Paclitaxel-Coated Balloon Dilation for Adults With Recurrent Bulbar Urethral Stricture: A Health Technology Assessment.

Abstract

Background: Bulbar urethral stricture is the narrowing of the bulbar segment of the urethra, which causes urinary symptoms and difficulty in voiding. Surgical urethroplasty is the gold standard treatment, but usually the first-line treatment is using either a simple (uncoated) balloon, a rigid dilator, or performing direct vision internal urethrotomy, which uses a blade or laser to make a cut in the stricture. Treatment with a balloon that is coated with paclitaxel has been offered as a second-line treatment when the stricture recurs. This health technology assessment looked at how safe, effective, and cost-effective paclitaxel-coated balloon dilation is for adults with recurrent bulbar urethral stricture. It also looked at the budget impact of publicly funding paclitaxel-coated balloon dilation and at the experiences, preferences, and values of people with bulbar urethral stricture.

Methods: We performed a systematic literature search and reviewed the clinical evidence and the economic evidence. We assessed the risk of bias in the study using RoB 2 and JBI tools and the quality of the body of clinical evidence according to the Grading of Recommendations Assessment, Development, and Evaluation (GRADE) Working Group criteria. We developed a probabilistic state-transition (Markov) model to conduct a cost-effectiveness analysis over the 5-year horizon from a public payer perspective. We compared urethral dilation with the paclitaxel-coated balloon catheter to usual care (i.e., endoscopic management represented by a mix of urethral dilation procedures and direct vision internal urethrotomy) for adult males with recurrent bulbar urethral strictures. We also analyzed the 5-year budget impact of publicly funding this technology in eligible adult males in Ontario. To contextualize the potential value of paclitaxel-coated balloon dilation, we aimed to speak with adults and care partners in Ontario who had lived experience with bulbar urethral strictures, including those with and without direct experience with this procedure.

Results: There is currently no evidence for head-to-head comparison between paclitaxel-coated balloon dilation and direct vision internal urethrotomy (the most common treatment method for bulbar recurrent urethral stricture in Ontario) or between paclitaxel-coated balloon dilation and surgical urethroplasty (the gold standard treatment). We identified 1 randomized controlled trial that compared outcomes of treatment with paclitaxel-coated balloon with other endoscopic methods in patients with short bulbar urethral stricture (≤ 3 cm; ROBUST III trial). This trial used Kaplan-Meier analysis and reported a statistically significant difference in freedom from repeat intervention at 1 year, favouring the intervention group (GRADE: Low). However, this estimate was likely skewed by the fact that there were participants in the intervention group who failed the treatment but did not undergo reintervention. These cases were excluded (censored) from the analysis, which made the intervention look more effective than it might actually be. Furthermore, outcomes for each of the various endoscopic methods included in the control group were not analyzed individually. Paclitaxel-coated balloon treatment may improve bothersome urinary symptoms and urine flow rate (GRADE: Low). Sexual function was not affected by the treatment in either group (GRADE: Moderate). The rate of hematuria and dysuria during the first month after treatment was higher in the intervention group than in the control group (GRADE: Moderate).We identified 2 economic studies which found that paclitaxel-coated balloon dilation was potentially cost saving at 5 years compared with usual endoscopic procedures. However, these studies were not directly applicable to the Ontario context. Our economic evaluation from the Ministry of Health perspective found that, compared with usual care over 5 years, paclitaxel-coated balloon dilation could be less costly (mean: -$1,476.44; 95%; credible interval [CrI]: -$3,217.15 to $112.40 per person) and more effective (showing a decrease in the recurrence of urethral strictures at 5 years; mean: 69%; 95% CrI: 68% to 70%). In the reference case analysis, the new treatment was cost-saving about 97% of the time. However, currently published clinical evidence that informed modeling of the effectiveness of this technology was limited and of low quality. In scenario analyses, the cost-effectiveness results were sensitive to changes in the effectiveness of paclitaxel-coated balloon dilation, duration of time horizon, and device cost.The 5-year budget impact of publicly funding paclitaxel-coated balloon dilation in eligible males is potentially cost saving, with net savings of about $0.74 million from treating 2,747 adult males in Ontario. Assuming a high rate of the procedure uptake from 50% in year 1 to 100% in year 5, we found additional costs of about $0.28 million in the first year of funding and annual savings for the remaining years ranging between $0.02 million and $0.58 million.The people with bulbar urethral strictures with whom we spoke reported hesitancy about undergoing urethroplasty and viewed paclitaxel-coated balloon dilation favourably due to it being a minimally invasive procedure. Barriers to access included lack of awareness of the procedure, the out of pocket cost when accessing it through a private clinic, and distance from hospitals or clinics performing the procedure.

Conclusions: There is currently no evidence for head-to-head comparison between paclitaxel-coated balloon dilation and direct vision internal urethrotomy or between paclitaxel-coated balloon dilation and surgical urethroplasty. While freedom from reintervention in ROBUST III trial favoured the intervention group, this may have been overestimated. However, paclitaxel-coated balloon dilation may improve urinary symptoms and urine flow rate. The rate of hematuria and dysuria during the first month after treatment was higher in the intervention group than in the control group.Paclitaxel-coated balloon dilation may be more effective and less costly than usual care for adult males with unsuccessfully treated recurrent and symptomatic bulbar urethral strictures. We estimate that publicly funding paclitaxel-coated balloon dilation in Ontario may result in cost savings of about $0.74 million over the next 5 years. Our economic analysis results remain uncertain and ought to be interpreted with caution because of limitations and low quality of the currently published clinical evidence. People with recurrent bulbar urethral strictures reported viewing paclitaxel-coated balloon dilation favourably because it is minimally invasive, but noted barriers to access.