Department of Pharmaceutical Sciences, College of Pharmacy, University of Illinois, Chicago, IL, USA.
Drug Des Devel Ther. 2022 Aug 24;16:2803-2815. doi: 10.2147/DDDT.S378813. eCollection 2022.
After 18 years and the administration of billions of doses, there is little doubt about biosimilars' safety and efficacy. Yet, only 14 molecules in the EU and 9 in the US are available as biosimilars, among the 200+ targets, due mainly to the high development cost attributed to clinical efficacy testing after extensive analytical assessment, nonclinical testing, and clinical pharmacology comparisons. So far, none of the hundreds of clinical efficacy testing has failed because it cannot fail due to its lack of sensitivity for multiple reasons, as argued in this paper. This analysis is unique since biosimilars are the first category of products that are put to comparative testing as if these were new biological drugs. Clinical efficacy testing used to overcome differences in the analytical, nonclinical, and clinical pharmacology comparisons can lead to the approval of unsafe products. Only recently the regulatory agencies have begun to talk about this risk and shown their willingness to waive these studies. However, a clear change in the regulatory guidelines is required to change the mindset of all biosimilar stakeholders to bring a pivotal change in the availability of affordable biosimilars.
经过 18 年的时间，已经施行了数十亿剂药物，生物类似药的安全性和疗效已毋庸置疑。然而，在 200 多个靶点中，仅有 14 种分子在欧盟和 9 种在美国被批准为生物类似药，这主要是由于在广泛的分析评估、非临床测试和临床药理学比较之后，开发成本很高。到目前为止，数百次临床疗效测试中没有一次失败，因为正如本文所讨论的，由于多种原因，其缺乏敏感性，因此不能失败。这种分析是独特的，因为生物类似药是第一批被进行对比测试的产品，就好像这些是新的生物药物一样。用于克服分析、非临床和临床药理学比较差异的临床疗效测试可能会导致不安全产品的批准。监管机构最近才开始谈论这种风险，并表示愿意放弃这些研究。然而，需要对监管指南进行明确的更改，以改变所有生物类似药利益相关者的思维方式，从而在提供负担得起的生物类似药方面带来重大变化。
