Biomedical Ethics Research Program, Mayo Clinic, Rochester, MN, USA and School of Medicine, University of North Carolina - Chapel Hill, Chapel Hill, NC, USA.
Biomedical Ethics Research Program and Alix School of Medicine, Mayo Clinic, Rochester, MN, USA.
J Natl Cancer Inst. 2021 Jun 1;113(6):735-741. doi: 10.1093/jnci/djaa137.
The federal Right-to-Try (RTT) Act created an alternate regulatory pathway for preapproval access to investigational drugs. A few studies have examined the experiences of physicians with the Food and Drug Administration's Expanded Access Programs, but to our knowledge, no study has yet to examine their attitudes and experiences toward RTT.
This study explored the views of 21 oncologists at a major cancer center with 3 main sites across the United States using semi-structured interviews and qualitative analysis. Participants were selected to have experience with Expanded Access Programs.
Most oncologists had limited familiarity with RTT, and several reported confusion about the legislation, including whether patients have a right to investigational drugs and an obligation for companies to provide them. Although oncologists were interested in decreased regulatory burdens, 3 areas of concern were articulated: lack of safety and oversight, unclear structure and no provision for data collection, and potential heightening of patient expectations. Only 4 oncologists had experience discussing RTT, and none formally attempted to obtain the drug through this mechanism. Participants questioned the practicality of RTT legislation and suggested alternative ways to improve access.
The study provides foundational empirical data underlying challenging ambiguities by experienced oncologists familiar with off-trial use of investigational therapeutics and reaffirms the role of physicians and regulatory bodies in mitigating the risks of investigational drugs. Our findings highlight the need for medical centers to inform oncologists about RTT and other preapproval pathways so that they are able to address questions from patients interested in nontrial investigational drugs.
联邦“有权利尝试”（RTT）法案为获得试验性药物的预批准提供了替代监管途径。有几项研究考察了医生在食品和药物管理局扩大准入计划方面的经验，但据我们所知，还没有研究考察他们对 RTT 的态度和经验。
本研究采用半结构式访谈和定性分析，在美国主要癌症中心的 3 个主要地点，对 21 名肿瘤学家进行了调查，以探讨他们的观点。参与者的选择标准是具有扩大准入计划的经验。
大多数肿瘤学家对 RTT 的了解有限，有几位报告说对立法感到困惑，包括患者是否有权获得试验性药物以及公司是否有义务提供药物。尽管肿瘤学家对减少监管负担感兴趣，但有 3 个关注领域得到了明确：缺乏安全性和监督、结构不清晰且没有规定数据收集，以及可能提高患者的期望。只有 4 名肿瘤学家有过讨论 RTT 的经验，而且没有人正式试图通过这种机制获得药物。参与者对 RTT 立法的实用性提出了质疑，并提出了改善准入的替代方法。
该研究为经验丰富的肿瘤学家提供了基础的实证数据，阐明了他们对试验性治疗药物的非试验使用所熟悉的挑战性模棱两可之处，并再次确认了医生和监管机构在减轻试验性药物风险方面的作用。我们的研究结果强调了医疗中心有必要向肿瘤学家介绍 RTT 和其他预批准途径，以便他们能够回答对非试验性试验性药物感兴趣的患者的问题。
