Abstract
The recent U.S. Food and Drug Administration (FDA) coapprovals of several therapeutic compounds and their companion diagnostic devices (FDA News Release, 2011, 2013) to identify patients who would benefit from treatment have led to considerable interest in incorporating predictive biomarkers in clinical studies. Yet, the translation of predictive biomarkers poses unique technical, logistic, and regulatory challenges that need to be addressed by a multidisciplinary team including discovery scientists, clinicians, biomarker experts, regulatory personnel, and assay developers. These issues can be placed into four broad categories: sample collection, assay validation, sample analysis, and regulatory requirements. In this paper, we provide a primer for drug development teams who are eager to implement a predictive patient segmentation marker into an early clinical trial in a way that facilitates subsequent development of a companion diagnostic. Using examples of nucleic acid-based assays, we briefly review common issues encountered when translating a biomarker to the clinic but focus primarily on key practical issues that should be considered by clinical teams when planning to use a biomarker to balance arms of a study or to determine eligibility for a clinical study.
Highlights
At many biopharmaceutical companies, predictive biomarker assays are developed and validated either internally or externally with partner companies with expertise in molecular analyses
Regular team meetings are highly recommended and are intended in part to facilitate communication and to ensure the team is able to adapt to both major changes and minor changes that may affect assay development or validation
The final procedure for collecting, BioMed Research International processing, storing, and shipping the clinical samples is typically documented in a Procedures Manual, which is a set of detailed instructions for clinical sites and the central laboratory and which typically is a collaborative effort between the assay developer and the clinical research associate
Summary
Predictive biomarker assays are developed and validated either internally or externally with partner companies with expertise in molecular analyses. A multidisciplinary internal biomarker team will be needed to define assay requirements, select a diagnostic company partner, develop a workplan, and oversee the assay development and validation processes (reviewed in [1]). Regular team meetings are highly recommended and are intended in part to facilitate communication and to ensure the team is able to adapt to both major changes (such as a change in clinical development timeline or target indication) and minor changes (such as changes to the list of clinical sites or to the specimen collection method) that may affect assay development or validation
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