Current medical research and opinion
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The increasing use of RWE in regulatory and reimbursement decision-making indicates the significant progress that has been made in building trust in RWE through greater transparency. This review of the published literature and key online sources was conducted to provide an update on progress towards greater transparency in RWE, based on four key barriers to trust identified in a 2016 paper and applying learnings from transparency initiatives established for RCTs, such as the US FDA Amendments Act (FDAAA) 2007 Final Rule. ⋯ Programs such as the REPEAT Initiative and RWE DUPLICATE are investigating reproducibility of RWE studies and improving understanding of the circumstances when valid inference on treatment effects can be obtained from RWE studies. Further work is needed to embed and to implement the new tools and guidance that are available, and thus raise standards for RWE transparency towards the levels expected for RCTs.
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Observational Study
Development, validation and implementation of the medical affairs pharmaceutical physician work-related quality of life instrument.
Medical affairs pharmaceutical physicians (MAPPs) are at risk for low work-related quality of life (WRQoL). The aim of this study was to develop, validate and implement the first WRQoL instrument for this population. ⋯ Discriminant validity of the 39-item MAPPWRQoL instrument was confirmed. The Jandhyala method successfully developed and validated a specific WRQoL instrument and may be applied to similar populations, such as junior doctors and UK general practitioners.
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Although dosing regimens of targeted therapies (TT) for ulcerative colitis (UC) and Crohn's disease (CD) are guided by market authorizations and clinical guidelines, little is known about clinical guideline adherence or outcomes in patients receiving escalated doses of TT due to lack of response. This real-world study explored the prevalence of dose escalation and compared outcomes between patients receiving standard and escalated TT doses. ⋯ Dose escalation is not always an effective approach to resolve inadequate or loss of response in UC and CD, highlighting a need for more therapeutic options or alternative treatment strategies in patients unresponsive to TT.
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The post-acute (long) COVID-19 Quality of Life instrument is the only specific instrument designed to assess the quality of life in long COVID patients. The present study aims to make a transcultural adaptation and validation into Spanish of the disease-specific (long COVID) quality of life instrument, post-acute (long) COVID-19 Quality of Life, to have a tool for objective measurement of quality of life in this population. ⋯ This study shows that the Spanish version of the post-acute (long) COVID-19 Quality of Life instrument is an appropriate and valid tool for assessing the quality of life of long COVID patients.
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In the study on triglyceride-induced pancreatitis (TG-IAP), a core clinical dataset using the Jandhyala method was developed to collect the minimum amount of information for each patient presenting with TG-IAP globally. This approach offered a unified framework for observing multiple populations of TG-IAP patients using the same set of indicators, resulting in a considerably larger and uniform real-world population. It was understood that when this core dataset is implemented in a patient registry it could address the issue of missing data in observational studies and produce higher-quality research. In this paper, the protocol used to design and implement a patient registry for this core dataset to generate real-world evidence from multiple sites is described. ⋯ Global epidemiological data on hypertriglyceridemia and its role in acute pancreatitis is limited. Using real-world evidence, this registry, along with others like it, may help healthcare providers understand the variability, progression, natural history, and burden of the disease, and improve the diagnosis and management of HTG and TG-IAP.