Validation of Instruments for Pragmatic Clinical Trials for Overactive Bladder
Status:
Completed
Trial end date:
2013-12-01
Target enrollment:
Participant gender:
Summary
A. Objectives & Hypothesis Overactive bladder (OAB) affects over 10 million adults in the
United States and each year substantial costs are incurred from private and public funds to
test the efficacy of new and existing drugs for OAB.14 However, effectiveness and adherence
data from traditional clinical trials are not generalizable to clinical practice.3, 4 In an
era of limited resources and competing demands, it is essential that cost-effectiveness data
from clinical trials be generalizable to the clinical world. Pragmatic clinical trials
measure the effectiveness of treatments in real clinical practice, and in the full spectrum
of patients that require treatment.5 Pragmatic trials require patient reported outcomes that
are valid but have low patient burden.5 The conduct of pragmatic clinical trials for OAB has
been limited by a lack of instruments that have demonstrable validity and reliability in the
typical clinical setting Pill counts, used to measure primary outcomes in most traditional
trials, are burdensome in the clinical setting.1610 The optimal instrument for measuring
utility scores, general quality of life scores for cost-effectiveness analyses for OAB, is
also not known.
Although poor adherence carries the potential for continued suffering for patients and wasted
health care resources, there is a lack of data on the clinical and economic impact of poor
adherence to treatment for OAB in real-world clinical practice. We have published preliminary
data that underscores the role of adherence and utilities in the treatment of OAB. 3 Our
hypothesis is that effectiveness, adherence, and utility preference scores can be measured
using pragmatic patient reported instruments and that poor adherence is associated with lower
effectiveness and quality of life in adults with OAB in a clinical setting. Specifically, the
overriding goal of this proposal is to validate pragmatic instruments to measure
effectiveness, adherence and utility preference scores and obtain preliminary data on the
effect of adherence and quality of life in adults undergoing treatment for overactive bladder
in a clinical setting.