Real-world evidence (RWE) is derived from real-world data (RWD) sources including electronic health records, claims data, registries (disease, product) and pragmatic clinical trials. The importance of RWE derived from RWD has been once again demonstrated during the coronavirus disease 2019 (COVID-19) pandemic, as it can improve patient care by complementing information obtained from traditional clinical trial programs. Additionally, RWE can generate insights into disease mechanisms, epidemiology, patient flows in and out of healthcare systems, and drivers and barriers to optimal clinical care in realworld settings. Identifying unmet medical needs is crucial as it often can inform which investigational new drugs enter clinical trial testing, and RWE studies from hospital settings have contributed substantial progress here. RWE can also optimize the design of clinical studies, inform benefit risk assessments and use networks of pragmatic studies to help with clinical trial feasibilities and eventual trial initiation. The challenges of RWD include data quality, reproducibility and accuracy which may affect validity. RWD
Background:
Proprotein convertase subtilisin-kexin type 9 (PCSK9) inhibitors are a novel
class of non-statin lipid lowering therapy that reduce LDL-cholesterol by 50 - 60%. PCSK9 inhibitors
decrease LDL-cholesterol by preventing intracellular degradation of LDL receptors; subsequently, a
greater number of LDL-receptors are available on the cell surface to extract circulating LDL.
Objective:
To describe the origins of PCSK9 inhibitors and their current use in clinical practice.
Methods:
We performed a narrative review of the PCSK9 inhibitor class of drugs.
Results:
Current data indicate that PCSK9 inhibitors effectively reduce LDL-cholesterol and are well
tolerated and safe. PCSK9 inhibitors have also been shown to reduce cardiovascular event rates in
patients with stable atherosclerotic cardiovascular disease and in patients with a recent (up to one year)
acute coronary syndrome. Given the costs, chronicity of the treatment and the potential budget impact,
PCSK9 inhibitors are often limited to patients with the highest absolute risk for major adverse cardiovascular
events despite optimal treatment with high-intensity statin and ezetimibe.
Conclusion:
PCSK9 inhibitors have a favorable safety, efficacy and tolerability profile. Postmarketing
safety surveillance and real-world studies are needed to further support the long-term safety
profile of this class of medicine.
Background: Neuroendocrine tumours are known to impact patients’ quality of life because of the symptoms caused by hypersecretion of serotonin and other peptides, in particular diarrhoea and flushing.Aim: The Q-SYMTU study was a prospective, observational registry that included 24 symptomatic patients with gastroenteropancreatic neuroendocrine tumours.Setting: Multiple oncology practices in South Africa.Method: Patients’ level of satisfaction was evaluated for a 6-month period from initiation of treatment with lanreotide Autogel.Results: The number of patients who had greater than 50% self-reported reduction in daily episodes of diarrhoea and flushing were 67% and 80%, respectively, over a 6-month period.Conclusion: Treatment with lanreotide Autogel was generally well tolerated, as demonstrated by low occurrence of Grade 3 and Grade 4 adverse events (AEs). None of the Grade 4 AEs were related to the study treatment. No Grade 5 AEs were reported.
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