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Economic analysis of basiliximab in renal transplantation.

BACKGROUND: Basiliximab is a chimeric monoclonal directed against the alpha-chain of the interleukin-2 receptor. International studies have shown that it is highly effective in preventing acute rejection in patients receiving Neoral, and causes no measurable incremental toxicity, but its economic value remains unknown. METHODS: This study employed an economic model to examine the potential economic benefit of basiliximab. Parameter estimates were derived from a randomized, prospective, double-blind study conducted in 21 renal transplant centers in seven countries in which 380 adult primary allograft recipients were randomized within center to receive basiliximab (20 mg i.v.) on days 0 and 4 or placebo in addition to dual immunosuppression with Neoral and steroids. Key clinical events included primary hospitalization, immunosuppressive drug use, patient and graft survival, graft rejection, treatment of rejection, dialysis, and repeat hospitalization. Health resources were valued via a comprehensive electronic cost dictionary, based upon a detailed economic evaluation of renal transplantation in Canada. Medication costs were calculated from hospital pharmacy acquisition costs; basiliximab was assessed a zero cost. RESULTS: The average estimated cost per patient for the first year after transplant was $55,393 (Canadian dollars) for placebo and $50,839 for basiliximab, rising to $141,690 and $130,592, respectively, after 5 years. A principal component of the cost in both groups was accrued during the initial transplant hospitalization ($14,663 for standard therapy and $14,099 for basiliximab). An additional $15,852 and $14,130 was attributable to continued care, graft loss, and dialysis in the two groups, whereas follow-up hospitalization consumed an additional $15,538 for placebo and $13,916 for basiliximab. The mean incremental cost of dialysis was $5,397 for placebo compared with $3,821 for basiliximab, whereas incremental costs of graft loss were $2,548 compared with $2,295 in the two treatment groups. The principal costs associated with repeat admission to the transplant ward and the general ward were marginally higher for placebo ($7,395 vs. $6,300 and $5,986 vs. $4,625). Treatment of acute rejection and maintenance immunosuppressive drug use were associated with only limited savings as a result of basiliximab (savings <$200 each). Sensitivity analysis indicated that the most influential parameters affecting the savings as a result of using basiliximab were a reduction in the duration of initial and repeat hospitalization followed by the reduced risks of acute rejection and graft loss. CONCLUSIONS: Before accounting for the cost of the therapy itself, basiliximab produces an estimated economic saving of $4,554 during the first year after transplant, of which $3,344 is attributable to the reduced costs of graft dysfunction, including graft loss and dialysis ($1,722) and follow-up hospitalizations ($1,622). When marketed, basiliximab is expected to cost approximately $3,000 per course (two doses of 20 mg), resulting in a net first-year saving of $1,554. Under these circumstances, basiliximab can be considered a dominant therapy in renal transplantation.

Antibodies, Monoclonal↗

Different approaches to modelling the cost-effectiveness of schistosomiasis control.

This paper reviews three different approaches to modelling the cost-effectiveness of schistosomiasis control. Although these approaches vary in their assessment of costs, the major focus of the paper is on the evaluation of effectiveness. The first model presented is a static economic model which assesses effectiveness in terms of the proportion of cases cured. This model is important in highlighting that the optimal choice of chemotherapy regime depends critically on the level of budget constraint, the unit costs of screening and treatment, the rates of compliance with screening and chemotherapy and the prevalence of infection. The limitations of this approach is that it models the cost-effectiveness of only one cycle of treatment, and effectiveness reflects only the immediate impact of treatment. The second model presented is a prevalence-based dynamic model which links prevalence rates from one year to the next, and assesses effectiveness as the proportion of cases prevented. This model was important as it introduced the concept of measuring the long-term impact of control by using a transmission model which can assess reduction in infection through time, but is limited to assessing the impact only on the prevalence of infection. The third approach presented is a theoretical framework which describes the dynamic relationships between infection and morbidity, and which assesses effectiveness in terms of case-years prevented of infection and morbidity. The use of this model in assessing the cost-effectiveness of age-targeted treatment in controlling Schistosoma mansoni is explored in detail, with respect to varying frequencies of treatment and the interaction between drug price and drug efficacy.

Cost-Benefit Analysis↗

An assessment of the accuracy of a regional economic-demographic projection model.

A large number of regional economic-demographic projection models have been developed but their accuracy has seldom been evaluated. This article examines the accuracy of one such model in projecting total populations for 1980, using 1970 base data, for 106 counties and 553 places in two states. Comparisons of the model's projections to 1980 Census counts reveal mean percentage absolute differences of 10 percent for counties and 14 percent for places. In addition, the model's accuracy was comparable to that for alternative projection systems. When projections for places of less than 1,000 are excluded, differences are substantially reduced. Economic-demographic models appear to be quite useful and deserve further attention.

Demography↗

Cost-effectiveness of using prognostic information to select women with breast cancer for adjuvant systemic therapy.

OBJECTIVES: To investigate the cost-effectiveness of using prognostic information to identify patients with breast cancer who should receive adjuvant therapy. DATA SOURCES: Electronic databases from 1980 through to February 2002. A survey of clinical practice in UK cancer centres and units. Large retrospective dataset containing data on prognostic factors, treatments and outcomes for women with early breast cancer treated in Oxford. REVIEW METHODS: Between six and nine databases were searched by an information expert. Evidence-based methods were used to review and select those studies and the quality of each included paper was assessed using standard assessment tools reported in the literature or piloted and developed for this study. A survey of clinical practice in UK cancer centres and units was carried out to ensure that conclusions drawn from the report could be implemented. These data, along with the information gathered in the systematic reviews, informed the methodological approach adopted for the health economic modelling. An illustrative framework was developed for incorporating patient-level prediction within a health economic decision model. This framework was applied to a large retrospective dataset containing data on prognostic factors, treatments and outcomes for women with early breast cancer treated in Oxford. The data were used to estimate directly a parametric regression-based risk equation, from which a prognostic index was developed, and prognosis-specific estimates of the baseline breast cancer hazard could be observed. Published estimates of treatment effects, health service treatment costs and utilities were used to construct a decision analytic framework around this risk equation, thus enabling simulation of the effectiveness and cost-effectiveness of adjuvant therapy for all possible combinations of prognostic factors included in the model. RESULTS: The lack of good-quality systematic reviews and well-conducted studies of prognostic factors in breast cancer is a striking finding. There are no registers of studies of prognostic factors or of reviews of prognostic studies. Many of the reviews used weak methods, primary studies are similar with poor methodology and reporting of results. In addition, there is much variation in patient populations, assay methods, analysis of results, definitions used and reporting of results. Most studies appear to be retrospective and some use inappropriate methods likely to inflate outcomes such as optimising cut points and failing to test the results in an independent population. Very few reviews used meta-analysis to conduct a pooled analysis and to provide an estimate of the average size of any association. Instead, most reviews relied on vote counting. Although many prognostic models for breast cancer have been published, remarkably few have been re-examined by independent groups in independent settings. The few validation studies have been carried out on ill-defined samples, sometimes of smaller size and short follow-up, and sometimes using different patient outcomes when validating a model. The evidence from the validation studies shows support for the prognostic value of the Nottingham Prognostic Index (NPI). No new prognostic factors have been shown to add substantially to those identified in the 1980s. Improvement of this index depends on finding factors that are as important as, but independent of, lymph node, stage and pathological grade. The NPI remains a useful clinical tool, although additional factors may enhance its use. We accepted that hormone receptor status (ER) for hormonal therapy such as tamoxifen and prediction of response to trastuzumab by HER2 did not require systematic review, as the mechanism of action of these drugs requires intact receptors. There was no clear evidence that other factors were useful predictors of response and survival. The survey confirmed pathological nodal status, tumour grade, tumour size and ER status as the most clinically important factors for consideration when selecting women with early breast cancer for adjuvant systemic therapy in the UK. The protocols revealed that although UK cancer centres appear to be using the same prognostic and predictive factors when selecting women to receive adjuvant therapy, much variation in clinical practice exists. Some centres use protocols based upon the NPI whereas others do not use a single index score. Within NPI and non-NPI users, between-centre variability exists in guidelines for women for whom the benefits are uncertain. Consensus amongst units appears to be greatest when selecting women for adjuvant hormone therapy with the decision based primarily upon ER or progesterone receptor status rather than combinations of a number of factors. Guidelines as to who should receive adjuvant chemotherapy, however, were found to be much less uniform. Searches of the literature revealed only five published papers that had previously examined the cost-effectiveness of using prognostic information for clinical decision-making. These studies were of varying quality and highlight the fact that economic evaluation in this area appears still to be in its infancy. By combining methodologies used in determining prognosis with those used in health economic evaluation, it was possible to illustrate an approach for simulating the effectiveness (survival and quality-adjusted survival) and the cost-effectiveness associated with the decision to treat individual women or groups of women with different prognostic characteristics. The model showed that effectiveness and cost-effectiveness of adjuvant systemic therapy have the potential to vary substantially depending upon prognosis. For some women therapy may prove very effective and cost-effective, whereas for others it may actually prove detrimental (i.e. the reductions in health-related quality of life outweigh any survival benefit). CONCLUSIONS: Outputs from the framework constructed using the methods described here have the potential to be useful for clinicians, attempting to determine whether net benefits can be obtained from administering adjuvant therapy for any presenting woman; and also for policy makers, who must be able to determine the total costs and outcomes associated with different prognosis based treatment protocols as compared with more conventional treat all or treat none policies. A risk table format enabling clinicians to look up a patient's prognostic factors to determine the likely benefits (survival and quality-adjusted survival) from administering therapy may be helpful. For policy makers, it was demonstrated that the model's output could be used to evaluate the cost-effectiveness of different treatment protocols based upon prognostic information. The framework should also be valuable in evaluating the likely impact and cost-effectiveness of new potential prognostic factors and adjuvant therapies.

Adjuvants, Pharmaceutic↗

Local and global alcohol supply: economic and geographic models of community systems.

This paper discusses how economic and geographic distributions of local supply affect patterns of alcohol problems in state and community settings. It is argued that characteristics of local supply directly affect the economic and social behaviors of consumers when purchasing and using alcohol. For example, although taxes may be raised in order to lower alcohol use, the manner in which tax increases are translated into price increases, and the way consumers respond to price increases through alterations in purchase patterns, may strongly mitigate price effects. Similarly, although overall alcohol availability may be reduced in order to lower alcohol use, the tendency for greater numbers of outlets to be focused in low-income areas and the manner in which consumers bundle alcohol purchases with other routine activities (e.g. shopping) may also strongly mitigate such effects.

Alcohol Drinking↗

A cost study of new media supported near oral anticoagulant treatment follow-up.

OBJECTIVE: In this study we sought to develop a comparative cost evaluation between conventional and new media, e.g. web, mobile communication technology and digital television, and near patient testing supported anticoagulant (ac) treatment follow-up in a primary health care setting. METHOD: The comparison was done for two patient groups, self-care and home-care patients, on oral ac treatment in the primary health care centre of the rural and sparsely populated municipality of Ikaalinen. In practise case analysis was used to develop cost functions from collected economic data, which were analysed to determine the break-even point in total cost between conventional and new media supported follow-up for the two patient groups. RESULTS: In the home-care setting the break-even point is 14 patients; in the self-care setting new media supported follow-up is always more cost-effective. CONCLUSION: The results illustrate that the use of new media and near patient testing in ac treatment follow-up brings about an economic benefit even with a small number of patients in the Ikaalinen setting. However, the sensitivity of break-even to perturbations in the individual costs of the used economic models remains high. Still, when the economic benefits are considered together with the clinical and practical benefits shown to result from self-testing, self-management and use of new media technologies the new service models can be said to provide noticeable benefits both in terms of quality of care and economics in our specific setting.

Administration, Oral↗

Cost-effectiveness of treatment for threshold retinopathy of prematurity.

OBJECTIVE: Retinopathy of prematurity (ROP) is a leading cause of adverse visual outcomes in premature infants. Both laser photocoagulation and cryotherapy have been demonstrated in clinical trials to be efficacious in reducing the incidence of visual loss occurring secondary to threshold ROP. Visual data recently have become available concerning the long-term clinical efficacy of both treatments, as have data concerning the utility value of visual states in general. Accordingly, we undertook an analysis to ascertain the cost-effectiveness of laser photocoagulation and cryotherapy in the treatment of threshold ROP. DESIGN: A computer simulation economic model is presented to evaluate the cost-effectiveness of cryotherapy and laser photocoagulation therapy, compared with the natural course of the disease, for treating premature infants with threshold ROP. The model applies long-term visual data from previous clinical trials, utility analysis, decision analysis, and economic principles, such as present value analysis, to account for the time value of money to arrive at a cost per quality-adjusted life-year (QALY) gained. OUTCOME MEASURES: Cost per QALY gained from laser therapy and cryotherapy. RESULTS: Laser photocoagulation therapy for threshold ROP costs $678 1998 US dollars (at a 3% discount rate to account for the time value of money) for each QALY gained from treatment. Cryotherapy for the same disease costs $1801 per QALY at a similar discount rate. CONCLUSIONS: From the point of view of cost-effectiveness, laser therapy seems to have an advantage over cryotherapy for the treatment of threshold ROP.

Computer Simulation↗

Allocating global package pricing. The example of bypass surgery.

When health care organizations announce a change in rates paid to physicians, determining how to equitably distribute the new allocation can be a problem. Package pricing requires practitioners to justify their fees or costs to their colleagues and negotiate for a "specific piece of the pie." While this approach has many models, there are few actual examples. When some successful ones are developed, they should include an economic model which allows a practice to make business decision about marginal production opportunities that are in the best interest of all parties involved. Steps include determining actual costs, historical charge patterns, historical payment patterns and Medicare payment rates.

Coronary Artery Bypass↗

Pharmacoeconomic aspects of non-steroidal anti-inflammatory drug gastropathy.

Non-steroidal anti-inflammatory drugs are commonly used to reduce inflammation and pain associated with arthritis. However, non-steroidal anti-inflammatory drugs induce gastrointestinal side-effects such as dyspeptic symptoms, duodenal or gastric ulcers and, in some cases, serious complications. The aim has been to compare the benefits with the drawbacks of non-steroidal anti-inflammatory drug treatment using a hypothetical population representing patients with arthritis. A problem description was made on the basis of a literature review, and a simple and hypothetical health economic model was constructed. Including direct and indirect costs, the annual total costs in Sweden for gastrointestinal side-effects per non-steroidal anti-inflammatory drug user were estimated to be 3,420 SEK (438 US$), and the approximated costs of arthritis were 60,000 SEK (7,692 US$). The benefits of non-steroidal anti-inflammatory drug treatment were found to outweigh the drawbacks if the patient's arthritis symptoms, expressed as a difference in utility value between having and not having symptoms of arthritis, are improved by 6% or more. Costs for non-steroidal anti-inflammatory drug-induced gastrointestinal side-effects should be evaluated in relation to the benefits of non-steroidal anti-inflammatory drugs in the treatment of inflammation and pain. A simple modelling approach indicated that treatment with non-steroidal anti-inflammatory drugs may be highly cost-effective as both the clinical and economic benefits for patients responding to such treatment out-weighed possible drawbacks.

Anti-Inflammatory Agents, Non-Steroidal↗

Problems with the interpretation of pharmacoeconomic analyses: a review of submissions to the Australian Pharmaceutical Benefits Scheme.

CONTEXT: Pharmacoeconomic analyses are being used increasingly as the basis for reimbursement of the costs of new drugs. Reports of these analyses are often published in peer-reviewed journals. However, the analyses are complex and difficult to evaluate. OBJECTIVE: To describe the nature of problems encountered in the evaluation and interpretation of pharmacoeconomic analyses used as a basis for reimbursement decisions. DATA SOURCES: All major submissions to the Department of Health and Aged Care (DHAC) by the pharmaceutical industry for funding made under the Australian Pharmaceutical Benefits Scheme. Specifically, the DHAC's database of submissions that were received between January 1994 and December 1997 were reviewed. STUDY SELECTION: Of a total of 326 submissions, 218 had serious problems of interpretation and were included in the analysis. The nature of the serious problems reviewed were classified as estimates of comparative clinical efficacy, comparator issues, modeling issues, and calculation errors. DATA EXTRACTION: All submissions in the DHAC's database were reviewed and data were extracted if both the DHAC evaluators and technical subcommittee considered problems to have a significant bearing on the decisions of the parent committee. DATA SYNTHESIS: Of a total of 326 submissions, 218 (67%) had significant problems and 31 had more than 1 problem. Of the 249 problems identified, 154 (62%) related to uncertainty in the estimates of comparative clinical efficacy, and 71 (28.5%) related to modeling issues, which included clinical assumptions or cost estimates, used in the construction of the economic models. There were 15 instances of disagreement over the choice of comparator, and serious calculation errors were found on 9 occasions. Overall, 159 problems (64%) were considered to be avoidable. CONCLUSIONS: Significant problems were identified in these pharmacoeconomic analyses. The intensive evaluation process used in the Australian Pharmaceutical Benefits Scheme allowed for identification and correction of pharmacoecomomic analysis problems, but the resources that are required may be beyond the capacity of many organizations, including peer-reviewed journals.

Australia↗

Striking out! Federal enforcers losing streak in antitrust cases shows need for new game plan.

The Federal Trade Commission and the U.S. Justice Department are on a four-case losing streak with hospital mergers they have tried to stop for antitrust reasons. Theories explaining why the trustbusters can't hit a home run abound. Some point to the loss of key players who have signed on with the private sector. Others say the complex economic models introduced in court can throw judges a curve ball.

Antitrust Laws↗

Cost-effectiveness analysis of treatment alternatives for beef bulls with preputial prolapse.

OBJECTIVE: To develop an economic model for comparing cost-effectiveness of medical and surgical treatment versus replacement of beef bulls with preputial prolapse. DESIGN: Economic analysis. SAMPLE POPULATION: Estimates determined from medical records of bulls treated for preputial prolapse at our hospital and from information about treatment of bulls published elsewhere. PROCEDURE: Annual depreciation cost for treatment (ADC(T)) and replacement (ADC(R)) were calculated. Total investment for an injured bull equaled the sum of salvage value, maintenance cost, and expected cost of the treatment option under consideration. Total investment for a replacement bull was purchase price. Net present value of cost was calculated for each year of bull use. Sensitivity analyses were constructed to determine the value that would warrant treatment of an injured bull. RESULTS: The decision to treat was indicated when ADC(T) was less than ADC(R). In our example, it was more cost-effective for owners to cull an injured bull. The ADC(R) was $97 less than ADC(T) for medical treatment ($365 vs $462) and $280 less than ADC(T) for surgical treatment ($365 vs $645). Likewise, net present value of cost values indicated that it was more cost-effective for owners to cull an injured bull. Sensitivity analysis indicated treatment decisions were justified on the basis of replacement value or planned number of breeding seasons remaining for the bull. CLINICAL IMPLICATIONS: The model described here can be used by practitioners to provide an objective basis to guide decision making of owners who seek advice on whether to treat or replace bulls with preputial prolapse.

Animals↗

Guidelines for pragmatic assessment for health planning in developing countries.

Although the primary health care strategy implemented since the Alma Ata declaration of 'health for all' appears to have contributed to improvements in selected health outcomes, the current ad hoc approach to health assessment and planning has impeded more substantial gains. A comprehensive yet pragmatic framework for country-level health programmers is needed that would permit consideration of the multiple steps involved in policy formulation and implementation. In the present paper, drawing upon an epidemiologic model (Iterative Measurement Loop) and an economic model (Cost-Effectiveness Analysis), we present guidelines for a pragmatic assessment for health planning. A format is provided for the conduct of these tasks which is operational in nature, is specific to the target country (or relevant region), can simultaneously consider multiple interventions, and is comprehensible to persons without sophisticated medical and/or economic backgrounds. Such a format enables articulation and consideration of local concerns as well as national and global considerations.

Cost-Benefit Analysis↗