BIOMARKERS/BIOSIGNATURES
Standards 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 Clinical
Trials 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)