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The use of treatment effects derived from nonrandomized studies (NRS) in health technology assessment (HTA) is growing. NRS carry an inherently greater risk of bias than randomized controlled trials (RCTs). Although bias can be mitigated to some extent through appropriate approaches to study design and analysis, concerns around data availability and quality and the absence of randomization mean residual biases typically render the interpretation of NRS challenging. Quantitative bias analysis (QBA) methods are a range of methods that use additional, typically external, data to understand the potential impact that unmeasured confounding and other biases including selection bias and time biases can have on the results (i.e., treatment effects) from an NRS. QBA has the potential to support HTA bodies in using NRS to support decision-making by quantifying the magnitude, direction, and uncertainty of biases. However, there are a number of key aspects of the use of QBA in HTA which have received limited discussion. This paper presents recommendations for the use of QBA in HTA developed using a multi-stakeholder workshop of experts in HTA with a focus on QBA for unmeasured confounding.
This study estimates the maximum price at which mesenchymal stem cell (MSC) therapy is deemed cost-effective for septic shock patients and identifies parameters that are most important in making treatment decisions.
We developed a probabilistic Markov model according to the sepsis care trajectory to simulate costs and quality-adjusted life years (QALYs) of septic shock patients receiving either MSC therapy or usual care over their lifetime. We calculated the therapeutic headroom by multiplying the gains attributable to MSCs with willingness-to-pay (WTP) threshold and derived the maximum reimbursable price (MRP) from the expected net monetary benefit and savings attributable to MSCs. We performed scenario analyses to assess the impact of changes to assumptions on the study findings. A value of information analysis is performed to identify parameters with greatest impact on the uncertainty around the cost-effectiveness of MSC therapy.
At a WTP threshold of $50,000 per QALY, the therapeutic headroom and MRP of MSC therapy were $20,941 and $16,748, respectively; these estimates increased with the larger WTP values and the greater impact of MSCs on in-hospital mortality and hospital discharge rates. The parameters with greatest information value were MSC's impact on in-hospital mortality and the baseline septic shock in-hospital mortality.
At a common WTP of $50,000/QALY, MSC therapy is deemed to be economically attractive if its unit cost does not exceed $16,748. This ceiling price can be increased to $101,450 if the therapy significantly reduces both in-hospital mortality and increases hospital discharge rates.
An improved understanding of diagnostic and treatment practices for patients with rare primary mitochondrial disorders can support benchmarking against guidelines and establish priorities for evaluative research. We aimed to describe physician care for patients with mitochondrial diseases in Canada, including variation in care.
We conducted a cross-sectional survey of Canadian physicians involved in the diagnosis and/or ongoing care of patients with mitochondrial diseases. We used snowball sampling to identify potentially eligible participants, who were contacted by mail up to five times and invited to complete a questionnaire by mail or internet. The questionnaire addressed: personal experience in providing care for mitochondrial disorders; diagnostic and treatment practices; challenges in accessing tests or treatments; and views regarding research priorities.
We received 58 survey responses (52% response rate). Most respondents (83%) reported spending 20% or less of their clinical practice time caring for patients with mitochondrial disorders. We identified important variation in diagnostic care, although assessments frequently reported as diagnostically helpful (e.g., brain magnetic resonance imaging, MRI/MR spectroscopy) were also recommended in published guidelines. Approximately half (49%) of participants would recommend “mitochondrial cocktails” for all or most patients, but we identified variation in responses regarding specific vitamins and cofactors. A majority of physicians recommended studies on the development of effective therapies as the top research priority.
While Canadian physicians’ views about diagnostic care and disease management are aligned with published recommendations, important variations in care reflect persistent areas of uncertainty and a need for empirical evidence to support and update standard protocols.
In March 2017, CADTH released the fourth edition of the Guidelines for the Economic Evaluations of Health Technologies. As part of the update a few notable changes were made to topics such as discount rate, target population, modeling, effectiveness, analysis, and the theoretical foundations for the Guidelines. In this presentation we will describe: the implementation of the Guidelines; approaches taken to facilitate the adoption of the Guideline statements; and the tools provided to assist users and doers in using cost-effectiveness information in healthcare decision making.
Given some of the changes made to the Guidelines, CADTH identified the need to engage stakeholders early in preparation for the release of the fourth edition. Feedback on topics was sought from various stakeholders (researchers in the field, industry, patient groups, and decision makers) throughout the process. Also, suggestions for tools to support the understanding and implementation of the Guidelines were noted by CADTH. To further support use of the Guidelines, CADTH committed to undertake a number of activities including: workshops for decision makers and researchers; worked examples to illustrate the approaches; and development of tools to assist in the use of recommended methods. Updates to align drug submission guidance with the Guidelines are ongoing.
The final version of the Guidelines was greatly influenced by the stakeholder feedback received, with a focus on greater clarity. Whilst efforts to increase acceptance and adoption of the guidelines are ongoing, we present preliminary findings with respect to engagement with stakeholders and adoption of new guidance in drug submissions.
The plan to engage stakeholders continues to be effective. As such, there has been general acceptance of the changes and an interest in education and tools to assist with implementation of the Guidelines.
We performed a return-on-investment analysis comparing the investment in surgical site infection (SSI) prevention programs in a hospital setting to the savings from averted SSI cases.
A retrospective case costing study using aggregated patient data to determine the incidence and costs of SSI infection in surgical departments over time. We calculated return on investment to the hospital and conducted several sensitivity and scenario analyses.
Data were compiled for the Ottawa Hospital (TOH), a Canadian tertiary-care teaching institution.
We used aggregated records for all hospital patients who underwent surgical procedures between April 2010 and January 2015.
We estimated the potential cost savings of the hospital’s surgical quality improvement program, namely the Surgeons National Surgical Quality Improvement Program (NSQIP) and the Comprehensive Unit-based Safety Program (CUSP).
From 2010 to 2016, TOH invested C$826,882 (US$624,384) in surgical quality improvement programs targeting SSI incidence and accrued C$1,885,110 (US$1,423,460) in cumulative savings from averted SSI cases, generating a return of $2.28 (US$3.02) per dollar invested (95% confidence interval [CI], −0.67 to 7.37). The study findings are sensitive to the estimated cost to the hospital per SSI case and the rate reduction attributable to the prevention program.
The NSQIP and CUSP have produced a positive return on investment at TOH; however, the result rests on several assumptions. This positive return on investment is expected to continue if the hospital can continue to reduce SSI incidence at least 0.25% annually without new investments. Findings from this study highlight the need for continuous program evaluation of the quality improvement initiatives.
Objectives: A recent randomized, controlled trial in chronic heart failure patients showed that NT-proBNP-guided, intensive patient management (BMC) on top of multidisciplinary care reduced all-cause mortality and heart failure hospitalizations compared with multidisciplinary care (MC) or usual care (UC). We now performed a cost-utility analysis of these interventions from a payer's perspective.
Methods: Costs related to hospitalizations, ambulatory physician and nurse visits, and NT-proBNP testing for the three management strategies were acquired for both Austria (€) and Canada ($) and combined with the survival and quality of life data from the clinical trial for cost-effectiveness analysis. Data on long-term survival, costs, and quality-adjusted life-years (QALY) were extrapolated for a 20-year time horizon using a Markov model, which simulated the progression of disease through beta-blocker use, hospitalizations, and mortality.
Results: BMC was the most cost-effective strategy as it was dominant (cost-saving with improved health outcome) over both MC and UC based on both Austrian and Canadian costs. Incremental cost-effectiveness ratios for MC relative to UC were €3,746 and $5,554 per QALY gained for Austrian and Canadian costs, respectively. The probabilities for BMC being the most cost-effective strategy were 92 percent at a threshold value of Austrian €40,000 and 93 percent at a threshold value of Canadian $50,000.
Conclusions: NT-proBNP-guided, intensive HF patient management in addition to multidisciplinary care not only reduces death and hospitalization but also proves to be cost-effective.
Background: A randomized controlled trial has shown that supervised, facility-based exercise training is effective in improving glycemic control in type 2 diabetes. However, these programs are associated with additional costs. This analysis assessed the cost-effectiveness of such programs.
Methods: Analysis used data from the Diabetes Aerobic and Resistance Exercise (DARE) clinical trial which compared three different exercise programs (resistance, aerobic or a combination of both) of 6 months duration with a control group (no exercise program). Clinical outcomes at 6 months were entered for individual patients into the UKPDS economic model for type 2 diabetes adapted for the Canadian context. From this, expected life-years, quality-adjusted life-years (QALYs) and costs were estimated for all patients within the trial.
Results: The combined exercise program was the most expensive ($40,050) followed by the aerobic program ($39,250), the resistance program ($38,300) and no program ($31,075). QALYs were highest for combined (8.94), followed by aerobic (8.77), resistance (8.73) and no program (8.70). The incremental cost per QALY gained for the combined exercise program was $4,792 compared with aerobic alone, $8,570 compared with resistance alone, and $37,872 compared with no program. The combined exercise program remained cost-effective for all scenarios considered within sensitivity analysis.
Conclusions: A program providing training in both resistance and aerobic exercise was the most cost-effective of the alternatives compared. Based on previous funding decisions, exercise training for individuals with diabetes can be considered an efficient use of resources.
Objectives: The research objectives were two-fold: first, to systematically review the literature on the cost-effectiveness of home telehealth for chronic diseases, and second to develop a framework for the conduct of economic evaluation of home telehealth projects for patients with chronic diseases.
Methods: A comprehensive literature search identified twenty-two studies (n = 4,871 patients) on home telehealth for chronic diseases published between 1998 and 2008. Studies were reviewed in terms of their methodological quality and their conclusions.
Results: Home telehealth was found to be cost saving from the healthcare system and insurance provider perspectives in all but two studies, but the quality of the studies was generally low. An evaluative framework was developed which provides a basis to improve the quality of future studies to facilitate improved healthcare decision making, and an application of the framework is illustrated using data from an existing program evaluation of a home telehealth program.
Conclusions: Current evidence suggests that home telehealth has the potential to reduce costs, but its impact from a societal perspective remains uncertain until higher quality studies become available.
Objectives: The aim of this study was to demonstrate how value of information analysis can measure the upper limit on returns to future research and identify the research priorities for computer-assisted total knee replacement (CAS-TKR).
Methods: Using a previous economic analysis of CAS-TKR compared with conventional TKR, the population expected value of perfect information (EVPI) was calculated using Monte Carlo simulation to provide an estimate of the upper limit on returns to future research. The population expected value of partial perfect information (EVPPI) for both individual parameters and groups of parameters was estimated to inform specific future research priorities.
Results: The UK individual EVPI would be £21.4 if the willingness to pay for one QALY (quality-adjusted life-year) were £30,000. The population EVPPI would be £8.3 million, assuming a 10-year time horizon for CAS-TKR. In this instance, the expected value of information is positively related to willingness to pay for one QALY for the range of £0 to £50,000. Although each individual parameter had an EVPPI of £0, groups of utility parameters had positive EVPPI. Population EVPPI was £5.6 million for utility parameters, £20,000 for transition probabilities relating to CAS-TKR, and £5,000 for transition probabilities related to conventional TKR.
Conclusions: The study provides evidence on which parameters further information may be of most value. Focusing research on the utility values associated with health states relating to TKR would be of greatest value.
Background: Despite a clear call for greater input from health technology assessment (HTA) in the areas of clinical practice and policy making, there are currently very few formal training programs. The objectives of our Consortium were to (i) develop a master's level program in HTA, (ii) test its content with a group of Canadian and European students, and (iii) evaluate the Program's strengths and weaknesses.
Objectives: This study presents the results of our evaluation of the first edition of the Master's Program (2001–2003).
Methods: The evaluation relied on (i) a self-administered student questionnaire for each course (n = 142), (ii) interviews with students (n = 10), and (iii) interviews with internship supervisors (n = 5).
Results: A vast majority of students were satisfied with the course content and particularly appreciated the exercises and materials presented in an intensive format. However, they needed more systematic feedback from faculty members and recommended increasing the methodology content. The six key characteristics of the program are (i) flexible format adapted to the needs of skilled professionals, (ii) continuous interaction between HTA users and producers, (iii) international academic and professional collaboration, (iv) partnership with HTA agencies, (v) global approach to evidence-based methods and practices, and (vi) multidisciplinary approach.
Conclusions: Despite the numerous organizational barriers inherent to creating an international program and several areas for improvement in the Program itself, the Ulysses Project was successful in attaining its objectives. Because there is a growing need for human resources with special training in HTA, further efforts need to be devoted to strengthening the international research capacity in HTA.
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