scholarly journals Monoclonal Antibodies Targeting CGRP: From Clinical Studies to Real-World Evidence—What Do We Know So Far?

2021 ◽  
Vol 14 (7) ◽  
pp. 700
Author(s):  
Theodoros Mavridis ◽  
Christina I. Deligianni ◽  
Georgios Karagiorgis ◽  
Ariadne Daponte ◽  
Marianthi Breza ◽  
...  
Keyword(s):  
Monoclonal Antibodies ◽  
Real World ◽  
Large Scale ◽  
Randomized Clinical Trials ◽  
Clinical Investigation ◽  
Phase Iii ◽  
Real World Evidence ◽  
Symptomatic Management ◽  
Repurposed Drugs ◽  
Cephalic Pain

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.

The impact of anti-CGRP monoclonal antibodies in resistant migraine patients: a real-world evidence observational study

2021 ◽  
Author(s):  
Marta Torres-Ferrús ◽  
Victor J. Gallardo ◽  
Alicia Alpuente ◽  
Edoardo Caronna ◽  
Eulalia Gine-Cipres ◽  
...  
Keyword(s):  
Monoclonal Antibodies ◽  
Observational Study ◽  
Real World ◽  
Real World Evidence ◽  
The Impact

scholarly journals Awareness, Knowledge, and Utility of RCT Data vs RWE: Results From a Survey of US Cardiologists: Real-world Evidence in Clinical Decision Making

2020 ◽  
Vol 14 ◽  
pp. 117954682095341 ◽  
Author(s):  
Todd C Villines ◽  
Mark J Cziraky ◽  
Alpesh N Amin
Keyword(s):  
Decision Making ◽  
Clinical Practice ◽  
Real World ◽  
Randomized Clinical Trials ◽  
Clinical Decision Making ◽  
Clinical Decision ◽  
The Body ◽  
Representative Sampling ◽  
Real World Evidence ◽  
Wide Range

Real-world evidence (RWE) provides a potential rich source of additional information to the body of data available from randomized clinical trials (RCTs), but there is a need to understand the strengths and limitations of RWE before it can be applied to clinical practice. To gain insight into current thinking in clinical decision making and utility of different data sources, a representative sampling of US cardiologists selected from the current, active Fellows of the American College of Cardiology (ACC) were surveyed to evaluate their perceptions of findings from RCTs and RWE studies and their application in clinical practice. The survey was conducted online via the ACC web portal between 12 July and 11 August 2017. Of the 548 active ACC Fellows invited as panel members, 173 completed the survey (32% response), most of whom were board certified in general cardiology (n = 119, 69%) or interventional cardiology (n = 40, 23%). The survey results indicated a wide range of familiarity with and utilization of RWE amongst cardiologists. Most cardiologists were familiar with RWE and considered RWE in clinical practice at least some of the time. However, a significant minority of survey respondents had rarely or never applied RWE learnings in their clinical practice, and many did not feel confident in the results of RWE other than registry data. These survey findings suggest that additional education on how to assess and interpret RWE could help physicians to integrate data and learnings from RCTs and RWE to best guide clinical decision making.

Survival among metastatic colorectal patients in clinical trials compared to the real world.

2015 ◽  
Vol 33 (3_suppl) ◽  
pp. 673-673
Author(s):  
Ziwei Wang ◽  
Lindsay Hwang ◽  
James Don Murphy
Keyword(s):  
Colorectal Cancer ◽  
Clinical Trial ◽  
Clinical Trials ◽  
Metastatic Colorectal Cancer ◽  
Median Survival ◽  
Real World ◽  
Randomized Clinical Trials ◽  
Phase Iii ◽  
The Real ◽  
Clinical Trial Results

673 Background: Randomized clinical trials play a central role in clinical research though only a small fraction of patients partake in clinical studies. Questions thus arise regarding the generalizability of clinical trial results to the remainder of the population. This study evaluated whether patient survival from randomized clinical trials in metastatic colorectal cancer reflects real world outcomes. Methods: A Pubmed search was used to identify randomized phase III clinical trials of first-line treatment for metastatic colorectal cancer published between 2005 and 2010. We excluded secondary or pooled analyses, second-line treatments, non-metastatic patients, non-English language, and non-randomized studies. Thirty-one clinical trials met these criteria, comprised of 79 distinct clinical trial arms. Overall survival among clinical trial patients was compared to metastatic colorectal cancer patients within the Surveillance, Epidemiology, and End Results (SEER) program. Within SEER, we restricted the analysis time-period and age of patients to match the enrollment period and age of patients within each individual clinical trial. Results: The clinical trials enrolled a total of 16,614 patients. Among all clinical trial arms the median survival ranged from 6.7-62 months, 1-year survival ranged from 30-97%, and 2-year survival ranged from 6-88%. Compared to SEER, the median survival was higher in 95% of the individual clinical trial arms by an average of 5.4 months (p<0.0001). The 1-year survival was higher in 94% of the clinical trial arms by an average of 16.7% (p<0.0001). The 2-year survival was higher in 71% of the clinical trial arms by an average of 7.2% (p<0.0001). Conclusions: This study found substantially improved survival among clinical trial participants compared to patients in the SEER database suggesting that survival estimates from clinical trials may not generalize to the “real world.” Potential patient factors such as differences in underlying comorbidity, performance status, disease burden, as well as variation in treatment could not be addressed in this study, though these factors likely explain some of the observed survival differences.

scholarly journals Principles for the use of large-scale medical databases to generate real-world evidence

2020 ◽  
Vol 2 (1) ◽  
pp. 27-32
Author(s):  
Hiraku Kumamaru ◽  
Shingo Fukuma ◽  
Hiroki Matsui ◽  
Ryo Kawasaki ◽  
Hironobu Tokumasu ◽  
...  
Keyword(s):  
Real World ◽  
Large Scale ◽  
Medical Databases ◽  
Real World Evidence

Real-world evidence of male breast cancer (BC) patients treated with palbociclib (PAL) in combination with endocrine therapy (ET).

2019 ◽  
Vol 37 (15_suppl) ◽  
pp. 1055-1055 ◽  
Author(s):  
Cynthia Huang Bartlett ◽  
Jack Mardekian ◽  
Michelle Yu-Kite ◽  
Matthew James Cotter ◽  
Sindy Kim ◽  
...  
Keyword(s):  
Real World ◽  
Randomized Clinical Trials ◽  
Treatment Guidelines ◽  
Healthcare Providers ◽  
First Line ◽  
Duration Of Treatment ◽  
Real World Data ◽  
The Real ◽  
Real World Evidence ◽  
Line Setting

1055 Background: The rarity of BC in men limits the feasibility of randomized clinical studies in this population. Treatment guidelines recommend that men with BC be treated similarly to postmenopausal women. PAL, a cyclin-dependent kinase 4/6 inhibitor, is used in men with metastatic BC (mBC) in real-world clinical practice, presenting an opportunity to utilize real-world evidence to enable healthcare providers to assess novel agents in this space. Methods: Two parallel approaches were taken. In the first approach, pharmacy and medical claims data from IQVIA Inc were retrospectively analyzed to describe the treatment patterns and duration of PAL + ET (aromatase inhibitor or fulvestrant) compared to ET in men with mBC. The second approach was a retrospective analysis of data derived from electronic health records in the Flatiron Health database to understand real-world clinical response to PAL + ET vs ET alone. Median duration of treatment (mDOT) was estimated by the Kaplan-Meier method. Results: Between Feb 2015 and Apr 2017, 12.9% (147/1139 [IQVIA dataset]) of men receiving treatment for mBC were prescribed PAL + ET for any line of therapy. The mDOT in the first-line setting was numerically longer in the PAL cohort (n=37) compared with the non-PAL cohort (n=214; 8.5 vs 4.3 mo, respectively). In particular, mDOT in the first-line setting was longer with PAL + letrozole (LET; n=26) than with LET alone (n=63; 9.4 vs 3.0 mo, respectively). In the Flatiron Health dataset between Feb 2015 and July 2017, the real-world maximum response rate in the PAL + ET cohort across all lines of therapy in the mBC setting (n=12) was 33.3% (2 complete responses [CR], 2 partial responses [PR]) vs 12.5% (0 CR, 1 PR) for the ET alone cohort (n=8). Conclusions: The real-world data sources used in this study support that men with mBC derive clinical benefit from the addition of PAL to ET. Given the challenges of conducting randomized clinical trials in men with mBC, noninterventional, real-world evidence data appear to be useful to delineate the benefit of such therapies in this setting. Funding: Pfizer.

scholarly journals HEALTH TECHNOLOGY PERFORMANCE ASSESSMENT: REAL-WORLD EVIDENCE FOR PUBLIC HEALTHCARE SUSTAINABILITY

2017 ◽  
Vol 33 (2) ◽  
pp. 279-287 ◽  
Author(s):  
Augusto Afonso Guerra-Júnior ◽  
Lívia Lovato Pires de Lemos ◽  
Brian Godman ◽  
Marion Bennie ◽  
Cláudia Garcia Serpa Osorio-de-Castro ◽  
...  
Keyword(s):  
Performance Assessment ◽  
Real World ◽  
Real Life ◽  
Health Technology ◽  
Public Consultation ◽  
Phase Iii ◽  
Public Healthcare ◽  
Extensive Literature ◽  
Controlled Conditions ◽  
Real World Evidence

Objectives:Health technology financing is often based on randomized controlled trials (RCTs), which are often the same ones used for licensing. Because they are designed to show the best possible results, typically Phase III studies are conducted under ideal and highly controlled conditions. Consequently, it is not surprising that technologies do not always perform in real life in the same way as controlled conditions. Because financing (and price paid) decisions can be made with overestimated results, health authorities need to ask whether health systems achieve the results they expect when they choose to pay for a technology. The optimal way to answer this question is to assess the performance of financed technologies in real-world settings. Health technology performance assessment (HTpA) refers to the systematic evaluation of the properties, effects, and/or impact of a health intervention or health technology in the real world to provide information for investment/disinvestment decisions and clinical guideline updates. The objective is to describe the development and principal aspects of the Guideline for HTpA commissioned by the Brazilian Ministry of Health.Methods:Our methods used include extensive literature review, refinement with experts across countries, and public consultation.Results:A comprehensive guideline was developed, which has been adopted by the Brazilian government.Conclusion:We believe the guideline, with its particular focus on disinvestment, along with the creation of a specific program for HTpA, will allow the institutionalization and continuous improvement of the scientific methods to use real-world evidence to optimize available resources not only in Brazil but across countries.

scholarly journals Atrial fibrillation: Importance of real world data from regional registries. A focus on the BALKAN-AF registry

2020 ◽  
Vol 26 (1) ◽  
pp. 5-9
Author(s):  
Monika Kozieł ◽  
Gregory Y. H. Lip ◽  
Tatjana S. Potpara
Keyword(s):  
Atrial Fibrillation ◽  
Clinical Trials ◽  
Real World ◽  
Randomized Clinical Trials ◽  
European Population ◽  
Real World Data ◽  
The Balkans ◽  
Real World Evidence ◽  
Patient Groups ◽  
End Stage

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).

scholarly journals Real World Evidence: welcome to reality!

2020 ◽  
Vol 23 (1) ◽  
pp. 75
Author(s):  
Pintaudi, B.
Keyword(s):  
Real World ◽  
Randomized Clinical Trials ◽  
Relevant Information ◽  
Administrative Databases ◽  
Real World Data ◽  
World Data ◽  
Real World Evidence ◽  
History Of ◽  
The Times ◽  
Concrete Face

Real World Data (RWD) constitute a set of information sources on which the very current line of research of Real World Evidence (RWE) is based. RWE studies are based on data from observational studies, administrative databases, population or disease registers, insurance registers, electronic medical records, population health surveys and, more recently, social media and data from mobile devices and apps. While Randomized Clinical Trials (RCTs) answer the question “Can it work?” “Is it safe?” RWDs are more interested in answering the question “Does it work?”. We therefore move from a question of “efficacy / safety” to one of “effectiveness”. RWE studies allow to evaluate the safety of a treatment in a longer period than that of the RCTs, verify its quality and cost effectiveness, allow us to trace the natural history of a disease conditioned or not by a treatment, give us relevant information on compliance and on adherence to treatments and allow us to identify service models and patient preferences. Given the exciting perspective that the “real” vision of things outlines, AMD has decided to keep up with the times by forming a Group of work on “Real World Evidence”. The activities that the RWE Working Group is already promoting and carrying out are: 1) support for the publication of clinical cases; 2) support for the drafting of research protocols; 3) analysis of the Annals database. Addressing Real World Evidence in the widest possible way by collaborating with interested AMD Members, aligning with the need to give a concrete face to things, represents the vision of this Group. “Welcome to reality”! KEY WORD Real World Data, Real World Evidence, effectiveness.

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