Investigator's Handbook A Manual for Participants in Clinical Trials of Investigational Agents Sponsored by DCTD, NCI Table of Contents Part A 1 2 3 Part B 4 5 6 Part C 7 8 9 10 11 Part D 12 13 14 15 16 Part E 17 18 19 Appendices Part E: Nonresearch or Treatment Referral Protocol Use of Investigational Agents 18. Special Exceptions 18.1 Definition 18.2 Criteria for Approval of a Special Exception Request 18.3 Requesting a Special Exception Agent 18.4 Responsibilities of Physicians Administering Special Exception Agents 18. Special Exceptions Physicians with patients who are refractory to standard measures, who are not eligible for an ongoing research protocol, and who have a cancer diagnosis for which an investigational agent has demonstrated activity may receive the agent from CTEP as a Special Exception to the policy of administering investigational agents only under a research protocol. If the requested agent is approved for Group C for that indication, it should be requested under the Group C Guidelines (Section 17). 18.1 Definition The Special Exception mechanism is the functional equivalent of a compassionate IND but differs from it in that the investigator may obtain investigational agents directly from CTEP, instead of having to obtain an IND from FDA. CTEP provides this mechanism as a service to the oncologic community and to cancer patients. Substantial effort on the part of CTEP professional and support staff is committed to maintaining the Special Exception Program. We expect that patients treated under the Special Exception mechanism are not eligible for established research protocols. Agents available for Special Exception are always in Phase 2 or Phase 3 trials. Special Exceptions are not granted for Phase 1 agents. The purpose of the Special Exception mechanism is to make unapproved investigational agents that have a significant activity against specific malignancies available to cancer patients and investigators who otherwise can not participate in a clinical trial. 18.2 Criteria for Approval of a Special Exception Request Pharmacists of the Pharmaceutical Management Branch and physician staff members of the Investigational Drug Branch review each Special Exception request and make the decision on a patient-by-patient basis, based on the following considerations: Is there a research protocol for which the patient is eligible? Have standard therapies been exhausted? Is there objective evidence that the investigational agent is active in the disease for which the request is being made? A review of the experience with Special Exception protocols in the past indicated that patients experienced considerable adverse events with little significant benefit. As a result, CTEP has attempted to improve the selection criteria for patients treated under Special Exception. Considerable evidence must attest to the activity of the agent for the requested indication. There should be sufficient data available to provide a reasonable expectation that the agent will prolong survival or improve the quality of life in a cohort of similar patients so treated. Reports of low response rates, or responses of brief duration, or anecdotal reports of an occasional response are not sufficient to justify approval. Is the agent likely to benefit this patient? Even if the agent has been reported to be active in the disease, the specific circumstances of the patient must be weighed by both his or her physicians and CTEP physicians. Please note that the Special Exception service may not be used as a means to obtain agents to treat a series of patients on protocol, or to do pilot work for an intended study. CTEP tracks these requests and will take whatever measures are necessary to discontinue such practices by an investigator. (Similar considerations apply to Group C). Agents distributed under Special Exception Service are investigational and are subject to FDA regulation and CTEP policy. 18.3 Requesting a Special Exception Agent Requests for Special Exceptions may be made in writing or by telephone to the Pharmaceutical Management Branch. You should indicate the patient's diagnosis, previous cancer therapy, current clinical status, intended dose and schedule of the requested agent, any proposed concomitant cancer agents or other therapies, and pertinent laboratory data. You should explain why the proposed use of the investigational agent is a better selection than a commercially available agent. In addition, the age, sex, and date of diagnosis should be included. 18.4 Responsibilities of Physicians Administering Special Exception Agents See Appendix XV. Table of Contents Part A 1 2 3 Part B 4 5 7 Part C 7 8 9 10 11 Part D 12 13 14 15 16 Part E 17 18 19 Appendices

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