J Natl Cancer Inst:FDA批准的抗癌新药 一半以上然并卵

2018-06-07 吴星 环球医学

抗癌新药上市一直是医药行业最为关注的问题之一。为了让新药尽快来到患者身边,美国食品和药物管理局(FDA)启动了优先审评与突破性疗法认定等一系列政策,加速这些新药上市的过程。近几年来,FDA的审批速度总体而言很快,可谓成绩斐然。然而,审批过快会不会出现浑水摸鱼的情况,导致没有很好的临床获益的药物也上市呢?

抗癌新药上市一直是医药行业最为关注的问题之一。为了让新药尽快来到患者身边,美国食品和药物管理局(FDA)启动了优先审评与突破性疗法认定等一系列政策,加速这些新药上市的过程。近几年来,FDA的审批速度总体而言很快,可谓成绩斐然。然而,审批过快会不会出现浑水摸鱼的情况,导致没有很好的临床获益的药物也上市呢?

2018年5月,西班牙、瑞士和加拿大科学家在《J Natl Cancer Inst》发表一项研究,考察了FDA批准的抗癌药物的临床获益程度,用经验证的量表(如欧洲肿瘤内科学会临床获益程度量表[ESMO-MCBS])确定FDA批准的药物是否具有临床有意义的获益。

研究者在@FDA网站检索了2006年1月~2016年12月的抗癌药物申请。从药品说明书和注册试验报告中收集研究特征、结局和监管途径信息。对于随机对照试验(RCTs),使用ESMO-MCBS分级。有意义的获益定义为(新)辅助用药的级别为A或B,姑息治疗为4或5。所有统计学检验均为双侧。

研究者鉴别出用于118个适应症的63种药物。这些批准受到了135项研究的支持,其中的105项为可使用ESMO-MCBS量表评估的RCTs。

结果显示,仅有46项RCTs(43.8%)符合ESMO-MCBS有意义的获益阈值[(新)辅助试验为100%,姑息试验为38.8%]。姑息治疗试验中,有意义的ESMO-MCBS分级与III期研究、将总生存期作为首要终点的研究和具有伴随诊断的靶向药物试验相关。随着时间推移,符合ESMO-MCBS阈值的试验数量增加。(新)辅助研究的数量不足以进行统计学分析。

这表明,符合ESMO-MCBS临床获益阈值的试验数量已经随时间推移有所改善。然而,仍仅仅少于一半的支持FDA批准的RCTs符合临床有意义获益的阈值。

尽管肿瘤协会和其他专业组织越来越认识到建立癌症疗法临床有意义阈值的重要性,但对于监管机构使用的阈值是否与临床医生接受阈值的吻合度知之甚少。在当前研究中,研究者系统地将ESMO-MCBS应用于过去十年支持FDA药物批准的RCTs。研究者分析了药物的首次和补充适应症,并基于提交的批准数据评估了临床获益。结果显示,仅43.8%的RCTs达到了有意义获益的阈值,这可能反映了既往FDA对癌症药物证据标准的缓和。

抗癌新药承载了大家相当多的期待,但实际上,其中一半以上抗癌新药的临床获益都没有太大意义。

原始出处:

Tibau A, Molto C, Ocana A, et al. Magnitude of Clinical Benefit of Cancer Drugs Approved by the US Food and Drug Administration. J Natl Cancer Inst. 2018 May 1;110(5):486-492. doi: 10.1093/jnci/djx232.

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    2019-05-01 bugit
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    2018-06-07 drj2003
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    2018-09-12 qidongfanjian
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    2018-12-29 liye789132251

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Mol Cancer Ther:改变癌细胞代谢的抗癌新药

Manchester科学家们发现一种新的药物,能抑制肿瘤的生长,并且将其与放射疗法结合后其有效性得到改进,这表明其可以在临床中有效治疗肿瘤。 许多肿瘤都缺氧,并且肿瘤能量生成过程会发生变化,从有氧呼吸切换到糖酵解并生成乳酸作为副产物。 为了防止乳酸对肿瘤细胞造成毒性,这种乳酸必须被单羧酸转运蛋白(MCT)分子转运出肿瘤细胞。 阿斯利康药物的新药AZD3965抑制肺癌细胞中单羧酸转运蛋白中

7年折戟沉沙,这名小城医生终将抗癌新药推向了临床

在7年时间里,杰夫·特罗斯基(Jeff Toretsky)医生炒掉了3名CEO。从结果来看,他相信一切都很值。经历了2000多个日夜的等待后,一款治疗罕见肿瘤的在研新药,终于走上了临床试验。