Early Access to Investigational Agents through the National Cancer Institute's Treatment Referral Center.

Background: 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.

Objectives: 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.

Methods: 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.

Results: 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.

Conclusion: 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.