在抗肿瘤药物临床试验中运用替代终点的审评考量

Evaluation considerations for using surrogate endpoints in anticancer clinical trials

患者的总生存时间延长是评价抗肿瘤药物临床价值的金标准。但在抗肿瘤新药的研发过程中,对于预后较好的癌种,如果进行药物临床试验时以总生存时间为主要终点,随访时间太长,药物上市时间也会相应延迟。此外,总生存时间还常受到后续治疗等混杂因素的干扰。因此,药品监管机构针对抗肿瘤新药审批上市建立了采用替代终点的加速审评模式,但在抗肿瘤药物临床试验中运用替代终点还存在一些问题。文章从新药审评的角度,深入阐述了在抗肿瘤药物临床试验中确证和合理运用替代终点的要点,有助于提高抗肿瘤新药临床试验的水平,加快抗肿瘤药物的研发。

The prolongation of patient′s overall survival is the accepted as gold standard to prove clinical values of anti-cancer drugs.However,if overall survival is taken as the primary endpoint in clinical trials for cancer types with a relatively good prognosis in the process of new anti-cancer drug research and development,the time to market the drugs will be prolonged due to the long follow-up time.In addition,overall survival is often interfered by confounding factors such as follow-up treatment.Therefore,regulatory agencies have established an accelerated review model using surrogate endpoints for the approval of new anti-cancer drugs,but there are still some problems in the use of surrogate endpoints in cancer clinical trials.From the perspective of new drug review,the authors expounds the key points of confirming and rationally using surrogate endpoints in clinical trials of anti-cancer drugs,which will improve the level of clinical trials of new anti-cancer drugs and accelerate the development of anti-tumor drugs.