Effective Incorporation of Biomarkers into Phase II Trials

Abstract The incorporationof biomarkers into the drugdevelopment processwill improveunderstandingof hownew therapeuticswork andallow formore accurate identificationof patientswhowill benefit from those therapies. Strategically plannedbiomarker evaluations inphase II studies may allow for the designofmore efficient phase III trials andbetter screeningof therapeutics for entry intophase III development, hopefully leading to increased chances of positive phase III trial results. Some examples of roles that a biomarker can play in a phase II trial include predictor of response or re-sistance to specific therapies, patient enrichment, correlative endpoint, or surrogate endpoint. Considerations for using biomarkers most effectively in these roles are discussed in the context of several examples. The substantial technical, logistic, and ethical challenges that can be faced when trying to incorporate biomarkers into phase II trials are also addressed. A rational and coor-dinated approach to the inclusion of biomarker studies throughout the drug development process will be the key to attaining the goal of personalized medicine. It is widely believed that incorporation of biomarkers into the drug development process will improve understanding of how new therapeutics work and allow for more accurate identifica