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.
Background:Thyroid nodules are common, but most are benign and do not require treatment. However, intervention is warranted in people who experience compressive symptoms or hyperthyroidism, or based on risk of malignancy. Ablative technologies may offer a minimally invasive option for those in whom surgery is being considered. We conducted a health technology assessment of ablative technologies for adults with symptomatic benign thyroid nodules, cystic thyroid nodules, autonomously functioning thyroid nodules (AFTNs), or small, low-risk papillary thyroid cancer, which included an evaluation of effectiveness, safety, cost-effectiveness, the budget impact of publicly funding ablative technologies, and patient preferences and values. Methods:We performed a systematic literature search of the clinical evidence to retrieve systematic reviews; we then complemented the chosen systematic reviews with a literature search to identify primary studies published from January 2022. We assessed the risk of bias of each included study using the Risk of Bias in Systematic Reviews tool for systematic reviews and the Cochrane Risk-of-Bias Tool 2 for primary studies. We assessed the quality of the body of evidence according to the Grading of Recommendations, Assessment, Development, and Evaluation (GRADE) Working Group criteria. We performed a systematic economic literature search and conducted cost-utility analyses with a lifetime horizon from a public payer perspective. We also analyzed the budget impact of publicly funding radiofrequency ablation (RFA) in adults with symptomatic benign thyroid nodules, AFTNs, and small, low-risk papillary thyroid cancer in Ontario. To contextualize the potential value of RFA, we spoke with people who had thyroid nodules. Results:We included 4 systematic reviews and 1 randomized controlled trial in the clinical evidence review. In patients with symptomatic benign thyroid nodules, thermal ablations (including RFA, microwave ablation, and high-intensity focused ultrasound ablation) may be as effective as surgery in reducing nodule volume and improving symptoms, and they may result in better quality of life (GRADEs: Very low to Low). In patients with cystic thyroid nodules, RFA may be as effective as ethanol ablation in reducing nodule volume and improving symptoms (GRADEs: Low). In patients with AFTNs, RFA may reduce nodule volume, improve symptoms, and normalize thyroid-stimulating hormone levels (GRADEs: Low). In patients with small, low-risk papillary thyroid cancer, thermal ablations may be as effective as surgery in terms of tumour disappearance but have a lower tumour recurrence rate; they may require less surgical time and a shorter length of hospital stay, and they may be associated with less postprocedural pain and better postprocedural quality of life than surgery (GRADEs: Low to Very low). The effectiveness of the different thermal ablations may be similar, but the evidence is very uncertain. Compared with surgery, thermal ablations may not result in hypothyroidism and may lead to fewer adverse events (GRADEs: Low to Very low). Thermal ablations are reasonably safe, and their safety profiles are comparable (GRADEs: Low to Very low).In patients with symptomatic benign thyroid nodules and AFTNs, RFA is more effective and less costly than surgery. In patients with small, low-risk papillary thyroid cancer, RFA is more effective and less costly than surgery and cost-effective compared with active surveillance at an incremental cost-effectiveness ratio of $1,574 per QALY gained. Publicly funding RFA for patients with symptomatic benign thyroid nodules, AFTNs, and small, low-risk papillary thyroid cancer in Ontario would lead to cost savings of $5.42 million, $0.64 million, and $4.03 million over 5 years, respectively.The quantitative preference evidence demonstrated that physicians preferred interventions that were safe and effective, and patients placed more value on quality of life after interventions. Patient preferences for interventions for papillary thyroid microcarcinoma were driven by aversion to complications rather than by interest in a particular treatment pathway. The people we interviewed noted their preference for minimally invasive options such as RFA for the treatment of thyroid nodules. Those who had experience with RFA noted benefits such as improvement of their symptoms, shorter recovery time, and less scarring. Conclusions:Ablative technologies may be as effective as surgery for patients with symptomatic benign thyroid nodules or small, low-risk papillary thyroid cancer, and they may also be safer. Effectiveness and safety among the different ablative technologies may be similar, but the evidence was inconclusive. In patients with symptomatic thyroid nodules and AFTNs, RFA is more effective and less costly than surgery. In patients with small, low-risk papillary thyroid cancer, RFA is more effective and less costly than surgery and cost-effective compared with active surveillance. We estimate that publicly funding RFA for patients with symptomatic benign thyroid nodules, AFTNs, and small, low-risk papillary thyroid cancer in Ontario would result in cost savings of $5.42 million, $0.64 million, and $4.03 million over the next 5 years, respectively. Thyroid nodules have a negative impact on people's physical and emotional well-being, affecting daily activities, work, and overall quality of life. The people we interviewed expressed a preference for minimally invasive treatment options such as RFA, noting benefits such as shorter recovery times and less reliance on lifelong medication as a result of preserved thyroid function.
Background:Hepatitis C virus (HCV) infection causes liver inflammation that, if left untreated, can lead to scarring of the liver (cirrhosis), liver failure, liver cancer (hepatocellular carcinoma [HCC]), and death. For most people, this process usually progresses slowly, over 10 to 20 years or more, during which time the person may remain asymptomatic despite the ongoing process of liver damage. HCV screening aims to identify people with an HCV infection so that they can be linked to care and treatment. We conducted a health technology assessment of (1) one-time HCV screening for all adults in addition to risk-based HCV screening and (2) one-time HCV screening for people born between 1945 and 1975 (1945-1975 birth cohort) in addition to risk-based HCV screening, compared with risk-based HCV screening alone. This included an evaluation of effectiveness, cost-effectiveness, the budget impact of publicly funding onetime HCV screening in those populations, and patient preferences and values. Methods:We performed a systematic literature search of the clinical evidence. We assessed the risk of bias of each included study using the Risk of Bias in Nonrandomized Studies - of Interventions (ROBINS-I) tool and the quality of the body of evidence according to the Grading of Recommendations Assessment, Development, and Evaluation (GRADE) Working Group criteria. We performed a literature search of the quantitative evidence on the preferences for HCV screening of the adult population and health care providers. We performed a systematic economic literature search and conducted a cost-utility analysis with a lifetime horizon from a public payer perspective to compare (1) one-time HCV screening for all adults plus risk-based HCV screening and (2) one-time HCV screening for the 1945-1975 birth cohort plus risk-based HCV screening, against risk-based HCV screening alone. We also analyzed the budget impact of publicly funding one-time HCV screening for all adults plus risk-based HCV screening and one-time HCV screening for the 1945-1975 birth cohort plus risk-based HCV screening in Ontario. We performed a literature search of the quantitative evidence on the preferences of adults and health care providers for HCV screening. To contextualize the potential value of expanding HCV screening, we spoke with people with HCV. Results:We included 3 observational studies in the clinical evidence review. The study findings suggest that one-time HCV screening for all adults plus risk-based HCV screening may identify more people with HCV and may result in more people with HCV linked to care compared with risk-based HCV screening alone (GRADE: Very low). No studies were identified for the assessment of one-time HCV screening for the 1945-1975 birth cohort or for the assessment of the quantitative preferences of adults and health care providers for HCV screening. One-time HCV screening for all adults plus risk-based HCV screening and one-time HCV screening for the 1945-1975 birth cohort plus risk-based HCV screening are less costly and more effective than risk-based HCV screening alone. The probability of one-time HCV screening for all adults plus risk-based HCV screening and one-time HCV screening for the 1945-1975 birth cohort plus risk-based HCV screening being cost-effective versus risk-based HCV screening alone is 100% at a willingness-to-pay of $50,000 per quality-adjusted life-year (QALY) gained and 100% at a willingness-to-pay of $100,000 per QALY gained. The annual budget impact of publicly funding one-time HCV screening for all adults plus risk-based HCV screening in Ontario over the next 5 years ranges from an additional $22 million in year 1 to $14 million in year 5. The annual budget impact of publicly funding one-time HCV screening for the 1945-1975 birth cohort plus risk-based HCV screening in Ontario over the next 5 years ranges from an additional $9 million in year 1 to $1 million in year 5. The people with HCV with whom we spoke reported that HCV negatively affected their health and social well-being, and they highlighted the emotional distress caused by the stigma associated with the infection. Conclusions:One-time HCV screening for all adults plus risk-based screening may identify more people with HCV and may result in more people with HCV linked to care compared with risk-based HCV screening alone, but the evidence is very uncertain due to concerns with generalizability of the study findings to the Ontario context. One-time HCV screening for all adults plus risk-based screening and one-time HCV screening for the 1945-1975 birth cohort plus risk-based screening are both less costly and more effective than riskbased HCV screening alone. We estimate that publicly funding one-time HCV screening for all adults plus risk-based screening and one-time HCV screening for the 1945-1975 birth cohort plus risk-based screening in Ontario would result in additional costs of $111 million and $32 million, respectively, over the next 5 years. People with HCV emphasized the need to expand HCV screening beyond traditionally defined high-risk groups to enable earlier diagnosis and treatment.
Background:When blood pressure remains elevated despite treatment, a person is considered to have uncontrolled hypertension, which increases the risk of serious health outcomes (e.g., cardiovascular disease, stroke, kidney failure, and death) over time. Renal denervation, a minimally invasive procedure targeting sympathetic nerves in the wall of renal arteries, has emerged as a promising adjunctive treatment to standard care (e.g., health behaviour modifications and antihypertensive medications). We conducted a health technology assessment of renal denervation for adults with uncontrolled hypertension, which included an evaluation of effectiveness, safety, cost-effectiveness, the budget impact of publicly funding renal denervation, and patient and provider preferences and values. Methods:We performed a systematic literature search of the clinical evidence. We assessed the risk of bias of each included systematic review using the Risk of Bias in Systematic Reviews (ROBIS) tool. We performed a systematic economic literature search and conducted a cost-utility analysis with a lifetime horizon from a public payer perspective. We also analyzed the budget impact of publicly funding renal denervation in adults with uncontrolled hypertension in Ontario. To contextualize the potential value of renal denervation, we conducted a review of the quantitative evidence of patient and provider preferences and values, and we spoke with people with hypertension. Results:We included 10 systematic reviews of randomized controlled trials in our clinical evidence review, all of which showed that renal denervation statistically significantly lowered systolic blood pressure more than standard care (by a mean of 2.1-6.3 mmHg), regardless of the type of renal denervation system used, the blood pressure end points assessed, and whether people were taking antihypertensive medications at the time of the procedure. Renal denervation in addition to standard care is more effective and more expensive than standard care alone. The incremental cost-effectiveness ratio of renal denervation in addition to standard care compared with standard care alone is $121,237 per quality-adjusted life-year (QALY) gained over a lifetime horizon. The probability of renal denervation in addition to standard care being cost-effective versus standard care alone is 0% at a willingness-to-pay (WTP) of $50,000 per QALY gained, 18.02% at a WTP of $100,000 per QALY gained, and 80.50% at a WTP of $150,000 per QALY gained. The cost-effectiveness results were sensitive to changes in time horizon, assumptions about the duration of treatment effect, and the cost of the renal denervation procedure (including the cost of the renal denervation system). The annual budget impact of publicly funding renal denervation for adults with uncontrolled hypertension in Ontario over the next 5 years ranges from an additional $0.42 million in year 1 to an additional $3.78 million in year 5. Our review of the quantitative evidence of patient and provider preferences and values found that about 30% of patients preferred renal denervation over drug therapy, with younger individuals and those with poor medication adherence more likely to favour it. All interview participants expressed a positive view of renal denervation. Those we spoke with who had undergone the procedure reported lower blood pressure, fewer doctor's visits, and greater peace of mind compared with those who had not, and some reported a reduction in medication. Others reported being open to renal denervation if it were recommended by their physician after other treatments had failed. Barriers to accessing renal denervation included limited awareness of the procedure and limited geographic access. Conclusions:In our overview of reviews, we found that renal denervation consistently lowers blood pressure more than standard care in adults with uncontrolled hypertension, including treatment-resistant hypertension. No statistically significant differences in safety outcomes or adverse events between groups were reported in the included reviews. Renal denervation in addition to standard care is more effective and more expensive than standard care alone. We estimate that publicly funding renal denervation for adults with uncontrolled hypertension in Ontario would result in additional annual costs of between $0.42 million and $3.78 million over the next 5 years. Our review of the quantitative evidence of patient and provider preferences and values and our direct patient engagement findings highlight renal denervation as a potential treatment option for adults with uncontrolled hypertension. Renal denervation was viewed favourably by all those we interviewed, particularly when other treatments have failed.
Background:Respiratory conditions encompass a wide range of disorders that affect the lungs and other parts of the respiratory system. These conditions vary greatly in severity and impact, from mild, transient issues to chronic, life-threatening diseases. Treatment options include medications, pulmonary rehabilitation, surgery, and respiratory therapies such as mechanical ventilation, continuous positive airway pressure, bilevel positive airway pressure, conventional oxygen therapies, and heated humidified high-flow therapy (HHHFT). We conducted a health technology assessment of home-based HHHFT for (1) children with pediatric obstructive sleep apnea (OSA) who cannot tolerate conventional respiratory therapies at home, and (2) adults and children hospitalized for respiratory conditions who will need treatment at home once they are discharged and who have no at-home alternatives that offer an equivalent level of support. This assessment included an evaluation of effectiveness, safety, the budget impact of publicly funding home-based HHHFT, and patient preferences and values. Methods:We performed a systematic literature search of the clinical evidence of the effectiveness and safety of home-based HHHFT for the populations described above. We performed a systematic economic literature search, but we did not conduct a primary economic evaluation because of a lack of evidence. We analyzed the budget impact of publicly funding home-based HHHFT in children with pediatric OSA and in adults and children with other respiratory conditions in Ontario. To contextualize the potential value of home-based HHHFT, we aimed to speak with adults and care partners of children in Ontario who had lived experience of respiratory conditions, including those with and without direct experience of HHHFT. Results:We did not identify any studies that met the eligibility criteria for our clinical evidence review. The estimated annual budget impact of publicly funding home-based HHHFT in Ontario over the next 5 years ranges from cost savings of $185,981 for children with pediatric OSA to an additional $2.5 million for adults and children with other respiratory conditions. We estimate that publicly funding home-based HHHFT would result in 99 fewer inpatient visits and 127 fewer outpatient visits related to pediatric OSA. It would also result in 653 inpatient days avoided for adults and children with other respiratory conditions. Due to data limitations, these budget impact estimates are highly uncertain. Care partners discussed the difficulties of caring for a child with complex care needs - particularly those of caring for a child with a tracheostomy. They shared their experiences with alternative treatment options that were ineffective in managing their child's symptoms. People with direct experience using home-based HHHFT highlighted its positive impact on their child's respiratory symptoms, improving quality of life and reducing hospital and specialist visits. Initial and ongoing costs were the biggest barriers to accessing HHHFT. Conclusions:We did not identify any studies that specifically evaluated the comparative effectiveness and safety of home-based HHHFT in relation to our research questions, but we did identify several studies conducted in other contexts that demonstrated the benefits of HHHFT, including improved oxygenation, reduced respiratory rates, decreased OSA severity, and fewer acute exacerbations of chronic obstructive pulmonary disease when used in hospital and at home. As well, HHHFT is used widely in Ontario hospitals, is generally considered to be clinically effective, and is standard care in such settings. We estimate that publicly funding home-based HHHFT in Ontario over the next 5 years would result in cost savings for children with pediatric OSA and additional costs of $2.5 million for adults and children with other chronic respiratory conditions. We estimate that publicly funding home-based HHHFT would result in fewer inpatient visits, fewer outpatient visits, and inpatient days avoided. Care partners of children with respiratory conditions viewed home-based HHHFT positively; for many, it became an essential treatment when other options failed. However, cost was a substantial barrier to accessing this treatment.
