Johnson Tali M, Boron Matthew J
National Cancer Institute, National Institutes of Health, Rockville, MD.
J Hematol Oncol Pharm. 2012 Dec;2(4):120-127.
The National Cancer Institute's (NCI) Division of Cancer Treatment and Diagnosis (DCTD), as an investigational new drug sponsor, may provide early access to investigational agents for treatment use. Until recently, the NCI had 3 protocol mechanisms for distributing investigational agents through the Treatment Referral Center (TRC), a service provided by the Pharmaceutical Management Branch (PMB) within the Cancer Therapy Evaluation Program of the NCI's DCTD. The first mechanism is the Group C protocol, the second mechanism is the TRC protocol, and the third, and most common, mechanism is the Special Exception protocol.
The purpose of this article is to describe and report on the activities of the TRC at the PMB since 2000 through the end of 2011.
Capital Technology Information Services performed PMB data mining for all treatment protocols from January 1, 2000, to December 31, 2011. Requests to PMB were sorted in spreadsheet format by disposition, either as referred, approved, or denied, and were counted by type, either as Group C, TRC, or Special Exception.
More than 60% of requests were either referred or approved between 2000 and 2011. The peak number of requests was 1664 between 2000 and 2011 and occurred in 2003. The peak was mostly a result of Special Exception requests; however, more than 400 TRC requests and 20 Group C requests were approved that year. The total number of requests dropped precipitously after 2003, and since 2008 have totaled fewer than 50 annually. All Group C and TRC protocols were completed by March 2006. The lowest number of treatment use requests occurred in 2011.
Providing agents through the Special Exception mechanism is one way that promising investigational new drug agents can get to patients with life-threatening illnesses. In general, the PMB's TRC is a useful drug information resource for sites conducting clinical research in oncology, and it provides a valuable service to the oncology community.
美国国立癌症研究所（NCI）的癌症治疗与诊断司（DCTD）作为研究性新药申办方，可为治疗用途提供研究性药物的早期获取途径。直到最近，NCI有3种方案机制通过治疗转诊中心（TRC）分发研究性药物，TRC是由NCI的DCTD癌症治疗评估项目内的药物管理部门（PMB）提供的一项服务。第一种机制是C组方案，第二种机制是TRC方案，第三种也是最常见的机制是特殊例外方案。
本文旨在描述并报告2000年至2011年底PMB的TRC的活动情况。
资本技术信息服务公司对2000年1月1日至2011年12月31日期间所有治疗方案进行了PMB数据挖掘。向PMB提出的请求按处置情况以电子表格形式分类，分为转诊、批准或拒绝，并按类型计数，即C组、TRC或特殊例外。
2000年至2011年期间，超过60% 的请求被转诊或批准。2000年至2011年期间请求数量的峰值为1664，出现在2003年。该峰值主要是特殊例外请求导致的；然而，当年有400多个TRC请求和20个C组请求获得批准。2003年后请求总数急剧下降，自2008年以来每年总计少于50个。所有C组和TRC方案于2006年3月完成。2011年治疗用途请求数量最少。
通过特殊例外机制提供药物是使有前景的研究性新药能够惠及危及生命疾病患者的一种方式。总体而言，PMB的TRC是肿瘤学临床研究机构有用的药物信息资源，它为肿瘤学界提供了有价值的服务。
