Zhaohui Su, VP, Strategic Consulting at Veristat, shared a post on LinkedIn:
“In a recent study, Francesco Pignatti and colleagues concluded that when evaluating the overall survival (OS) of new therapies in oncology, studies based on high quality real-world data (RWD) are considered more convincing than single-arm trials (SAT) without a formal control.
In advanced cancers, where randomized clinical trials (RCT) can be challenging to conduct, external comparator cohorts (ECC) are increasingly used to contextualize outcomes such as OS. This method is particularly valuable in single-arm trials, rare diseases, or situations with high unmet needs.
It is essential to carefully balance opportunity and rigor:
– RWD can provide meaningful context for survival outcomes and enhance the evidence base.
– However, credibility relies heavily on design choices, including patient selection, alignment of inclusion criteria, and careful control of bias.
– Methods such as matching, weighting, and thoughtful endpoint definition are critical to ensure valid comparisons.
The paper emphasizes that real-world evidence (RWE) can significantly demonstrate treatment benefits, but only when it is methodologically sound and fit for purpose.
As we see an increasing application of external controls in regulatory and health technology assessment (HTA) settings, achieving the right balance will be important.
If this topic interests you, consider joining my upcoming webinar, where I will share practical approaches and lessons learned in generating high-quality RWE for regulatory submissions.
Register here. I hope to see you there.”
Title: Assessing Overall Survival Benefits in Advanced Cancers: The Role of External Comparator Cohort Studies with Real-World Data
Authors: Francesco Pignatti, Tarec Christoffer El-Galaly, Martin Kaiser, Kimmo Porkka, Robin Doeswijk, Peter Mol, Donna R. Rivera, Catherine C. Lerro, Ulrich-Peter Rohr, Patrice Verpillat, Antonios Valachis, Dario Trapani, Massimo Di Maio, Nicola Latino, Raul Cordoba, Nathan Cherny, Miriam Koopman, Diogo Martins-Branco, George Pentheroudakis, Douwe Postmus
Read the article here.
Other articles about Zhaohui Su on OncoDaily.