title: NCCN Working Group report: designing clinical trials in the era of multiple biomarkers and targeted therapies.
abstract: Defining treatment-susceptible or -resistant populations of patients with cancer through the use of genetically defined biomarkers has revolutionized cancer care in recent years for some disease/patient groups. Research continues to show that histologically defined diseases are diverse in their expression of unique mutations or other genetic alterations, however, which presents opportunities for the development of personalized cancer treatments, but increased difficulty in testing these therapies, because potential patient populations are divided into ever smaller numbers. To address some of the growing challenges in biomarker development and clinical trial design, NCCN assembled a group of experts across specialties and solid tumor disease types to begin to define the problems and to consider alternate ways of designing clinical trials in the era of multiple biomarkers and targeted therapies. Results from that discussion are presented, focusing on issues of clinical trial design from the perspective of statisticians, clinical researchers, regulators, pathologists, and information developers.
authors.forename: Alan P
authors.initials: AP
authors.lastname: Venook
authors.forename: Maria E
authors.initials: ME
authors.lastname: Arcila
authors.forename: Al B
authors.initials: AB
authors.lastname: Benson
authors.forename: Donald A
authors.initials: DA
authors.lastname: Berry
authors.forename: David Ross
authors.initials: DR
authors.lastname: Camidge
authors.forename: Robert W
authors.initials: RW
authors.lastname: Carlson
authors.forename: Toni K
authors.initials: TK
authors.lastname: Choueiri
authors.forename: Valerie
authors.initials: V
authors.lastname: Guild
authors.forename: Gregory P
authors.initials: GP
authors.lastname: Kalemkerian
authors.forename: Razelle
authors.initials: R
authors.lastname: Kurzrock
authors.forename: Christine M
authors.initials: CM
authors.lastname: Lovly
authors.forename: Amy E
authors.initials: AE
authors.lastname: McKee
authors.forename: Robert J
authors.initials: RJ
authors.lastname: Morgan
authors.forename: Anthony J
authors.initials: AJ
authors.lastname: Olszanski
authors.forename: Mary W
authors.initials: MW
authors.lastname: Redman
authors.forename: Vered
authors.initials: V
authors.lastname: Stearns
authors.forename: Joan
authors.initials: J
authors.lastname: McClure
authors.forename: Marian L
authors.initials: ML
authors.lastname: Birkeland
mesh_terms: D015415:Biomarkers / Q000378:metabolism*; D002986:Clinical Trials as Topic; D006801:Humans; D009369:Neoplasms / Q000188:drug therapy* / Q000378:metabolism*; D057285:Precision Medicine / Q000379:methods; D012107:Research Design
chemical_list: D015415:Biomarkers