The use of emerging safety biomarkers in nonclinical and clinical safety assessment – The current and future state: An IQ DruSafe industry survey

• Published in: Regulatory toxicology and pharmacology 2021-03, Vol.120, p.104857, Article 104857
• Main Authors: Zabka, Tanja S., Burkhardt, John, Reagan, William J., Gautier, Jean-Charles, Glaab, Warren E., Guffroy, Magali, Harding, Joanna, Brees, Dominique, McDuffie, Eric, Ramaiah, Lila, Schultze, A. Eric, Smith, James D., Wolfreys, Alison, Dalmas, Deidre A.
• Format: Article
• Language: English
• Subjects: Clinical; Drug development; DruSafe; Emerging safety biomarkers; Nonclinical; Qualification; Survey
• ISSN: 0273-2300; 1096-0295
• DOI: 10.1016/j.yrtph.2020.104857

Pharmaceutical and biotechnology companies rarely disclose their use of translational emerging safety biomarkers (ESBs) during drug development, and the impact of ESB use on the speed of drug development remains unclear. A cross-industry survey of 20 companies of varying size was conducted to understand current trends in ESB use and future use prospects. The objectives were to: (1) determine current ESB use in nonclinical and clinical drug development and impact on asset advancement; (2) identify opportunities, gaps, and challenges to greater ESB implementation; and (3) benchmark perspectives on regulatory acceptance. Although ESBs were employed in only 5–50% of studies/programs, most companies used ESBs to some extent, with larger companies demonstrating greater nonclinical use. Inclusion of ESBs in investigational new drug applications (INDs) was similar across all companies; however, differences in clinical trial usage could vary among the prevailing health authority (HA). Broader implementation of ESBs requires resource support, cross-industry partnerships, and collaboration with HAs. This includes generating sufficient foundational data, demonstrating nonclinical to clinical translatability and practical utility, and clearly written criteria by HAs to enable qualification. If achieved, ESBs will play a critical role in the development of next-generation, translationally-tailored standard laboratory tests for drug development. •83% of companies use ESB in some capacity, but only in ≤50% of studies/program).•ESB are used more by large companies and before candidate selection.•Companies generate non-program specific foundational knowledge.•ESB strategy was not impacted by assay validation, study location, or HA requests.•ESB as next generation tailored standard labs will require resources and partnerships.