Current medical research and opinion
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Low rates of inclusion of real-world evidence (RWE) during regulation may arise from lack of clarity and consensus on its definition. A conceptually mature definition of RWE may have pragmatic utility, increasing its inclusion during regulation. The aim was to develop a definition of RWE to promote inclusion in regulatory submissions and assess its conceptual maturity. ⋯ A definition of RWE was generated that may increase its inclusion during medicines regulation, especially with further refinement from regulators and other stakeholders.
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Parkinson's Disease (PD) is a common progressive neurodegenerative disorder that leads to an imbalance of various neurotransmitters and affects cognitive, motor and non-motor function. Safinamide inhibits monoamine oxidase B in a highly selective and reversible manner and beyond that has anti-glutamatergic properties, with positive effects on motor and non-motor symptoms. The aim of the study was to obtain data about the effectiveness and tolerability of safinamide under routine clinical practice conditions in unselected patients with Parkinson's disease (PD). ⋯ The benefit-risk profile of safinamide was favourable and consistent with the total cohort of the SYNAPSES study. In the subgroups, findings were congruent with the total population, which allows the clinical utilisation of safinamide also in more vulnerable patient groups.
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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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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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Time To Discontinuation (TTD) is defined as the time from the start of treatment to the end of treatment, usually occurring due to loss of efficacy or occurrence of adverse events. It has become an important surrogate efficacy endpoint especially in real-world studies due to its correlation with endpoints such as Progression Free Survival (PFS). The aim of the study is to conduct a literature review of all studies reporting TTD in first-line therapy of Non-Small Cell Lung Cancer (NSCLC). ⋯ Studies reporting TTD are increasing, most notably real-world studies. Given the increasing importance of TTD as an efficacy endpoint, it becomes critical to measure and monitor it in various therapeutic settings such as NSCLC. This is the first study to review all TTD values of drugs used in first-line NSCLC.