A novel biomarker assay qualification strategy for rare human matrices - a case study of biomarkers in aqueous humor.

ABSTRACT

Background: Measuring pharmacodynamic biomarkers near the therapeutic site of action presents considerable challenges for sites with limited matrix volume or difficult access. Bioanalytical method qualification requires the use of numerous matrix samples, which is problematic for rare matrices. The aim of this study was to design and implement a streamlined, fit-for-purpose strategy for qualification of biomarker assays in rare matrices. Materials & methods: A multiplexed biomarker immunoassay was developed in human aqueous humor. Results: Our strategy was successfully implemented, providing characterization of assay performance while reducing number of samples in assay qualification. Our assay was used in clinical trial support for an ophthalmic drug candidate. Conclusion: Our results indicate this approach can be applied to other early stage drug development programs facing similar challenges.

Bioanalytical assay development and qualification requires the use of numerous matrix samples, which is problematic for rare matrices. We designed and successfully implemented a streamlined strategy for qualification of biomarker assays in rare matrices.