Advancing Oncology Innovation With Adaptive Trial Designs

A biopharmaceutical company developing a novel therapy for solid tumor cancers partnered with Veristat for assistance in designing and executing a Phase 3, open-label, randomized, multi-regional trial comparing the therapy to standard of care. To support regulatory submissions in the US, EU, and Asia, the study required balanced geographic enrollment to ensure population diversity and generalizable results. Veristat applied FDA Multi-Regional Clinical Trial guidance, advising real-time monitoring of enrollment by region, pacing site activations, and setting diversity targets to prevent over-representation from any single geography.

Given the open-label design, there was a risk of control arm patients withdrawing early to pursue alternative treatments, threatening data integrity for the primary endpoint, progression-free survival (PFS). To mitigate bias, Veristat recommended increasing the sample size, implementing strong patient and site engagement strategies, and planning for multiple missing data scenarios within the estimated framework to maintain robustness. These measures aimed to ensure representative enrollment, preserve follow-up completeness, and generate credible efficacy and safety evidence for global regulatory review.