Background The scientific aims of phase I oncology trials are to evaluate the safety and tolerability of new therapeutic agents. Subjects who enrol in phase I oncology trials generally are advanced cancer patients (ACPs) for whom standard treatments have either failed, have low expectation of benefit, or do not exist. Although all ACPs undergo an informed consent process prior to trial participation, there is evidence that ACPs may not appreciate the primary aims of a phase I trial and also have unrealistic expectations of therapeutic benefit. However, these perceptions of benefit have not been well described. A pilot study was performed to examine and evaluate these perceptions from ACPs who participated in phase I oncology trials.
Methods In this pilot study, patients who were enrolled in phase I trials were interviewed retrospectively regarding their perceptions of benefit after withdrawal from a phase I trial. To date, a total of nine ACPs have been interviewed: median age 72 years (range 59-82 years); 55% male; 77% Caucasian. Their qualitative responses were recorded and analyzed for content and themes. The interview included open-ended questions assessing patient experiences with the trial, including reasons for enrolment, impressions of improvement in quality of life, costs incurred, and other benefits.
Results Stated reasons for enrolment include hope, prior treatment failure, altruism, and family influence. Described patient perceptions of anticipated trial benefit included stabilization of disease and close monitoring of their disease. Unexpected costs incurred during trial participation were time spent during clinic visits, financial costs with transportation, time taken off work by family members to support trial participation, time spent away from the family, and additional procedures associated with the trial. Issues of quality of life during the trial revolved around side effects from the investigational agent. Many interviewed patients (44%) describe their current quality of life off the trial as being improved compared with quality of life during phase I trial participation. The majority of interviewed patients (67%) acknowledged that the trial did not impact their overall survival.
Conclusions Our data suggest that ACPs had perceptions of significant therapeutic benefit prior to enrolment, including stabilization of disease, close monitoring of their disease, and an improvement in overall survival and quality of life. There would appear to be significant and unanticipated out-of-pocket expenses incurred by subjects and their families as a result of phase I trial enrolment. Further research is needed to examine the multiple factors, such as side effects, unexpected costs, and lack of improved survival, that contribute to these individuals' perceptions of benefit from trial participation.
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