Department of Pharmacology, Faculty of Pharmacy, Ankara University, Ankara, Turkey.
Department of Pharmacology, Faculty of Pharmacy, Izmir Katip Celebi University, Izmir, Turkey.
Naunyn Schmiedebergs Arch Pharmacol. 2022 Aug;395(8):867-885. doi: 10.1007/s00210-022-02250-2. Epub 2022 May 11.
The second year of the COVID-19 pandemic had no adverse effect on the number of new drug approvals by the US Food and Drug Administration (FDA). Quite the contrary, with a total of 50 new drugs, 2021 belongs to the most successful FDA years. We assign these new drugs to one of three levels of innovation: (1) first drug against a condition ("first-in-indication"), (2) first drug using a novel molecular mechanism ("first-in-class"), and (3) "next-in-class", i.e., a drug using an already exploited molecular mechanism. We identify 21 first-in-class, 28 next-in-class, and only one first-in-indication drugs. By treatment area, the largest group is once again cancer drugs, many of which target specific genetic alterations. Every second drug approved in 2021 targets an orphan disease, half of them being cancers. Small molecules continue to dominate new drug approvals, followed by antibodies and non-antibody biopharmaceuticals. In 2021, the FDA continued to approve drugs without strong evidence of clinical effects, best exemplified by the aducanumab controversy.
新冠疫情第二年对美国食品药品监督管理局（FDA）批准的新药数量并无负面影响。恰恰相反，2021 年共批准了 50 种新药，属于 FDA 最成功的年份之一。我们将这些新药分为三个创新级别：（1）治疗某一特定病症的首个药物（“同类首创”），（2）采用新颖分子机制的首个药物（“同类首创”），以及（3）“同类最佳”，即使用已有开发的分子机制的药物。我们鉴定出 21 种同类首创、28 种同类最佳，以及仅 1 种同类首创药物。按治疗领域划分，最大的类别仍然是癌症药物，其中许多针对特定的遗传改变。2021 年获批的每二种药物都针对一种孤儿病，其中一半是癌症。小分子继续主导新药批准，其次是抗体和非抗体生物制药。2021 年，FDA 继续批准没有明显临床效果证据的药物，阿杜卡努单抗争议就是最好的例证。
