Faculty of Health, York University, Toronto, Ontario, Canada.
Faculty of Medicine, Dalhousie University, Halifax, Nova Scotia, Canada.
Int J Health Serv. 2021 Jan;51(1):5-13. doi: 10.1177/0020731420979824.
Medicine regulators rely on pivotal clinical trials to make decisions about approving a new drug, but little is known about how they judge whether pivotal trials justify the approval of new drugs. We explore this issue by looking at the positions of 3 major regulators: the European Medicines Agency, Food and Drug Administration, and Health Canada. Here we report their views and the implications of those views for the approval process. On various points, the 3 regulators are ambiguous, consistent, and demonstrate flexibility. The range of views may well reflect different regulatory cultures. Although clinical trial information from pivotal trials is becoming more available, regulators are still reluctant to provide detailed information about how that information is interpreted. As medicines and vaccines come up for approval for treatment of COVID-19, transparency in how pivotal trials are interpreted will be critical in determining how these treatments should be used.
药品监管机构依赖关键性临床试验来决定是否批准新药,但对于他们如何判断关键性试验是否证明新药的批准是合理的,人们知之甚少。我们通过研究欧洲药品管理局、美国食品和药物管理局以及加拿大卫生部这 3 个主要监管机构的立场来探讨这个问题。在此,我们报告他们的观点,以及这些观点对审批程序的影响。在不同的方面,这 3 个监管机构的立场是模棱两可的、一致的,并且具有灵活性。观点的多样性很可能反映了不同的监管文化。尽管关键性试验的临床试验信息变得更加容易获得,但监管机构仍然不愿意提供有关如何解释这些信息的详细信息。随着治疗 COVID-19 的药物和疫苗申请批准,关键性试验如何解释的透明度将是决定这些治疗方法应如何使用的关键。