Clinical benefit in oncology trials: Is this a patient-centered or tumor-centered endpoint?

6564 Background: Clinical benefit (CB) was first successfully used as an endpoint in 1997 in the pivotal study of gemcitabine in advanced pancreas cancer. In the trial by Burris et al (J Clin Oncol. 1997) CB was a composite measure of pain, performance status, and weight. Here we describe how CB has been used in oncology trials since that time. Methods: We performed an electronic search ( www.jco.org ) for reports of all clinical trials (phase I, II, III) published in the Journal of Clinical Oncology 1997–2008 citing ‘clinical benefit.‘ Eligible trials were those reporting clinical benefit as an endpoint. Details related to study methodology, sponsorship, and endpoints were abstracted. Use of CB was classified as patient-centered if it referred to improvement in the clinical parameters used by Burris et al or in other disease-related symptoms. CB was classified as tumor-centered if it related to objective tumor criteria for partial/complete response and/or stable disease. Descriptive statistics were used to summarize findings and the chi-square test used to compare proportions. Results: 71 trials reporting CB as an endpoint were identified: 37 in breast, 8 in pancreas, and 26 in other cancers. The definition of CB was patient-centered in 21 trials (30%) and tumor-centered in 50 trials (70%). Only 20% (14/71) of trials (including all 8 pancreas studies) used the original Burris definition. In the second half of the study period there was an increase in the number of trials using CB as an endpoint (17 to 54 trials) and in the proportion of trials with a tumor-centered definition (10/17, 59% to 41/54, 76%, p = 0.09). Study variables associated with the use of a tumor-centered definition include: disease site (breast 35/37, 95%; all others 16/34, 47%, p < 0.001) and intervention (hormone or targeted agent 38/40, 95%; chemotherapy 13/31, 42%, p < 0.001). There was no association with sponsorship (industry 41/56, 73%; non-industry 10/15, 67%, p = 0.86) or phase of trial (phase I/II 36/48, 75%; phase III 15/23, 65%, p = 0.56). Conclusions: Despite its initial definition, clinical benefit is often used to describe objective tumor findings. Clinical trials should use endpoints in a consistent manner to enable clear communication between investigators, clinicians, and patients about the benefit of novel therapies. No significant financial relationships to disclose.

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