Opportunities and Challenges of Safety Biomarker Qualification: Perspectives from the <scp>P</scp>redictive <scp>S</scp>afety <scp>T</scp>esting <scp>C</scp>onsortium

Abstract

Abstract Preclinical Research Biomarkers hold tremendous promise to improve the drug development and evaluation process, advance patient care, and reduce health‐care costs. However, understanding the characteristics of novel biomarkers and developing the robust evidentiary packages to support incorporating them into drug development and clinical practice is an enormous undertaking requiring significant resources and commitment from a wide range of stakeholders, including regulators, the biopharmaceutical industry, academia, governmental agencies, patients, and payors. The P redictive S afety T esting C onsortium ( PSTC ) is a unique public–private partnership formed by C ritical P ath I nstitute ( C ‐ P ath) in collaboration with the U nited S tates F ood and D rug A dministration ( FDA ) to identify new and improved drug safety testing methods and submit them for formal regulatory qualification by the FDA , the E uropean M edicines A gency, and the Japanese P harmaceuticals and M edical D evices A gency. In 2008, the PSTC obtained the first regulatory qualification of seven urinary renal preclinical safety biomarkers for use in rodent studies and on a case‐by‐case basis for inclusion into clinical development programs. These qualified biomarkers are now successfully informing drug discovery and development decisions. PSTC has expanded their qualification efforts into dogs, nonhuman primates, and humans and focuses on six areas of organ toxicity. The collaborative effort of multiple stakeholders through PSTC has resulted in significant cost savings and more rapid scientific consensus, leading to greater acceptance of these biomarkers by health authorities and pharmaceutical companies. Regulatory qualification of a biomarker for a defined context of use provides scientifically robust assurances to sponsors and regulators that should accelerate appropriate adoption of biomarkers into drug development and, ultimately, clinical practice.