Real-World Evidence in Oncology: Opportunities, Challenges, and Clinical Impact
The Clinical Pathways Congress and Cancer Care Business Exchange (CPC+CBEx) session titled “Real-World Evidence Doesn’t Move the Needle. Or Does It?” explored the evolving role of real-world evidence (RWE) in oncology, highlighting both its promise in complementing clinical trials and the challenges that must be addressed to ensure rigor, transparency, and meaningful impact on patient care.
Aimee Ginsburg Chesnick, PharmD, BCPS, director of clinical content strategy at The US Oncology Network/McKesson, opened by establishing the growing importance of RWE in shaping health care decisions, regulatory approvals, and value-based care. She explained the distinction between real-world data (RWD) and RWE: While RWD consists of raw information from electronic health records, billing claims, registries, mobile apps, and patient-reported outcomes, RWE arises only after rigorous statistical methods transform this data into clinically meaningful insights. RWE complements randomized controlled trials (RCTs) by addressing questions of long-term safety, cost-effectiveness, and treatment effectiveness in diverse populations often excluded from traditional trials.
Challenges with RWD include incomplete or inaccurate documentation, data not collected with research intent, and ethical concerns regarding use outside trial structures. Ginsburg Chesnick emphasized solutions such as improving point-of-care data capture, incentivizing accurate documentation, applying artificial intelligence (AI) to harness structured and unstructured records, and reducing physician burden in clinical workflows. She advocated for longitudinal patient data systems, possibly via a national database, to unify fragmented health records.
Regulatory modernization was also highlighted in the session. Agencies such as the US Food and Drug Administration (FDA), empowered by the 21st Century Cures Act, must evolve with data science and AI capabilities. The FDA’s framework stresses 2 quality dimensions: relevance (ensuring the data answers the right clinical question) and reliability (accuracy, completeness, provenance, and timeliness). Done well, RWE supports faster approvals, expanded drug indications, reimbursement strategies, and refined commercial targeting strategies, ultimately improving personalized medicine and value-based care.
The second presenter, Lalan Wilfong, MD, senior vice president of value-based care at Thyme Care, built upon this foundation, focusing on study design, quality standards, and practical application in oncology. He stressed that RWE must derive from diverse, high-quality data sources and be transparent about origins, limitations, and biases. Dr Wilfong described collaboration among clinicians, academics, and community partners as critical. Beyond efficacy, RWE helps assess drug toxicity, patient adherence, economic burden, and health inequities—issues often invisible in RCTs. He encouraged use of external comparative arms, analysis of combination therapies, and target trial approaches to mitigate confounding and bias. He also noted transparency in reporting methodology, statistical analysis, and results are essential for credibility.
The speakers presented 2 illustrative case studies. One was a small retrospective Brazilian study on low-dose abiraterone taken with meals for prostate cancer. This study was critiqued for its methodological flaws, including limited sample size, lack of control group, short follow-up, and reliance on prostate-specific antigen (PSA) response rather than survival endpoints. The speakers concluded that such hypothesis-generating studies are insufficient to change clinical standards. In contrast, a robust US Department of Veterans Affairs study of sotorasib in KRAS-mutated lung cancer demonstrated strong methodological rigor, clinically meaningful endpoints, and insights into dose-response relationships, supporting broader use of RWE in regulatory and clinical decision-making.
The session concluded with an interactive debate on a hypothetical rare mutation therapy. Participants wrestled with whether weak RWE should influence treatment pathways in the absence of alternatives. Concerns included missing toxicity data, lack of comparative arms, and limited quality-of-life assessment. The discussion emphasized the balance between clinical need in rare diseases and evidentiary rigor. Ultimately, both presenters reinforced that while RWE is indispensable in modern oncology, its value hinges on quality, transparency, and thoughtful integration with clinical practice to enable better informed consent, shared decision-making, and patient-centered care.
Reference
Ginsburg Chesnick A, Wilfong L. Real-world evidence in oncology: opportunities, challenges, and clinical impact. Presented at the Clinical Pathways Congress; September 6, 2025; Boston, MA.
