重组人凝血因子Ⅶ的临床试验分析

Clinical trial analysis of recombinant human coagulation factor Ⅶ

目前,多家医药企业利用基因重组技术研发重组人凝血因子Ⅶ,其中NovoSeven和Sevenfact已获批上市,我国有多个研发中的重组凝血因子Ⅶ处于临床试验阶段。本文简要介绍NovoSeven和Sevenfact的临床试验内容,重点分析其针对血友病A/B的临床试验设计关键要素,包括临床试验开展顺序、药动学和药效学研究、给药方案探索、有效性评价指标、安全性评价关注问题等。希望国内医药企业借鉴已上市产品研发经验,结合自身特点和临床实际情况,完成合理的临床试验方案设计并获得足够的临床试验数据,进而充分说明在研产品的临床有效性和安全性特征,最终实现国产重组人凝血因子Ⅶ的成功研制。

At present, a number of pharmaceutical companies use gene recombinant technology to develop products of recombinant human coagulation factor Ⅶ, of which NovoSeven and Sevenfact have been approved for market. China has a number of products of recombinant coagulation factor Ⅶ in the research and development stage entering the clinical trial stage. This paper briefly introduces the clinical trials of NovoSeven and Sevenfact, focusing on the key elements of their clinical trial design for hemophilia A and hemophilia B, including the sequence of clinical trials, pharmacokinetic and pharmacodynamics studies, drug administration regimen exploration, efficacy evaluation indicators, and safety evaluation concerns, etc. It is hoped that domestic pharmaceutical enterprises can learn from their research and development experience, combine their own characteristics and clinical actual situation, complete reasonable clinical trial scheme design and obtain enough clinical trial data, and then fully explain the efficacy and safety characteristics of the product, and finally realize the successful development of domestic recombinant human coagulation factor Ⅶ.