The Effective Statistician - in association with PSI

How can we build a patient-reported outcome strategy that captures the patient voice and strengthens clinical development?

In this episode of The Effective Statistician Podcast, I speak with Julia Poritz, Senior Research Consultant on Cytel’s Evidence, Value, and Access team, about developing and implementing an effective patient-reported outcome (PRO) strategy for clinical trials.

We discuss how PROs help us understand the effects of a disease and its treatment from the patient’s perspective—including symptoms, physical functioning, health perceptions, and overall quality of life. Julia explains how the Wilson and Cleary model helps us connect these different outcomes and create a clear measurement strategy.

We also explore the four essential parts of a PRO strategy: selecting appropriate PRO measures, implementing them within the study, analyzing the data, and reporting the results. Throughout our conversation, we emphasize the importance of early planning, validated measures, patient relevance, regulatory expectations, and comparability with previous clinical trials.

In this episode, I talk with Ning Leng, Ph.D., Director II, Data and AI Acceleration Group, Data & Statistical Sciences at AbbVie, about the growing role of the R Consortium in the pharmaceutical industry.

Ning brings extensive experience in statistics, computational genomics, open-source technology, and the adoption of R across the pharmaceutical industry. Before joining AbbVie, she spent 10 years at Roche Genentech, where she helped drive the adoption of R, cloud technologies, Git, and Shiny.

We discuss how the R Consortium creates a platform for statisticians, programmers, pharmaceutical companies, and regulators to collaborate on practical challenges—and how that collaboration is changing the way we approach regulatory submissions.

Cancer treatments have changed dramatically over the past decade, but have our dose-finding strategies kept pace?

In this episode, I speak with Dr. Ayon Mukherjee, who leads statistical innovation in early oncology development at Eli Lilly. Together, we explore Project Optimus, the FDA initiative that is changing how we think about dose optimization in oncology.

Instead of simply finding the highest dose patients can tolerate, Project Optimus encourages us to identify the dose that provides the best balance between efficacy, safety, pharmacokinetics, pharmacodynamics, and long-term tolerability.

Ayon explains why the traditional maximum tolerated dose approach worked well for chemotherapy but often falls short for targeted therapies and immunotherapies. We also discuss how statisticians can help lead this transformation by designing better dose optimization studies and collaborating more effectively with clinicians, pharmacologists, and regulators.

In this insightful interview, Emma May and Alun Bedding explore the nuances of coaching and mentoring, sharing personal stories, frameworks, and practical tips to enhance professional growth. Discover how these powerful tools can transform statisticians' careers and foster leadership development.

External control arms are becoming increasingly important in drug development, but creating valid comparisons requires more than matching patient populations.

In this episode, I speak with Ben Ackerman, Director of Real-World Biostatistics at GSK, about one of the most overlooked challenges in external control arm studies: endpoint bias. We discuss why differences in how outcomes are measured can influence study results, what researchers should consider when designing studies, and how the field is evolving to address these challenges.

If you work with real-world evidence, causal inference, or innovative clinical trial designs, this episode offers valuable insights into improving the credibility and transparency of external control arm analyses.