title: [Methodology and management of clinical trials with small number of patients for rare diseases].
authors.affiliation: OrphanDev, CIC-Centre de pharmacologie clinique et d'évaluations thérapeutiques, CeNGEPS, université de Méditerranée, CHU Hôpital de la Timone, 264, rue Saint-Pierre, 13385 Marseille cedex 5, France. Joelle.micallef@ap-hm.fr
authors.forename: Joëlle
authors.initials: J
authors.lastname: Micallef
mesh_terms: D002986:Clinical Trials as Topic / Q000379:methods* / Q000592:standards / Q000706:statistics & numerical data; D003198:Computer Simulation / Q000706:statistics & numerical data; D003299:Cooperative Behavior; D005060:Europe / Q000453:epidemiology; D005602:France / Q000453:epidemiology; D006801:Humans; D018579:Patient Selection; D035583:Rare Diseases / Q000453:epidemiology* / Q000628:therapy*; D012017:Referral and Consultation / Q000706:statistics & numerical data; D018401:Sample Size