title: Adaptive Clinical Trial Design: An Overview and Potential Applications in Dermatology.
abstract: The challenges of drug development, including increasing costs, late-stage drug failures, and the decline in the number of drugs being approved by the US Food and Drug Administration over time, have generated interest in adaptive study designs that have the potential to address these problems. Adaptive trial designs use interim data analysis to amend trials, and have been recognized for more than a decade as a way to increase trial efficiency, partly by the increased probability of demonstrating a drug effect if one exists. In this article, we define adaptive trials; give examples of the most common types; highlight the pros, cons, and ethical considerations of these designs; and illustrate how these tools can be applied to drug development in dermatology.
authors.affiliation: Harvard Medical School, Boston, Massachusetts, USA.
authors.forename: Scott A
authors.initials: SA
authors.lastname: Elman
authors.affiliation: Department of Biostatistics, Harvard T.H. Chan School of Public Health, Boston, Massachusetts, USA.
authors.forename: James H
authors.initials: JH
authors.lastname: Ware
authors.affiliation: Department of Dermatology, Tufts Medical Center, Boston, Massachusetts, USA.
authors.forename: Alice B
authors.initials: AB
authors.lastname: Gottlieb
authors.affiliation: Harvard Medical School, Boston, Massachusetts, USA; Department of Dermatology and Department of Medicine, Division of Rheumatology, Brigham and Women's Hospital, Boston, Massachusetts, USA. Electronic address: jfmerola@bwh.harvard.edu.
authors.forename: Joseph F
authors.initials: JF
authors.lastname: Merola
mesh_terms: D002986:Clinical Trials as Topic / Q000379:methods*; D003880:Dermatology / Q000379:methods*; D015195:Drug Design; D004992:Ethics, Medical; D006801:Humans; D012107:Research Design; D018401:Sample Size; D012871:Skin Diseases / Q000188:drug therapy*; D014481:United States; D014486:United States Food and Drug Administration