Ontario Health Technology Assessment Series最新文献

{"title":"Portable Normothermic Cardiac Perfusion System in Donation After Cardiocirculatory Death: A Health Technology Assessment.","authors":"","doi":"","DOIUrl":"","url":null,"abstract":"<p><strong>Background: </strong>Heart transplantation is the most effective treatment for people experiencing end-stage heart failure whose quality of life and life expectancy are unacceptable. However, there is a chronic shortage of donor hearts to meet the demand, so it is essential to expand the donor pool and increase supply. Heart donation mainly occurs after brain death (neurological determination of death [NDD]), but it may also be feasible after cardiocirculatory death (when the heart has stopped beating and there is no longer blood flow or a pulse), provided specialized preservation techniques are used. An investigational device, a portable normothermic cardiac perfusion system, could make it possible to procure, preserve, and transport hearts donated after cardiocirculatory death (DCD). We conducted a health technology assessment of a portable normothermic cardiac perfusion system for the preservation and transportation of DCD hearts for adult transplantation. This included an evaluation of the effectiveness, safety, value for money, and budget impact of publicly funding this system, as well as an evaluation of patient preferences and values.</p><p><strong>Methods: </strong>We performed a systematic review of the clinical literature published since 1998 that examined the clinical safety and effectiveness of a portable normothermic cardiac perfusion system for DCD heart transplantation. We assessed the risk of bias of each included study and the quality of the body of evidence according to the Grading of Recommendations Assessment, Development, and Evaluation (GRADE) Working Group criteria. We also reviewed the economic evidence published during the same time period for the cost-effectiveness of a portable normothermic cardiac perfusion system for DCD hearts compared with cold storage for NDD hearts. We further estimated the 5-year net budget impact of publicly funding a normothermic cardiac perfusion system for DCD heart transplantation for adults on Ontario's waitlist. To contextualize the potential value of a portable normothermic cardiac perfusion system, we spoke with people waiting for a heart transplant, people who had received a heart transplant, and family members of organ donors.</p><p><strong>Results: </strong>We screened 2,386 clinical citations. One study and two case reports met the inclusion criteria. The survival of recipients of DCD hearts procured with a portable normothermic cardiac perfusion system did not differ significantly from the survival of recipients of hearts donated after NDD at 30 days or 90 days, nor was there a significant difference in cumulative survival at 1 year post-transplant (GRADE: Very Low). The occurrence of rejection and graft failure also did not significantly differ between the groups (GRADE: Very Low). Cardiac function in the early post-operative period was better in DCD hearts than NDD hearts (GRADE: Very Low). There were no differences in outcomes between DCD procurement techniques.The eco","PeriodicalId":39160,"journal":{"name":"Ontario Health Technology Assessment Series","volume":"20 3","pages":"1-90"},"PeriodicalIF":0.0,"publicationDate":"2020-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7077939/pdf/ohtas-20-1.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"37753428","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Auditory Brainstem Implantation for Adults With Neurofibromatosis 2 or Severe Inner Ear Abnormalities: A Health Technology Assessment.","authors":"","doi":"","DOIUrl":"","url":null,"abstract":"<p><strong>Background: </strong>Neurofibromatosis 2 (NF2) is a rare genetic disorder that causes vestibular schwannomas to develop in both eighth cranial nerves. Almost all people with NF2 eventually become completely deaf as a result of progressive tumour enlargement or following surgical or radiotherapy treatment. Other rare abnormal conditions in the inner ears can also cause complete deafness. For people with either indication who are not candidates for cochlear implantation, auditory brainstem implantation is the only treatment option to restore some functional hearing. We conducted a health technology assessment of auditory brainstem implantation for adults with NF2 and severe inner ear abnormalities, which included an evaluation of effectiveness, safety, cost-effectiveness, the budget impact of publicly funding auditory brainstem implantation, and patient preferences and values.</p><p><strong>Methods: </strong>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 Non-randomized 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 systematic economic literature search. We did not conduct a primary economic evaluation because the outcomes identified in our clinical evidence review were difficult to translate into measures appropriate for health economic modelling. We also analyzed the net budget impact of publicly funding auditory brainstem implantation over the next 5 years in Ontario, including the device, presurgical assessment, surgical procedure, and postsurgical rehabilitation. To contextualize the potential value of auditory brainstem implants, we spoke with six people with lived experience of NF2 and severe inner ear abnormalities.</p><p><strong>Results: </strong>We included 22 publications (16 in NF2, five in severe inner ear abnormalities, and one in complications of auditory brainstem implantation) in the clinical evidence review. In adults with NF2, auditory brainstem implantation when compared with no intervention allows any degree of improvement in sound recognition (GRADE: High), allows any degree of improvement in speech perception when used in conjunction with lip-reading (GRADE: High), and provides subjective benefits of hearing (GRADE: High). It likely allows any degree of improvement in speech perception when using the implant alone (GRADE: Moderate) and may improve quality of life (GRADE: Low). In adults with severe inner ear abnormalities, auditory brainstem implantation when compared with no intervention likely allows any degree of improvement in sound recognition (GRADE: Moderate) and in any speech perception when using the implant alone (GRADE: Moderate). It may allow any degree of improvement in speech perception when used in conjunction with lip-reading (GRADE: Low),","PeriodicalId":39160,"journal":{"name":"Ontario Health Technology Assessment Series","volume":"20 4","pages":"1-85"},"PeriodicalIF":0.0,"publicationDate":"2020-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7077937/pdf/ohtas-20-1.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"37753429","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Implantable Devices for Single-Sided Deafness and Conductive or Mixed Hearing Loss: A Health Technology Assessment.","authors":"","doi":"","DOIUrl":"","url":null,"abstract":"<p><strong>Background: </strong>Single-sided deafness refers to profound sensorineural hearing loss or non-functional hearing in one ear, with normal or near-normal hearing in the other ear. Its hallmark is the inability to localize sound and hear in noisy environments. Conductive hearing loss occurs when there is a mechanical problem with the conduction of sound vibrations. Mixed hearing loss is a combination of sensorineural and conductive hearing loss. Conductive and mixed hearing loss, which frequently affect both ears, create additional challenges in learning, employment, and quality of life. Cochlear implants and bone-conduction implants may offer objective and subjective benefits of hearing for people with these conditions who are deemed inappropriate candidates for standard hearing aids and do not meet the current indication (i.e., bilateral deafness) for publicly funded cochlear implants in Canada.</p><p><strong>Methods: </strong>We conducted a health technology assessment, which included an evaluation of clinical benefits and harms, cost-effectiveness, budget impact, and patient preferences and values related to implantable devices for single-sided deafness and conductive or mixed hearing loss. We performed a systematic literature search for systematic reviews and cost-effectiveness studies of cochlear implants and bone-conduction implants, compared to no interventions, for these conditions in adults and children. We conducted cost-utility analyses and budget impact analyses from the perspective of the Ontario Ministry of Health to examine the impact of publicly funding both types of hearing implants for the defined populations. We also interviewed 22 patients and parents of children about their experience with hearing loss and hearing implants.</p><p><strong>Results: </strong>We included 20 publications in the clinical evidence review. For adults and children with single-sided deafness, cochlear implantation when compared with no treatment improves speech perception in noise (% correct responses: 43% vs. 15%, <i>P</i> < .01; GRADE: Moderate), sound localization (localization error: 14° vs. 41°, <i>P</i> < .01; GRADE: Moderate), tinnitus (Visual Analog Scale, loudness: 3.5 vs. 8.5, <i>P</i> < .01; GRADE: Moderate), and hearing-specific quality of life (Speech Spatial and Qualities of Hearing Scale, speech: 5.8 vs. 2.6, <i>P</i> = .01; spatial: 5.7 vs. 2.3, <i>P</i> < .01; GRADE: Moderate); for children, speech and language development also improve (GRADE: Moderate). For those with single-sided deafness in whom cochlear implantation is contraindicated, bone-conduction implants when compared with no intervention provide clinically important functional gains in hearing thresholds (36-41 dB improvement in pure tone audiometry and 38-56 dB improvement in speech reception threshold, <i>P</i> < .05; GRADE: Moderate) and improve speech perception in noise (signal-to-noise ratio -2.0 vs. 0.6, <i>P</i> < .05 for active percutaneous devices; signa","PeriodicalId":39160,"journal":{"name":"Ontario Health Technology Assessment Series","volume":"20 1","pages":"1-165"},"PeriodicalIF":0.0,"publicationDate":"2020-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7080453/pdf/ohtas-20-1.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"37756649","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"5-Aminolevulinic Acid Hydrochloride (5-ALA)-Guided Surgical Resection of High-Grade Gliomas: A Health Technology Assessment.","authors":"","doi":"","DOIUrl":"","url":null,"abstract":"<p><strong>Background: </strong>High-grade gliomas are a type of malignant brain tumour. Optimal management often includes maximal surgical resection. 5-aminolevulinic acid hydrochloride (5-ALA) is an imaging agent that makes a high-grade glioma fluoresce under blue light, which can help guide the surgeon when removing the tumour. We conducted a health technology assessment of 5-ALA-guided surgical resection of high-grade gliomas, which included an evaluation of effectiveness, safety, the budget impact of publicly funding 5-ALA, and patient preferences and values.</p><p><strong>Methods: </strong>We performed a systematic literature search of the clinical evidence to retrieve systematic reviews, and selected and reported results from one review that was recent, of high quality, and relevant to our research question. We complemented the identified systematic review with a literature search to identify randomized controlled trials published after the review. We reported the risk of bias of each included study 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 systematic economic literature search to identify economic studies that compared 5-ALA-guided surgical resection of high-grade gliomas with standard surgical care or other intraoperative imaging modalities. We did not conduct a primary economic evaluation due to lack of high-quality published clinical evidence evaluating 5-ALA-guided surgical resection. From the perspective of the Ontario Ministry of Health, we analyzed the 5-year budget impact of publicly funding 5-ALA-guided surgical resection for adults with newly diagnosed, primary, high-grade gliomas for which resection is considered feasible. To contextualize the potential value of 5-ALA, we spoke with someone who had experience with high-grade glioma, 5-ALA-guided resection, and standard surgical treatment.</p><p><strong>Results: </strong>We included one systematic review reporting on a single randomized controlled trial in the clinical evidence review. 5-ALA increased the proportion of patients achieving complete tumour resection compared with standard care (relative risk of incomplete resection 0.55, 95% confidence interval 0.42-0.71; GRADE: Low). Evidence was uncertain for an effect on overall survival with 5-ALA (hazard ratio for death 0.82, 95% confidence interval 0.62-1.07; GRADE: Low), but there may be an improvement in 6-month progression-free survival (GRADE: Very low). Adverse events between groups was insufficiently reported, but appeared similar between groups for overall and neurological adverse events, with an observed increase in neurological deficits 48 hours after surgery with 5-ALA (GRADE: Very low). The economic literature search identified five studies that met our inclusion criteria because they evaluated the cost-effectiveness of 5-ALA-guided surgical resection as compared with surgery with a ","PeriodicalId":39160,"journal":{"name":"Ontario Health Technology Assessment Series","volume":"20 9","pages":"1-92"},"PeriodicalIF":0.0,"publicationDate":"2020-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7077938/pdf/ohtas-20-1.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"37756614","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Continual Long-Term Physiotherapy After Stroke: A Health Technology Assessment.","authors":"","doi":"","DOIUrl":"","url":null,"abstract":"<p><strong>Background: </strong>Stroke is a serious health issue in which an interruption in blood flow to any part of the brain damages brain cells. About 83% of people survive with substantial morbidity after their first stroke. We conducted a health technology assessment of continual long-term physiotherapy for people with a diagnosis of stroke, which included an evaluation of effectiveness, safety, cost-effectiveness, the budget impact of publicly funding continual long-term physiotherapy for people with a diagnosis of stroke, and patient preferences and values.</p><p><strong>Methods: </strong>We performed a systematic literature search of the clinical evidence. We also performed a systematic literature search of the economic evidence. We did not conduct a primary economic evaluation because there was insufficient clinical evidence. We also analyzed the budget impact of publicly funding continual long-term physiotherapy after stroke in Ontario. To contextualize the potential value of continual long-term physiotherapy after stroke, we spoke with people who had been diagnosed with stroke, as well as their caregivers.</p><p><strong>Results: </strong>We did not find any published studies that met the specific clinical inclusion criteria. We did not identify any studies that compared the cost-effectiveness of continual long-term versus short-term physiotherapy after stroke. The budget impact of publicly funding continual long-term physiotherapy after stroke in Ontario over the next 5 years ranges from $445,000 in year 1 at an uptake rate of 8% to $888,000 in year 5 at an uptake rate of 16%. The people who had been diagnosed with stroke with whom we spoke reported that they had benefitted from continual long-term physiotherapy.</p><p><strong>Conclusions: </strong>We did not identify studies that addressed the specific research question. Based on the clinical evidence review, we are unable to determine the benefits of continual long-term compared with short-term physiotherapy after stroke. The cost-effectiveness of continual long-term physiotherapy after stroke in Ontario is unknown. We estimate that publicly funding continual long-term physiotherapy after stroke in Ontario would result in additional costs of between $445,000 and $888,000 annually over the next 5 years. Patients and caregivers who we spoke with felt that patients who have experienced a stroke should be able to continue with physiotherapy.</p>","PeriodicalId":39160,"journal":{"name":"Ontario Health Technology Assessment Series","volume":"20 7","pages":"1-70"},"PeriodicalIF":0.0,"publicationDate":"2020-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7077936/pdf/ohtas-20-1.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"37756613","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Extracorporeal Membrane Oxygenation for Cardiac Indications in Adults: A Health Technology Assessment.","authors":"","doi":"","DOIUrl":"","url":null,"abstract":"<p><strong>Background: </strong>Extracorporeal membrane oxygenation (ECMO) is a rescue therapy used to stabilize patients with hemodynamic compromise such as refractory cardiogenic shock or cardiac arrest. When used for cardiac arrest, ECMO is also known as extracorporeal cardiopulmonary resuscitation (ECPR). We conducted a health technology assessment of venoarterial ECMO for adults (aged ≥ 18 years) with cardiac arrest refractory to conventional cardiopulmonary resuscitation (CPR) or with cardiogenic shock refractory to conventional medical management (i.e., drugs, mechanical support such as intra-aortic balloon pump and temporary ventricular assist devices). Our assessment included an evaluation of effectiveness, safety, cost-effectiveness, the budget impact of publicly funding ECMO for these indications, and patient preferences and values.</p><p><strong>Methods: </strong>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 Systematic Reviews (ROBIS) tool for systematic reviews and the Risk of Bias Among Nonrandomized Trials (ROBINS-I) tool for observational studies, 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 economic literature search and conducted a cost-effectiveness analysis with a lifetime horizon from a public payer perspective. We also analyzed the budget impact of publicly funding ECMO in Ontario for patients with refractory cardiogenic shock or cardiac arrest. To contextualize the potential value of ECMO for cardiac indications, we spoke with patients and caregivers with direct experience with the procedure.</p><p><strong>Results: </strong>We included one systematic review (with 13 observational studies) and two additional observational studies in the clinical review. Compared with traditional CPR for patients with refractory cardiac arrest, ECPR was associated with significantly improved 30-day survival (pooled risk ratio [RR] 1.54; 95% CI 1.03 to 2.30) (GRADE: Very Low) and significantly improved long-term survival (pooled RR 2.17; 95% CI 1.37 to 3.44) (GRADE: Low). Overall, ECPR was associated with significantly improved 30-day favourable neurological outcome in patients with refractory cardiac arrest compared with traditional CPR; pooled RR 2.02 (95% CI 1.29 to 3.16) (GRADE: Very Low). For patients with cardiogenic shock, ECMO was associated with a significant improvement in 30-day survival compared with intra-aortic balloon pump (pooled RR 2.11; 95% CI 1.23 to 3.61) (GRADE: Very Low). Compared with temporary percutaneous ventricular assist devices, ECMO was not associated with improved survival (pooled risk ratio 0.94; 95% CI 0.67 to 1.30) (GRADE: Very Low).We estimated the incremental cost-effectiveness ratio of ECPR compared with conventional CPR is $18,722 and $28,792 per life-year gained (LYG) fo","PeriodicalId":39160,"journal":{"name":"Ontario Health Technology Assessment Series","volume":"20 8","pages":"1-121"},"PeriodicalIF":0.0,"publicationDate":"2020-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7143364/pdf/ohtas-20-1.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"37828823","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Genome-Wide Sequencing for Unexplained Developmental Disabilities or Multiple Congenital Anomalies: A Health Technology Assessment.","authors":"","doi":"","DOIUrl":"","url":null,"abstract":"<p><strong>Background: </strong>People with unexplained developmental disabilities or multiple congenital anomalies might have had many biochemical, metabolic, and genetic tests for a period of years without receiving a diagnosis. A genetic diagnosis can help these people and their families better understand their condition and may help them to connect with others who have the same condition. Ontario Health (Quality), in collaboration with the Canadian Agency for Drugs and Technologies in Health (CADTH) conducted a health technology assessment about the use of genome-wide sequencing for patients with unexplained developmental disabilities or multiple congenital anomalies. Ontario Health (Quality) evaluated the effectiveness, cost-effectiveness, and budget impact of publicly funding genome-wide sequencing. We also conducted interviews with patients and examined the quantitative evidence of preferences and values literature to better understand the patient preferences and values for these tests.</p><p><strong>Methods: </strong>Ontario Health (Quality) performed a systematic literature search of the clinical evidence. We assessed the risk of bias of each included study using the Risk of Bias Assessment tool for Non-randomized Studies (RoBANS) 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 search of the quantitative evidence and undertook direct patient engagement to ascertain patient preferences for genetic testing for unexplained developmental disabilities or multiple congenital anomalies. CADTH performed a review of qualitative literature about patient perspectives and experiences, and a review of ethical issues.Ontario Health (Quality) performed an economic literature review of genome-wide sequencing in people with unexplained developmental disabilities or multiple congenital anomalies. Although we found eight published cost-effectiveness studies, none completely addressed our research question. Therefore, we conducted a primary economic evaluation using a discrete event simulation model. Owing to its high cost and early stage of clinical implementation, whole exome sequencing is primarily used for people who do not have a diagnosis from standard testing (referred to here as whole exome sequencing after standard testing; standard testing includes chromosomal microarray and targeted single-gene tests or gene panels). Therefore, in our first analysis, we evaluated the cost-effectiveness of whole exome sequencing after standard testing versus standard testing alone. In our second analysis, we explored the cost-effectiveness of whole exome and whole genome sequencing used at various times in the diagnostic pathway (e.g., first tier, second tier, after standard testing) versus standard testing. We also estimated the budget impact of publicly funding genome-wide sequencing in Ontario for the next 5 years.</p><p><strong>Result","PeriodicalId":39160,"journal":{"name":"Ontario Health Technology Assessment Series","volume":"20 11","pages":"1-178"},"PeriodicalIF":0.0,"publicationDate":"2020-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7080457/pdf/ohtas-20-1.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"37756650","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Transcatheter Aortic Valve Implantation in Patients With Severe, Symptomatic Aortic Valve Stenosis at Intermediate Surgical Risk: A Health Technology Assessment.","authors":"","doi":"","DOIUrl":"","url":null,"abstract":"<p><strong>Background: </strong>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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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.</p><p><strong>Conclusions: </strong>In people with","PeriodicalId":39160,"journal":{"name":"Ontario Health Technology Assessment Series","volume":"20 2","pages":"1-121"},"PeriodicalIF":0.0,"publicationDate":"2020-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7080451/pdf/ohtas-20-1.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"37756651","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Gene Expression Profiling Tests for Early-Stage Invasive Breast Cancer: A Health Technology Assessment.","authors":"","doi":"","DOIUrl":"","url":null,"abstract":"<p><strong>Background: </strong>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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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 ","PeriodicalId":39160,"journal":{"name":"Ontario Health Technology Assessment Series","volume":"20 10","pages":"1-234"},"PeriodicalIF":0.0,"publicationDate":"2020-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7143374/pdf/ohtas-20-1.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"37828824","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cell-Free Circulating Tumour DNA Blood Testing to Detect <i>EGFR</i> T790M Mutation in People With Advanced Non-Small Cell Lung Cancer: A Health Technology Assessment.","authors":"","doi":"","DOIUrl":"","url":null,"abstract":"<p><strong>Background: </strong>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 (<i>EGFR</i>) 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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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 <i>EGFR</i> 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 ","PeriodicalId":39160,"journal":{"name":"Ontario Health Technology Assessment Series","volume":"20 5","pages":"1-176"},"PeriodicalIF":0.0,"publicationDate":"2020-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7082730/pdf/ohtas-20-1.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"37765644","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}