Abstract A015: Characterization of pharmacogenomic biomarkers in FDA oncology/hematology drug labelling: A descriptive regulatory review

Abstract Background: Pharmacogenomic biomarkers have become essential in precision oncology, enabling targeted therapies, optimising drug efficacy, and minimising adverse effects. Drug regulatory agencies such as the U.S. Food and Drug Administration (FDA) integrate biomarker information into labelling to inform treatment decisions. This review aims to systematically characterise pharmacogenomic biomarkers in FDA-approved oncology and haematology drug labelling, using the BEST (Biomarkers, EndpointS, and other Tools) Resource framework developed by the FDA-NIH Biomarker Working Group (BEST Resource, 2016). Methods: A review of FDA-labelled pharmacogenomic biomarkers was conducted using publicly available datasets, including the FDA’s Table of Pharmacogenomic Biomarkers in Drug Labelling (2000–2024). The dataset was filtered to include only oncology and haematology indications. Variables included drug name, biomarker, labelling section, therapeutic area, and biomarker function according to the BEST classification (predictive, prognostic, diagnostic, etc.). Drug names were normalised for consistency. Descriptive statistics were used to summarise frequencies and proportions across variables by using IBM SPSS version 29. Results: A total of 305 oncology and haematology pharmacogenomic entries were identified. The most frequently reported biomarkers were ERBB2 (HER2) (24 entries; 7.9%), EGFR (19; 6.2%), ALK (15; 4.9%), hormone receptors such as ESR and PGR (15; 4.9%), and BRAF (12; 3.9%). Most biomarkers served predictive roles, followed by diagnostic or prognostic purposes. The most frequently referenced drugs in labels were Venetoclax, which appeared in 8 entries (2.6%), followed by Pembrolizumab in 7 (2.3%), and Avatrombopag, Nivolumab, and Olaparib, each with 6 entries (2.0%). Drug names were successfully normalised, enabling pharmacologic class identification, with the most common being tyrosine kinase inhibitors, checkpoint inhibitors, and PARP inhibitors. Pharmacogenomic content was most frequently referenced in the “Clinical Studies” section of drug labels (72 entries; 23.6%), followed by integrated sections combining Indications and Usage with dosing or safety content. Regarding therapeutic area, 84.6% were categorised under oncology, with the majority associated with breast cancer, while 9.8% were specific to haematology. Conclusions: This review highlights that predictive biomarkers dominate FDA oncology labelling, especially in breast, lung, and colorectal cancers. The application of the BEST framework provides a valuable structure to classify biomarker function from a regulatory perspective. Findings support further standardisation in biomarker labelling to enhance clarity and global harmonisation. Citation Format: Alhanouf Y. Alnafisah, Tariq H. Alqurayshah. Characterization of pharmacogenomic biomarkers in FDA oncology/hematology drug labelling: A descriptive regulatory review [abstract]. In: Proceedings of the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics; 2025 Oct 22-26; Boston, MA. Philadelphia (PA): AACR; Mol Cancer Ther 2025;24(10 Suppl):Abstract nr A015.