Abstract
BackgroundIn recent years there has been an increasing, partially also critical interest in understanding the potential benefits of generating real-world evidence (RWE) in medicine.ObjectivesThe benefits and limitations of RWE in the context of randomized controlled trials (RCTs) are described along with a view on how they may complement each other as partners in the generation of evidence for clinical oncology. Moreover, challenges and success factors in building an effective RWE network of cooperating cancer centers are analyzed and discussed.Material and methodsThis article is based on a selective literature search (predominantly 2015–2017) combined with our practical experience to date in establishing European oncology RWE networks.ResultsRWE studies can be highly valuable and complementary to RCTs due to their high external validity. If cancer centers successfully address the various challenges in the establishment of an effective RWE study network and in the consequent execution of studies, they may efficiently generate high-quality research findings on treatment effectiveness and safety. Concerns pertaining to data privacy are of utmost importance and discussed accordingly. Securing data completeness, accuracy, and a common data structure on routinely collected disease and treatment-related data of patients with cancer is a challenging task that requires high engagement of all participants in the process.ConclusionBased on the discussed prerequisites, the analysis of comprehensive and complex real-world data in the context of a RWE study network represents an important and promising complementary partner to RCTs. This enables research into the general quality of cancer care and can permit comparative effectiveness studies across partner centers. Moreover, it will provide insights into a broader optimization of cancer care, refined therapeutic strategies for patient subgroups as well as avenues for further research in oncology.
Highlights
The U.S Food and Drug Administration (FDA) defines real-world evidence (RWE) as “the clinical evidence regarding the usage, and potential benefits or risks, of a medical product derived from analysis of real-world data” [34]
The American Society of Clinical Oncology (ASCO) has recently published a research statement that discusses the potential of RWE and provides recommendations on how RWE may be utilized in conjunction with randomized controlled trials (RCTs) [35]
This article is based on a selective literature search combined with our practical experience to date in establishing European oncology RWE networks
Summary
The U.S Food and Drug Administration (FDA) defines RWE as “the clinical evidence regarding the usage, and potential benefits or risks, of a medical product derived from analysis of real-world data” [34]. The American Society of Clinical Oncology (ASCO) has recently published a research statement that discusses the potential of RWE and provides recommendations on how RWE may be utilized in conjunction with RCTs [35]. In addition to a higher external validity, RWE studies have the potential to address a number of further limitations of RCTs. For example, RCTs often underestimate (long-term) toxicity and they rarely, or with a delay, explore certain research topics such as head-to-head comparisons of novel medications or interventions. RWE studies canextend ourknowledge of treatment effectiveness and safety by generalizing the findings of prior RCTs [29] They may further describe underutilization of therapies or reveal overtreatment [3], and foster research of rare tumors because they may allow the use of data sets with sufficiently large patient cohorts [13].
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