Economics notes: cost effectiveness calculations and sample size.
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Biomedical subjects
Publications and source records attributed to D J Torgerson.
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OBJECTIVE: To identify and synthesise the evidence for efficacy and cost effectiveness of topical treatments for superficial fungal infections of the skin and nails of the feet. DESIGN: Systematic review. INTERVENTIONS: Topical treatments for superficial fungal infections. MAIN OUTCOME MEASURES: Cure confirmed by culture and microscopy for skin and by culture for nails in patients with clinically diagnosed fungal infections. RESULTS: Of 126 trials identified in 121 papers, 72 (57.1%) met the inclusion criteria. Placebo controlled trials yielded pooled relative risks of failure to cure skin infections: allylamines (0.30, 95% confidence interval 0.24 to 0.38); azoles (0.54, 0.42 to 0.68); undecenoic acid (0.28, 0. 11 to 0.74); and tolnaftate (0.46, 0.17 to 1.22). Although meta-analysis of 11 trials comparing allylamines and azoles showed a relative risk of failure to cure of 0.88 (0.78 to 0.99) in favour of allylamines, there was evidence of language bias. Seven reports in English favoured allylamines (0.79, 0.69 to 0.91), but four reports in foreign languages showed no difference between the two drugs (1. 01, 0.90 to 1.13). Neither trial of nail infections showed significant differences between alternative topical treatments. CONCLUSIONS: Allylamines, azoles, and undecenoic acid were efficacious in placebo controlled trials. There are sufficient comparative trials to judge relative efficacy only between allylamines and azoles. Allylamines cure slightly more infections than azoles but are much more expensive than azoles. The most cost effective strategy is first to treat with azoles or undecenoic acid and to use allylamines only if that fails.
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Economic evaluations are increasingly being conducted alongside clinical trials of health interventions, with resource consequences being estimated from stochastic data. It is, therefore, important that economic evaluation results, like the clinical results, reflect the underlying variance within the sample data. A statistical methodology, known as bootstrapping, has recently been put forward as a potential method for calculating confidence intervals for cost-effectiveness ratios, yet it is still unusual to see economic evaluations reporting confidence intervals. In this paper we demonstrate the practical application of bootstrapping using real data from clinical trials, and conclude that bootstrapping is easily transferable from theory to practice for the estimation of confidence intervals for cost-effectiveness ratios. We encourage further investigation into its applicability and use.
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Hormone replacement therapy (HRT) is a highly cost-effective treatment for symptoms of the menopause such as hot flushes (flashes). A number of economic evaluations have indicated that it may also be a cost-effective therapy for the prevention of cardiovascular disease and osteoporosis. However, these evaluations are based on the premise that HRT will reduce cardiovascular disease by 30 to 50%. Recent evidence casts doubt on its effectiveness at preventing cardiovascular disease, certainly as a secondary preventive therapy. Furthermore, HRT is likely to increase the incidence of breast cancer. If the effect of HRT on the cardiovascular system is slight or nonexistent, but its effect on breast cancer is modest or strong, then HRT is unlikely to be a cost-effective treatment for asymptomatic women at low risk of osteoporosis. However, the unwanted effects of HRT on the breast may be significantly reduced by targeting therapy to those women with low bone mass and who have other risk factors for fracture. Such a strategy is likely to be more cost effective than a strategy which allows asymptomatic women with low fracture risk to take HRT in the long term. As selective estrogen receptor modulators (SERMs) aggravate menopausal symptoms they are not likely to be an alternative for most perimenopausal women. Therefore, SERMs are more likely to be a competitor to existing and forthcoming bisphosphonates rather than HRT.
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