title: Statistical considerations for a trial of Ebola virus disease therapeutics.
abstract: The 2014 West African outbreak of Ebola virus ravaged Liberia, Sierra Leone, and Guinea, causing hemorrhagic fever and death. The need to identify effective therapeutics was acute. The usual drug development paradigm of phase I, followed by phase II, and then phase III trials would take too long. These and other factors led to the design of a clinical trial of Ebola virus disease therapeutics that differs from more conventional clinical trial designs. This article describes the Ebola virus disease medical countermeasures trial design and the thinking behind it.
authors.affiliation: National Institute of Allergy and Infectious Diseases (NIAID), Bethesda, MD, USA ProschaM@niaid.nih.gov.
authors.forename: Michael A
authors.initials: MA
authors.lastname: Proschan
authors.affiliation: National Institute of Allergy and Infectious Diseases (NIAID), Bethesda, MD, USA.
authors.forename: Lori E
authors.initials: LE
authors.lastname: Dodd
authors.affiliation: US Food and Drug Administration, Silver Spring, MD, USA.
authors.forename: Dionne
authors.initials: D
authors.lastname: Price
mesh_terms: D000911:Antibodies, Monoclonal / Q000627:therapeutic use*; D000998:Antiviral Agents / Q000627:therapeutic use*; D001499:Bayes Theorem; D003627:Data Interpretation, Statistical; D019142:Hemorrhagic Fever, Ebola / Q000188:drug therapy* / Q000401:mortality; D006801:Humans; D016032:Randomized Controlled Trials as Topic / Q000379:methods*; D012107:Research Design; D016896:Treatment Outcome
chemical_list: D000911:Antibodies, Monoclonal; D000998:Antiviral Agents; C000594176:ZMapp
keywords: Barnard’s test; Bayesian methods; Fisher’s exact test; beta-binomial distribution; conditional power; emerging infectious diseases; group-sequential monitoring; non-informative prior