Real-World Evidence in Oncology: Opportunities and Limitations
- Randomized controlled trials (RCTs) are traditionally viewed as the gold standard for evaluating treatment efficacy, but real-world evidence (RWE) is gaining traction in oncology for addressing questions not fully answered by RCTs.
- RWE is derived from health records, cancer registries, and other sources, and while valuable, it has intrinsic limitations that require careful consideration to avoid misleading conclusions.
- RWE is particularly useful for evaluating treatment effectiveness in daily practice, assessing complex procedures, and exploring interventions in rare subgroups where RCTs are not feasible.
- There are notable differences between RCTs and RWE, such as the highly selective patient criteria in RCTs versus the broader, more diverse populations in RWE studies.
- RWE can provide insights into treatment safety and long-term adverse events, especially in patient populations not well-represented in RCTs.
- Despite its benefits, RWE has limitations such as potential selection bias and the absence of control groups, which can affect the accuracy of treatment efficacy comparisons.
- Combining RWE with RCTs offers a more comprehensive understanding of treatment impacts, although RWE should not replace RCTs.
Traditionally, randomized controlled clinical trials (RCTs) have been considered the highest level of evidence to define the efficacy of treatments, before their adoption in clinical practice. However, in oncology, like in other fields of medicine, the analysis of real-world evidence (RWE) to answer clinical and policy-relevant questions that cannot be directly or completely answered using data from RCTs has rapidly gained increased interest in recent years [1–3]. It is clear that data obtained from health records, cancer registries, and other RWE sources can produce valuable insights into treatments and their outcomes in routine, daily oncology practice. However, caution must be paid to the intrinsic limitations of such data, to avoid a misleading and potentially harmful use of them....
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