Pragmatic clinical trials

2020 ◽  
pp. 52-60
Author(s):  
O. R. Shevchenko ◽  
A. S. Kolbin
Keyword(s):  
Clinical Trials ◽  
Informed Consent ◽  
Real World ◽  
Randomized Clinical Trials ◽  
Inclusion Criteria ◽  
Unified Approach ◽  
Scientific Rigor ◽  
Real World Evidence ◽  
Pragmatic Clinical Trials ◽  
Routine Therapy

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.

scholarly journals Trial in Progress: Real-World Safety and Effectiveness of Brentuximab Vedotin in Adults with CD30+ Lymphoma in China

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 4552-4552
Author(s):  
Pengpeng Xu ◽  
Mingci Cai ◽  
Wendy Zhang ◽  
Wei Li Zhao
Keyword(s):  
Clinical Trials ◽  
Clinical Practice ◽  
Informed Consent ◽  
Survival Rate ◽  
Real World ◽  
Brentuximab Vedotin ◽  
Routine Clinical Practice ◽  
Inclusion Criteria ◽  
Real World Data

Abstract Background: Clinical trials have demonstrated the effectiveness of the CD30-targeted antibody-drug conjugate brentuximab vedotin (BV) for the treatment of classical Hodgkin lymphoma (HL) and non-Hodgkin lymphoma(e.g. ALCL, PTCL-NOS, AITL, CTCL and etc.). While clinical trials are critical for establishing efficacy, collection of real-world data outside of the controlled trial setting is important to evaluate how interventions are applied and assess the effectiveness of new treatments in routine clinical practice. Inclusion criteria are often rather restrictive compared with the patient populations seen by physicians in daily practice. There are limited real-world data related to treatment with BV in China. Our study aims to obtain timely real-world knowledge in terms of safety and effectiveness of BV in CD30+ lymphoma patients in China. Study Design and Methods: The study (NCT04837222) is a real-world, prospective, multicenter study to evaluate the safety and effectiveness of BV in patients with CD30+ lymphoma in China. Consecutive CD30+ lymphoma patients treated with BV as a part of standard clinical practice will be enrolled. Key inclusion criteria includes adult patients undergoing treatment with BV or to be received with BV, patient/legal guardian must be able to read, understand, and sign the Informed Consent Form, CD30+ lymphoma by INV (any CD30 expression). Exclusion criteria includes patient who currently participates in or with plan to participate in any interventional clinical trial, any other reason that, in the investigator's opinion, makes the patient unsuitable to participate in this study. As CD30+ lymphoma is not a common disease and the affordability of novel treatment is limited, 1000 patients with CD30+ lymphoma will be recruited from almost 30 hematology centers. The physician will determine the treatment regimen, as well as the frequency of laboratory and clinical assessment according to her/his routine practice. All patients will be followed up per routine clinical practice and data will be documented at baseline/3/6/9/12/18/24 months unless withdrawal of Informed Consent, death or loss of follow-up, whichever comes first. Loss to follow-up will be minimized through active contact with participating patients thereafter to ensure almost all clinically relevant outcomes will be captured. The primary endpoint is serious adverse events. Secondary endpoints include adverse events, adverse drug reaction, dose adjustment, characteristics of patients receiving BV, use of BV, number of BV cycles administered, disease characteristics, time to next treatment, overall response rate, duration of response, progression free survival rate, overall survival rate, quality of life and cost-effectiveness ratio. Descriptive analysis will be performed for data analysis. Disclosures Zhang: Takeda Pharmaceuticals: Current Employment.

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).

Systematic literature review of cross-protective effect of HPV vaccines based on data from randomized clinical trials and real-world evidence

Vaccine ◽  
2021 ◽  
Vol 39 (16) ◽  
pp. 2224-2236 ◽  
Author(s):  
Darron R. Brown ◽  
Elmar A. Joura ◽  
Glorian P. Yen ◽  
Smita Kothari ◽  
Alain Luxembourg ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Literature Review ◽  
Protective Effect ◽  
Systematic Literature Review ◽  
Real World ◽  
Randomized Clinical Trials ◽  
Hpv Vaccines ◽  
Real World Evidence

scholarly journals Pragmatic Clinical Trials for Real-World Evidence: Concept and Implementation

2020 ◽  
Vol 2 (3) ◽  
pp. 85
Author(s):  
Na-Young Jeong ◽  
Seon-Ha Kim ◽  
Eunsun Lim ◽  
Nam-Kyong Choi
Keyword(s):  
Clinical Trials ◽  
Real World ◽  
Real World Evidence ◽  
Pragmatic Clinical Trials

Comparative Effectiveness Research, Learning Health Systems, and Pragmatic Randomized Controlled Trials

2020 ◽  
Author(s):  
Scott Y. H. Kim
Keyword(s):  
Informed Consent ◽  
Real World ◽  
Standard Of Care ◽  
Minimal Risk ◽  
Analysis Model ◽  
Research Risks ◽  
Real World Evidence ◽  
Rigorous Research ◽  
Pragmatic Clinical Trials ◽  
Research Analysis

Pragmatic clinical trials (PCTs) comparing interventions “within the standard of care” can efficiently yield important real-world evidence for healthcare practice and policymaking. But since PCTs attempt to mimic real-world procedures, any requirements added to the PCTs—even ethical requirements such as informed consent—can compromise the pragmatic nature of the trials. Many therefore treat such PCTs as ethically exceptional and propose that if PCTs compare two standard interventions, by that mere fact they pose no more than minimal risk—making them candidates for waivers of informed consent. This chapter argues that such an approach misunderstands research risks, is dangerous, and is unnecessary. Using a rigorous research analysis model, the chapter argues that instead of discarding the research–treatment distinction, it is better to pragmatically integrate research and clinical ethics.

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