PS27: Harmonizing the Estimand Framework and Product Labeling: An Industry and Regulatory Perspective

The estimand framework introduced in ICH E9 (R1) has become an integral component of clinical trial planning and is routinely applied in confirmatory study protocols. While this framework has improved trial design by providing systematic alignment among trial objectives, conduct, data collection, and statistical analyses, challenges remain. One such challenge is ensuring that the precise clinical questions addressed through estimands are accurately translated into product labeling that guides clinical practice, thereby preventing misalignment between the evidence generated in clinical trials and the information communicated to healthcare providers and patients in product labels. This session will discuss practical approaches to enhance trial reporting and labeling practices, ensuring that the benefits of the estimand framework extend to improved healthcare decision-making through more precise and informative product labeling. The session will feature two complementary presentations (representing industry and FDA perspectives) followed by an interactive panel discussion: • Industry Perspective (speaker: Bharani Dharan - Novartis): A case study demonstrating the application of the estimand framework from protocol development through study reporting to final labeling and manuscript publication, including challenges in ensuring clear communication of results. The presenter will outline key considerations for achieving the appropriate balance between clearly describing results in the product label while avoiding overly technical language, ensuring that labels remain clear and easily understood by both patients and prescribers. • Regulatory Perspective (speaker: Alexei Ionan - FDA): An examination of how estimands influence the interpretation and presentation of efficacy and safety results in labeling, addressing alignment approaches and the role of statisticians in ensuring consistency and scientific rigor in label development. • International Panel Discussion (Lei Nie - FDA, Miya Paterniti - FDA, Catherine Njue - Health Canada, Florian Lasch - EMA): A moderated discussion featuring clinicians and statisticians from FDA, EMA, and Health Canada, exploring specific scenarios, current best practices, and how evolving estimand frameworks may shape future labeling standards across regulatory jurisdictions. Key Takeaways: Attendees will gain practical strategies for implementing the estimand framework throughout the product development lifecycle, learn best practices for translating complex study results into clear product labels, and understand regulatory perspectives across major international agencies to support more effective product labeling. Co-organizers: Abel Eshete - FDA, Elena Polverejan - Johnson & Johnson