回顾 2005 年 7 月至 2007 年 12 月期间美国食品药品监督管理局批准的肿瘤学和血液学药物产品。

Review of oncology and hematology drug product approvals at the US Food and Drug Administration between July 2005 and December 2007.

机构信息

Division of Biometrics V, Office of Translational Sciences, Center for Drug Evaluation and Research, United States Food and Drug Administration, Silver Spring, MD, USA.

出版信息

J Natl Cancer Inst. 2010 Feb 24;102(4):230-43. doi: 10.1093/jnci/djp515. Epub 2010 Jan 29.

DOI:10.1093/jnci/djp515

PMID:20118413

Abstract

BACKGROUND

The Office of Oncology Drug Products (OODP) in the Center for Drug Evaluation and Research at the US Food and Drug Administration began reviewing marketing applications for oncological and hematologic indications in July 2005. We conducted an overview of products that were reviewed by the OODP for marketing approval and the regulatory actions taken during July 2005 to December 2007.

METHODS

We identified all applications that were reviewed by the OODP from July 1, 2005, through December 31, 2007, and reviewed the actions that OODP took. We also sought the basis for the actions taken, including the clinical trial design, endpoints used, patient accrual in the trial(s) supporting approval, and the type of regulatory approval.

RESULTS

During the study period, the OODP reviewed marketing applications for 60 new indications and took regulatory action on 58 indications. Regulatory action was based on a risk-benefit evaluation of the data submitted with each application. Products that demonstrated efficacy and had an acceptable risk-benefit ratio were granted either regular or accelerated marketing approval for use in the specific indication that was studied. Regular approval was based on endpoints that demonstrated that the drug provided clinical benefit as evidenced by a longer or better life or a favorable effect on an established surrogate for a longer or better life. Accelerated approval was based on a less well-established surrogate endpoint that was reasonably likely to predict a longer or better life. Of the 53 new indications that were approved during the study period, 39 received regular approval, nine received accelerated approval, and five were converted from accelerated to regular approval. Five applications were not approved, and two applications were withdrawn before any regulatory action was taken. Eighteen of the 53 indications that were approved were for new molecular entities.

CONCLUSION

During the study period, regulatory action was taken on 58 of the 60 marketing applications. Fifty-three applications were approved. A variety of clinical trial endpoints were used in the approval trials.

摘要

背景

美国食品药品监督管理局（FDA）药品评估和研究中心的肿瘤药物产品办公室（OODP）于 2005 年 7 月开始审查肿瘤学和血液学适应证的营销申请。我们对 OODP 审查的用于营销批准的产品和 2005 年 7 月至 2007 年 12 月期间采取的监管行动进行了概述。

方法

我们确定了 OODP 从 2005 年 7 月 1 日至 2007 年 12 月 31 日审查的所有申请，并审查了 OODP 采取的行动。我们还寻找了采取这些行动的依据，包括临床试验设计、使用的终点、支持批准的试验中的患者入组情况以及监管批准的类型。

结果

在研究期间，OODP 审查了 60 个新适应证的营销申请，并对 58 个适应证采取了监管行动。监管行动是基于对每份申请提交的数据进行风险效益评估。证明有效且具有可接受的风险效益比的产品获得了特定适应证的常规或加速营销批准。常规批准基于证明药物提供临床益处的终点，例如延长或改善生命或对既定替代终点产生有利影响，以延长或改善生命。加速批准基于不太成熟的替代终点，该终点合理地有可能预测延长或改善生命。在研究期间批准的 53 个新适应证中，有 39 个获得了常规批准，9 个获得了加速批准，5 个从加速批准转换为常规批准。5 个申请未获得批准，2 个申请在采取任何监管行动之前撤回。在获得批准的 53 个适应证中，有 18 个是新的分子实体。