BMJ open
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The efficacy of pharmaceuticals is most often demonstrated by randomised controlled trials (RCTs); however, in some cases, regulatory applications lack RCT evidence. ⋯ Despite the frequency with which approvals are granted without RCT results, there is no systematic monitoring of such treatments to confirm their effectiveness or consistency regarding when this form of evidence is appropriate. We recommend a more open debate on the role of marketing authorisations granted without RCT results, and the development of guidelines on what constitutes an acceptable data package for regulators.
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Review Meta Analysis
Efficacy and safety of bevacizumab plus erlotinib versus bevacizumab or erlotinib alone in the treatment of non-small-cell lung cancer: a systematic review and meta-analysis.
Bevacizumab and erlotinib inhibit different tumour growth pathways, and both exhibit beneficial effects in the treatment of non-small-cell lung cancer (NSCLC). However, the efficacy of bevacizumab in combination with erlotinib remains controversial. Therefore, we conducted a meta-analysis to compare combination treatment with bevacizumab and erlotinib to bevacizumab or erlotinib monotherapy in the treatment of NSCLC. ⋯ The addition of bevacizumab to erlotinib can significantly improve PFS and the ORR in the second-line treatment of NSCLC with an acceptable and manageable risk of rash and diarrhoea. Further well-conducted, large-scale trials are needed to validate these findings.
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Observational Study
Regional variation of patient behaviour and reasons for consultation in the general practice of Northern Germany: protocol for an observational study.
Inappropriate supply and an increasing demand on the healthcare system have been of concern for health policy in Germany for at least 15 years. In the primary care setting, this especially relates to an undersupply of general practitioners (GPs) in the countryside. In addition, there seem to be other regional differences, for example, a difference in accessing primary and secondary care between rural and urban areas. Despite these findings, regional differences in health services have not been studied extensively in Germany. Therefore, this study aims to explore regional variations of patient populations and reasons for accessing primary medical care. ⋯ Our study was approved by the Ethics Committee of the Medical Association of Hamburg and is conducted in accordance with the Declaration of Helsinki. Study participants give written informed consent before data collection and data is pseudonymised. Survey data and person identifiers are stored separately in locked cabinets and have restricted availability. The results of our study will be presented at conferences and published in peer-reviewed journals.
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Randomized Controlled Trial Multicenter Study
Study protocol for the randomised controlled trial: combined multimarker screening and randomised patient treatment with ASpirin for evidence-based PREeclampsia prevention (ASPRE).
Pre-eclampsia (PE) affects 2-3% of all pregnancies and is a major cause of maternal and perinatal morbidity and mortality. Prophylactic use of low-dose aspirin in women at risk for PE may substantially reduce the prevalence of the disease. Effective screening for PE requiring delivery before 37 weeks (preterm PE) can be provided by a combination of maternal factors, uterine artery Doppler, mean arterial pressure, maternal serum pregnancy-associated plasma protein A and placental growth factor at 11-13 weeks' gestation, with a detection rate of 75% at a false-positive rate of 10%. We present a protocol (V.6, date 25 January 2016) for the ASpirin for evidence-based PREeclampsia prevention (ASPRE) trial, which is a double-blinded, placebo-controlled, randomised controlled trial (RCT) that uses an effective PE screening programme to determine whether low-dose aspirin given to women from 11 to 13 weeks' gestation will reduce the incidence of preterm PE. ⋯ All eligible women attending for their first trimester scan will be invited to participate in the screening study for preterm PE. Those found to be at high risk of developing preterm PE will be invited to participate in the RCT. Further scans will be conducted for assessment of fetal growth and biomarkers. Pregnancy and neonatal outcomes will be collected and analysed. The first enrolment for the pilot study was in April 2014. As of April 2016, 26 670 women have been screened and 1760 recruited to the RCT. The study is registered on the International Standard Randomised Controlled Trial Number (ISRCTN) registry.
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Survivors of critical illness experience multidimensional disabilities that reduce quality of life, and 25-30% require unplanned hospital readmission within 3 months following index hospitalisation. We aim to understand factors associated with unplanned readmission; develop a risk model to identify intensive care unit (ICU) survivors at highest readmission risk; understand the modifiable and non-modifiable readmission drivers; and develop a risk assessment tool for identifying patients and areas for early intervention. ⋯ Data linkage has approval but does not require ethical approval. The qualitative study has ethical approval. Dissemination with key healthcare stakeholders and policymakers is planned.