title: [Therapeutic uses of investigational drugs: research extension, compassionate use, and expanded access].
abstract: This article describes the methodological, regulatory, and ethical aspects of the different therapeutic uses of investigational drugs--research extension, compassionate use, and expanded access. Worldwide, the principle challenges of this kind of treatment are: setting minimum quality standards for researchers, as well as institutions, so that projects can include drugs at various stages of development; training of evaluation and assessment committees on the methodological, regulatory, and ethical aspects of new drug research; clearly outlining the relationship between researchers and funding organizations and between researchers and study participants; and understanding the opposition to the recent proposal to enable drug manufacturers to charge for drugs used in research studies.
authors.affiliation: Universidade Federal do Rio Grande do Sul (UFRGS), Hospital de Clínicas de Porto Alegre (HCPA), e Pontifícia Universidade Católica do Rio Grande do Sul (PUCRS), Faculdade de Medicina, Brasil. jrgoldim@gmail.com
authors.forename: José Roberto
authors.initials: JR
authors.lastname: Goldim
mesh_terms: D000328:Adult; D001938:Brazil; D016022:Case-Control Studies; D002648:Child; D002986:Clinical Trials as Topic; D015331:Cohort Studies; D015507:Drugs, Investigational / Q000627:therapeutic use*; D004645:Empathy; D028744:Ethics Committees, Research; D032984:Ethics, Research; D005062:European Union; D006801:Humans; D017387:Investigational New Drug Application; D012109:Research Support as Topic; D014481:United States; D014486:United States Food and Drug Administration
chemical_list: D015507:Drugs, Investigational