Biomarkers for Precision Patient Selection in Cancer Therapy Approvals in the US, from 2011 to 2023.

Autor: Dou YN; Oncology Regulatory Science, Strategy & Excellence, AstraZeneca, Gaithersburg, Maryland, USA., Grimstein C; Oncology Regulatory Science, Strategy & Excellence, AstraZeneca, Gaithersburg, Maryland, USA., Mascaro J; Oncology Regulatory Science, Strategy & Excellence, AstraZeneca, Gaithersburg, Maryland, USA., Wang J; Oncology Regulatory Science, Strategy & Excellence, AstraZeneca, Gaithersburg, Maryland, USA.
Jazyk: angličtina
Zdroj: Clinical pharmacology and therapeutics [Clin Pharmacol Ther] 2024 Aug; Vol. 116 (2), pp. 304-314. Date of Electronic Publication: 2024 May 15.
DOI: 10.1002/cpt.3306
Abstrakt: During the period of 2011-2023, the US Food and Drug Administration (US FDA) granted 139 accelerated and 329 regular approvals for 86 and 152 cancer therapeutic products, respectively. The percentage of approvals for a biomarker-defined population was numerically higher in accelerated approvals in comparison to regular approvals, that is, 48% vs. 40%. From 2011-2016 to 2017-2023, there was an increasing number of approvals with biomarker-defined populations in lung and breast cancers, serving as the primary driver for the overall increase in the percentage of approvals for biomarker-defined populations in solid tumors relative to hematological malignancies. Over the years, approvals were incorporating a more diverse collection of distinct biomarkers, from 3 in 2011 to 16 in 2022. Overall, HER2, hormone receptor (HR), EGFR, ALK, BRAF, and PD-L1-defined populations received the highest numbers of approvals. The FDA decision on approving a biomarker-defined or an all-comers population may depend on a number of factors and may evolve over time based on emerging evidence. The review discusses selected FDA approvals where a pivotal trial enrolled an all-comers population but the approved indication was restricted to a biomarker-defined population, as well as challenges in clinical trial design in the context of precision medicine. The prominent role of biomarkers in optimizing trial design and identifying a population most likely to benefit from treatment underlines the significance of a comprehensive understanding of disease biology and drug mechanisms. Our review illustrates that biomarker-driven approaches enhance the likelihood of identifying optimal patient populations, potentially streamlining trials through accelerated approval pathways for cancer drug development.
(© 2024 Astrazeneca. Clinical Pharmacology & Therapeutics © 2024 American Society for Clinical Pharmacology and Therapeutics.)
Databáze: MEDLINE
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