Expanded Access of Investigational Drugs: The Experience of the Center of Drug Evaluation and Research Over a 10-Year Period
Expanded Access of Investigational Drugs: The Experience of the Center of Drug Evaluation and Research Over a 10-Year Period
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DOI：
10.1177/2168479016656030
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发表时间：
2016-11-01
影响因子：
1.5
通讯作者：
Moscicki, Richard
中科院分区：
文献类型：
--
作者：
Jarow, Jonathan P.;Lemery, Steven;Moscicki, Richard
背景资料：本研究的目的是描述的经验，中心的药物评价和研究（CDER）与扩大访问的investigateddrugs.Methods：多次搜索的CDER的文件跟踪系统进行识别的数量，类型和适应症的所有扩大访问的请求超过10年的时间段，2005年1月至2014年12月。进行了额外的检索，以确定该时间段内所有正在进行的商业研究药物开发项目，以及临床项目是否被搁置。这两个搜索，然后交叉引用，以确定那些商业研究药物开发计划放置在临床搁置，由于严重不良事件发生在扩大访问programmes.Results：CDER收到超过1000申请扩大访问每年。大多数是为单身病人，大致平均分配之间的紧急和非紧急使用。绝大多数，99.7%，被允许继续进行。所有商业研究药物开发计划的临床持有的发病率为7.9%，相比之下，只有0.2%的患者接受药物治疗下观察到的不良事件expandedaccess.Conclusions：从FDA的角度来看，大量的应用程序处理，并允许进行每年的基础上，扩大访问程序被视为一个成功。然而，希望在扩大可及性下获得药物治疗的患者及其医疗保健提供者的实际人数尚不清楚。这是非常罕见的严重不良事件下扩大访问影响该药物的开发计划。
Background: The purpose of this study was to describe the experience of the Center of Drug Evaluation and Research (CDER) with expanded access of investigational drugs.Methods: Multiple searches of CDER's document tracking system were performed to identify the number, type, and indication for all expanded access requests over the 10-year time period of January 2005 through December 2014. An additional search was performed to identify all active commercial investigational drug development programs during that time period and whether or not the clinical program was placed on hold. The two searches were then cross-referenced to identify those commercial investigational drug development programs placed on clinical hold due to serious adverse events occurring within expanded access programs.Results: CDER receives over 1000 applications for expanded access each year. The majority are for single patients, roughly evenly split between emergency and nonemergency use. The vast majority, 99.7%, are allowed to proceed. The incidence of clinical holds for all commercial investigational drug development programs is 7.9%, as compared to only 0.2% related to adverse events observed in patients receiving drug treatments under expanded access.Conclusions: The expanded access program is viewed as a success from FDA's perspective based on the large number of applications processed and allowed to proceed each year. However, the actual number of patients and their health care providers that desire drug treatments available under expanded access is not known. It is exceedingly rare for a serious adverse event under expanded access to affect the development program for that drug.