Highlights
- •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.
ABSTRACT
Objective
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.
Methods
A 28-question anonymous online survey was posted on the OCNA website (www.ovariancancer.org). 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.
Results
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.
Conclusions
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.
Keywords
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Article info
Publication history
Published online: November 26, 2015
Accepted:
November 25,
2015
Received in revised form:
November 23,
2015
Received:
September 7,
2015
Identification
Copyright
© 2015 Elsevier Inc. Published by Elsevier Inc. All rights reserved.
