Establishing the Research Infrastructure To Develop and Classify
Clinical Biomarkers
Objectives
•
Explore the development of a conceptual model that describes the
future for biomarker development and their application as surrogate
endpoints
•
Establish an action plan throughout the clinical trials enterprise
to apply this model
•
Evaluate classification systems for surrogate endpoints
•
Consider broad infrastructure needs in basic and clinical research
areas to apply fundamental biology and technological advances to foster
development of novel clinical measuring tools
•
Characterize the need to organize scientific disciplines that will
utilize biomarkers in evaluation of therapeutic safety and efficacy
AGENDA
Moderators:
Bert Spilker, Ph.D., M.D., PhRMA (Pharmaceutical Research and
Manufacturers of America)
Discussants:
Adam Cohen, M.D., Ph.D., Centre for Human Drug Research, The
Netherlands
ABSTRACTS
Establishing
the Research Infrastructure To Develop and Classify
Clinical
Biomarkers
Bert
Spilker, Ph.D., M.D.; Scott L. Zeger, Ph.D.; John Oates, M.D.; and Adam
Cohen, M.D., Ph.D.
The purpose of the session will be to propose an agenda for advancing the scientific approaches by which clinical biomarkers will be discovered, evaluated, and in appropriate instances, developed as effective surrogate endpoints. This session will be organized as a free‑flowing discussion designed to address several topics. The first is the research infrastructure in support of developing clinical biomarkers, to include (1) Centers of Excellence that provide an environment in which fundamental scientific inquiry is integrated with clinical investigation in the process of translational research; (2) technological resources (e.g., chip technology, mass spectrometry, proteomics, magnetic resonance imaging); (3) training and the training environment; (4) requests for proposals that encourage development of biomarkers in designated areas; and (5) other infrastructure support. The second topic, research infrastructure in support of classifying clinical biomarkers, will include (1) What evidence is necessary to establish the utility of a surrogate endpoint for a particular treatment and disease? (2) What new methodologies will enhance our ability to generate the evidence necessary to establish the utility of a surrogate endpoint? (3) Who should be systematically evaluating surrogate endpoints? How can such research be stimulated and disseminated for maximal benefit? Should there be a repository of such information on surrogate endpoints? If so, where should it be located, and who should manage it? and, (4) What will research on biomarkers and surrogate endpoints look like over the next 5 to 10 years? Participants in this session will include bench scientists, clinical investigators, and statisticians with both scientific and regulatory perspectives. Session participants will draft a tentative agenda and action plan for research and activities needed in the future.