Division of Medical Ethics, NYU School of Medicine, New York City, New York, USA.
Brookfield, Connecticut, USA.
J Med Ethics. 2018 Nov;44(11):761-767. doi: 10.1136/medethics-2016-103917. Epub 2018 Jul 7.
Patients have received experimental pharmaceuticals outside of clinical trials for decades. There are no industry-wide best practices, and many companies that have granted compassionate use, or 'preapproval', access to their investigational products have done so without fanfare and without divulging the process or grounds on which decisions were made. The number of compassionate use requests has increased over time. Driving the demand are new treatments for serious unmet medical needs; patient advocacy groups pressing for access to emerging treatments; internet platforms enabling broad awareness of compelling cases or novel drugs and a lack of trust among some that the pharmaceutical industry and/or the FDA have patients' best interests in mind. High-profile cases in the media have highlighted the gap between patient expectations for compassionate use and company utilisation of fair processes to adjudicate requests. With many pharmaceutical manufacturers, patient groups, healthcare providers and policy analysts unhappy with the inequities of the status quo, fairer and more ethical management of compassionate use requests was needed. This paper reports on a novel collaboration between a pharmaceutical company and an academic medical ethics department that led to the formation of the Compassionate Use Advisory Committee (CompAC). Comprising medical experts, bioethicists and patient representatives, CompAC established an ethical framework for the allocation of a scarce investigational oncology agent to single patients requesting non-trial access. This is the first account of how the committee was formed and how it built an ethical framework and put it into practice.
几十年来，患者一直在临床试验之外接受实验性药物治疗。目前还没有全行业最佳实践，许多给予同情用药（或“预批准”）以获取其研究性产品的公司都没有大肆宣扬，也没有透露做出决定的过程或依据。随着时间的推移，同情用药的请求数量有所增加。推动这种需求的因素包括针对严重未满足医疗需求的新治疗方法；患者权益倡导团体争取获得新兴治疗方法的机会；互联网平台使人们广泛了解引人注目的案例或新型药物，以及一些人对制药行业和/或 FDA 以患者的最大利益为中心的缺乏信任。媒体上的一些备受瞩目的案例突显了患者对同情用药的期望与公司利用公平流程来裁决请求之间的差距。由于许多制药制造商、患者团体、医疗保健提供者和政策分析师对现状的不平等感到不满，因此需要更公平、更符合伦理的同情用药请求管理。本文报告了制药公司和学术医学伦理部门之间的一项新颖合作，该合作促成了同情用药咨询委员会（CompAC）的成立。CompAC 由医学专家、生物伦理学家和患者代表组成，为向单个请求非试验性获取稀缺肿瘤学研究药物的患者分配药物制定了伦理框架。这是首次描述该委员会是如何成立的，以及它是如何建立伦理框架并付诸实践的。
