Jeffrey Frankel: Hi, I'm Dr. Jeffrey Frankel, a urologist. I'm in Seattle, Washington. I'm also the Medical Director with Seattle Urology Research Center.

Diane Newman: Diane Newman, I'm a nurse practitioner at the University of Pennsylvania and the Division of Urology here in Philadelphia, Pennsylvania.

As we've seen in practice, with patients, men, and women who have overactive bladder, it has a profound impact on their lives. Patients are embarrassed. The bladder is somewhat stigmatized, so people don't want to bring it up. But overactive bladder symptoms of urgency, frequency, incontinence, can really impair daily functioning. It affects relationships. I hear it all the time. And what's disturbing is that the patient plans their whole day around their symptoms, and basically, the location of toilets. And this can really have a negative impact on quality of life in most people that we see.

Jeffrey Frankel: A lot of the patients we see, we rule out medically significant condition. Once that's relieving the patient of that burden, they do worry about their quality of life. So these treatments are in place, statistically, to help the patients, and the drugs get approved by the FDA, but how important is it to the patient? Do they perceive improvement themselves, as compared to the statistical analysis, which shows statistically significance against placebo, for instance?

This clinical trial was designed to look at vibegron versus placebo and an active comparator. And these trials are very specific protocols. Patients have to meet certain criteria. They're enrolled in a prospective trial, and this is using vibegron, which is a β3-adrenergic receptor agonist.

In addition to looking at the two primary endpoints of micturition frequency, and urgency incontinence, as well as the important secondary endpoint of urgency, the EMPOWUR trial did also utilize the patient global impression scale and their perception going forward to look at meaningfulness.

Diane Newman: Prior to the start of the study, qualitative patient interviews were conducted, to try to get a patient perspective of what was meaningful in other symptoms, and what they saw was meaningful improvement.

Jeffrey Frankel: Well, the patients interviewed showed that, approximately 50% improvement in urgency was felt to give the patients a meaningful perception of improvement. And as far as urgency incontinence, it was approximately 75% improvement through the trial.

Diane Newman: In this study, an anchor based approach was used to interpret the clinical meaningfulness of reductions in certain key elements that was tracked, such as voting diaries.

Jeffrey Frankel: All of these graphs showed results that were pooled over all treatment groups, including by vibegron, placebo, and the active comparator, tolterodine.

The first graph shows improvement in median frequency, and the patients and the investigators are blinded as to the drug versus placebo. And the patients are reporting their improvement. You can see a clear correlation between their improvement in micturition frequency, and how they felt they were improved. And even though these are small amounts of improvements in actual voiding, voiding less often, they felt a strong improvement in the meaningfulness.

The next graph shows the median reduction in urgency. Again, similar response, you can see a continued improvement. The less urgency they had, the more they perceived that their medication was helping them.

Then the last is urge urinary incontinence, similar results showing again, less incontinence, which was significant. You're talking about 75 to 90% reduction in the urgency incontinence episodes, resulted in the patient's perception, this was clinically meaningful to them

Diane Newman: As is shown in these three graphs, the greater percentage of these patients who received by vibegron experienced at least a 15% reduction in voids or micturitions, at least 50% reduction urgency episodes, and about 75 to 90% reduction in urge urinary incontinent episodes, when compared with placebo

As a healthcare provider, and a clinician who has been practicing now for many years in this area, I feel that clinical meaningfulness data is really very important. We try to ask patients what their goals are prior to say, prescribing a medication, or instituting some other type of treatment for overactive bladder. So this study really shows us that, what patients may say, which is clinical meaningful, may not always be with the provider. So this has tremendous applications to my practice. Understanding what the patient wants and what the patient expects to see with treatment.

Jeffrey Frankel: It shows me that, the study's statistics correlate with what the patients perceive. And even a small change in the reduction in micturitions can make a significant difference in the patient's quality of life, as it relates to the bladder function. So when you see the correlation between the statistical improvement and the patient's perception of clinical improvement, or their global impression, that's important. If you're going to use a product you want satisfied patients, and that's what it's all about.

When you present these trials in a presentation, and you see the data, and you see a reduction of urinary frequency from 1.7 times to 1.3 times, when you're looking at the pool data, we know that patients void either one time or two times. But yet, when they tell you they're feeling better, it's more important than the statistic.

Diane Newman: We're asking patients what they want. What do they expect? And I think that's a really important thing in practice that we tend to forget about. What our goals may be with, say, prescribing something, or recommending something to a patient, it's not what the patient wants. What would they find clinically significant in their lives with a treatment for overactive bladder, which is so very devastating to them?