Health technology assessment : HTA
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Health Technol Assess · Jun 2006
ReviewA systematic review of the clinical effectiveness and cost-effectiveness of enzyme replacement therapies for Fabry's disease and mucopolysaccharidosis type 1.
To determine the clinical effectiveness and cost-effectiveness of the administration of intravenous enzyme replacement therapy (ERT) to symptomatic patients for the prevention of long-term damage and symptoms in Fabry's disease and in mucopolysaccharidosis type 1 (MPS1). ⋯ Although ERT for treating the 'average' patient with Fabry's disease exceeds the normal upper threshold for cost-effectiveness seen in NHS policy decisions by over sixfold, and the value for MPS1 is likely to be of a similar order of magnitude, clinicians and the manufacturers argue that, as the disease is classified as an orphan disease under European Union legislation, it has special status, and the NHS has no option but to provide ERT. More information is required before the generalisability of the findings can be determined. Although data from the UK have been used wherever possible, this was very thin indeed. Nonetheless, even large errors in assumptions made will not reduce the ICER to anywhere near the upper level of treatments usually considered cost-effective. In order to overcome limited evidence on the natural history of the disease and the clinical effectiveness of the intervention, the establishment of disease-specific data registries is suggested to facilitate the process of technology assessment and improving patient care. These registries should attempt to include all affected patients in the UK, and collect longitudinal patient level data on clinically relevant problems, interventions received and quality of life in a utility format.
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Health Technol Assess · Jun 2006
ReviewDiagnostic tests and algorithms used in the investigation of haematuria: systematic reviews and economic evaluation.
To determine the most effective diagnostic strategy for the investigation of microscopic and macroscopic haematuria in adults. ⋯ There are insufficient data currently available to derive an evidence-based algorithm of the diagnostic pathway for haematuria. A hypothetical algorithm based on the opinion and practice of clinical experts in the review team, other published algorithms and the results of economic modelling is presented in this report. This algorithm is presented, for comparative purposes, alongside current US and UK guidelines. The ideas contained in these algorithms and the specific questions outlined should form the basis of future research. Quality assessment of the diagnostic accuracy studies included in this review highlighted several areas of deficiency.
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Health Technol Assess · Jun 2006
Randomized Controlled TrialCognitive behavioural therapy in addition to antispasmodic therapy for irritable bowel syndrome in primary care: randomised controlled trial.
To determine whether cognitive behavioural therapy (CBT) in addition to antispasmodic treatment offers a cost-effective benefit to primary care patients with irritable bowel syndrome (IBS) and to identify predictors of outcome. ⋯ Specially trained practice nurses can provide effective CBT to primary care patients with a clinical diagnosis of IBS, which although effective does not reduce service or social costs. Using a variety of measures the beneficial therapeutic effects of the addition of CBT to antispasmodic therapy persist for up to 6 months. Future research might include studies of the long-term follow-up of IBS patients treated with CBT, cost-benefit analyses comparing CBT with other therapeutic approaches to IBS, and evaluating means of training both non-specialist health professionals and GPs to deliver CBT.