Kesselheim A S, Darrow J J
Brigham & Women's Hospital and Harvard Medical School, Boston, Massachusetts, USA; Program On Regulation, Therapeutics, And Law, Division of Pharmacoepidemiology and Pharmacoeconomics.
Clin Pharmacol Ther. 2015 Jan;97(1):29-36. doi: 10.1002/cpt.1. Epub 2014 Dec 1.
New prescription drugs receive approval from the US Food and Drug Administration (FDA) based on tests establishing safety and adequate and well-controlled trials demonstrating "substantial evidence" of efficacy. However, a number of legislative and regulatory initiatives, the most recent being the breakthrough therapy designation created in 2012, give the FDA flexibility to approve drugs on the basis of less rigorous data in situations of greater clinical need. These expedited development and review pathways now contribute to a majority of all new drug approvals and have important benefits in encouraging efficient availability of transformative drugs. They also have a number of risks, including a heightened possibility that the drugs will be discovered to be ineffective or unsafe after widespread use, and confusion by patients and physicians over what it means for a product to be "FDA approved."
新处方药需经美国食品药品监督管理局（FDA）批准，批准依据是确定安全性的测试以及充分且严格控制的试验，这些试验能证明药物有效性的“充分证据”。然而，一系列立法和监管举措，最近的一项是2012年设立的突破性疗法认定，赋予了FDA在临床需求更大的情况下，依据不那么严格的数据批准药物的灵活性。这些加速开发和审评途径如今促成了大多数新药的批准，在鼓励变革性药物高效上市方面具有重要益处。它们也存在一些风险，包括药物在广泛使用后被发现无效或不安全的可能性增加，以及患者和医生对产品“FDA批准”的含义感到困惑。
