Bridging the Gap Between Preclinical and Clinical Evaluation of Therapeutic Candidates

Session 5

Qualification and Acceptance of Biomarkers, Surrogate Endpoints and Diagnostics

Stephen A. Williams, Ph.D.
Pfizer Global Research and Development

For at least the past decade, biomarkers have contributed to decisions in clinical trial designs in all phases of drug development. Utilities in exploratory development and beyond include confirmation of mechanism [or conversely proof of non-viability], improved dose-ranging, improved detection of safety issues, improved understanding of disease and patient population selection, and many biomarkers have become accepted as diagnostics in medical practice. Indirect effects of these uses include cost and time savings in clinical trials, improved safety, and earlier access to new medicines for patients. Although there are many examples of success, which appear to be more numerous than the examples of failure, it is difficult to determine whether biomarker use is truly optimized. This is because of remaining issues around confusing language and a haphazard approach to determination of fitness for purpose [“qualification”] in the absence of a framework for developing evidentiary standards. In the presence of attempting to optimize tradeoffs between conflicting factors – such as sensitivity, specificity or economics, this leads to substantial inefficiency in the R&D process for biomarkers and diagnostics and unpredictable acceptance. I will discuss these issues together with some emerging approaches to their solution.

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