Pragmatic randomized clinical trials: a proposal to enhance evaluation of new cancer therapies with early signs of exceptional activity

被引：12
作者：
Koehler, M. [1 ]
Donnelly, E. T. [2 ]
Kalanovic, D. [3 ]
Dagher, R. [4 ]
Rothenberg, M. L. [1 ]
机构：
[1] Pfizer Oncol, New York, NY 10017 USA
[2] Pfizer Oncol, Cambridge, MA USA
[3] Pfizer Oncol, Reg Med Affairs, Berlin, Germany
[4] Pfizer, Worldwide Safety & Regulatory, Groton, CT USA
来源：
ANNALS OF ONCOLOGY | 2016年 / 27卷 / 07期
关键词：
WORLD EFFECTIVENESS TRIAL; MULTIPLE-MYELOMA; DRUG DEVELOPMENT; SALFORD LUNG; BORTEZOMIB; PROTOCOL; AGE;
D O I：
10.1093/annonc/mdw143
中图分类号：
R73 [肿瘤学];
学科分类号：
100214 ;
摘要：
We propose that for oncology drugs that demonstrate "exceptional activity" in Phase 1 or Phase 2 trials and receive accelerated/conditional approval and/or Breakthrough Therapy Designation, and for certain expanded indications, regulatory authorities should consider accepting data from prospectively-agreed pragmatic randomized clinical trials to grant full regulatory approval.
引用
收藏
页码：1342 / 1348
页数：7
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