Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research
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Outcomes research literature has many examples of high-quality, reliable patient-reported outcome (PRO) data entered directly by electronic means, ePRO, compared to data entered from original results on paper. Clinical trial managers are increasingly using ePRO data collection for PRO-based end points. Regulatory review dictates the rules to follow with ePRO data collection for medical label claims. ⋯ It is a guide on the requirements and documentation needed from a data collection systems provider to demonstrate systems validation. It is a practical source of information for study teams to ensure that ePRO providers are using system validation and implementation processes that will ensure the systems and services: operate reliably when in practical use; produce accurate and complete data and data files; support management control and comply with any existing regulations. Furthermore, this short report will increase user understanding of the requirements for a technology review leading to more informed and balanced recommendations or decisions on electronic data collection methods.
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Review
Measuring the quality of a childhood cancer care delivery system: quality indicator development.
A set of indicators to assess the quality of a childhood cancer system has not been identified in any jurisdiction internationally, despite the movement toward increased accountability and provision of high-quality care with limited health care resources. This study was conducted to develop a set of quality indicators (QIs) of a childhood cancer control and health care delivery system in Ontario, Canada. ⋯ The subset of 33 QIs developed on the basis of a systematic literature review and consensus provides the basis for the selection of a set of QIs for ongoing, standardized monitoring of various dimensions of quality in a childhood cancer system.
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Meta Analysis Comparative Study
Cost-effectiveness of new oral anticoagulants compared with warfarin in preventing stroke and other cardiovascular events in patients with atrial fibrillation.
The primary objective was to assess the cost-effectiveness of new oral anticoagulants compared with warfarin in patients with nonvalvular atrial fibrillation. Secondary objectives related to assessing the cost-effectiveness of new oral anticoagulants stratified by center-specific time in therapeutic range, age, and CHADS2 score. ⋯ Results were highly sensitive to patient characteristics. Rivaroxaban and dabigatran 110 mg were unlikely to be cost-effective. For different characteristics, apixaban or dabigatran 150 mg were optimal. Thus, the choice between these two options may come down to the price of apixaban and further evidence on the impact of major and minor bleeds with dabigatran.
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Randomized Controlled Trial Comparative Study
Cost-effectiveness of trabectedin plus pegylated liposomal doxorubicin for the treatment of women with relapsed platinum-sensitive ovarian cancer in the UK: analysis based on the final survival data of the OVA-301 trial.
To estimate the cost-effectiveness of trabectedin plus pegylated liposomal doxorubicin (PLD) compared with PLD alone for the treatment of patients with relapsed platinum-sensitive ovarian cancer who are not expected to benefit from retreatment with platinum-based therapies based on the final survival data published in October 2012. ⋯ The analysis estimated a significant improvement in mean overall survival and incremental cost per QALY compared with that calculated in the original National Institute for Health and Clinical Excellence assessment, which was based on immature survival data.
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Patient-reported outcome (PRO) instruments for children and adolescents are often included in clinical trials with the intention of collecting data to support claims in a medical product label. ⋯ Additional research is needed to provide methodological guidance for future studies, especially for studies involving young children and parents' observational reports. As PRO data are increasingly used to support pediatric labeling claims, there will be more information regarding the standards by which these instruments will be judged. The use of PRO instruments in clinical trials and regulatory submissions will help ensure that children's experience of disease and treatment are accurately represented and considered in regulatory decisions.