‘Statistics in Biopharmaceutical Research’ to Publish Special Section on Overall Survival, Drug Development

Statistics in Biopharmaceutical Research will publish a special section titled “Overall Survival in Oncology Trials and Drug Development,” led by guest editors Ruixiao Lu of Alumis, Lisa R. Rodriguez of GSK, and Ying Lu of Stanford University.

This topic was inspired by the 2023 workshop Overall Survival in Oncology Clinical Trials, co-hosted by the US Food and Drug Administration, American Association for Cancer Research, and American Statistical Association. While overall survival remains the gold standard for evaluating treatment benefit and safety, the increasing use of earlier endpoints, such as progression-free survival, has introduced new challenges in assessing long-term outcomes.

Following the workshop and built on its discussions, a new perspective article published in 2024 in the Journal of Clinical Cancer Research listed best practices, novel statistical methods, and suggestions for improving benefit-risk assessments. It outlined considerations for clinical trial design to enhance the collection and analysis of overall survival in the context of modern therapeutic treatments. The article was co-authored by researchers, clinicians, statisticians, industry representatives, and patient advocates—the key stakeholders across regulatory agencies, industry, and academia involved in drug development. The goal was to explore how to overcome hurdles associated with traditional analyses of overall survival.

Furthermore, the FDA’s August 2025 draft guidance, “Approaches to Assessment of Overall Survival in Oncology Clinical Trials,” reinforced overall survival as the “gold standard” primary endpoint to be used whenever feasible. It advises collecting overall survival data to evaluate potential long-term safety concerns to ensure initial observed benefits don’t translate to long-term harm.

With the growing use of early endpoints for submissions, such as progression-free survival or durable response rates, the FDA often requires companies to share overall survival data following an accelerated approval or traditional approval. However, several factors pose challenges for monitoring long-term overall survival, including differences between subpopulations, crossover trial designs, subsequent lines of therapy after a patient leaves a trial, and fluctuations in the magnitude of benefit compared to the control arm over time.

“This special section reflects the vital collaboration between regulators, industry, and academia to address the complexities of monitoring overall survival. By engaging in these discussions, statisticians ensure their research translates into robust evidence and meaningful benefits for patients,” said Ying Lu.

The special section features six articles that provide deeper insights, design frameworks, and practical guidance for implementation, with a range of topics:

• Benefit and harm using overall survival endpoint
• Safety assessment with sparse overall survival data
• Pre-specified threshold for safety analysis
• Safety monitoring for overall survival
• Overall survival interim evaluation for pivotal oncology trials

“It is exciting to see the progress that has been made and the different perspectives shared, culminating in this collection, which also highlights the important role statisticians can take in owning strategy and contributing to benefit-risk evaluation in drug development,” said Rodriquez.

“We hope these contributions will extend the ongoing dialogue on this important topic and continue to spark future innovation in the field,” said Ruixiao Lu. “We look forward to seeing the field evolve as discussions around this topic advance, supporting robust clinical evidence generation and ultimately delivering meaningful benefit to patients