When Randomized Clinical Trials and Real‐World Evidence Say the Same: Tocilizumab and Its Cardiovascular Safety

2019 ◽  
Vol 72 (1) ◽  
pp. 4-6
Author(s):  
Seoyoung C. Kim ◽  
Sebastian Schneeweiss
2020 ◽  
Vol 26 (1) ◽  
pp. 5-9
Author(s):  
Monika Kozieł ◽  
Gregory Y. H. Lip ◽  
Tatjana S. Potpara

Real world registries of patients with atrial fi brillation (AF) have provided important evidence on contemporary AF management and adherence to guidelines in real-world patients across most of regions in Europe. While prospective randomized clinical trials are the ‘gold standard’ of evidence, we recognize that trials have specifi c inclusion/exclusion criteria and many groups of patients can be under-represented. Thus, real world evidence is needed to supplement and augment the evidence, especially for the under-represented patient groups (eg. the very elderly and frail, ethnic minorities, end stage renal failure, those in nursing homes, cognitive impairment, etc) that have been largely under-represented or excluded from clinical trials. The BALKAN-AF survey is the largest prospective, multicenter (a total of 49 centres), observational AF dataset from the Balkans, a European region inhabited by about 10% of the European population that has been under-represented in many prior clinical trials or registries. In BALKAN-AF, data regarding consecutive subjects with electrocardiographically documented non-valvular AF were collected in seven Balkan countries (Albania, Bosnia & Herzegovina, Bulgaria, Croatia, Montenegro, Romania and Serbia) by a cardiologist or an internal medicine specialist where cardiologist was not available. The Serbian Atrial Fibrillation Association created and conducted the BALKAN-AF survey (performed from December 2014 to February 2015).


Vaccine ◽  
2021 ◽  
Vol 39 (16) ◽  
pp. 2224-2236 ◽  
Author(s):  
Darron R. Brown ◽  
Elmar A. Joura ◽  
Glorian P. Yen ◽  
Smita Kothari ◽  
Alain Luxembourg ◽  
...  

Author(s):  
O. R. Shevchenko ◽  
A. S. Kolbin

Pragmatic clinical trials (PCTs) allow combining the advantages of observational trials in real-world evidence with the scientific rigor of randomized clinical trials (RCTs), and thereby provide more effective answers to questions of real-world evidence.Aim. Assessment of differences in conducting RCTs and PCTs, as well as analysis of the features related to conducting PCTs at different stages.Methods. An analysis of publications in the period from 1999 to 2017 was conducted to identify data on PCTs.Results. There are significant differences in conducting classic RCTs and PCTs. First, PCTs use more flexible inclusion criteria and differ in the approach to choosing an investigator’s site. Also, the procedure for obtaining informed consent has significant differences from that of classical RCTs; alternative options are proposed but a unified approach has not yet been developed. When conducting PCTs, monitor intervention should be minimal in order not to interfere in the routine therapy, which, however, can lead to a violation of reporting. A possible solution may be remote data collection.Conclusion. PCTs represent a huge potential for studying the effectiveness of drugs in real-world evidence. However, despite a significant increase in the number of such trials, there are still a sufficient number of points that need to be resolved.


2021 ◽  
Vol 14 (7) ◽  
pp. 700
Author(s):  
Theodoros Mavridis ◽  
Christina I. Deligianni ◽  
Georgios Karagiorgis ◽  
Ariadne Daponte ◽  
Marianthi Breza ◽  
...  

Now more than ever is the time of monoclonal antibody use in neurology. In headaches, disease-specific and mechanism-based treatments existed only for symptomatic management of migraines (i.e., triptans), while the standard prophylactic anti-migraine treatments consist of non-specific and repurposed drugs that share limited safety profiles and high risk for interactions with other medications, resulting in rundown adherence rates. Recent advances in headache science have increased our understanding of the role of calcitonin gene relate peptide (CGRP) and pituitary adenylate cyclase-activating polypeptide (PACAP) pathways in cephalic pain neurotransmission and peripheral or central sensitization, leading to the development of monoclonal antibodies (mAbs) or small molecules targeting these neuropeptides or their receptors. Large scale randomized clinical trials confirmed that inhibition of the CGRP system attenuates migraine, while the PACAP mediated nociception is still under scientific and clinical investigation. In this review, we provide the latest clinical evidence for the use of anti-CGRP in migraine prevention with emphasis on efficacy and safety outcomes from Phase III and real-world studies.


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