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非劣效性试验在设计和解读方面的挑战
Challenges in the Design and Interpretation of Noninferiority Trials


Laura Mauri ... 其他 • 2017.10.05

非劣效性临床试验已成为评估药物、器械、生物制剂和其他疗法的主要工具。当一项有效的治疗已经确立时,研究中采用安慰剂对照或无治疗对照是不符合伦理的。许多医学症状都存在有效的治疗方法,它们是新疗法需要超越的标杆。虽然一些新的治疗方法产生了更好的疗效,但还有一些疗法可能在疗效相似的情况下更为安全、便捷或低价。设计非劣效性试验的初衷是寻找一个好的替代品(即评估一种与既定疗法疗效相近的新疗法)。最近,非劣效性试验方法也被应用于评估一种有效的治疗是否足够安全。评估非劣效性的随机试验数量是10年前的6倍。2005年,在MEDLINE数据库输入“noninferiority”进行一般检索,只有不到100项试验,然而在2015年,这类试验有将近600项。这些试验涵盖了多个内、外学科和多种治疗策略。

在本文中,我们提供了一个框架来思考非劣效性研究的特征,包括易犯错误。我们以心血管治疗试验举例说明,但非劣效性试验可以在许多领域进行。这些试验包括为使新疗法获得监管机构批准而设计的研究,以及与既定疗法相比较的试验。此外,我们还思考了将非劣效性概念和设计应用在临床研究的新兴领域。“安慰剂”一词被用来表示真正的安慰剂对照,或真正的安慰剂对照无法实施情况下所采用的无治疗对照。





作者信息

Laura Mauri, M.D., and Ralph B. D’Agostino, Sr., Ph.D.
From the Division of Cardiovascular Medicine, Brigham and Women’s Hospital (L.M.), Harvard Medical School (L.M.), the Department of Mathematics and Statistics, Boston University (R.B.D.), and Baim Institute for Clinical Research (L.M., R.B.D.) — all in Boston. Address reprint requests to Dr. Mauri at Brigham and Women’s Hospital, Dept. of Cardiovascular Medicine, 75 Francis St., Boston, MA 02115, or at lmauri@bwh.harvard.edu.

 

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