Rationale, Strengths, and Limitations of Real-World Evidence in Oncology: A Canadian Review and Perspective
Data obtained from real-world studies have an integral role in evidence-based medicine, serving as an essential source of safety information and a complement to efficacy data from RCTs. RWE is particularly useful for expanding the evidence base to encompass populations of patients who are not well represented in RCTs but who may benefit from the interventions in question. RWE is also critical in the setting of complex or rapidly evolving treatments, where RCT design cannot answer all the relevant questions. Defining the role of treatments for rare cancers or rare subtypes is another key function of RWE. RWE is essential to better defining a treatment’s safety, particularly over the long term.
These uses enhance clinical knowledge and patient care with approved agents. Moreover, RWE is becoming widely accepted by regulators for new drug approvals or line extensions. While RWE has many uses, it also has many limitations. Efforts are being made by regulators and other groups to develop best practices for the mitigation of common biases in the design of real-world studies.
Randomized controlled trials (RCTs) remain the gold standard for evaluating the efficacy of cancer therapies. Their design, however, limits the amount and type of information they can. For clinicians, researchers, and regulators to develop a more complete and in-depth understanding of therapeutic agents and their safety profiles, data obtained from real-world evidence (RWE) are essential to complement the data obtained from RCTs. RWE encompasses data obtained through various research types, essentially anything that is not a traditional RCT. The US 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.”
Potential sources include electronic health records, claims and billing data, disease- or product-specific registries, and digital health solutions outside of conventional clinical trials. Regulators worldwide have acknowledged RWE as a key component of the evidence base to review in the approval of novel interventions, both during initial approval and for line extensions...
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