Hastings Cent Rep. 2019 Mar;49(2):36-39. doi: 10.1002/hast.991.
The U.S. federal government provides two tracks for eligible patients to obtain access outside clinical trials to investigational interventions currently under study for potential clinical benefits: the Food and Drug Administration's expanded access pathway and the pathway created by the more recent Right to Try Act. In this issue of the Hastings Center Report, with a critical focus on patients, industry, and the research enterprise, Kelly Folkers and colleagues frame the inherent challenges that these pathways are meant to solve and have also inadvertently created. But an additional key focus is how the relevant situations should be managed at the bedside and how the system risks both inefficient and inequitable access to options at the institutional level. Although either pathway could be helpful to patients, the challenges of having the pathways coexist are greater than the sum of their parts. Individual clinicians represent the front line of the regulatory and eligibility challenges of expanded access and right to try, making clinical education a critical component of a comprehensive approach to using them well. But it is medical institutions that must take the lead on supporting access to investigational options in the most equitable and effective manner possible.
美国联邦政府为符合条件的患者提供了两种途径，使其能够在临床试验之外获得正在研究的潜在临床益处的研究性干预措施：一是食品和药物管理局的扩大准入途径，二是最近的《尝试权法案》所创建的途径。在本期《 Hastings Center 报告》中，Kelly Folkers 及其同事重点关注患者、行业和研究企业，阐述了这些途径旨在解决但也无意中造成的固有挑战。但另一个关键重点是如何在床边管理相关情况，以及该系统如何在机构层面面临低效和不公平地获取选择的风险。尽管这两种途径都可能对患者有所帮助，但共存的途径带来的挑战大于其各部分的总和。个别临床医生代表着扩大准入和尝试权的监管和资格挑战的第一线，因此开展临床教育是综合使用这些途径的重要组成部分。但医疗机构必须带头以最公平、最有效的方式支持获取研究性选择。
