Health Economic Data Research Articles

UROLOGIC DISEASES IN AMERICA PROJECT: TRENDS IN RESOURCE USE FOR URINARY TRACT INFECTIONS IN WOMEN

Urinary tract infection (UTI) is one of the most common clinical diagnoses in women. In this study we examined epidemiological, economic and health care use trends for UTI in women in the United States. The analytical methods used to generate these results have been described previously. The lifetime risk for UTI in women is high (greater than 50%). Between 1988 and 1994 the overall lifetime prevalence of UTI was estimated to be 53,067/100,000 women. Prescribing patterns demonstrated an increase in the trend toward using fluoroquinolones as first line therapy for UTI, which was associated with increased costs. Composite data revealed that overall expenditures for the treatment of UTIs in women in the United States, excluding spending on outpatient prescriptions, were approximately 2.47 billion dollars in 2000. Diagnosis and treatment of UTI in women is performed in various clinical settings. Inpatient hospitalization for UTI care has generally decreased in younger women but increased in elderly women. There has been a sharp increase in emergency room use by younger women, which may reflect disparities in access to health insurance or primary care providers. Most outpatient care of women with UTIs is performed in physician offices. Analysis of health care use and economic data on UTIs in women revealed various interesting trends. These findings will help shape understanding of UTI treatment in relation to other urological disorders in women. The results raise various important future research questions.

Collection of Health-Economic Data Alongside Clinical Trials: Is There a Future for Piggyback Evaluations?

Objective The objective of this article is to discuss issues surrounding the conduct of “piggyback evaluations,” in which health-economic data are collected within an otherwise typical clinical trial. Methods We review the methodologic literature on piggyback economic evaluations, as well as selected empiric studies. We summarize the challenges encountered in the conduct of these studies, alternative ways of addressing these challenges, and their future role in pharmacoeconomic research. Results Piggyback evaluations have certain advantages over other types of pharmacoeconomic studies. An economic evaluation can benefit from the experimental design that maximizes the trial's internal validity, and it is often more practical to collect economic data alongside a trial rather than to fund a stand-alone economic study. However, piggyback evaluations are subject to problems deriving from the competing nature of clinical versus economic study objectives, which can give rise to tension in such fundamental aspects of study design as the selection of study subjects and sites; the extent of protocol-mandated health-care services; and the determination of sample size, length of follow-up, and the study comparator(s). Many solutions have been put forth in the literature to address these challenges. Conclusions Piggyback evaluations can be an appropriate means to measure the economic impact of medical interventions, provided that the methodologic challenges are acknowledged and addressed within the context of each individual study. As long as a desire for patient-level data from clinical trials exists, there will be a need for piggyback economic evaluations in the future.

Editorial Comment—What Can Models Teach Us About Stroke Treatment?

The report in this issue of Stroke on the cost-effectiveness of recombinant tissue plasminogen activator (rt-PA)1 suggests – with notable uncertainty – that, when compared with standard acute stroke care, thrombolytic therapy provides, on average, greater health benefits (in terms of average quality adjusted life years [QALYs]) at a reasonable average medical cost. This is not itself a unique finding; other similar efforts suggest – with notably greater certainty – that rt-PA is a good value for money.2–4 What have we learned from these modeling exercises? Is it true, as the authors propose, that we need another trial of rt-PA? Before considering the answer, it is useful to briefly review a few basics of disease models – what they are and what they can teach us. Disease models are mathematical representations of a clinical condition, its development, and its outcome, and models are often used to evaluate the impact of potential diagnostic or therapeutic strategies. These models are usually implemented in computer code, ranging from something as simple as a spreadsheet formula to sophisticated clinical event simulations. Sandercock and colleagues1 frame the question of rt-PA use as a decision tree, with a Markov model as a “calculation engine.” The inputs to this model are epidemiological, clinical trial, and health economic data (specifically applicable to the context of the …

A Computer Simulation Model of the Natural History and Economic Impact of Chronic Obstructive Pulmonary Disease

Objective Chronic obstructive pulmonary disease (COPD) is a major health problem with high societal costs. The Global Initiative for Chronic Lung Disease (GOLD) has identified a need for health economics data for COPD. For chronic diseases, such as COPD, where the natural history of disease is lifetime, a modeling approach for economic evaluation may be more realistic than prospective, piggy-backed clinical trials or specific COPD cohort studies. Simulation models can be used to extrapolate clinical data beyond the limited time frame of clinical trials, to analyze subgroups of patients or to explore uncertainty regarding the results by using sensitivity analysis techniques. Our purpose has been to develop a flexible computer simulation model for COPD that will represent disease progression and GOLD recommendations, useful for economic evaluations of new medicines to meet the needs of various payer requirements for reimbursement and resource allocation. Methods This article describes a two-dimensional Markov model, which uses data from multiple sources about disease progression, exacerbation frequency and duration, mortality, costs, burden of illness, and the relationships between those variables. The model is evaluated using stochastic uncertainty analysis, it allows comparison of treatments affecting different disease mechanisms, and it uses primary data validated against published sources. Results We have evaluated two hypothetical interventions treating different features of the disease (lung function decline and acute exacerbations). These analyses show that reducing lung function decline must be a long-term strategy compared to reducing the number of exacerbations. It was necessary to have a long term like 30 years, with 10,000 patients and 20% increase in price, or 20 years with equal prices to show cost-effectiveness with statistical significance for a treatment that reduces lung function decline. Conclusions Our study shows the value of modeling as a tool for evaluating different scenarios and for combining several sources of data, to provide estimates that would otherwise be unavailable. Clinical trials of this size and duration would be unrealistic.

A real-life prospective health economic study of elective single embryo transfer versus two-embryo transfer in first IVF/ICSI cycles.

We analysed the difference in maternal, neonatal and total costs after single (SET) versus double day 3 embryo transfer (DET). We performed a two-centre prospective study of women in their first IVF/ICSI cycle choosing between SET or DET. Infertility treatment data were gathered from a database; maternal and neonatal outcome data from a case report form (CRF); health economic data from medical acts registered in the CRF for the outpatient part and from hospital bills. SET was performed in 206/367 (56.1%) and DET in 161/367 (43.9%) women. In all, 367 transfers yielded 186 positive pregnancy tests, 148 ongoing pregnancies and 136 live deliveries (50.7, 40.3 and 37.1% per embryo transfer) of which 15 (11.0%) were twins. Live birth rate was 37.4% for SET, 36.6% for DET. Intention-to-treat analysis showed differences for: duration of pregnancy (SET: 39.0 +/- 1.4 versus DET: 38.3 +/- 2.2 weeks; P = 0.055), percentage prematurity (8.5 versus 23.8%; P = 0.033), percentage of neonates hospitalized (5.7 versus 17.9%; P = 0.121) and duration of neonatal hospitalization (6.3 +/- 2.2 versus 10.3 +/- 10.1 days; P = 0.01). Total cost after DET was higher (SET: 4700 +/- 3239 versus DET: 8613 +/- 10 105; P = 0.105), due to significantly higher neonatal costs (451 +/- 957 versus 3453 +/- 8154; P < 0.001) and not to differences in maternal costs (4250 +/- 2882 versus 5160 +/- 4106; P = 0.152). This prospective health economic study shows that transfer of a single top quality embryo is equally effective as, but substantially cheaper than, double embryo transfer in women <38 years of age in their first IVF/ICSI cycle.

Health care reform in six Central European countries

This study gives an overview of the health care reform in six Central European countries after the transition from a central planning system to a regulated market economy. We focused on cost containment policies for drugs, especially the requirements for submitting health economic data in the pricing and/or reimbursement processes. The literature review was supplemented with a survey with decision makers at national health authorities in each country. The study covered Croatia, Czech Republic, Hungary, Poland, Slovakia, and Slovenia. All countries had in common that health economic information was used in reimbursement rather than in pricing processes. Differences between the six countries were mainly variations in the relative importance of health economic data and the presence of explicit requirements and guidelines. Published health economic guidelines exist in two countries and one of the six countries applies a mandatory submission system for a selected range of new drugs. In most of the Central European countries it is more typical that authorities issue a brief list of required data for reimbursement submissions that include health economic information among other data. There is a generally widespread expectation towards more systematic and formalized requirements for health economic and outcomes research data appearing within the next 3-5 years in the region.

An Updated View of the National Anesthesia Personnel Shortfall

Reports of anesthesia personnel shortages in 2001 led to the first comprehensive analysis of labor supply and demand for anesthesiologists since 1993. We now update this analysis and forecast, incorporating newly available data about residency composition, American Board of Anesthesiology and Certified Registered Nurse Anesthetist certification, the 2002 residency match, surgical facilities, and the US physician workforce. In addition, US residency programs were surveyed; national health care utilization and economic data were reviewed. Adjusted for the new information, our model still shows an anesthesiologist shortfall in 2002, projected to continue through 2005. We now estimate a current shortage of 1100–3800 anesthesiologists in 2002, on the basis of past service demand growth assumptions of 2%–3%, respectively. By 2005 this number is expected to be 500–3900, depending on a future service demand growth of 1.5%–2%, respectively. To avoid a surplus of anesthesiologists in 2006–2010, our model suggests that the number of graduates should level out at 1600 yearly, with a 1.5% service demand growth. To forecast the anesthesia personnel market more accurately, thereby helping supply match demand, substantially better quantification of future demand for anesthesia services is needed. If sustained growth in service demand >1.5% is likely, entry into the specialty should be encouraged beyond the current level.

An updated view of the national anesthesia personnel shortfall.

Reports of anesthesia personnel shortages in 2001 led to the first comprehensive analysis of labor supply and demand for anesthesiologists since 1993. We now update this analysis and forecast, incorporating newly available data about residency composition, American Board of Anesthesiology and Certified Registered Nurse Anesthetist certification, the 2002 residency match, surgical facilities, and the US physician workforce. In addition, US residency programs were surveyed; national health care utilization and economic data were reviewed. Adjusted for the new information, our model still shows an anesthesiologist shortfall in 2002, projected to continue through 2005. We now estimate a current shortage of 1100-3800 anesthesiologists in 2002, on the basis of past service demand growth assumptions of 2%-3%, respectively. By 2005 this number is expected to be 500-3900, depending on a future service demand growth of 1.5%-2%, respectively. To avoid a surplus of anesthesiologists in 2006-2010, our model suggests that the number of graduates should level out at 1600 yearly, with a 1.5% service demand growth. To forecast the anesthesia personnel market more accurately, thereby helping supply match demand, substantially better quantification of future demand for anesthesia services is needed. If sustained growth in service demand >1.5% is likely, entry into the specialty should be encouraged beyond the current level. With updates from training programs, surgical activity, and other sources, our previously described model estimates a continuing shortfall of 1000-3800 anesthesiologists in 2002 and 500-3900 in 2005, assuming that service demand growth is 1.5% or 2% annually. If service growth >1.5% is likely, entry into the specialty should be encouraged beyond current levels.

Can varicella be eliminated by vaccination? Potential clinical and economic effects of universal childhood varicella immunisation in Germany.

Varicella is a potentially serious infection not only in immunocompromised individuals but also in otherwise healthy adults and children. Vaccination plays an important role in preventing the disease and its sequelae. A universal vaccination in childhood is expected to reduce substantially the number of uncomplicated cases of varicella and decrease the number of complicated cases requiring hospitalisation. To generate data as basis for decisions of the health authorities concerning prevention of varicella, epidemiological and health-economic data were collected in two studies. Using an age-structured decision analytic model the benefits, costs and cost effectiveness of a varicella immunisation program for a period of 30 years were assessed. It was shown that after the first year of life seroprevalence rates increased steadily and reached 62% among the 4- to 5-year olds and 94% among the 10- to 11-year olds, respectively; 90% of varicella patients were younger than 12 years. A severe course was assessed for 16.3% of the cases. Overall incidence of complications was estimated to be 5.7%. A routine varicella vaccination program targeting healthy children could prevent 82.7% of varicella cases and over 4,700 major complications per year provided the coverage level was 85%. Under these conditions the elimination of varicella is predicted to be achievable within 18 years. It is expected that a combined measles, mumps, rubella and varicella vaccine could provide the required coverage. Average yearly discounted net cost savings of universal childhood vaccination are 51 million Euro with a benefit-cost ratio of 4.12. Childhood vaccination with catch-up of adolescents provides additional clinical benefits. The break-even point indicating first net savings could be achieved already 3 years after the implementation of the vaccination program. In summary, routine childhood varicella vaccination appears to be a highly efficient strategy to significantly reduce the sizeable burden of varicella and would lead to net savings from both the societal but also the payer perspective.

Counting the cost of proton pump inhibitors

See article on page 488 Proton pump inhibitors (PPIs) have become an indispensable part of the armoury of treating gastro-oesophageal reflux disease (GORD). They are more effective than H2 receptor...

PHP7: APPLICATION OF HEALTH ECONOMICS IN THE CANADIAN PHARMACEUTICAL INDUSTRY

OBJECTIVES: The field of health economics (HE) has undergone vast growth over the last decade. One continually evolving main factor is the motivation for performing HE studies, in particular in Canada because of changing requirements for provincial formulary submissions. The objective of this study was to determine how HE data is being applied by the Canadian pharmaceutical industry to date. METHODS: A survey was sent to key personnel working in HE in the Canadian pharmaceutical industry and followed-up. Data from returned surveys were compiled in a database, tabulated, and evaluated. RESULTS: Twenty-one (84%) of 25 surveys sent to companies reporting previous experience with HE were returned; 90% of these companies were firms with more than 100 full-time employees. Of the respondents, 67% currently have a department or unit whose sole responsibility is HE, up from 12% reported in a previous study for 1995. The application of HE information by Canadian pharmaceutical companies is most often for provincial formulary submissions (100%), marketing (86%) and pricing (81%). It is also used by 62% of respondents for internal training and medical education programs. Relatively few Canadian companies apply HE information to managed care decision-making (38%) and patient education (24%). Health economic research data was published by 3/4 of the respondents in the form of scientific posters or conference presentations and by 2/3 in the biomedical, peer-reviewed literature. Ninety percent of respondents cited plans for new HE studies in Canada within the next year. CONCLUSION: Canadian companies have expanded their use of HE data in recent years. The focus has shifted from preferential internal use (pricing and marketing) to more external purposes such as provincial and hospital formulary submissions. Health economic research data also find a place in training within pharmaceutical companies and in medical education.

WW10: MULTINATIONAL ECONOMIC STUDIES: LESSONS LEARNED FROM TRIAL-BASED AND MODELLING STUDIES

WORKSHOP OBJECTIVE: The interest in multinational health economic studies has grown with the number of countries requiring economic data on new pharmaceuticals. The workshop will explore and illustrate challenges in the conduct of international studies using examples from the presenters' research while providing an opportunity for participants to share their experiences. PARTICIPANTS WHO WOULD BENEFIT: Researchers who are involved in the design, execution or commission of multinational health economic studies. As health care expenditures have risen, so too has scrutiny over the economic value of new pharmacological interventions. Many countries now require that the cost-effectiveness of new agents be addressed in formal analyses as part of the regulatory approval process. Given these widespread requirements, there has been a growing interest in undertaking multinational health economic studies that can be used as the basis for submissions in multiple countries. Two main strategies to provide health economic data include: creation of decision analytic models that utilize existing clinical, epidemiological and cost data, and collection of economic data alongside multinational clinical trials. Each raises methodological challenges, which will be discussed using examples from studies in rheumatology, vaccination, stroke, and cardiovascular disease. For example, in modeling studies, how does one accommodate local clinical practice patterns? In trial-based studies, how does one decide whether economic data can be pooled or not? Multinational studies also raise a number of practical issues, such as the involvement of a cross-section of international experts to avoid the introduction of country-specific biases, creation of buy-in among national offices and decision experts, availability of resource use and unit cost data, and requirements for a multinational publication strategy. These issues will also be discussed.

Changes in health economic data bases

Changes in health economic data bases

The Vein Graft Surveillance Trial: Rationale, Design and Methods

Objectives to compare the amputation rates, quality of life and health care costs in patients receiving duplex ultrasound scanning against clinical surveillance following femoropopliteal and femorocrural vein bypass.Design multi-centre, prospective, randomised controlled trial. Methods 1200 patients with a patent vein graft at 30 days postoperatively will be randomised to either clinical or duplex follow-up. All patients are seen in an out-patient clinic at 6 weeks, then 3, 6, 9, 12 and 18 months postoperatively. At each appointment patients are examined clinically; palpable pulses in the graft and crural vessels, presenting symptoms and their ankle–branchial pressure indices (ABPIs) measured. In the duplex group only, the results of the scan are monitored. The incidence of radiological and/or surgical interventions throughout the follow-up period are also noted. Quality of life is measured using the SF-36 and EuroQol questionnaires at the 6 and 18 month appointments. Hospital stays and resource use are documented for health economic analysis. Results the primary endpoint of this study is amputation or death from vascular causes; however, graft patency rates will also be compared between the groups. Quality of life and health economic data will be used to determine if there is any benefit in either arm in these outcomes between follow-up strategies. Conclusions this large, randomised-controlled trial will hopefully provide direct evidence on the benefit of duplex surveillance for vein grafts in terms of limb salvage, quality of life of the patients and cost-benefit to the purchaser.

Measuring costs in multisite randomized controlled trials: lessons from the VA Cooperative Studies Program.

The interest in the economic impact of new health care interventions has increased dramatically over recent years; however, the results can be highly variable depending upon the economic assumptions made and the approaches taken in collecting the data and in conducting the analyses. This paper describes experiences from the VA Cooperative Studies Program in measuring health care utilization and costs for studies that evaluate clinical interventions. Experiences from two multisite randomized clinical trials (RCTs) are highlighted to illustrate strategies used to measure costs by directly measuring health care utilization and economic data within the context of the trials. Despite the substantial resources required to gather evidence about the cost of care for health care innovations, future VA multisite studies should include accepted health economic approaches to make important contributions to health planning and health policy within and outside the VA health care system.

Cost-effectiveness of budesonide Turbuhaler in the treatment of mild-to-moderate asthma in Japan

ABSTRACT Asthma therapy including inhaled corticosteroids (ICS) has been shown to be effective in many parts of the world, but its use is still limited in Japan. A 6-week, randomized, placebo-controlled, clinical trial was therefore undertaken to assess the efficacy of the ICS budesonide Turbuhaler (budesonide administered via the dry-powder inhaler Turbuhaler) in patients in Japan with mild-to-moderate asthma. Prior to, and during, the study, all concomitant medications, except corticosteroid preparations, were allowed (i.e. all pre-study medication was to be maintained throughout the study). In total, 218 patients randomized to receive budesonide Turbuhaler 200μg/day ( n = 53), 400μg/day ( n = 55), 800μg/day ( n = 57) or placebo ( n = 53) were included in the analysis. Due to the increased demand and interest for health economic data in Japan, this retrospective cost-effectiveness analysis reports on the economic impact of budesonide Turbuhaler compared with placebo (i.e. usual care), based on this clinical trial. At each dosage, budesonide Turbuhaler was significantly more effective than the placebo, according to the number of symptom-free and episode-free days. The number of emergency visits, days of lost production and days of hospitalization were all lower in the budesonide Turbuhaler groups, leading to significantly ( P

Economic analysis of secondary prevention of coronary heart disease with simvastatin (Zocor) in Germany

Coronary artery disease (CAD) has enormous financial, medical, as well as economical consequences in Germany. Important risk factors include raised total cholesterol and LDL cholesterol blood levels. The 4S Study has demonstrated the clinical effectiveness in the secondary prevention of CAD. Based on the health economics data of this study, we undertook a cost-effectiveness analysis of the secondary prevention of CAD with simvastatin in Germany. On average, the costs per life year gained were DM 18,500.-(sensitivity analysis: DM 9,340.- to DM 29,374.-). The consequences of this result are discussed. It is necessary from a health economists' point of view to assess the efficiency of a clinically effective measure in a standardised manner. This permits a comparison of efficiency with other, competing forms of health care, which is necessary in areas like CAD where there are different approaches to combat the disease. Simvastatin is highly efficacious in the secondary prevention of CAD in a defined patient population and, in comparison to other interventions in this area, it also proves to be cost-efficient.

Concept and Clinical Application of Platelet Glycoprotein IIb/IIIa Inhibition with Abciximab (c7E3 Fab; ReoPro) for the Prevention of Acute Ischemic Syndromes

The platelet membrane glycoprotein (GP) IIb/IIIa integrin receptor is the final common pathway leading to platelet aggregation. Local aggregation commonly occurs following atherosclerotic plaque rupture or other injury to the vascular wall. When GP IIb/IIIa is activated, fibrinogen and von Willebrand factor bind to the receptor with high affinity, crosslinking platelets and locking them to the vessel surface and to each other. This process is central to arterial thrombus formation and consequent acute coronary syndromes, such as myocardial infarction (MI), unstable angina, and abrupt closure following revascularization procedures. Abciximab (c7E3 Fab; ReoPro) is a chimeric monoclonal antibody fragment developed specifically to inhibit GP IIb/IIIa receptor activity and thus prevent platelet aggregation and thrombosis. Abciximab has been evaluated in several clinical studies, the largest of which was the Evaluation of Abciximab for the Prevention of Ischemic Complications (EPIC) trial. This randomized, multicenter, placebo-controlled trial enrolled 2,099 patients at high risk for ischemic complications following coronary revascularization. The patients were randomized into three treatment groups: placebo, abciximab bolus (0.25 mg/kg), or abciximab bolus plus 12-h infusion (10 μg/min). Patients in the abciximab bolus plus infusion group had significant reductions, compared with placebo, in a composite end point of death, nonfatal MI, and urgent coronary intervention within 30 days. These positive, short-term findings were maintained at 6 months of follow-up. Bleeding complications and transfusions were significantly increased in abciximab patients, although there was no increase in bleeding-related death, stroke, or surgery. Retrospective secondary analyses suggested that many of the bleeding events observed in the EPIC trial may have been associated with concomitant high-dose heparin therapy, particularly in lighter weight patients. Subsequent clinical trials have shown that bleeding events can be reduced in patients treated with abciximab by using weight-adjusted heparin dosing without affecting the efficacy of the abciximab bolus plus infusion regimen. Examination of health economic data from the EPIC trial showed that abciximab bolus plus infusion is cost effective as well as clinically beneficial. These results confirm the importance of platelet GP IIb/IIIa receptor blockade in the treatment of acute thrombotic syndromes. Key Words: Platelet aggregation—GP IIb/IIIa receptor—Coronary revascularization— Ischemia.

9 Health economics in rheumatology

Economic evaluations of health-care technologies are playing an increasingly central role in determining which therapies are available to clinicians in the treatment of a whole range of conditions. In rheumatology, a large body of work has already been done on the cost effectiveness of alternative non-steroidal anti-inflammatory drugs (NSAIDs), and much of the current work on disease modifying therapies incorporates economic evaluations. This chapter describes the main techniques of economic evaluation and reviews the strengths and weaknesses of each. Two published economic evaluations are discussed in order to highlight what economic evaluations can offer to the care of people with rheumatoid arthritis, as well as the current limitations of economic evaluation. The objective of this chapter is to equip readers with a critical understanding of economic evaluation that can be used in considering the increasing volume of health economic data that they encounter in their clinical work.

A model for estimating the impact of changes in children's vaccines.

To assist in strategic planning for the improvement of vaccines and vaccine programs, an economic model was developed and tested that estimates the potential impact of vaccine innovations on health outcomes and costs associated with vaccination and illness. A multistep, iterative process of data extraction/integration was used to develop the model and the scenarios. Parameter replication, sensitivity analysis, and expert review were used to validate the model. The greatest impact on the improvement of health is expected to result from the production of less reactogenic vaccines that require fewer inoculations for immunity. The greatest economic impact is predicted from improvements that decrease the number of inoculations required. Scenario analysis may be useful for integrating health outcomes and economic data into decision making. For childhood infections, this analysis indicates that large cost savings can be achieved in the future if we can improve vaccine efficacy so that the number of required inoculations is reduced. Such an improvement represents a large potential "payback" for the United States and might benefit other countries.