Research Article| Volume 140, ISSUE 2, P193-198, February 2016

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Endpoints in clinical trials: What do patients consider important? A survey of the Ovarian Cancer National Alliance

Published:November 26, 2015DOI:


      • Clinicians have debated the selection of ovarian clinical trial endpoints.
      • Optimal endpoint selection should reflect true patient benefit.
      • We surveyed patients to discern what constitutes meaningful clinical trials outcomes.



      In order to understand the patient's perspective in regards to meaningful surrogate clinical trial endpoints and the impact of treatment-related toxicity, and quality of life, we surveyed women with gynecological cancers to ascertain their preferences.


      A 28-question anonymous online survey was posted on the OCNA website ( Survey questions included demographic factors, tumor data, and patients' preference regarding side effects and therapy endpoints. Data was analyzed for frequency and percentage of each response. Student t-test, Fisher's exact test and Wilcoxon rank sums were preformed.


      There were 1413 survey responses. Participants reported that for a new agent to be meaningful, the minimum extension of progression-free survival (PFS) and overall survival (OS) should be five or more months, 77% and 85% of the time, respectively. Most subjects (55%, n = 612) were interested in an agent that would keep tumor growth relatively static without change in OS. Addressing the impact of adverse aspects from a hypothetical new agent as a function of response, there was significant migration (p < 0.0001) to acceptance of greater toxicity and cost under the scenario of a 5–6 months OS gain, despite three-fold higher neurotoxicity, as compared to a PFS gain of 3–4 months/no OS gain without toxicity. Response patterns weren't altered by recurrence status.


      Herein, we show that magnitude of outcome is a desired effect, even given the prospect of significant toxicity and cost. However, these preferences appear to differ between those with primary and recurrent disease.


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