Sean Khozin

This work included studies of IGF-1R-targeted therapy in thymoma, survivin suppression in NSCLC, the identification of ALK rearrangements in rare lung cancer histologies, characterization of germline EGFR T790M mutations, and the use of FDG-PET imaging as a predictive and prognostic tool in thymic malignancies. At the FDA, Dr. Khozin led several landmark accelerated and regular approvals that defined the modern paradigm of biomarker-driven oncology, including ceritinib (ALK-positive NSCLC), erlotinib (EGFR-mutant NSCLC), osimertinib (EGFR T790M NSCLC), and atezolizumab (metastatic NSCLC).

His work demonstrated that real-world endpoints can meaningfully correlate with overall survival and be implemented at scale with scientific rigor. He has also advanced hybrid trial designs that integrate randomization with external data sources, developed approaches to improve the interpretation of clinical trials for underrepresented populations, and established practical frameworks for incorporating real-world evidence into both regulatory and treatment decisions.

At the FDA, he established Information Exchange and Data Transformation (INFORMED), the agency’s first data science and technology incubator, which laid the groundwork for integrating AI and digital health into drug development and regulatory decision-making.

A central focus of Dr. Khozin’s current work is modernizing how tumor burden is measured in clinical trials. In a large FDA-led meta-analysis of more than 14,000 patients enrolled in registration trials, he found that two independent radiologists reviewing the same scans agreed on tumor response classification only about 70% of the time. In ovarian and pancreatic cancers, discordance reached as high as 45%, with striking cases in which one reader determined a complete response while the other concluded progressive disease in the very same patient.