title: Need for alternative trial designs and evaluation strategies for therapeutic studies of invasive mycoses.
abstract: Studies of invasive fungal infections have been and remain difficult to implement. Randomized clinical trials of fungal infections are especially slow and expensive to perform because it is difficult to identify eligible patients in a timely fashion, to prove the presence of the fungal infection in an unequivocal fashion, and to evaluate outcome in a convincing fashion. Because of these challenges, licensing decisions for antifungal agents have to date depended heavily on historical control comparisons and secondary advantages of the new agent. Although the availability of newer and potentially more effective agents makes these approaches less desirable, the fundamental difficulties of trials of invasive fungal infections have not changed. Therefore, there is a need for alternative trial designs and evaluation strategies for therapeutic studies of invasive mycoses, and this article summarizes the possible strategies in this area.
authors.affiliation: Division of Infectious Diseases, Department of Internal Medicine, Center for the Study of Emerging and Reemerging Pathogens, University of Texas Medical School, Houston, TX, USA. John.H.Rex@uth.tmc.edu
authors.forename: J H
authors.initials: JH
authors.lastname: Rex
authors.forename: T J
authors.initials: TJ
authors.lastname: Walsh
authors.forename: M
authors.initials: M
authors.lastname: Nettleman
authors.forename: E J
authors.initials: EJ
authors.lastname: Anaissie
authors.forename: J E
authors.initials: JE
authors.lastname: Bennett
authors.forename: E J
authors.initials: EJ
authors.lastname: Bow
authors.forename: A J
authors.initials: AJ
authors.lastname: Carillo-Munoz
authors.forename: P
authors.initials: P
authors.lastname: Chavanet
authors.forename: G A
authors.initials: GA
authors.lastname: Cloud
authors.forename: D W
authors.initials: DW
authors.lastname: Denning
authors.forename: B E
authors.initials: BE
authors.lastname: de Pauw
authors.forename: J E
authors.initials: JE
authors.lastname: Edwards
authors.forename: J W
authors.initials: JW
authors.lastname: Hiemenz
authors.forename: C A
authors.initials: CA
authors.lastname: Kauffman
authors.forename: G
authors.initials: G
authors.lastname: Lopez-Berestein
authors.forename: P
authors.initials: P
authors.lastname: Martino
authors.forename: J D
authors.initials: JD
authors.lastname: Sobel
authors.forename: D A
authors.initials: DA
authors.lastname: Stevens
authors.forename: R
authors.initials: R
authors.lastname: Sylvester
authors.forename: J
authors.initials: J
authors.lastname: Tollemar
authors.forename: C
authors.initials: C
authors.lastname: Viscoli
authors.forename: M A
authors.initials: MA
authors.lastname: Viviani
authors.forename: T
authors.initials: T
authors.lastname: Wu
mesh_terms: D000935:Antifungal Agents / Q000627:therapeutic use*; D018849:Controlled Clinical Trials as Topic / Q000379:methods*; D006801:Humans; D009181:Mycoses / Q000188:drug therapy*; D016032:Randomized Controlled Trials as Topic / Q000379:methods*; D012107:Research Design; D016896:Treatment Outcome
chemical_list: D000935:Antifungal Agents