Ethics review in compassionate use

BMC Medicine

Opinion

Jan Borysowski¹  Andrzej Górski²  Hans-Jörg Ehni³ 

[1] Department of Clinical Immunology, Medical University of Warsaw, Nowogrodzka Str. 59, 02-006, Warsaw, Poland;Department of Clinical Immunology, Medical University of Warsaw, Nowogrodzka Str. 59, 02-006, Warsaw, Poland;Ludwik Hirszfeld Institute of Immunology and Experimental Therapy, Polish Academy of Sciences, Weigla Str. 12, 53-114, Wrocław, Poland;Institute of Ethics and History of Medicine, Eberhard Karls Universität, Gartenstr. 47, 72074, Tübingen, Germany;

关键词: Compassionate use;    Expanded access;    Research ethics committee;    Institutional review board;    Informed consent;    Clinical trial;   

DOI  :  10.1186/s12916-017-0910-9

 received in 2017-04-20, accepted in 2017-07-05,  发布年份 2017

来源: Springer

【 摘 要 】

BackgroundCompassionate use is the use of unapproved drugs outside of clinical trials. So far, compassionate use regulations have been introduced in the US, Canada, many European countries, Australia and Brazil, and treatment on a compassionate use basis may be performed in Japan and China. However, there are important differences between relevant regulations in individual countries, particularly that approval by a research ethics committee (institutional review board) is a requirement for compassionate use in some countries (e.g. the US, Spain, and Italy), but not in others (e.g. Canada, the UK, France, and Germany).DiscussionThe main objective of this article is to present aspects of compassionate use that are important for the discussion of the role of research ethics committees in the review of compassionate use. These aspects include the nature of compassionate use, potential risks to patients associated with the use of drugs with unproven safety and efficacy, informed consent, physicians’ qualifications, and patient selection criteria. Our analysis indicates that the arguments for mandatory review substantially outweigh the arguments to the contrary.ConclusionsApproval by a research ethics committee should be obligatory for compassionate use. The principal argument against mandatory ethical review of compassionate use is that it is primarily a kind of treatment rather than biomedical research. Nonetheless, compassionate use is different from standard clinical care and should be subject to review by research ethics committees. First, in practice, compassionate use often involves significant research aspects. Second, it is based on unapproved drugs with unproven safety and efficacy. Obtaining informed consent from patients seeking access to unapproved drugs on a compassionate use basis may also be difficult. Other important problems include the qualifications of the physician who is to perform treatment, and patient selection criteria.

【 授权许可】

CC BY   
© The Author(s). 2017