title: Statistics in clinical trials.
abstract: Statistical developments over the past several years are described in this review. Efforts in phase I studies have focused on efficient estimation of maximum tolerated dose. Issues investigated for phase II trials include incorporation of multiple endpoints and randomization. For phase III trials, methods to reduce time or use the sample size more efficiently have been investigated. However, design innovations come with costs, including possible increased risk of incorrect conclusions. Other recent challenging statistical developments in clinical trials relate to use of complementary outcomes such as quality of life and to associated biologic questions, including the emergence of the field of genomics.
authors.affiliation: Fred Hutchinson Cancer Research Center, 1100 Fairview Avenue North MP-557, Seattle, WA 98109, USA. stephani@crab.org
authors.forename: Stephanie J
authors.initials: SJ
authors.lastname: Green
authors.forename: Donna K
authors.initials: DK
authors.lastname: Pauler
mesh_terms: D002986:Clinical Trials as Topic / Q000379:methods* / Q000706:statistics & numerical data*; D017321:Clinical Trials, Phase I as Topic / Q000379:methods / Q000706:statistics & numerical data; D017322:Clinical Trials, Phase II as Topic / Q000379:methods / Q000706:statistics & numerical data; D017326:Clinical Trials, Phase III as Topic / Q000379:methods / Q000706:statistics & numerical data; D023381:Endpoint Determination / Q000379:methods / Q000706:statistics & numerical data; D006801:Humans; D009369:Neoplasms / Q000628:therapy; D011788:Quality of Life; D012107:Research Design / Q000706:statistics & numerical data