title: Multiplicity in confirmatory clinical trials: a case study with discussion from a JSM panel.
abstract: An invited panel session was conducted in the 2012 Joint Statistical Meetings, San Diego, California, USA, to stimulate the discussion on multiplicity issues in confirmatory clinical trials for drug development. A total of 11 expert panel members were invited and 9 participated. Prior to the session, a case study was previously provided to the panel members to facilitate the discussion, focusing on the key components of the study design and multiplicity. The Phase 3 development program for this new experimental treatment was based on a single randomized controlled trial alone. Each panelist was asked to clarify if he or she responded as if he or she were a pharmaceutical drug sponsor, an academic panelist or a health regulatory scientist.
authors.affiliation: US Food and Drug Administration, U.S.A.
authors.forename: Sue-Jane
authors.initials: SJ
authors.lastname: Wang
authors.affiliation: Novartis, Basel, Switzerland.
authors.forename: Frank
authors.initials: F
authors.lastname: Bretz
authors.affiliation: Quintiles, U.S.A.
authors.forename: Alex
authors.initials: A
authors.lastname: Dmitrienko
authors.affiliation: Ohio State University, Columbus, OH, U.S.A.
authors.forename: Jason
authors.initials: J
authors.lastname: Hsu
authors.affiliation: US Food and Drug Administration, U.S.A.
authors.forename: H M James
authors.initials: HM
authors.lastname: Hung
authors.affiliation: University of North Carolina, NC, U.S.A.
authors.forename: Gary
authors.initials: G
authors.lastname: Koch
authors.affiliation: Novartis, Basel, Switzerland.
authors.forename: Willi
authors.initials: W
authors.lastname: Maurer
authors.affiliation: AbbVie, IL, U.S.A.
authors.forename: Walt
authors.initials: W
authors.lastname: Offen
authors.affiliation: US Food and Drug Administration, U.S.A.
authors.forename: Robert
authors.initials: R
authors.lastname: O'Neill
mesh_terms: D017326:Clinical Trials, Phase III as Topic / Q000706:statistics & numerical data*; D003226:Congresses as Topic; D003627:Data Interpretation, Statistical; D055808:Drug Discovery / Q000706:statistics & numerical data*; D023381:Endpoint Determination / Q000379:methods*; D006801:Humans; D007231:Infant, Newborn; D012107:Research Design / Q000706:statistics & numerical data*; D012127:Respiratory Distress Syndrome, Newborn / Q000188:drug therapy*; D016896:Treatment Outcome
keywords: accelerated approval; biomarker endpoint; decision theoretic; multiplicity in regions; primary and key secondary endpoints