Leyland-Jones B, Davies B R, Clagett-Carr K, Shoemaker D, Macfarlane D, Fortner C, O'Dwyer P J, Sarosy G, Foster B J, Chun H G
Investigational Drug Branch, National Cancer Institute, Bethesda, MD.
Ann Oncol. 1992 Jan;3(1):59-62. doi: 10.1093/oxfordjournals.annonc.a058072.
The special exception mechanism was established by the Division of Cancer Treatment (DCT), National Cancer Institute (NCI), for the provision of anticancer drugs not yet approved by the Food and Drug Administration (FDA) to patients on a compassionate basis. Strict guidelines have been established for the distribution of drugs through this mechanism and for the reporting of adverse drug reactions (ADRs) with investigational drugs. These guidelines have been used to format the data base which is maintained on all ADRs submitted by investigators. In this paper, the incidence of ADRs with the eleven investigational drugs most frequently administered on special exception protocols was determined for a twelve month time period, January 1, 1985 through December 31, 1985. On special exception protocols, the overall incidence rate of ADRs was significantly greater than that seen on research protocols for the time period. For three drugs, Methyl-G, DBD, and AMSA, the ADR incidence rate was seven to fifteen-fold greater on special exception protocols than on research protocols. In an analysis of all ADRs submitted to the FDA for the twelve months time period, no difference was found in the frequency of distribution of either types of adverse effects or the causal assessments of ADRs on special exception and research protocols.
癌症治疗司（DCT）、国立癌症研究所（NCI）设立了特殊例外机制，以便在同情的基础上向患者提供尚未获得美国食品药品监督管理局（FDA）批准的抗癌药物。已经针对通过该机制分发药物以及报告研究用药物的药物不良反应（ADR）制定了严格的指导方针。这些指导方针已用于构建数据库，该数据库保存着研究人员提交的所有ADR数据。在本文中，我们确定了1985年1月1日至1985年12月31日这十二个月期间，在特殊例外方案中最常使用的11种研究用药物的ADR发生率。在特殊例外方案中，ADR的总体发生率显著高于该时间段内研究方案中的发生率。对于三种药物，即甲基-G、DBD和氨茴霉素，特殊例外方案中的ADR发生率比研究方案中的高7至15倍。在对提交给FDA的十二个月期间的所有ADR进行分析时，未发现特殊例外方案和研究方案在不良反应类型的分布频率或ADR的因果评估方面存在差异。
