Lifetime cost effectiveness of simvastatin in a range of risk groups and age groups derived from a randomised trial of 20,536 people.

Objectives: To evaluate the cost effectiveness of 40 mg simvastatin daily continued for life in people of different ages with differing risks of vascular disease.

Design: A model developed from a randomised trial was used to estimate lifetime risks of vascular events and costs of treatment and hospital admissions in the United Kingdom.

Setting: 69 hospitals in the UK.

Pharmacoeconomics and pharmacoepidemiology: curious bedfellows or a match made in heaven?

Pharmacoepidemiology is an established subdiscipline of epidemiology concerned with estimating the efficacy, effectiveness and safety of pharmaceutical products. Pharmacoeconomics is an established subdiscipline of health economics concerned with the evaluation of pharmaceutical products in terms of their value for money. Despite a common focus on the evaluation of pharmaceuticals, practitioners in the two disciplines appear to work largely in isolation of each other, as evidenced by lines of reasoning developed in one field that do not appear in the other.

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.

The electromyographic activity of thoracic paraspinal muscles identified as abnormal with palpation.

Objective: The aim of this study was to compare the electromyographic (EMG) activity of deep muscles in the thoracic paravertebral gutter (PVG) detected as abnormal to palpation (AbPT) and reported as tender by the subject with muscles underlying nontender (NT), normal to palpation sites under various experimental conditions.

Methods: Twelve subjects (mean age, 25.42 years; range, 22-43 years) participated in this study.

Estimating the cost-effectiveness of fluticasone propionate for treating chronic obstructive pulmonary disease in the presence of missing data.

Objectives: To explore the cost-effectiveness of fluticasone propionate (FP) for the treatment of chronic obstructive pulmonary disease (COPD), we estimated costs and quality-adjusted life-years (QALYs) over 3 years, based on an economic appraisal of a previously reported clinical trial (Inhaled Steroids in Obstructive Lung Disease in Europe [ISOLDE]).

Methods: Seven hundred forty-two patients enrolled in the ISOLDE trial who received either FP or placebo had data available on health-care costs and quality of life over the period of the study. The SF-36-based utility scores for quality of life were used to calculate QALYs.

The effect of osteoporotic vertebral fracture on predicted spinal loads in vivo.

The aetiology of osteoporotic vertebral fractures is multi-factorial, and cannot be explained solely by low bone mass. After sustaining an initial vertebral fracture, the risk of subsequent fracture increases greatly. Examination of physiologic loads imposed on vertebral bodies may help to explain a mechanism underlying this fracture cascade.

Cost-effectiveness of asthma control: an economic appraisal of the GOAL study.

Background: The Gaining Optimal Asthma ControL (GOAL) study has shown the superiority of a combination of salmeterol/fluticasone propionate (SFC) compared with fluticasone propionate alone (FP) in terms of improving guideline defined asthma control.

Methods: Clinical and economic data were taken from the GOAL study, supplemented with data on health related quality of life, in order to estimate the cost per quality adjusted life year (QALY) results for each of three strata (previously corticosteroid-free, low- and moderate-dose corticosteroid users). A series of statistical models of trial outcomes was used to construct cost effectiveness estimates across the strata of the multinational GOAL study including adjustment to the UK experience.

Treatment benefit by perindopril in patients with stable coronary artery disease at different levels of risk.

Aims: Patients with stable coronary artery disease (CAD) are at increased risk. Estimation of individual risk is difficult. We developed a cardiovascular risk model based on the EUROPA study population and investigated whether benefit of long-term administration of the angiotensin-converting enzyme (ACE)-inhibitor perindopril was modified by risk level.

Discounting and cost-effectiveness in NICE - stepping back to sort out a confusion.

Brouwer and colleagues [1] argue that the reasons for specifying an equal discount rate for health outcomes and costs in the recent guidance on methods of technology appraisal issued by the National Institute for Clinical Excellence (NICE) [2] is both opaque and wrong. They argue that a lower rate should apply to health outcomes like QALYs. It is also claimed that the guidance on discounting represents a step backwards, that is both inconsistent with current theoretical insights and will prejudice the outcome of cost-effectiveness studies of preventive interventions.

Economic evaluation of treating chronic obstructive pulmonary disease with inhaled corticosteroids and long-acting beta2-agonists in a health maintenance organization.

Objectives: In light of recent results from observational studies showing prolonged survival in subjects taking long-acting beta2-agonists (LABA) and/or inhaled corticosteroids (ICS) for chronic obstructive pulmonary disease (COPD), we investigated their cost-effectiveness (CE).

Methods: Costs and survival data were collected for a sample of members enrolled in a large Health Maintenance Organization in the United States. An observational study design was used to evaluate cumulative costs and health benefits of LABA, ICS, ICS+LABA, or comparison drugs.

Good research practices for cost-effectiveness analysis alongside clinical trials: the ISPOR RCT-CEA Task Force report.

Objectives: A growing number of prospective clinical trials include economic end points. Recognizing the variation in methodology and reporting of these studies, the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) chartered the Task Force on Good Research Practices: Randomized Clinical Trials-Cost-Effectiveness Analysis. Its goal was to develop a guidance document for designing, conducting, and reporting cost-effectiveness analyses conducted as a part of clinical trials.

In vivo intrarater and interrater precision of measuring apparent bone mineral density in vertebral subregions using supine lateral dual-energy x-ray absorptiometry.

Analysis of apparent bone mineral density (BMD) in the lumbar spine is commonly based on anteroposterior (AP) scanning using dual-energy X-ray absorptiometry (DXA). Although not widely used, clinically important information can also be derived from lateral scanning. Vertebral bone density, and therefore strength, can may vary in different subregions of the vertebral body.

Development of an economic model to assess the cost effectiveness of treatment interventions for chronic obstructive pulmonary disease.

Objective: To develop a Markov model that allows the cost effectiveness of interventions in patients with chronic obstructive pulmonary disease (COPD) to be estimated, and to apply the model to investigate the cost effectiveness of an inhaled corticosteroid/long-acting beta(2)-adrenoceptor agonist (beta(2)-agonist) combination (salmeterol/fluticasone propionate) versus usual care.

Methods: A Markov model consisting of four mutually exclusive disease states was constructed (mild, moderate and severe disease, and death). The transition probabilities of disease progression (for smokers and ex-smokers) and death were derived from the published medical literature.

Incorporation of uncertainty in health economic modelling studies.

In a recent leading article in PharmacoEconomics, Nuijten described some methods for incorporating uncertainty into health economic models and for utilising the information on uncertainty regarding the cost effectiveness of a therapy in resource allocation decision-making. His proposals are found to suffer from serious flaws in statistical and health economic reasoning.Nuijten's suggestions for incorporating uncertainty: (a) wrongly interpret the p-value as the probability that the null hypothesis is true; (b) represent this probability wrongly by truncating the input distribution; and (c) in the specific example of an antiparkinsonian drug uses a completely inappropriate p-value of 0.

A review of anatomical and mechanical factors affecting vertebral body integrity.

Background: The aetiology of osteoporotic vertebral fracture is multifactorial and may be conceptualised using a systems framework. Previous studies have established several correlates of vertebral fracture including reduced vertebral cross-sectional area, weakness in back extensor muscles, reduced bone mineral density, increasing age, worsening kyphosis and recent vertebral fracture. Alterations in these physical characteristics may influence biomechanical loads and neuromuscular control of the trunk and contribute to changes in subregional bone mineral density of the vertebral bodies.

Cost-effectiveness of simvastatin in people at different levels of vascular disease risk: economic analysis of a randomised trial in 20,536 individuals.

Background: Statin therapy reduces the rates of heart attack, stroke, and revascularisation among a wide range of individuals. Reliable assessment of its cost-effectiveness in different circumstances is needed.

Methods: 20,536 adults (aged 40-80 years) with vascular disease or diabetes were randomly allocated 40 mg simvastatin daily (10,269) or placebo (10,267) for an average of 5 years.

Conducting economic evaluations alongside multinational clinical trials: toward a research consensus.

Demand for economic evaluations in multinational clinical trials is increasing, but there is little consensus about how such studies should be conducted and reported. At a workshop in Durham, North Carolina, we sought to identify areas of agreement about how the primary findings of economic evaluations in multinational clinical trials should be generated and presented. In this paper, we propose a framework for classifying multinational economic evaluations according to (a) the sources of an analyst's estimates of resource use and clinical effectiveness and (b) the analyst's method of estimating costs.

Cost-utility analyses of intensive blood glucose and tight blood pressure control in type 2 diabetes (UKPDS 72).

Aims/hypothesis: This study estimated the economic efficiency (1) of intensive blood glucose control and tight blood pressure control in patients with type 2 diabetes who also had hypertension, and (2) of metformin therapy in type 2 diabetic patients who were overweight.

Methods: We conducted cost-utility analysis based on patient-level data from a randomised clinical controlled trial involving 4,209 patients with newly diagnosed type 2 diabetes conducted in 23 hospital-based clinics in England, Scotland and Northern Ireland as part of the UK Prospective Diabetes Study (UKPDS). Three different policies were evaluated: intensive blood glucose control with sulphonylurea/insulin; intensive blood glucose control with metformin for overweight patients; and tight blood pressure control of hypertensive patients.

Probabilistic sensitivity analysis for NICE technology assessment: not an optional extra.

Recently the National Institute for Clinical Excellence (NICE) updated its methods guidance for technology assessment. One aspect of the new guidance is to require the use of probabilistic sensitivity analysis with all cost-effectiveness models submitted to the Institute. The purpose of this paper is to place the NICE guidance on dealing with uncertainty into a broader context of the requirements for decision making; to explain the general approach that was taken in its development; and to address each of the issues which have been raised in the debate about the role of probabilistic sensitivity analysis in general.

The use of probabilistic decision models in technology assessment : the case of total hip replacement.

There is increasing recognition that decision modelling is central to health technology assessment and, in particular, to analyses to support formal decision making regarding the funding of the use of new technologies. In part, the key role of decision analysis stems from the need to handle multiple sources of uncertainty in the available evidence. The use of probabilistic decision analysis is a means of reflecting the parameter uncertainty in models and presenting this in a comprehensible manner to decision makers.

Parametric modelling of cost data: some simulation evidence.

Recently, commentators have suggested that the distributional form of cost data should be explicitly modelled to gain efficiency in estimating the population mean. We perform a series of simulation experiments to evaluate the usual sample mean and the mean estimator of a lognormal distribution, in the context of both theoretical distributions and three large empirical datasets. The sample mean is always unbiased, but is somewhat less efficient when the population distribution is truly lognormal.

Treatment costs in Canada of health conditions resulting from chronic hepatitis B infection.

Goals: To estimate resource use and expected annual cost of care for subjects with chronic hepatitis B and resulting complications in Canada.

Background: Patients chronically infected with hepatitis B virus are at an increased risk of progressing to complications from deteriorating liver function.

Study: The direct medical costs for six disease states associated with chronic hepatitis B virus infection were estimated for the year 2001.