Current medical research and opinion
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To update on and describe the role of Disease Specific Programmes (DSPs), a multi-perspective real-world data (RWD) source, in the context of the evolution of the value and acceptance of real-world evidence (RWE) in clinical, regulatory and guideline decision-making. ⋯ DSPs provide a wealth of RWD in addition to evidence generated by registries, clinical trials and observational research, with wide use for the pharmaceutical industry, government, funding/regulatory bodies, clinical practice guideline insights and, most importantly, informing improvements in people's lives. The depth, breadth and heritage of information collected via DSPs since 1995 is unparalleled, extending understanding of how diseases are managed by physicians in routine clinical practice and why treatment choices are made, patients' perceptions of their disease management, and caregiver burden.
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Priority setting in health research has been described as essential due to disparities within and between countries and populations. Commercial benefits to the pharmaceutical industry may increase the generation and use of regulatory Real-World Evidence which has recently been reported in the literature. Research must be steered by valuable priorities. This study's objective is to identify key gaps in the knowledge of triglyceride-induced acute pancreatitis by generating a list of potential research priorities for a Hypertriglyceridemia Patient Registry. ⋯ The TG-IAP core dataset and research priorities combined can develop a globally harmonized framework where TG-IAP patients can be observed simultaneously using the same set of indicators. This will increase knowledge of the disease and facilitate higher-quality research by addressing issues related to incomplete data sets in observational studies. Furthermore, validation of new tools will be enabled, and diagnosis and monitoring will be improved as well as the detection of changes in disease severity and subsequent disease progression, improving the management of patients with TG-IAP overall. This will inform personalized patient management plans and improve patient outcomes along with their quality of life.
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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.