Biomarkers in Medicines Development-From Discovery to Regulatory Qualification and Beyond.

Autor:	Hendrikse NM; Regulatory Science and Innovation Task Force, European Medicines Agency, Amsterdam, Netherlands., Llinares Garcia J; Regulatory Science and Innovation Task Force, European Medicines Agency, Amsterdam, Netherlands., Vetter T; Scientific Advice Office, European Medicines Agency, Amsterdam, Netherlands., Humphreys AJ; Regulatory Science and Innovation Task Force, European Medicines Agency, Amsterdam, Netherlands., Ehmann F; Regulatory Science and Innovation Task Force, European Medicines Agency, Amsterdam, Netherlands.
Jazyk:	angličtina
Zdroj:	Frontiers in medicine [Front Med (Lausanne)] 2022 Apr 26; Vol. 9, pp. 878942. Date of Electronic Publication: 2022 Apr 26 (Print Publication: 2022).
DOI:	10.3389/fmed.2022.878942
Abstrakt:	Biomarkers are important tools in medicines development and clinical practice. Besides their use in clinical trials, such as for enrichment of patients, monitoring safety or response to treatment, biomarkers are a cornerstone of precision medicine. The European Medicines Agency (EMA) emphasised the importance of the discovery, qualification, and use of biomarkers in their Regulatory Science Strategy to 2025, which included the recommendation to enhance early engagement with biomarker developers to facilitate regulatory qualification. This study explores the journey of biomarkers through the EU regulatory system and beyond, based on a review of interactions between developers and the EMA from 2008 to 2020, as well as the use of qualified biomarkers in clinical trials. Of applicants that used early interaction platforms such as the Innovation Task Force, less than half engaged in fee-related follow-up procedures. Results showed that, as compared to companies, consortia were more likely to opt for the Qualification of Novel Methodologies procedure and engage in follow-up procedures. Our results highlight the importance of early engagement with regulators for achieving biomarker qualification, including pre-submission discussions in the context of the qualification procedure. A review of clinical trials showed that all qualified biomarkers are used in practice, although not always according to the endorsed context of use. Overall, this study highlights important aspects of biomarker qualification, including opportunities to improve the seamless support for developers by EMA. The use of qualified biomarkers in clinical trials underlines the importance of regulatory qualification, which will further enable precision medicine for the benefit of patients. Competing Interests: The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest. (Copyright © 2022 Hendrikse, Llinares Garcia, Vetter, Humphreys and Ehmann.)
Databáze:	MEDLINE
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