Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2018 Feb;243(3):222-227.
doi: 10.1177/1535370217743949. Epub 2017 Nov 24.

Preclinical biomarker qualification

Affiliations
Review

Preclinical biomarker qualification

John-Michael Sauer et al. Exp Biol Med (Maywood). 2018 Feb.

Abstract

Biomarkers are ubiquitously used within drug development programs in both nonclinical species and in humans to assess safety and efficacy of novel compounds. To routinely apply such novel biomarkers with certainty, a well-defined data package is necessary for review and endorsement by regulatory agencies including the US Food and Drug Administration, European Medicines Agency, and Japanese Pharmaceuticals and Medical Devices Agency. This type of endorsement is known as regulatory qualification. Novel approaches are being applied to speed the process, lower the resource intensity, and increase the accessibility of biomarker qualification data and it is likely that consortia will continue to play a fundamental role in the qualification process by bringing together like-minded stakeholders focused on specific tools to accelerate drug development. This article will focus on learnings from the previous three nonclinical biomarker qualification projects, as well as discuss the progression of preclinical biomarker projects into the clinical qualification space and the current strategy for the use of nonclinical biomarker data in the translational qualification of clinical biomarkers; much like nonclinical information is used in the approval of drug development candidates. Impact statement This minireview provides an overview of the history of preclinical biomarker qualification by summarizing the three examples of this type of qualification with US Food and Drug Administration, European Medicines Agency, and Japanese Pharmaceuticals and Medical Devices Agency. In addition, an overview of the biomarker qualification process is included to educate key stakeholders with links to relevant white papers that provide information on current evidentiary considerations. The manuscript also provides new information on the evolution of the role that preclinical qualification plays in clinical qualification of biomarkers and the novel approaches that are being utilized to improve the process.

Keywords: Biomarker; context of use; drug development tool; preclinical; regulatory qualification; safety.

PubMed Disclaimer

Figures

Figure 1.
Figure 1.
Stages of qualification for novel translational safety biomarkers for use in clinical trials.

References

    1. US Food and Drug Administration. FDA’s Critical Path Initiative Critical Path Initiative, www.fda.gov/ScienceResearch/SpecialTopics/Critical PathInitiative/ucm076... (2004, accessed 27 July 2016)
    1. Biomarker Qualification Program, www.fda.gov/Drugs/Development ApprovalProcess/DrugDevelopmentToolsQualif... (accessed 21 February 2017)
    1. Amur SG, Sanyal S, Chakravarty AG, Noone MH, Kaiser J, McCune S, Buckman-Garner SY. Building a roadmap to biomarker qualification: challenges and opportunities. Biomarkers Med 2015; 9:1095–105 - PubMed
    1. FDA-NIH Biomarker Working Group. BEST (Biomarkers, EndpointS, and other Tools) Resource, www.ncbi.nlm.nih.gov/books/NBK338448/ (2016, accessed 4 May 2016) - PubMed
    1. US Food and Drug Administration. Submission resources by stage. Biomarker Qualification Program, www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugDevelopmentToolsQualifi... (accessed 12 September 2017)

MeSH terms

Substances