[Therapeutic uses of investigational drugs: research extension, compassionate use, and expanded access].
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.
Goldim
José Roberto
JR
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
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
D015507:Drugs, Investigational