scholarly journals Role of real-world evidence in informing cancer care: lessons from colorectal cancer

2019 ◽  
Vol 26 (1) ◽  
Author(s):  
A. Batra ◽  
W. Y. Cheung
Keyword(s):  
Colorectal Cancer ◽  
Real World ◽  
Cancer Care ◽  
Clinical Care ◽  
Real World Data ◽  
Cancer Management ◽  
Randomized Controlled ◽  
Real World Evidence ◽  
Research Questions

The term “real-world evidence” (rwe) describes the analysis of data that are collected beyond the context of clinical trials. The use and application of rwe have become increasingly common and relevant, especially in oncology, because there is growing recognition that randomized controlled trials (rcts) might not be sufficiently representative of the entire patient population that is affected by cancer, and that specific clinical research questions might be best addressed by real-world data. In this brief review, our main aim is to highlight the role of rwe in informing cancer care, particularly focusing on specific examples from colorectal cancer. Our hope is to illustrate the ways in which rwe can complement rcts in improving the understanding of cancer management and how rwe can facilitate cancer treatment decisions for real-world patients encountered in routine clinical care.

scholarly journals The Expanding Role of Real-World Evidence Trials in Health Care Decision Making

2019 ◽  
Vol 14 (1) ◽  
pp. 174-179 ◽  
Author(s):  
David C. Klonoff
Keyword(s):  
Real World ◽  
Clinical Evidence ◽  
Real World Data ◽  
Medical Products ◽  
Advantages And Disadvantages ◽  
Randomized Controlled ◽  
Real World Evidence ◽  
Care Decision

Real-world evidence (RWE) is the clinical evidence about benefits or risks of medical products derived from analyzing real world data (RWD), which are data collected through routine clinical practice. This article discusses the advantages and disadvantages of RWE studies, how these studies differ from randomized controlled trials (RCTs), how to overcome barriers to current skepticism about RWE, how FDA is using RWE, how to improve the quality of RWE, and finally the future of RWE trials.

The role of real-world evidence in clinical care: A survey of oncology leaders.

2018 ◽  
Vol 36 (15_suppl) ◽  
pp. e18515-e18515
Author(s):  
Melissa Stahl ◽  
Alissa M Winzeler ◽  
Ali Zaman ◽  
Nicole Shelby ◽  
James Lin Chen ◽  
...  
Keyword(s):  
Real World ◽  
Clinical Care ◽  
Real World Evidence

scholarly journals Bridging the divide between clinical research and clinical care in oncology: An integrated real-world evidence generation platform

2021 ◽  
Vol 7 ◽  
pp. 205520762110599
Author(s):  
Ariel B. Bourla ◽  
Neal J. Meropol
Keyword(s):  
Clinical Research ◽  
Real World ◽  
Clinical Care ◽  
Routine Care ◽  
Trial Participation ◽  
Real World Data ◽  
Regulatory Decision ◽  
Real World Evidence ◽  
Level Data ◽  
Patient Health

Real world data (RWD) are data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources; real-world evidence (RWE) generated by RWD analyses can become an important component of drug development programs and, potentially, regulatory decision-making. As a RWD source, electronic health records (EHRs) can now provide patient-level data at unparalleled depth and granularity. We propose a RWE generation framework that could maximize the synergy between RWD and prospective clinical trials by capitalizing on an emerging data curation infrastructure that may be applied to both retrospective and prospective research. In this platform, centralized data collection and monitoring could be enabled via routine EHR use, and seamlessly integrated with select intentional data capture during prospective study periods. By bridging the divide between routine care and clinical research, this integrated platform aggregates retrospective and prospective data, collected both routinely and intentionally. This approach makes clinical trial participation more available to patients, increasing the potential depth of data, representativeness and efficiency of clinical research.

scholarly journals Role of real-world evidence studies in cardiology

2021 ◽  
Vol 26 (3S) ◽  
pp. 4636
Author(s):  
T. V. Pavlova
Keyword(s):  
Real World ◽  
Observational Studies ◽  
Safety Data ◽  
Randomized Controlled ◽  
Medication Effects ◽  
Real World Evidence ◽  
Methodological Problems ◽  
Based Medicine

The introduction of evidence-based medicine into practical healthcare provides physicians with the opportunity to use treatment approaches, which have been proven to be effective in randomized controlled trials. In this case, it is necessary to consider using the medication in a particular patient in accordance with the design of clinical trials and strictly follow the instructions. Drugs should be critically selected with focus on quality of available efficacy and safety data obtained in the population closest to a patient. In addition, clinicians should regularly review the available data with particular regard to its quality. The advantages, disadvantages, limitations and methodological problems of observational studies should be carefully considered during the interpretation of results. At the same time, the compliance of the results of real-world evidence studies with registration trial data indicates a high reproducibility of medication effects.

scholarly journals Regulatory-grade clinical trial design using real-world data

2020 ◽  
Vol 17 (4) ◽  
pp. 377-382
Author(s):  
Mark S Levenson
Keyword(s):  
Real World ◽  
Trial Design ◽  
Clinical Trial Design ◽  
Real World Data ◽  
World Data ◽  
Real World Evidence ◽  
Study Results ◽  
Potential Use ◽  
The U.S

Real-world data and evidence provide the potential to address the effectiveness and safety of drugs. The U.S. Food & Drug Administration has initiated a program to evaluate the potential use of real-world evidence for regulatory uses. Whether a study is designed for regulatory purposes or for other purposes, existing regulation and guidance provide a reference for high-quality studies. Clarifying the study objectives and the role of real-world data in the study are important considerations. Robustness and transparency of the analysis allow for greater understanding and acceptance of the study results.

scholarly journals Real world data on adjuvant chemotherapy for high-risk stage II colorectal cancer: The role of tumour side

2019 ◽  
Vol 30 ◽  
pp. v211-v212
Author(s):  
C S Araujo de Carvalho ◽  
C.M.V. Moniz ◽  
G.Y. Watarai ◽  
J.A.R. Crespo ◽  
P V D S Nogueira ◽  
...  
Keyword(s):  
Colorectal Cancer ◽  
High Risk ◽  
Adjuvant Chemotherapy ◽  
Real World ◽  
Stage Ii ◽  
Real World Data ◽  
World Data ◽  
Stage Ii Colorectal Cancer

scholarly journals The Prospective Dutch Colorectal Cancer (PLCRC) cohort: real-world data facilitating research and clinical care

2021 ◽  
Vol 11 (1) ◽  
Author(s):  
Jeroen W. G. Derksen ◽  
◽  
Geraldine R. Vink ◽  
Marloes A. G. Elferink ◽  
Jeanine M. L. Roodhart ◽  
...  
Keyword(s):  
Colorectal Cancer ◽  
Real World ◽  
Clinical Care ◽  
Daily Practice ◽  
Additional Patient ◽  
Real World Data ◽  
World Data ◽  
Oncology Research ◽  
Netherlands Cancer Registry ◽  
Patient Reported

AbstractReal-world data (RWD) sources are important to advance clinical oncology research and evaluate treatments in daily practice. Since 2013, the Prospective Dutch Colorectal Cancer (PLCRC) cohort, linked to the Netherlands Cancer Registry, serves as an infrastructure for scientific research collecting additional patient-reported outcomes (PRO) and biospecimens. Here we report on cohort developments and investigate to what extent PLCRC reflects the “real-world”. Clinical and demographic characteristics of PLCRC participants were compared with the general Dutch CRC population (n = 74,692, Dutch-ref). To study representativeness, standardized differences between PLCRC and Dutch-ref were calculated, and logistic regression models were evaluated on their ability to distinguish cohort participants from the Dutch-ref (AU-ROC 0.5 = preferred, implying participation independent of patient characteristics). Stratified analyses by stage and time-period (2013–2016 and 2017–Aug 2019) were performed to study the evolution towards RWD. In August 2019, 5744 patients were enrolled. Enrollment increased steeply, from 129 participants (1 hospital) in 2013 to 2136 (50 of 75 Dutch hospitals) in 2018. Low AU-ROC (0.65, 95% CI: 0.64–0.65) indicates limited ability to distinguish cohort participants from the Dutch-ref. Characteristics that remained imbalanced in the period 2017–Aug’19 compared with the Dutch-ref were age (65.0 years in PLCRC, 69.3 in the Dutch-ref) and tumor stage (40% stage-III in PLCRC, 30% in the Dutch-ref). PLCRC approaches to represent the Dutch CRC population and will ultimately meet the current demand for high-quality RWD. Efforts are ongoing to improve multidisciplinary recruitment which will further enhance PLCRC’s representativeness and its contribution to a learning healthcare system.

Use of cancer immunotherapies in the real-world in the setting of microsatellite instability.

2018 ◽  
Vol 36 (5_suppl) ◽  
pp. 30-30
Author(s):  
Vineeta Agarwala ◽  
Anala Gossai ◽  
Gaurav Singal ◽  
Claire O'Connell ◽  
Gerald Li ◽  
...  
Keyword(s):  
Colorectal Cancer ◽  
Microsatellite Instability ◽  
Real World ◽  
Checkpoint Inhibitors ◽  
Clinical Care ◽  
Unknown Primary ◽  
Tumor Type ◽  
Real World Data ◽  
The Real ◽  
Genomic Marker

30 Background: In May 2017, the FDA approved for the first time a cancer therapy (pembrolizumab) for use in patients based on the presence of a genomic marker (microsatellite instability, or MSI) rather than anatomical tumor type. Real-world data on the rates and clinical impact of MSI on treatment selection and response are scant, especially outside of colorectal cancer. Methods: We performed a retrospective study of all patients treated in the Flatiron Health network (>265 oncology practices across the U.S.) between January 2011 and June 2017, and who underwent FoundationOne tumor sequencing as part of routine clinical care. Tumor type was determined by pathologist review of specimens submitted to Foundation Medicine. Data on therapy use was sourced from electronic health records (EHRs). Assessment of MSI was performed from DNA sequencing across the coding regions of >300 genes. Results: Our overall cohort included n=16,020 patients. Among patients in whom MSI status could be assessed (n=12,411), 207 patients had MSI-high tumors. The observed rate of MSI-high was 1.7% across all tumor types combined; tumor-specific rates varied significantly, from 4.9% in colorectal adenocarcinoma to 0.3% in breast and non-small cell lung cancer. The rate of MSI-high was 2.4% in patients with an unknown primary based on specimen review. A total of 1,329 patients received common checkpoint inhibitors (nivolumab, pembrolizumab, atezolizumab). Among the checkpoint-inhibitor treated patients with known MSI status (n=1,175), 14 (1.2%) had MSI-high tumors, and the majority of these patients (n=8) had colorectal cancer. Conclusions: Evidence of MSI-high is rare in real world cancer care settings. Early identification of patients with this biomarker is important in order to efficiently match them to treatment. Further evaluation of the real-world effectiveness of immune checkpoint inhibitors in the MSI-high population is still needed. Because most patients receiving these therapies today do not have high MSI, exploration of additional biomarkers for immunotherapy response is also critical.

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