title: Effective incorporation of biomarkers into phase II trials.
abstract: The incorporation of biomarkers into the drug development process will improve understanding of how new therapeutics work and allow for more accurate identification of patients who will benefit from those therapies. Strategically planned biomarker evaluations in phase II studies may allow for the design of more efficient phase III trials and better screening of therapeutics for entry into phase 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 resistance 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 coordinated approach to the inclusion of biomarker studies throughout the drug development process will be the key to attaining the goal of personalized medicine.
authors.affiliation: Biometric Research Branch, Division of Cancer Treatment and Diagnosis, National Cancer Institute, Bethesda, Maryland, USA. lm5h@nih.gov
authors.forename: Lisa M
authors.initials: LM
authors.lastname: McShane
authors.forename: Sally
authors.initials: S
authors.lastname: Hunsberger
authors.forename: Alex A
authors.initials: AA
authors.lastname: Adjei
mesh_terms: D014408:Biomarkers, Tumor / Q000032:analysis*; D017322:Clinical Trials, Phase II as Topic / Q000379:methods*; D023381:Endpoint Determination; D006801:Humans; D009369:Neoplasms / Q000175:diagnosis / Q000188:drug therapy*; D012107:Research Design; D013048:Specimen Handling
chemical_list: D014408:Biomarkers, Tumor