Tumor Agnostic Development

We believe that tumour agnostic approval will replace the traditional tumour and stage specific path. The FDA approval pembrolizumab for MSI tumours, and both Larotrectinib and Entrectinib for NTRK fusions has set the path.

Richard Pazdur, MD, Acting Director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research and Director of the FDA’s Oncology Center of Excellence quotes re the pembrolizumab approval: “The review of pembrolizumab for this indication was based on overall response rate and durability of response. Of the 149 patients who received pembrolizumab in these studies, 39.6% had a complete or partial response. For 78% of those patients, the response lasted 6 months or longer.”

To further quote Dr Pazdur: This new indication represents a shift in our evaluation of therapies. The approval moves us away from the strict disease-site indication, such as breast, colon, or lung cancer, to a site-agnostic indication. It changes how we may define cancer—the disease is no longer being solely determined by its site of origin or pathologic diagnosis. It is now defined by the presence of a specific biomarker. This will lead to much smaller trial sizes and accelerated drug development and approval.

Moderator Julie R. Gralow, MD, FACP, FASCO, EVP & CMO, American Society of Clinical Oncology Association for Clinical Oncology


  • David Hyman MD, CMO, Loxo Oncology at Lilly
  • Brian Leyland Jones, MB BS, PhD, CMO, National Foundation For Cancer Research
  • Jean-François Martini, PhD, MSc, Sr Director, Translational Oncology Lead, Global Product Development, Pfizer Inc
  • Mark Pegram, MD, Professor of Medical Oncology, Stanford Comprehensive Cancer Institute, Stanford University
  • Richard L. Schilsky, MD, FACP, FSCT, FASCO, Professor Emeritus, University of Chicago