Validation of Instruments for Pragmatic Clinical Trials for Overactive Bladder
| Status: | Completed |
|---|---|
| Conditions: | Overactive Bladder |
| Therapuetic Areas: | Gastroenterology |
| Healthy: | No |
| Age Range: | 18 - Any |
| Updated: | 2/7/2015 |
| Start Date: | May 2011 |
| End Date: | December 2013 |
| Contact: | LILY A ARYA, MS, MD |
| Email: | larya@obgyn.upenn.edu |
| Phone: | 215-615-6569 |
VALIDATION OF INSTRUMENTS FOR PRAGMATIC CLINICAL TRIALS FOR OVERACTIVE BLADDER
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.
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.
Specific Aim 1: To determine the validity and responsiveness of pragmatic patient reported
outcome instruments to measure clinical effectiveness, adherence and utility preference
scores in adults undergoing treatment with anti-cholinergic medications for overactive
bladder. We will perform a prospective cohort study of adults with OAB undergoing treatment
with anti-cholinergic medication. We will compare improvement in urinary symptoms (clinical
effectiveness), adherence, and change in utility preference scores from baseline to 3 months
of treatment using new and existing 'gold standard' instruments. The results will be
informative as to which, if any of these pragmatic instruments, is useful for measuring
effectiveness, adherence, and utility preference scores in the 'real world' clinical
setting.
Specific Aim 2: To use the newly validated pragmatic instruments to measure the impact of
adherence on clinical effectiveness and health related quality of life in a real world
clinical setting in adults undergoing treatment with anti-cholinergic medications for
overactive bladder. The impact of adherence on clinical effectiveness and health related
quality of life will be measured through comparisons in adults with high and low adherence
to anti-cholinergic medication. This aim will corroborate the findings of aim 1 and
establish if pragmatic instruments are useful in measuring outcomes of effectiveness,
adherence and utility preference scores. These results will also provide preliminary data on
the effectiveness of anti-cholinergic medication and the impact of adherence on clinical
effectiveness and quality of life in the clinical setting.
outcome instruments to measure clinical effectiveness, adherence and utility preference
scores in adults undergoing treatment with anti-cholinergic medications for overactive
bladder. We will perform a prospective cohort study of adults with OAB undergoing treatment
with anti-cholinergic medication. We will compare improvement in urinary symptoms (clinical
effectiveness), adherence, and change in utility preference scores from baseline to 3 months
of treatment using new and existing 'gold standard' instruments. The results will be
informative as to which, if any of these pragmatic instruments, is useful for measuring
effectiveness, adherence, and utility preference scores in the 'real world' clinical
setting.
Specific Aim 2: To use the newly validated pragmatic instruments to measure the impact of
adherence on clinical effectiveness and health related quality of life in a real world
clinical setting in adults undergoing treatment with anti-cholinergic medications for
overactive bladder. The impact of adherence on clinical effectiveness and health related
quality of life will be measured through comparisons in adults with high and low adherence
to anti-cholinergic medication. This aim will corroborate the findings of aim 1 and
establish if pragmatic instruments are useful in measuring outcomes of effectiveness,
adherence and utility preference scores. These results will also provide preliminary data on
the effectiveness of anti-cholinergic medication and the impact of adherence on clinical
effectiveness and quality of life in the clinical setting.
Inclusion Criteria:
1. urinary urgency for at least 3 months.
2. Subjects must have mean of ≥ 8 voids per 24 hours
3. and ≥ 1 urgency episode per 24 hours and a rating of at least moderate bother on
the single item
4. Patient Perception of Bladder Condition.
Exclusion Criteria:
1. < 18 years
2. predominant stress incontinence as measured by the validated 3-Incontinence
Questionnaire
3. current/recent use (≤6 m) or contra-indication to anti-cholinergic medication for OAB
4. severe voiding difficulties in the judgment of the investigator
5. men on unstable use of alpha blockers/5-alpha reductase inhibitors
6. severe neurologic disease
7. anti-incontinence or prolapse surgery
8. pregnancy ≤6 months
9. pelvic neuromodulation other lower urinary tract disorder such as calculus, urethral
diverticulum, current or recurrent urinary tract infections.
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