Borysowski Jan, Ehni Hans-Jörg, Górski Andrzej
Department of Clinical Immunology, Medical University of Warsaw, Nowogrodzka Str. 59, 02-006, Warsaw, Poland.
Institute of Ethics and History of Medicine, Eberhard Karls Universität, Gartenstr. 47, 72074, Tübingen, Germany.
BMC Med. 2017 Jul 24;15(1):136. doi: 10.1186/s12916-017-0910-9.
Compassionate 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).
The 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.
Approval 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.
同情用药是指在临床试验之外使用未经批准的药物。到目前为止，美国、加拿大、许多欧洲国家、澳大利亚和巴西已经出台了同情用药规定，日本和中国也可以进行同情用药治疗。然而，各个国家的相关规定存在重要差异，特别是一些国家（如美国、西班牙和意大利）要求同情用药需经研究伦理委员会（机构审查委员会）批准，而其他一些国家（如加拿大、英国、法国和德国）则无此要求。
本文的主要目的是阐述同情用药的一些方面，这些方面对于讨论研究伦理委员会在审查同情用药中的作用至关重要。这些方面包括同情用药的性质、使用安全性和有效性未经证实的药物给患者带来的潜在风险、知情同意、医生资质以及患者选择标准。我们的分析表明，支持强制审查的论据大大超过反对的论据。
同情用药应强制要求获得研究伦理委员会的批准。反对对同情用药进行强制伦理审查的主要理由是，它主要是一种治疗而非生物医学研究。尽管如此，同情用药不同于标准临床护理，应接受研究伦理委员会的审查。首先，在实践中，同情用药往往涉及重大的研究方面。其次，它基于安全性和有效性未经证实的未经批准的药物。在同情用药基础上，从寻求使用未经批准药物的患者那里获得知情同意也可能很困难。其他重要问题包括进行治疗的医生的资质以及患者选择标准。
