Patrick Hensley: Yeah, thanks so much for having me. As you know, non-muscle-invasive bladder cancer is a highly recurrent disease state. The morbidity of the treatments from a TURBT standpoint, bladder biopsies, intravesical therapy, some of the financial toxicity and the time toxicity associated with surveillance cystoscopies, may not be adequately captured in traditional clinical trial endpoints like RFS, PFS, complete response. There's been some emphasis from patient advocacy groups, the International Bladder Cancer Group, and the FDA provided some very formal guidance in 2021 on the use of patient-reported outcomes, specifically as predefined clinical trial endpoints. So what we did was, and this was a study that was led by two residents at our University of Kentucky, Kim Toumazos and Spencer Bell.
We queried the ClinicalTrials.gov database for interventional non-muscle-invasive bladder cancer clinical trials and looked at the incorporation of patient-reported outcomes as predefined primary or secondary endpoints. This study was over the course of about 30 years. We identified a hundred eligible trials and surprisingly, only about 22% of trials actually incorporated predefined PROs as primary secondary endpoints. And even more notably, I think that there was no linear trend towards higher implementation of PROs as predefined endpoints in trials over time. So this is really kind of a call to action and really kind of dovetails nicely to some of the work done by yourself and Sarah Psutka within the IBCG to really make fit-for-purpose PROs that are all-encompassing of the patient's lived experience, from diagnostic TURBT to intravesical therapy to survivorship.
Ashish Kamat: So again, it's disappointing that it's only 20%, right? But that is something that I might have expected. Was it surprising that there was no linear, there's some attempt at increase? That surprised me to be honest.
Patrick Hensley: I think so. And I think that there's a lot of lip service to incorporating PROs and the patient experience in these trials, but the data speaks for itself. And I think that we really need to, I guess, formally define these criteria. What is a PRO that should be specifically defined for non-muscle-invasive versus muscle-invasive disease for instance? We've incorporated a lot of general health-related quality of life PROs in bladder cancer trials, but we need PROs that are specific for bladder cancer trials, specifically intravesical non-muscle-invasive trials.
Ashish Kamat: Yeah. So I think you touch upon an important point, and again, you're on the board of the IBCG, so you know that we've talked about this, but I think more than lip service, I think it might be just, because I want to give credit to our fellow colleagues, right? I'm sure they're trying, but the lack of a well-defined, annotated, a structurally sound PRO might be what's hampering the efforts. Do you think that's more likely? Because otherwise, why would people not incorporate it?
Patrick Hensley: Yeah, I think so. And especially if you're going to use a PRO, for instance, as a co-primary endpoint, you need to identify the de facto standard of care PRO in a specific disease space, and that simply doesn't exist. There's a variety of PROs that have been validated and used in a number of trials, but there is no kind of standard of care PRO within a disease state.
Ashish Kamat: Right. Well, thank you for shedding some light on this and thanks for taking the time.
Patrick Hensley: Thank you.