PharmacoEconomics
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The health-related quality of life (HR-QOL) of children and adolescents is increasingly considered a relevant topic for research. Instruments to assess quality of life in children and adolescents of a generic as well as disease- or condition-specific nature are being developed and applied in epidemiological surveys, clinical studies, quality assurance and health economics. This paper attempts to give an overview on the state of the art of HR-QOL assessment in children as it relates to methodological and conceptual challenges. ⋯ It was concluded that (i) HR-QOL instruments are available to assess the dimensions of the construct relevant to children and adolescents; (ii) provided that an instrument was constructed in an appropriate way, the dimensions of HR-QOL can be measured in an interculturally comparable manner; (iii) the HR-QOL of children and adolescents can and should be ascertained by self-rating; (iv) the measurement instruments used have to consider maturity and cognitive development; (v) only generic quality-of-life instruments allow for an assessment of HR-QOL in both healthy and chronically ill children and adolescents; (vi) the representation of HR-QOL achieved through a singular index value is connected to strict psychometric conditions: the index instrument has to be tailored to these psychometric conditions; (vii) how far utility measures are employable with children and adolescents has to be investigated in further studies. The problem aspects identified indicate the necessity for further research. Nevertheless, instruments for assessing the HR-QOL of children and adolescents can be identified that meet the requirements mentioned above.
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Randomized Controlled Trial Multicenter Study Comparative Study
Economic evaluation of weekly epoetin alfa versus biweekly darbepoetin alfa for chemotherapy-induced anaemia: evidence from a 16-week randomised trial.
A 16-week, open-label, multicentre, randomised trial of weekly epoetin alfa 40 000 units versus biweekly darbepoetin alfa 200microg among 358 patients with solid-tumour cancers and chemotherapy-induced anaemia demonstrated superior haematological outcomes with epoetin alfa. We sought to compare resource use, costs and clinical outcomes between treatment groups and report the results using a cost-consequences framework. ⋯ Most clinical outcome measures suggested greater improvement with epoetin alfa relative to darbepoetin alfa, but most costs for both agents appeared similar. Decision makers must evaluate the differences in costs and efficacy measures that are most relevant from their perspectives.
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As modellers push to make their models more accurate, the ability of others to understand the models can decrease, causing the models to lose transparency. When this type of conflict between accuracy and transparency occurs, the question arises, "Where do we want to operate on that spectrum?" This paper argues that in such cases we should give absolute priority to accuracy: push for whatever degree of accuracy is needed to answer the question being asked, try to maximise transparency within that constraint, and find other ways to replace what we wanted to get from transparency. There are several reasons. ⋯ Transparency by itself can't answer this; only demonstrations that the model accurately calculates or predicts real events can. Rigorous simulations of clinical trials are a good place to start. This is the type of empirical validation we need to provide if the potential of mathematical models in pharmacoeconomics is to be fully achieved.
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Excellent treatment outcomes with long-term durability and few adverse effects are expectations of treatments for chronic conditions. The long-term cost effectiveness of newer treatments for benign prostatic hyperplasia (BPH), including high-energy transurethral microwave thermotherapy (TUMT) and combination pharmaceutical therapy, has not been sufficiently studied against existing alternatives. The objective of this study was to estimate the incremental cost effectiveness of BPH treatment alternatives. ⋯ Our model suggests that alpha-blockers and TURP appear to be the most cost-effective alternatives, from a US payer perspective, for BPH patients with moderate and severe symptoms, respectively. TUMT was promising for patients with moderate symptoms and the oldest patients with severe symptoms, but otherwise was dominated. Value of information analysis could be used to determine the net benefit of additional research.
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Clopidogrel (Plavix) is a selective inhibitor of adenosine diphosphate-induced platelet aggregation. In patients with acute coronary syndromes (ACS) [unstable angina or non-ST-segment elevation myocardial infarction], clopidogrel plus aspirin (acetylsalicylic acid) for up to 1 year significantly reduced the risk of cardiovascular events relative to placebo plus aspirin in the well designed clinical trial CURE (Clopidogrel in Unstable angina to prevent Recurrent Events) and its substudy in patients undergoing percutaneous coronary intervention (PCI) [PCI-CURE]. In pharmacoeconomic evaluations based on data from these trials conducted in a number of countries that used a variety of models, methods and/or type of costs, clopidogrel plus aspirin was consistently predicted to be cost effective relative to aspirin alone in the management of patients with ACS, including those undergoing PCI. ⋯ Cost-utility analyses based on the CURE trial suggest that, relative to lifelong aspirin alone, clopidogrel plus aspirin for 1 year followed by aspirin alone is associated with incremental costs per QALY gained that are below the traditional threshold of cost utility in Spain, the UK and the US. In patients with ACS, including those undergoing PCI, the addition of clopidogrel to standard therapy with aspirin is clinically effective in preventing cardiovascular events. Available pharmacoeconomic data from several countries, despite some inherent limitations, support the use of clopidogrel plus aspirin for up to 1 year as a cost-effective treatment relative to aspirin alone in this patient population.