从美国FDA批准新药看生物制品和化学药在临床审评方面考量的异同——以HER2阳性转移性乳腺癌药品帕妥珠单抗及拉帕替尼为例

Comparison of US FDA's clinical review and regulation for biologics and chemical drugs——a case study of type 2 HER2 positive metastatic breast cancer drugs pertuzumab and lapatinib

目的:研究FDA对用于HER2阳性转移性乳腺癌的帕妥珠单抗(pertuzumab,Perjeta)和拉帕替尼(lapatinib,Tykerb)在审批标准、考量以及后续监管措施方面的异同。方法:分析FDA新药审批卷宗的临床综述、审评会议纪要、标签说明及修订、上市后要求与承诺、相关法规和指南以及公开发表资料。结果:尽管帕妥珠单抗和拉帕替尼分别属于大分子的生物制品和小分子的化学药品,未发现帕妥珠单抗和拉帕替尼在临床试验规模、审批考量以及后续监管措施上有明显差异。FDA批准两药品均是基于可接受的风险-获益比。结论:未发现FDA对帕妥珠单抗和拉帕替尼审批标准及考量无明显差异。

Objective： To study FDA＇s approval criteria, considerations and post approval regulatory actions of pertuzumab and lapatinib for HER2 positive metastatic breast cancer. Methods： Analyze the drug approval packages and other public information with respect to clinical reviews, labels and revisions, minutes of review meetings, post-approval requirements and commitments, and regulatsory actions for pertuzumab and lapatinib. Results： Although pertuzumab and lapatinib are biologics and chemical drugs respectively, no remarkable difference is observed in the review consideration and post approval regulatory actions between pertuzumab and lapatinib. Both pertuzumab and lapatinib are approved by FDA based upon acceptable risk-benefit ratios. Conclusion：The clinical review criteria for pertuzumab and lapatinib are basically consistent.