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Executive Summary

BONE PAIN REDUCTION COULD BE ADDITIONAL EFFICACY CRITERIA FOR CANCER AGENTS, FDA Office of Drug Research and Review Director Robert Temple, MD, suggested at a June 28 Oncologic Drugs Advisory Cmte. meeting. Asking the cmte. what responses other than tumor shrinkage could be meaningful in terms of determining efficacy of a cancer agent, Temple suggested that bone pain might be one possible parameter. "The things that come to mind as, possibly, symptoms whose improvement would unequivocally represent a real drug effect are things like bone pain over site of evident metastasis, a change in appetite accompanied by an increase in nonfluid weight in a patient who had been obviously cachectic, perhaps an improvement in pulmonary function in a person who did not have a lung infection but did have identified pulmonary metastases, and things of that nature," Temple stated. Temple explained that "the purpose of identifying those things is to provide within a study -- that is, without reference to any external conclusions about how you think the active control works -- unequivocal evidence of a benefit to the patient." The reason, Temple continued, "one needs parameters like that as opposed to making use of quality-of-life scores is that it seems possible that quality-of-life scores are affected by a variety of influences, only some of which might be the tumor." Because cancer trials typically use an active control, Temple explained, "a showing of no difference in survival between the new agent and the control agent does not in fact prove anything about whether either agent had an effect on survival." Temple noted, "it is fairly apparent that an approval of such an application on the basis of that kind of trial would, in effect, be an approval based on [tumor] response rate." He added: "That is not necessarily an invalid criterion." While the cmte. came to no firm conclusions, cmte. member Susan Pitman, Md, Yale University School of Medicine, New Haven, Connecticut, commented: "I think things are going in the right direction . . . I think your suggestion that, in addition to objective tumor response or shrinkage, we have some other parameter, possibly, to add to that, to somehow assess the subjective improvement of the patient, is not a bad idea."

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