Clinical effectiveness and cost-effectiveness of growth hormone in children: a systematic review and economic evaluation.
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Child↗
Biomedical subjects
K Gerard
Publications and source records attributed to K Gerard.
Clinical effectiveness and cost-effectiveness of growth hormone in adults in relation to impact on quality of life: a systematic review and economic evaluation.
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Adult↗
Efficacy of a mixed amphetamine salts compound in adults with attention-deficit/hyperactivity disorder.
BACKGROUND: We report on a controlled trial of a mixed amphetamine salts compound (Adderall, dextroamphetamine sulfate, dextro-, levoamphetamine sulfate, dextroamphetamine aspartate, levoamphetamine aspartate, and dextroamphetamine saccharate) in the treatment of adult attention-deficit/hyperactivity disorder (ADHD). METHODS: This was a 7-week, randomized, double-blind, placebo-controlled, crossover study of Adderall in 27 well-characterized adults satisfying full DSM-IV criteria for ADHD of childhood onset and persistent symptoms into adulthood. Medication was titrated up to 30 mg twice a day. Outcome measures included the ADHD Rating Scale and the Clinical Global Impression Score. Comorbid psychiatric disorders were assessed to test for potential effects on treatment outcome. RESULTS: Treatment with Adderall at an average oral dose of 54 mg (administered in 2 daily doses) was effective and well tolerated. Drug-specific improvement in ADHD symptoms was highly significant overall (42% decrease on the ADHD Rating Scale, P<.001), and sufficiently robust to be detectable in a parallel groups comparison restricted to the first 3 weeks of the protocol (P<.001). The percentage of subjects who improved (reduction in the ADHD rating scale of > or =30%) was significantly higher with Adderall treatment than with a placebo (70% vs 7%; P =.001). CONCLUSIONS: Adderall was effective and well tolerated in the short-term treatment of adults with ADHD. More work is needed to evaluate the long-term effects of Adderall, or other amphetamine compounds, in the treatment of adults with ADHD.
Adult↗
Assessing efficiency in the UK breast screening programme: does size of screening unit make a difference?
The UK breast screening programme (UK BSP) is organised into a large number of individual screening units. Decision makers need to ensure these units are producing efficiently, particularly as the programme is anticipated to expand. Data envelopment analysis (DEA) was applied to investigate: the relative efficiency of screening units; the impact of screening unit size on efficiency; and how individual units could improve. Sixty-four screening units were categorised into 33 large and 31 small. Data were collected using a national survey and routinely collected data. The overall median efficiency score was 91%, 39 units were inefficient. Variation in efficiency scores was wide. Large units had a median efficiency score of 100% and 12 units were inefficient. Smaller units had a median efficiency score of 95% and 19 were inefficient. This difference was not statistically significant (Mann-Whitney, P=0.076). Forty-two percent of large units and 21% of small units were operating at constant returns to scale (mean difference 0.20, 95% CI: 0.15-0.43). Although there is no systematic difference in efficiency by size of screening unit there are inefficiencies in both large and small units and there is scope for many individual units to improve their use of current resources. It will be necessary for decision-makers to examine the practices of individual screening units before considering options for how best to improve their resource use. DEA can help to identify feasible options.
Breast Neoplasms↗
Clinical and cost-effectiveness of donepezil, rivastigmine and galantamine for Alzheimer's disease: a rapid and systematic review.
BACKGROUND: Alzheimer's disease is the most common cause of dementia and is characterised by an insidious onset and slow deterioration. The estimated prevalence of Alzheimer's disease for a standard health authority (500,000 people) is about 3330. Current service involves a wide range of agencies, and drug therapy for some patients. OBJECTIVES: To provide a rapid and systematic review of the clinical effectiveness and cost-effectiveness of donepezil, rivastigmine and galantamine in the symptomatic treatment of people suffering from Alzheimer's disease. METHODS: A systematic review of the literature was undertaken. METHODS - DATA SOURCES: Searches were made of electronic databases, including MEDLINE, EMBASE, The Cochrane Library, Database of Abstracts of Reviews of Effectiveness, NHS Economic Evaluation Database, National Research Register, Science Citation Index, BIOSIS, EconLit, MRC Trials database, Early Warning System, Current Controlled Trials, TOXLINE, Index of Scientific and Technical Proceedings, and Getting Easier Access to Reviews. All sources were searched over the period covered by the databases up to March/July 2000. Bibliographies of related papers were assessed for relevant studies and experts were contacted for advice and peer review, and to identify additional published and unpublished references. Manufacturer submissions to the National Institute for Clinical Excellence (NICE) were reviewed. METHODS - STUDY SELECTION: Studies were included if they fulfilled the following criteria: (1) INTERVENTION: donepezil, rivastigmine or galantamine used to treat Alzheimer's disease. (2) PARTICIPANTS: people diagnosed with Alzheimer's disease who meet the criteria for treatment with donepezil, rivastigmine and galantamine. (3) OUTCOMES: measures assessing changes in cognition, function, behaviour and mood, quality of life (including studies assessing carer well-being and carer-input), and time to institutionalisation. (4) DESIGN: systematic reviews of randomised controlled trials (RCTs) and RCTs comparing donepezil, rivastigmine or galantamine with placebo or each other or non-drug comparators were included in the review of effectiveness. Economic studies of donepezil, rivastigmine or galantamine used to treat Alzheimer's disease that included a comparator (or placebo) and both the costs and consequence (outcomes) of treatment were included in the review of cost-effectiveness. Studies in non-English language, and abstracts and conference poster presentations of systematic reviews, RCTs and economic evaluations were excluded. Two reviewers identified studies by independently screening study titles and abstracts, and then by examining the full text of selected studies to decide inclusion. METHODS - DATA EXTRACTION AND QUALITY ASSESSMENT: Data extraction and quality assessment were undertaken by one reviewer and checked by a second reviewer, with any disagreements resolved through discussion. The quality of RCTs was assessed using the Jadad scale and the quality of systematic reviews was assessed using criteria developed by the NHS Centre for Reviews and Dissemination. The quality of economic evaluation studies was assessed by their internal validity (i.e. the methods used) using a standard checklist, and external validity (i.e. the generalisability of the economic study to the population of interest) using a series of relevant questions. METHODS - DATA SYNTHESIS: The clinical effectiveness and cost-effectiveness of donepezil, rivastigmine and galantamine were synthesised through a narrative review with full tabulation of results of all included studies. In the economic evaluation, the reviewers assessed whether adjustments could be made to existing models to reflect the current situation in England and Wales. RESULTS - CLINICAL EFFECTIVENESS: (1) Donepezil--three systematic reviews and five RCTs (plus four studies from industry (unpublished data, submitted as commercial in confidence)) were found. Results suggest that donepezil is beneficial when assessed using global and cognitive outcome measures. (2) Rivastigmine--three systematic reviews and five RCTs (plus two studies from industry (unpublished data, submitted as commercial in confidence)) were found. Results suggest that rivastigmine is beneficial in terms of global outcome measures. (3) Galantamine--one systematic review and three RCTs (plus three studies from industry (unpublished data, submitted as commercial in confidence)) were found. Results suggest that galantamine is beneficial in terms of global, cognitive and functional scales. RESULTS - SUMMARY OF BENEFITS: It is difficult to quantify benefits from the evidence available in the literature. Statistically significant improvements in tests such as ADAS-cog (Alzheimer's Disease Assessment Scale cognitive subscale) may not be reflected in changes in daily life. (ABSTRACT TRUNCATED)
Alzheimer Disease↗
A tool to improve quality of reporting published economic analyses.
OBJECTIVES: To test the feasibility of obtaining a baseline level of quality of reporting for cost-utility analysis (CUA) studies using the British Medical Journal economic submissions checklist, test interrater reliability of this tool, and discuss its longer term implications. METHODS: CUA studies in peer-reviewed English language journals in 1996, assessed using the British Medical Journal checklist, a quality index, and interrater reliability correlations. RESULTS: Forty-three CUA studies were assessed, with 23 checklist items acceptable and 10 items inadequate. Lowest quality scores were reported in specialist medical journals. Proportional agreement between assessors was over 80%. CONCLUSIONS: The British Medical Journal checklist is a feasible tool to collect baseline information on the quality of reporting in journals other than the British Medical Journal. Editors of specialist medical journals are in greatest need of economic guidance. If handled carefully, they might consider adopting the British Medical Journal checklist.
Cost-Benefit Analysis↗
The role of a pre-scored multi-attribute health classification measure in validating condition-specific health state descriptions.
It is common to find specially constructed condition-specific health state descriptions used as the basis for benefit assessment in cost-utility analysis. For this approach to be valid it is necessary to have valid descriptors of health states. Yet the evidence demonstrating descriptive validity has been neglected in economic evaluation. This paper reports on the validity, reliability and feasibility of obtaining values from specially constructed condition-specific descriptions of breast cancer screening by mapping these descriptions into a pre-scored multi-attribute health classification measure (the EuroQol instrument) and comparing the values obtained with those derived from a time trade-off exercise. In doing so, it highlights the importance of descriptive validity in explaining why different valuation methods produce different results. Four descriptions typically associated with ex post true negative, false positive, true positive and false negative breast screening results were considered.
Adult↗
Raising the quality of cost-utility analyses: lessons learnt and still to learn.
Heightened awareness by health care funders of the need to find more efficient ways of using scarce health care resources has led to greater demand for evidence of cost-effectiveness. Implicit in this demand is that evidence is generated using clear reporting and accepted methods. The research reported here updates an earlier review of published cost-utility analyses (CUAs) to address whether previously identified gaps in reporting have diminished over time. Raising CUA standards requires systematic and regular reviews of published material to allow adequate monitoring and evaluation. There is also a need to 'appraise the appraisers' in the sense of reviewing peer-review processes. This is particularly so in those journals which are growing in importance as outlets for economic evaluation information. The findings from this study indicate continuing variation in the quality of reporting. At the lower end of this spectrum improvements could be made in the reporting of comparators, in the clarity of effectiveness evidence, in the assignment of utility weights to health states and in reporting of sensitivity analysis. CUAs published in peer-reviewed specialist medical journals were more likely to be lower in quality suggesting guidance on the appraisal of economic submissions needs to be extended to the editors of these particular journals. These findings could be used to help to target attempts to raise the quality of evidence-based CUA information.
Australia↗
Valuing temporary and chronic health states associated with breast screening.
The aim of this study was to derive quality of life values for the four key breast screening outcomes (true negative, false positive, true positive and false negative), including the quality of life effects of the screening and treatment processes. In doing so, methodological issues in health status measurement were explored, in particular the valuation of temporary health states. The true negative and false positive descriptions were temporary health states, lasting for short term durations (12 months) and the true positive and false negative outcomes were chronic health states lasting for long term durations (rest of life). Descriptions of breast screening outcomes were valued using the time trade-off technique and the visual analogue scale. Paired comparisons between TTO values for states with the same duration found a difference between the true negative and the false positive time trade-off values but no difference for true positive and false negative descriptions. The TTO values for the false positive were low. The study highlights several important methodological issues such as the use of the two stage procedure for valuing temporary health states, the impact of duration on values, the impact of anchor points, and the importance of qualitative analysis of respondents values. Further empirical testing of all these issues is recommended.
Adult↗
Generalizing costs from trials. Analyzing center selection bias in a breast screening trial.
With a growing number of economic evaluations being conducted alongside trials, there is a need to address, and if necessary, investigate the generalizability of results derived from trials. This paper explores the generalizability of costs from a trial investigating the frequency of breast cancer screening in the United Kingdom.
Bias↗
Less fog on the Tyne? Programme budgeting in Newcastle and North Tyneside.
Programme Budgeting (PB) has been widely promoted as a model for the better conduct of the work of Health Authorities in the National Health Service in the United Kingdom. This paper reports on a project which looked at the development of PB in Newcastle and North Tyneside Health Authority (NNTHA), concentrating on the construction of a computerised tool for the compilation and analysis of programme budgets. The main activities carried out were a survey of user requirements for PB, a survey of data availability, the collection of data to construct programme budgets, and development of a relational database for storing and manipulating PB information. The main source of data was the Contract Minimum Data Set, which was supplemented by data from a number of other sources to give comprehensive information on spending in NNTHA. Costed activity data were produced, which could be aggregated in a large number of ways, such as by care setting (inpatient, outpatient, community, general practice, etc.), disease group (ICD9 chapter headings), case mix (Healthcare Resource Groups) and socio-demographic variables (age/sex, locality of GPs practice).
Budgets↗
UK breast screening programme: how does it reflect the Forrest recommendations?
OBJECTIVE: To compare the UK breast screening programme with the Forrest Report recommendations of 1986. SETTING: The UK breast screening programme. METHODS: A postal survey of 97 local breast screening programmes in the United Kingdom. The main outcome measures were the frequency of screening, the use of two view screening on incident screens, reading of screening mammograms, assessment procedures and visits, staffing levels, and the use of building and equipment. RESULTS: Eighty two (85%) of the questionnaires were completed and returned. All programmes screen every three years, as Forrest intended, with the exception of one health region which screens more often. The national policy is to use two views on incident screens where there is a clinical indication. None the less, 14% of programmes are using, or intending to use, two views on all women. Double reading of mammograms is not recommended in the United Kingdom outside Scotland, but is used by 88% of programmes. All programmes have access to the equipment required for the assessment techniques recommended by Forrest. Variation exists between programmes in the procedures women can expect to receive at their initial assessment visit and in the total number of assessment visits. Sixty eight per cent of programmes' breast screening budgets cover the staff required for a multidisciplinary team as defined by the Forrest Report. Ninety three per cent of screening programmes are organised around static sites, with 86% of these also using mobile vans. CONCLUSIONS: The national programme is following recommendations about the frequency of screening, but there seems to be some divergence from policy as regards the use of double reading, two views at incident screening, and the multidisciplinary team covered by the programmes' breast screening budget. Further research is needed on the effectiveness and cost effectiveness of two view incidence screening, double reading, and non-radiologists as readers. Investigation is also needed of the costs and effects of the variation between programmes in the number of assessment visits a woman may have.
Breast Neoplasms↗
Clearing the fog on the Tyne: programme budgeting in Newcastle and North Tyneside Health Authority.
When the internal market was introduced, the National Health Service Management Executive envisaged purchasing as a process by which contracts would be developed from information concerning current services, modified in the light of strategic purchasing objectives, epidemiological needs assessment and indicators of comparative performance and efficiency. Our concern in this paper is with the promotion of efficiency. We distinguish between three levels and, in particular, discuss how the programme budgeting and marginal analysis framework can be used in the promotion of efficiency at 'top-level' decision making. PB/MA can be used to give a focus to needs assessment and forge explicit links between individual contracts within a well defined health strategy. The objectives of the current research and development ongoing within Newcastle and North Tyneside Health Authority are outlined. The intention is to achieve programme budgeting which is more responsive to decision makers' needs and is consistent with the contracting cycle. However, a number of constraints are expected to impede development. They include transferability of national and international information; absence of local information on epidemiology, effectiveness and cost-effectiveness; limitations on the accuracy and precision of programme budgets; and whether purchasers make strategic decisions based on macro budgets. The contribution of each of these constraints is explored.
Budgets↗
Will early detection of breast cancer reduce the costs of treatment?
A substantial investment in resources is required to provide a population-based mammography screening program. At the same time, screening may also reduce the costs of treating breast cancer. Empirical evidence to support such cost savings, however, is limited. This paper presents a simulation of the impact on treatment costs of a population-based mammography screening program in New South Wales. A 1991 cohort of women aged 45 to 69 years is followed for the period 1991 to 2023. With two-yearly screening, the present value of the total health service costs for this cohort would be approximately $112 million. Primary treatment, at $60 million, would cost $5 million more with screening than without. Treatment for advanced stages of the disease would cost $22 million less. Overall, this analysis suggests that savings in treatment costs are relatively small in relation to the overall resource requirements of organised screening.
Aged↗
QALY league tables: handle with care.
This paper examines some of the difficulties in using QALY league tables in priority setting. Such tables sometimes are seen as being 'the' way to prioritise in health care and in particular, at present, with respect to priority setting among purchasers in the UK NHS. However the paper highlights the fact that the base on which such tables is built is small--relatively few studies in the English language using CUA have been conducted anywhere. Further, four issues which require handling with care are set out: (i) the relevant measure of cost in QALY league tables has to be restricted to health service resource use; (ii) the relevant measure of benefit in QALY league tables is clearly restricted to QALYs, thereby the utility of health gains and indeed the maximisation of the utility of health gains; (iii) in incorporating the results of CUA studies into QALY league tables there is a need for greater clarification on what the margin constitutes; and (iv) those who might use CUA results in QALY league tables need to ascertain whether the original context of the study will allow the results to be transferred to the local context of the decision maker. The paper suggests that there is a need to be quite clear what goal QALY league tables serve. The authors argue that the only legitimate (and clearly important) goal of QALY league tables is the maximization of the utility of health gains within a health service budget.(ABSTRACT TRUNCATED AT 250 WORDS)
Cost-Benefit Analysis↗
Econometric analyses of national health expenditures: can positive economics help to answer normative questions?
The size of national health care expenditure is an important research and policy issue. This paper reviews theoretical and empirical analyses of an implied optimal size for a health sector. Various economic theories are explicitly or implicitly invoked, but none is fully satisfactory. Theory provides, at best, a loose justification for empirical specifications of health sector behaviour. Nevertheless, this has a large and growing empirical research industry. The complexity of the issues provides an excuse for reliance on empirical analyses using ad hoc models. The paper analyses aggregate time-series data, using the cointegration approach, on health, health care expenditures and national income. Only one national model met both statistical criteria and showed a significant relationship: between potential life years lost and health care expenditure in the UK. The case for any general relationships remains unproven. There is no objective scientific method to determine optimal health expenditure, nor should we expect one. However, positive analyses can help with normative questions. A better understanding of health expenditure determination would arise from better specification of the relationships, perhaps by analysis at a lower level of aggregation.
Bias↗
Setting priorities in the new NHS: can purchasers use cost-utility information?
It could be argued that the success of the new NHS depends, to some extent, on the production of accurate cost-utility information. This raises questions about the quality of this information, whether it can be transferred from one study setting to another and whether such information can reasonably be used 'at the negotiating table'. The quality of some studies is open to question. Some agreement is needed on issues of principle and more work is required to make cost-utility analyses more applicable locally.
Contract Services↗
Framing and labelling effects in health descriptions: quality adjusted life years for treatment of breast cancer.
At present there is a growing interest in the use of cost-utility analysis (CUA) to a point where it merits serious consideration by health care decision makers. However, there remain a number of theoretical and practical issues to be resolved including the way in which quality of life information is presented and described to subjects. Two potential sources of influence in the construction of the quality adjusted life year (QALY) values elicited for a recent Australian CUA of mammography screening have been investigated. 180 subjects were randomly allocated to nine different presentations of two breast cancer health descriptions to investigate the impact of some framing and labelling effects. No statistically significant differences were found in the valuations placed on these descriptions when framing and labelling effects were taken into account, either as separate framing and labelling factors or as interactions with one another. A significant difference was found in the particular values of descriptions that were written in the third person that differed in terms of whether the word "cancer" was used. The main contribution of these data is to the robustness of the health descriptions used in the cost-utility analysis of mammography screening.
Aged↗