title: Evidence of clinical utility: an unmet need in molecular diagnostics for patients with cancer.
abstract: This article defines and describes best practices for the academic and business community to generate evidence of clinical utility for cancer molecular diagnostic assays. Beyond analytical and clinical validation, successful demonstration of clinical utility involves developing sufficient evidence to demonstrate that a diagnostic test results in an improvement in patient outcomes. This discussion is complementary to theoretical frameworks described in previously published guidance and literature reports by the U.S. Food and Drug Administration, Centers for Disease Control and Prevention, Institute of Medicine, and Center for Medical Technology Policy, among others. These reports are comprehensive and specifically clarify appropriate clinical use, adoption, and payer reimbursement for assay manufacturers, as well as Clinical Laboratory Improvement Amendments-certified laboratories, including those that develop assays (laboratory developed tests). Practical criteria and steps for establishing clinical utility are crucial to subsequent decisions for reimbursement without which high-performing molecular diagnostics will have limited availability to patients with cancer and fail to translate scientific advances into high-quality and cost-effective cancer care. See all articles in this CCR Focus section, "The Precision Medicine Conundrum: Approaches to Companion Diagnostic Co-development."
authors.affiliation: Authors' Affiliations: New Enterprise Associates, Inc., Menlo Park; CCS Associates, Mountain View; Myraqa, Redwood Shores, California; Johnson & Johnson/Veridex, LLC, Raritan, New Jersey; University of Texas, MD Anderson Cancer Center, Houston, Texas; Center for Diagnostics and Radiologic Health, Office of In Vitro Diagnostics, Personalized Medicine Program, Silver Spring; Center for Medical Technology Policy, Baltimore; Opus Three LLC; National Cancer Institute, Division of Cancer Treatment and Diagnosis, Rockville, Maryland; and Kaiser Permanente Research Affiliates Evidence-Based Practice Center, Kaiser Permanente Center for Health Research, Portland, Oregon.
authors.forename: David R
authors.initials: DR
authors.lastname: Parkinson
authors.forename: Robert T
authors.initials: RT
authors.lastname: McCormack
authors.forename: Susan M
authors.initials: SM
authors.lastname: Keating
authors.forename: Steven I
authors.initials: SI
authors.lastname: Gutman
authors.forename: Stanley R
authors.initials: SR
authors.lastname: Hamilton
authors.forename: Elizabeth A
authors.initials: EA
authors.lastname: Mansfield
authors.forename: Margaret A
authors.initials: MA
authors.lastname: Piper
authors.forename: Patricia
authors.initials: P
authors.lastname: Deverka
authors.forename: Felix W
authors.initials: FW
authors.lastname: Frueh
authors.forename: J Milburn
authors.initials: JM
authors.lastname: Jessup
authors.forename: Lisa M
authors.initials: LM
authors.lastname: McShane
authors.forename: Sean R
authors.initials: SR
authors.lastname: Tunis
authors.forename: Caroline C
authors.initials: CC
authors.lastname: Sigman
authors.forename: Gary J
authors.initials: GJ
authors.lastname: Kelloff
mesh_terms: D057069:Diagnostic Test Approval / Q000592:standards / Q000639:trends; D006801:Humans; D025202:Molecular Diagnostic Techniques / Q000592:standards / Q000639:trends; D058990:Molecular Targeted Therapy / Q000379:methods; D009369:Neoplasms / Q000175:diagnosis* / Q000628:therapy; D017410:Practice Guidelines as Topic / Q000592:standards; D014481:United States