BMJ open
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Non-randomised clinical trial designs involving comparisons against external controls or specific standards can be used to support regulatory submissions for indications in diseases that are rare, with high unmet need, without approved therapies and/or where placebo is considered unethical. The objective of this review was to summarise the characteristics of non-randomised trials submitted to the European Medicines Agency (EMA) or Food and Drug Administration (FDA) for indications in haematological cancers, haematological non-malignant conditions, stem cell transplants or rare metabolic diseases. ⋯ There has been a large increase in submissions to the EMA and FDA using non-randomised study designs involving comparisons with external controls in recent years. This study demonstrated that regulators may be willing to approve such submissions, although approvals are often conditional on further confirmatory evidence from postapproval studies.
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We aim to evaluate the diagnostic test accuracy (DTA) of intraoperative evoked potential (EP) monitoring to detect cerebral injury during clipping of cerebral aneurysms. ⋯ CRD42015016884.
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Predicting successful liberation from mechanical ventilation (MV) among critically ill patients receiving MV can be challenging. The current parameters used to predict successful extubation have shown variable predictive value. Brain natriuretic peptide (BNP) has been proposed as a novel biomarker to help guide decision-making in readiness for liberation of MV following a spontaneous breathing trial (SBT). Current evidence on the predictive ability of BNP has been uncertain, and BNP has not been integrated into clinical practice guidelines. ⋯ CRD42018087474.
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To identify, characterise and explain common and specific features of the experience of treatment burden in relation to patients living with lung cancer or chronic obstructive pulmonary disease (COPD) and their informal caregivers. ⋯ CRD42016048191.