Zhaohui Su, VP, Strategic Consulting at Veristat, shared a post on LinkedIn:
“This research paper examines both successful and unsuccessful oncology regulatory submissions that incorporated real-world data (RWD). The findings demonstrate that high-quality data alone is not sufficient for regulatory success.
Successful submissions also depend on:
- Careful study design
- Pre-specified statistical analysis plans
- Appropriate adjustment for confounding
- Demonstration of patient comparability
- Robust sensitivity analyses
- Clinically meaningful treatment effects
Several high-profile oncology approvals successfully leveraged real-world evidence (RWE) to provide context for single-arm clinical studies, particularly in areas of high unmet medical need, such as relapsed or refractory hematologic malignancies.
Conversely, many regulatory submissions were unsuccessful because of:
- Missing covariates
- Endpoint inconsistencies
- Small matched sample sizes
- Insufficient control of bias
For organizations developing oncology therapies, statistical rigor, high-quality data, and strategic planning remain the foundation of successful real-world evidence programs.
For those exploring external control arms, clinical trial augmentation, natural history studies, or broader real-world evidence strategies for regulatory decision-making, the Strategic Consulting team at Veristat offers expertise in approaches and lessons learned from previous submissions.”
Other articles about RWD on OncoDaily.