Ontario Health Technology Assessment Series最新文献

Background: Surgical aortic valve replacement (SAVR) is the conventional treatment in patients at low or intermediate surgical risk. Transcatheter aortic valve implantation (TAVI) is a less invasive procedure, originally developed as an alternative for patients at high or prohibitive surgical risk.

Methods: We conducted a health technology assessment of TAVI versus SAVR in patients with severe, symptomatic aortic valve stenosis at intermediate surgical risk, which included an evaluation of effectiveness, safety, cost-effectiveness, budget impact, and patient preferences and values. We performed a literature search to retrieve systematic reviews and selected one that was relevant to our research question. We complemented the systematic review with a literature search to identify randomized controlled trials published after the review. Applicable, previously published cost-effectiveness analyses were available, so we did not conduct a primary economic evaluation. We analyzed the net budget impact of publicly funding TAVI in people at intermediate surgical risk in Ontario. To contextualize the potential value of TAVI for people at intermediate surgical risk, we spoke with people who had aortic valve stenosis and their families.

Results: We identified two randomized controlled trials; they found that in patients with severe, symptomatic aortic valve stenosis, TAVI was noninferior to SAVR with respect to the composite endpoint of all-cause mortality or disabling stroke within 2 years of follow-up (GRADE: High). However, compared with SAVR, TAVI had a higher risk of some complications and a lower risk of others. Device-related costs for TAVI (approximately $23,000) are much higher than for SAVR (approximately $6,000). Based on two published cost-effectiveness analyses conducted from the perspective of the Ontario Ministry of Health, TAVI was more expensive and, on average, more effective (i.e., it produced more quality-adjusted life-years) than SAVR. The incremental cost-effectiveness ratios showed that TAVI may be cost-effective, but the probability of TAVI being cost-effective versus SAVR was less than 60% at a willingness-to-pay value of $100,000 per quality-adjusted life-year. The net budget impact of publicly funding TAVI in Ontario would be about $2 million to $3 million each year for the next 5 years. This cost may be reduced if people receiving TAVI have a shorter hospital stay (≤ 3 days). We interviewed 13 people who had lived experience with aortic valve stenosis. People who had undergone TAVI reported reduced physical and psychological effects and a shorter recovery time. Patients and caregivers living in remote or northern regions reported lower out-of-pocket costs with TAVI because the length of hospital stay was reduced. People said that TAVI increased their quality of life in the short-term immediately after the procedure.

Conclusions: In people with

Background: Breast cancer is a disease in which cells in the breast grow out of control. They often form a tumour that may be seen on an x-ray or felt as a lump.Gene expression profiling (GEP) tests are intended to help predict the risk of metastasis (spread of the cancer to other parts of the body) and to identify people who will most likely benefit from chemotherapy. We conducted a health technology assessment of four GEP tests (EndoPredict, MammaPrint, Oncotype DX, and Prosigna) for people with early-stage invasive breast cancer, which included an evaluation of effectiveness, safety, cost effectiveness, the budget impact of publicly funding GEP tests, 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 either the Cochrane Risk of Bias tool, Prediction model Risk Of Bias ASsessment Tool (PROBAST), or Risk of Bias Assessment tool for Non-randomized Studies (RoBANS), depending on the type of study and outcome of interest, and the quality of the body of evidence according to the Grading of Recommendations Assessment, Development, and Evaluation (GRADE) Working Group criteria. We also performed a literature survey of the quantitative evidence of preferences and values of patients and providers for GEP tests.We performed an economic evidence review to identify published studies assessing the cost-effectiveness of each of the four GEP tests compared with usual care or with one another for people with early-stage invasive breast cancer. We adapted a decision-analytic model to compare the costs and outcomes of care that includes a GEP test with usual care without a GEP test over a lifetime horizon. We also estimated the budget impact of publicly funding GEP tests to be conducted in Ontario, compared with funding tests conducted through the out-of-country program and compared with no funding of tests in any location.To contextualize the potential value of GEP tests, we spoke with people who have been diagnosed with early-stage invasive breast cancer.

Results: We included 68 studies in the clinical evidence review. Within the lymph-node-negative (LN-) population, GEP tests can prognosticate the risk of distant recurrence (GRADE: Moderate) and may predict chemotherapy benefit (GRADE: Low). The evidence for prognostic and predictive ability (ability to indicate the risk of an outcome and ability to predict who will benefit from chemotherapy, respectively) was lower for the lymph-node-positive (LN+) population (GRADE: Very Low to Low). GEP tests may also lead to changes in treatment (GRADE: Low) and generally may increase physician confidence in treatment recommendations (GRADE: Low).Our economic evidence review showed that GEP tests are generally cost-effective compared with usual care.Our primary economic evaluation showed that all GEP test strategies were more effective

Background: Cell-free circulating tumour DNA blood testing (also called liquid biopsy) can determine if a person with advanced non-small cell lung cancer (NSCLC) whose disease is progressing has developed the epidermal growth factor receptor (EGFR) T790M resistance mutation. Identifying this resistance mutation can help physicians choose appropriate treatment (i.e., osimertinib if positive and chemotherapy if negative). Tissue biopsy is typically used to look for the resistance mutation, but this is an invasive test that might not be feasible if the patient is too ill. We conducted a health technology assessment of liquid biopsy for people with advanced NSCLC, which included an evaluation of the diagnostic accuracy, clinical utility, safety, cost-effectiveness, and the budget impact of publicly funding liquid biopsy, as well as an evaluation of 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 Risk of Bias in Systematic Reviews (ROBIS), Quality Assessment of Diagnostic Accuracy Studies (QUADAS-2), Risk of Bias Among Non-randomized Studies (RoBANS), and the Cochrane risk of bias (ROB) tool and assessed quality 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 short-term and long-term cost-effectiveness and cost-utility analyses comparing liquid biopsy as a triage test, liquid biopsy alone, and tissue biopsy alone from a public payer perspective. We also analyzed the budget impact of publicly funding liquid biopsy for people in Ontario with advanced NSCLC. To assess the potential value of liquid biopsy, we spoke with people with lung cancer and people with an understanding of the process of liquid biopsy.

Results: We included 19 studies (within a published systematic review) to examine diagnostic test accuracy and 12 studies to examine clinical utility. In patients with advanced NSCLC, liquid biopsy to detect the EGFR T790M resistance mutation demonstrated a positive and negative predictive value of 89% and 61%, respectively, a sensitivity of 68%, and specificity of 86%. No studies examined the clinical utility of liquid biopsy as a triage test. When NSCLC was treated appropriately, progression-free survival was similar in patients with and without the resistance mutation, as ascertained by liquid biopsy.We estimated that it costs about $700 to conduct a liquid biopsy and $2,500 to conduct a tissue biopsy. Our analyses showed that, when considering costs and effects directly related to testing, liquid biopsy (as a triage test, which means patients who test negative undergo a follow-up tissue biopsy, or alone, which means using only liquid biopsy) was less costly than tissue biopsy alone and led to fewer tissue

Background: Glaucoma is a condition that causes progressive damage to the optic nerve, which can lead to visual impairment and irreversible blindness. There is a spectrum of current treatments for glaucoma that aim to reduce intraocular pressure (IOP), including pharmacotherapy (eye drops), laser therapy, and the more invasive option of filtration surgery. A new class of treatments called minimally invasive glaucoma surgery (MIGS) may reduce IOP and offer a better safety profile than more invasive procedures. We conducted a budget impact analysis of MIGS for adults with glaucoma from the perspective of the Ontario Ministry of Health and Long-Term Care. We also conducted interviews with people with glaucoma and family members of people with glaucoma to determine patient preferences and values surrounding glaucoma and its treatment options, including MIGS. We completed this work to complement a health technology assessment conducted in collaboration with the Canadian Agency for Drugs and Technologies in Health (CADTH).

Methods: We analyzed the budget impact of publicly funding MIGS in adults with glaucoma in Ontario. We derived costs from the collaborative health technology assessment.¹ We assumed MIGS may be used in three subgroups: (1) MIGS in combination with cataract surgery as a replacement for cataract surgery alone in people with mild to moderate glaucoma; (2) MIGS alone as a replacement for other glaucoma treatments in people with mild to moderate glaucoma; and (3) MIGS (alone or in combination with cataract surgery) to replace filtration surgery (alone or in combination with cataract surgery) in people with advanced to severe glaucoma. We estimated the budget impact over 5 years for two possible uptake scenarios: a slow rate of uptake and a fast rate of uptake. To contextualize the lived experience of glaucoma and treatments for glaucoma, we also interviewed people with glaucoma and family members of people with glaucoma, some of whom had experience with surgical procedures such as MIGS and some of whom did not.

Results: Assuming a slow uptake scenario, the annual budget impact of publicly funding MIGS in Ontario over the next 5 years ranges from $1 million in year 1 to $18 million in year 5. Assuming a fast uptake scenario, the annual budget impact of publicly funding MIGS in Ontario over the next 5 years ranges from $6 million in year 1 to $70 million in year 5. The budget impact varies depending on the proportion of people in each of the three subgroups described above. Introducing a new MIGS billing code may reduce the overall expenditures. Interview participants felt that less invasive surgical procedures, such as MIGS, could control glaucoma progression with minimal side effects and recovery time needed.

Conclusions: We estimate that publicly funding MIGS in Ontario would result in additional costs over the next 5 years; however, this

Background: People with diabetes manage their condition by monitoring the amount of glucose (a type of sugar) in their blood, typically using a method called self-monitoring of blood glucose. Flash glucose monitoring is another method of assessing glucose levels; it uses a sensor placed under the skin and a separate touchscreen reader device. We conducted a health technology assessment of flash glucose monitoring for people with type 1 or type 2 diabetes, which included an evaluation of effectiveness and safety, the budget impact of publicly funding flash glucose monitoring, 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 Cochrane risk-of-bias tool for randomized controlled trials and the Cochrane ROBINS-I tool for nonrandomized studies, and 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 we analyzed the net budget impact of publicly funding flash glucose monitoring in Ontario for people with type 1 diabetes and for people with type 2 diabetes requiring intensive insulin therapy who are eligible for coverage under the Ontario Drug Benefit program. To contextualize the potential value of flash glucose monitoring, we spoke with adults with diabetes and parents of children with diabetes.

Results: Six publications met the eligibility criteria for the clinical evidence review. Compared with self-monitoring of blood glucose, people who used flash glucose monitoring spent on average 1 hour more in the target glucose range (95% confidence interval [CI] 0.41-1.59) and 0.37 hours (22 minutes) less in a high glucose range (95% CI -0.69 to -0.05) (GRADE: Moderate). Among adults with well-controlled type 1 diabetes, flash glucose monitoring was more effective than self-monitoring of blood glucose in reducing glucose variability (GRADE: Moderate). Flash glucose monitoring was more effective than self-monitoring of blood glucose in reducing the average time spent in hypoglycemia (-0.47 h [95% CI -0.73 to -0.21]) and the average number of hypoglycemia events (-0.16 [95% CI -0.29 to -0.03]) among adults with type 2 diabetes requiring intensive insulin therapy (GRADE: Moderate). Our certainty in the evidence for the effectiveness of flash glucose monitoring for other clinical outcomes, such as quality of life and severe hypoglycemia events, is low or very low. We identified no studies on flash glucose monitoring that included pregnant people, people with diabetes who did not use insulin, or children younger than 13 years of age.We identified two studies for the economic evidence review: one cost analysis and one cost-utility analysis. The cost analysis study, conducted from the perspective of United Ki

Background: Osseointegrated prosthetic implants are biocompatible metal devices that are inserted into the residual bone to integrate with the bone and attach to the external prosthesis, eliminating the need for socket prostheses and the problems that may accompany their use. We conducted a health technology assessment of osseointegrated prosthetic implants, compared with conventional socket prostheses, for people with lower-limb amputation who experience chronic problems with their prosthetic socket, leading to prosthesis intolerance and reduced mobility. Our analysis included an evaluation of effectiveness, safety, cost-effectiveness, the budget impact of publicly funding osseointegrated prosthetic implants, and patient preferences and values.

Methods: We performed a systematic literature search of the clinical evidence on the safety and effectiveness of the latest iterations of three implant systems: the Osseointegrated Prostheses for the Rehabilitation of Amputees (OPRA) Implant System, the Endo-Exo-Femur-Prosthesis, and the Osseointegration Group of Australia-Osseointegration Prosthetic Limb (OGAP-OPL). We assessed the risk of bias of individual studies and determined 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 a cost-utility analysis with a lifetime horizon from a public payer perspective. We also analyzed the net budget impact of publicly funding osseointegrated prosthetic implants in Ontario. To contextualize the potential value of osseointegrated prosthetic implants, we spoke with people with lower-limb amputations.

Results: We included nine studies in the clinical evidence review. All studies included patients with above-the-knee amputation who underwent two-stage surgery and mostly had short-term follow-up. With osseointegrated prosthetic implants, scores for functional outcomes improved significantly as measured by 6-Minute Walk Test (6MWT), Timed Up and Go (TUG) test, and Questionnaire for Persons with a Transfemoral Amputation (Q-TFA). The scores for quality of life measured by SF-36 showed significant improvement in the physical component summary but a nonsignificant decline for the mental component summary. The most frequently seen adverse event was superficial infection, occurring in about half of patients in some studies. Deep or bone infection was a serious adverse event, with variable rates among the studies depending on the length of follow-up. The treatment of deep or bone infection required long-term antibiotic use, surgical debridement, revision surgery, and implant extraction in some cases. Other adverse events included femoral bone fracture, implant breakage, issues with extramedullary parts that required replacement, and implant removal. Our assessment of the quality of the clinical

Background: Cervical degenerative disc disease is a multifactorial condition that begins with deterioration of the intervertebral disc and results in further degeneration within the spine involving the facet joints and ligaments. This health technology assessment examined the effectiveness, safety, durability, and cost-effectiveness of cervical artificial disc replacement (C-ADR) versus fusion for treating cervical degenerative disc disease.

Methods: We performed a systematic literature search of the clinical evidence comparing C-ADR with fusion. We assessed the risk of bias in each study 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 systematic review of the economic literature and assessed the cost-effectiveness of C-ADR compared with fusion. We also estimated the budget impact of publicly funding C-ADR in Ontario over the next 5 years. To contextualize the potential value of C-ADR, we spoke with people with cervical degenerative disc disease.

Results: Eight studies of C-ADR for one-level cervical degenerative disc disease and two studies of C-ADR for two-level disease satisfied the criterion of statistical noninferiority compared with fusion on the primary outcome of 2-year overall treatment success (GRADE: Moderate). In two studies of C-ADR for two-level disease, C-ADR was statistically superior to fusion surgery for the same primary outcome (GRADE: Moderate). C-ADR was also noninferior to fusion for perioperative outcomes (e.g., operative time, blood loss), patient satisfaction, and health-related quality of life (GRADE: Moderate). C-ADR was superior to fusion for recovery and return to work, had higher technical success, and had lower rates of re-operation at the index site (GRADE: Moderate). C-ADR also maintained motion at the index-treated cervical level (GRADE: Moderate), but evidence was insufficient to determine if adjacent-level surgery rates differed between C-ADR and fusion. Current evidence is also insufficient to determine the long-term durability of C-ADR.The primary economic analysis shows that C-ADR is likely to be cost-effective compared with fusion for both one-level ($11,607/quality-adjusted life-year [QALY]) and two-level ($16,782/QALY) degeneration. Various sensitivity and scenario analyses confirm the robustness of the results. The current uptake for one-level and two-level C-ADR in Ontario is about 8% of the total eligible. For one-level involvement, the estimated net budget impact increases from $7,243 (18 procedures) in the first year to $395,623 (196 procedures) in the fifth year following public funding, for a total budget impact over 5 years of $916,326. For two-level involvement, the corresponding values are $5,460 (7 procedures) in the first year and $283,689 (76 procedures) in the fifth year, for an estimated total budget impa

Background: Pregnant people have a risk of carrying a fetus affected by a chromosomal anomaly. Prenatal screening is offered to pregnant people to assess their risk. Noninvasive prenatal testing (NIPT) has been introduced clinically, which uses the presence of circulating cell-free fetal DNA in the maternal blood to quantify the risk of a chromosomal anomaly. At the time of writing, NIPT is publicly funded in Ontario for pregnancies at high risk of a chromosomal anomaly.

Methods: We completed a health technology assessment, which included an evaluation of clinical benefits and harms, value for money, budget impact, and patient preferences related to NIPT. We performed a systematic literature search for studies on NIPT for trisomies 21, 18, and 13, sex chromosome aneuploidies, and microdeletions in the average-risk or general population. We evaluated the cost-effectiveness of traditional prenatal screening, NIPT as a second-tier test (performed after traditional prenatal screening), and NIPT as a first-tier test (performed instead of traditional prenatal screening). We also conducted a budget impact analysis to estimate the additional costs of funding first-tier NIPT. We interviewed people who had lived experience with NIPT and people living with the conditions NIPT screens for, or their families.

Results: The pooled clinical sensitivity of NIPT in the average-risk or general population was 99.5% (95% confidence interval [CI] 81.8%-99.9%) for trisomy 21, 93.1% (95% CI 75.9%-98.3%) for trisomy 18, and 92.7% (95% CI 81.6%-99.9%) for trisomy 13. The clinical specificity for any trisomy was 99.9% (95% CI 99.8%-99.9%). Compared with traditional prenatal screening, NIPT was more accurate in detecting trisomies 21, 18, and 13, and decreased the need for diagnostic testing. We found limited evidence on NIPT for sex chromosome aneuploidies or microdeletions in the average-risk or general population. Positive NIPT results should be confirmed by diagnostic testing.Compared with traditional prenatal screening, second-tier NIPT detected more affected fetuses, substantially reduced the number of diagnostic tests performed, and slightly reduced the total cost of prenatal screening. Compared with second-tier NIPT, first-tier NIPT detected more affected cases, but also led to more diagnostic tests and additional budget of $35 million per year for average-risk pregnant people in Ontario.People who had undergone NIPT were largely supportive of the test and the benefits of earlier, more accurate results. However, many discussed the need for improved pre- and post-test counselling and raised concerns about the quality of the information they received from health care providers about the conditions NIPT can screen for.

Conclusions: NIPT is an effective and safe prenatal screening method for trisomies 21, 18, and 13 in the average-risk or general population. Compared with traditiona

Background: People with chronic urinary retention typically require intermittent catheterization. This review evaluates the effectiveness, safety, patient preference, cost-effectiveness, and budget impact of different types of intermittent catheter (IC). Specifically, we compared prelubricated catheters (hydrophilic, gel reservoir) and noncoated catheters, as well as their single use versus reuse (multiple use).

Methods: We performed a systematic literature search and included randomized controlled trials, cohort, and case-control studies that examined any type of single-use versus multiple-use IC, hydrophilic single-use versus noncoated single-use, or gel reservoir single-use versus noncoated single-use. The outcomes of interest were symptomatic urinary tract infection (UTI), hematuria, other serious adverse events, and patient satisfaction. The quality of the body of evidence was examined according to the Grading of Recommendations, Assessment, Development, and Evaluation (GRADE) Working Group criteria. We also completed an economic evaluation, using the perspective of the Ontario Ministry of Health and Long-Term Care, to determine the cost-effectiveness of various intermittent catheters used in Ontario. We determined the budget impact of fully and partially funding various intermittent catheters for outpatients with chronic urinary retention. To understand patient experiences with intermittent catheterization, we interviewed 34 adults and parents of children affected by chronic urinary retention.

Results: We found 14 randomized controlled trials that met the inclusion criteria. When comparing any type of single-use or multiple-use IC, we found no difference in UTI (RR = 0.98, 95% CI 0.70-1.39), hematuria, or serious adverse events, and inconclusive evidence on patient satisfaction.Our meta-analysis of studies on people living in the community showed that hydrophilic ICs may result in fewer UTIs than single-use noncoated ICs, but given the nature of the studies, we were uncertain about this conclusion.The nature of the available evidence also did not allow us to make definitive conclusions regarding whether one type of catheter was likely to result in less hematuria, fewer serious adverse events, or greater patient satisfaction.Our economic evaluation found that owing to small differences in quality-adjusted life-years and moderate to large incremental cost differences, the lowest-cost ICs-noncoated multiple-use (using one catheter per week or one catheter per day)-have the highest probability of being cost-effective. In a subpopulation of those clinically advised not to reuse ICs, single-use noncoated ICs have the highest probability of being cost-effective. As current funding is limited in the outpatient setting, publicly funding noncoated multiple-use catheters (one per day) would result in a total additional cost of $93 million over the first 5 years. People who use ICs repor