributions, but under the competitive model this may be compromised by incentives for the fundholders to select low-risk patients. Evidence drawn from regional fundholding in New South Wales (NSW, Australia), the US Veterans Health Agency and the literature on managed care in the USA illustrate these concerns. The problem of risk selection in the competitive model is a major theoretical concern, confirmed by the empirical evidence. This, together with concerns regarding other aspects of performance, suggests that the non-competitive model may be preferable, at least as an interim step in reform in public or mixed systems. Future research on this issue is clearly required.

Competitive Medical Plans↗

Consumer focus can spur group practice turnaround.

Many healthcare organizations have lost money on their employed group practices. The solution to this dilemma is not necessarily divestment of the group practices. Instead, some healthcare organizations should view their physicians as an asset. Healthcare organizations and physicians need to develop a new framework for their relationship to optimize their competitive advantage. Three guiding principles that will help accomplish this objective are to recast the healthcare organization-physician relationship to focus on the consumer, reconfigure the economic model to exceed consumer demands, and restructure the group practice to encourage fiscal and service excellence. In developing a new relationship framework, the stakeholders need to define the group practice's mission, strategic direction, composition, infrastructure, compensation model, and structure.

Delivery of Health Care, Integrated↗