DeTora Lisa M
Writing Studies and Rhetoric, Hofstra University, Hempstead, NY, USA.
Int J Clin Pract. 2017 Jul;71(7). doi: 10.1111/ijcp.12966. Epub 2017 Jun 8.
Public discourse is full of quick solutions to health care problems like cancer and rare diseases. Among these is Right to Try legislation for experimental therapies. Right to Try legislation is based on the premise that all experimental agents in clinical trials are safe and guaranteed to produce miracles. Unfortunately, this notion is at odds with expert understanding, which indicates that the benefits and risks of drug products can only be understood together and evaluated incrementally and over time. The current manuscript examines why benefit to risk considerations, a lynchpin of the ethical conduct of clinical research since the Nuremberg Code, might be easily elided from public discourse. This paper considers guidelines for regulatory writing, which routinely separate discussions of effectiveness and safety, as a possible source for some confusion. The internationally-accepted ICH M4E (Common Technical Document) guideline published in 2016 now provides additional guidance for composing Benefits and Risks Conclusions, which weigh and consider effectiveness and safety together. Yet fundamental differences in understanding the "safety" of medicinal products continue to exist between experts in biomedicine, politicians, and healthcare activists. Examining differences in the understanding of "safety" between experts and non-experts also may help explain the source for flawed logic about the safety of investigational products in Right To Try narratives. No drug product is 100% safe. Continuing to weigh benefits and risks together is an important intellectual practice necessary to safeguard human health worldwide, and testing clinical safety is the only way to provide meaningful protections to patients. Science, not miracles, can ensure the protection of patients in clinical research as well as clinical practice. Weighing benefits and risks is an essential intellectual act that informs public health. Science, not miracles, can guide this work.
公众话语中充斥着针对癌症和罕见病等医疗保健问题的快速解决方案。其中包括针对实验性疗法的“尝试权”立法。“尝试权”立法基于这样一个前提，即临床试验中的所有实验性药物都是安全的，并且肯定会产生奇迹。不幸的是，这一观念与专家的理解相悖，专家的理解表明，药品的益处和风险只能综合起来理解，并随着时间的推移逐步评估。当前的这篇论文探讨了为什么自《纽伦堡法典》以来一直是临床研究道德行为关键的益处与风险考量，可能很容易在公众话语中被忽略。本文认为，监管文件的撰写指南（该指南通常将有效性和安全性的讨论分开）可能是造成一些困惑的原因。2016年发布的国际认可的ICH M4E（通用技术文档）指南现在为撰写益处与风险结论提供了额外指导，该结论会综合权衡和考虑有效性与安全性。然而，生物医学专家、政治家和医疗保健活动家在对药品“安全性”的理解上仍然存在根本差异。审视专家和非专家在“安全性”理解上的差异，也可能有助于解释“尝试权”相关叙述中关于研究性产品安全性的错误逻辑的根源。没有任何药品是100%安全的。持续综合权衡益处和风险是保障全球人类健康所需的一项重要思维实践，而测试临床安全性是为患者提供有意义保护的唯一途径。科学而非奇迹，才能确保在临床研究以及临床实践中保护患者。权衡益处和风险是一项为公共卫生提供信息的重要思维行为。科学而非奇迹，才能指导这项工作。
