BIOMARKERS/BIOSIGNATURESStandards needed for Translational

Research

1. Standardized QUESTIONS in Clinical Trials

2. Standardized PRACTICES in advancing pilot studies

3. Standardized VALIDATION/VERIFICATION studies

Michael E. Berens, Ph.D.

Develop standardized QUESTIONS in ClinicalTrials that position success (“low hanging fruit”) from biomarkers/biosignatures

• patient stratification for balancing the trial arms for equivalency (survival biomarkers)• patient eligibility into clinical trial (is the target present?)• tumor response/non-response to therapy (futility marker)

to predict failure early• Include the ecosystem of players in the design of

biomarkers/biosignatures (academic, industry, advocacy, regulatory, philanthropic, etc)

Adopt standardized PRACTICES in advancing pilot studies using clinically-annotated specimens

•Use specimens from no more than 3 sites to ensure minimal deviation from SOPs•Use best-in-class analytical partner matched to the analyte•Work in close collaboration with PK and PD correlative

endpoints (best-in-class partner)•Operate in a parallel, blinded, limited competition model for

maximal learning• Include the ecosystem of players in the design of PRACTICES

that enable biomarkers/biosignatures (academic, industry, advocacy, regulatory, philanthropic, etc)

Conceive, deploy, test, iterate, and improve standardized VALIDATION/VERIFICATION studies with an eye to business development• Deploy Laboratory Developed Tests under CLIA (versus FDA) • Harmonize test validation requirements between CMS and FDA• Portability &reproducibility testing by >3 performance sites•Work with regulatory groups on “gold standards” for highly

complex or multi-plex analyte tests• Support as a thought-leader, FDA’s requirements to launch

companion diagnostics with drug trials • Include the ecosystem of players in the design of

VALIDATION/VERIFICATION studies for biomarkers/biosignatures (academic, industry, advocacy, regulatory, philanthropic, venture funding, etc)