美國食品藥品監(jiān)督管理局(FDA)于 2022 年 3 月發(fā)布了"主方案:加快抗腫瘤藥物和生物制品開發(fā)的高效臨床試驗設(shè)計策略供企業(yè)用的指導(dǎo)原則"。"主方案"是指同時評價成人和兒童癌癥的同一總體試驗體系(主方案)中的 1 種以上研究藥物和(或)1 種以上癌癥類型。與在 1 項臨床試驗中,單一藥物在單一疾病人群中試驗的傳統(tǒng)的試驗設(shè)計不同,主方案使用單一的基礎(chǔ)設(shè)施、試驗設(shè)計和方案,在多個子研究中同時評價多種藥物和(或)疾病人群,從而可高效和快速地開發(fā)抗腫瘤藥物。該指導(dǎo)原則對抗腫瘤藥主方案研究的試驗設(shè)計和實施的諸多方面提出了許多很有價值的建議,而中國目前還沒有類似的指導(dǎo)原則。詳細(xì)介紹 FDA 該指導(dǎo)原則,以期對我國開展抗腫瘤藥主方案研究及其監(jiān)管有所幫助。

US Food and Drug Administration (FDA) published the "Master Protocols: Efficient Clinical Trial Design Strategies to Expedite Development of Oncology Drugs and Biologics Guidance for Industry" in March 2022. The so-called "master protocol" refers to evaluate more than one investigational drug and/or more than one cancer type within the same overall trial structure (master protocols) in adult and pediatric cancers simultaneously. In contrast to traditional trial designs, where a single drug is tested in a single disease population in one clinical trial, master protocols use a single infrastructure, trial design, and protocol to simultaneously evaluate multiple drugs and/or disease populations in multiple substudies, allowing for efficient and accelerated drug development. This guidance has put forward many valuable suggestions in terms of trial design and implementation of research on the master protocol of oncology drugs. However, there is currently no such guidance in our country. The guidance of FDA are introduced in detail, and look forward to being helpful to the research and supervision of the master protocols of oncology drugs in my country.

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