Crohn's disease (CD) is a chronic inflammatory disease of the gastrointestinal tract. Symptoms include but are not limited to abdominal pain, nausea, emesis, and diarrhea. Anti-TNF-a drugs are increasingly being used in patients with CD who have inadequate response to conventional therapy. However, these medications are quite expensive. The objective of this study is to evaluate the cost-utility of two anti-TNF-a drugs (infliximab, adalimumab) for refractory CD.
A Markov model was used to estimate the costs and QALYs of three treatments (usual care, infliximab, adalimumab) over a 5 year time horizon. After initial treatment, patients achieve remission, achieve treatment response or remain in the drug refractory health state. Patients who achieve remission or treatment response are at risk of relapse each 3 month model cycle. Patients in the drug refractory health state either remain in the health state or have surgery in each cycle. Different costs and utility values were assigned to the various model health states. Model input parameters including initial response rates, relapse rates, utility values were derived from published literature.
Usual care had both the lowest expected costs ($17,017) and QALYs (2.555), while infliximab had both the highest expected costs ($54,084) and QALYs (2.721). The incremental cost per QALY moving from usual care to adalimumab and from adalimumab to infliximab was estimated to be to be $193,305 and $451,165, respectively.
Based on common willingness to pay thresholds, ant-TNF-a drugs would not be perceived as a cost effective treatment for refractory CD.
Idiopathic thrombocytopenic purpura (ITP) is a hematological disorder and can be classified as acute or chronic. The main goal of treatment for acute childhood ITP is the prevention of potentially fatal bleeding complications, the most serious of which is intracranial hemorrhage (ICH). Treatment options for acute childhood ITP include splenectomy, corticosteroids, and blood products such as intravenous immunoglobulin.
The objective was to evaluate, from a Canadian perspective, the cost-effectiveness of intravenous immunoglobulin (IVIG) compared to alternative inpatient treatments for acute childhood idiopathic thrombocytopenic purpura (ITP).
A Markov model with a lifelong time horizon was used to evaluate the costs and quality-adjusted life years (QALYs) for 5 treatments for children hospitalized for ITP: 1) no treatment; 2) IVIG; 3) Anti-D; 4) prednisone; and 5) methylprednisolone. The model predicted the probability of intracranial hemorrhage for each treatment strategy based on the time children spent with platelet counts
The Common Drug Review (CDR) was created to provide a single process to review the comparative clinical efficacy and cost effectiveness of new drugs, and then to make formulary listing recommendations to Canadian publicly funded drug benefit plans.
The objective was to conduct an in-depth analysis of Canadian Expert Drug Advisory Committee (CEDAC) recommendations to date, to explore predictors and possible explanatory factors associated with negative recommendations.
Final recommendations were identified from inception (September 2003) to 31 December 2009. Using only publicly available information, recommendations were analysed under the following categories: submission specifics, drug characteristics, clinical factors and economic factors. Descriptive analyses were conducted, followed by statistical analyses, to determine which factors independently predicted a 'do not list' (DNL) recommendation.
The database consisted of 138 unique final recommendations. The overall DNL rate was 48%. Significant differences in DNL rates were observed between therapeutic areas, ranging from 0% for HIV antivirals up to 88% for analgesic drugs. In the univariate analysis, several factors were significantly associated with a DNL recommendation, including first-in-class drugs and use of clinical scales as an outcome. In the multivariate regression, four factors were significantly predictive of a DNL recommendation: clinical uncertainty (odds ratio [OR] 14), price higher than comparators (OR 9), request for reconsideration (OR 10) and price as the only economic evidence used (OR 18). Incremental cost-effectiveness thresholds were not predictive of recommendations. The hypothesis that economic factors did not impact recommendations when clinical factors were included first was supported by the analysis.
This analysis documented an evidence-driven process that simultaneously weighted multiple factors. Clinical uncertainty and price considerations, but not economic results, had a strong impact on the recommendations. Insufficiency of clinical evidence may have resulted from the gap in evidence available at the time of product launch and the absence of demonstrated benefits to support innovative drugs.
To conduct a cost-effectiveness analysis of currently available nucleos(t)ide antiviral treatments (lamivudine, telbivudine, entecavir, and tenofovir) for chronic hepatitis B in Canada.
Markov modeling was used to project the lifetime health benefits and costs associated with the antiviral treatments. The hypothetical patient population was hepatitis B e antigen-positive chronic hepatitis B-infected patients aged 34 years. Quality-adjusted life-years were used as a measure of effectiveness. Long-term cumulative incidence of liver complications was also projected. Treatment effectiveness data were derived from the literature; meta-analysis was conducted when there was a large variance in reported effectiveness data. Costs were obtained from a cost analysis of treating chronic hepatitis B-related complications in Canada. Stochastic parameter uncertainty was examined in probabilistic sensitivity analysis by using second-order Monte Carlo simulation. Alternative modeling assumptions were assessed in scenario analysis. One-way sensitivity analysis was used to explore each parameter's impact on the uncertainty of the results.
In the base-case analysis, telbivudine was dominated by entecavir and tenofovir. Tenofovir strictly dominated lamivudine, telbivudine, and entecavir. Over the 72-year period of the model, the expected life expectancy (undiscounted) of lamivudine, telbivudine, entecavir, and tenofovir was 35.71, 36.94, 37.65, and 37.99 years, respectively. Tenofovir had the highest expected quality-adjusted life-years at 11.86 (discounted) in all comparisons. Scenario and sensitivity analyses proved the robustness of the base-case results. The projected 10-year cumulative incidence of cirrhosis and hepatocellular carcinoma was 11.40% and 3.05%, respectively, for tenofovir, which is significantly lower than that for lamivudine.
Tenofovir generated the best results compared with all other therapies under evaluation.
Potential cost and effectiveness of a nephrologist/nurse-based multifaceted intervention for stage 3 to 4 chronic kidney disease are not known. This study examines the cost-effectiveness of a chronic disease management model for chronic kidney disease.
Cost and cost-effectiveness were prospectively gathered alongside a multicenter trial. The Canadian Prevention of Renal and Cardiovascular Endpoints Trial (CanPREVENT) randomized 236 patients to receive usual care (controls) and another 238 patients to multifaceted nurse/nephrologist-supported care that targeted factors associated with development of kidney and cardiovascular disease (intervention). Cost and outcomes over 2 years were examined to determine the incremental cost-effectiveness of the intervention. Base-case analysis included disease-related costs, and sensitivity analysis included all costs.
Consideration of all costs produced statistically significant differences. A lower number of days in hospital explained most of the cost difference. For both base-case and sensitivity analyses with all costs included, the intervention group required fewer resources and had higher quality of life. The direction of the results was unchanged to inclusion of various types of costs, consideration of payer or societal perspective, changes to the discount rate, and levels of GFR.
The nephrologist/nurse-based multifaceted intervention represents good value for money because it reduces costs without reducing quality of life for patients with chronic kidney disease.
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In a practice setting where casting is considered the standard of care, the aim of this study was to assess the cost-effectiveness of wrist splints compared with routine casting in children with acceptably angulated distal radius greenstick or transverse fractures.
A cost-effectiveness analysis was conducted alongside a randomized controlled trial (RCT). One hundred children with acceptably angulated distal radius greenstick or transverse fractures received either a wrist splint or cast. Information on health care provider and patient and family resource use as well as productivity cost was collected. Resource use was costed using unit costs from local administrative data sources and expense diaries. Effectiveness was assessed at 6 weeks using the performance version of the Activities Scale for Kids (ASKp) questionnaire. Cost-effectiveness analysis related differential costs to differential ASKp scores.
Mean total cost was $877.58 in the splint group and $950.35 in the cast group, with a mean difference of $-72.76 (standard error [SE] 45.88). Mean total healthcare cost was $670.66 in the splint group and $768.22 in the cast group, with a mean difference of $-97.56 (SE 9.24). Mean (SE) ASKp was 92.8 in the splint group and 91.4 in the cast group, with a mean difference of 1.439 (SE 1.585). Therefore, splint management was more effective and cheaper. After accounting for uncertainty, the probability of splint being cost-effective compared with cast was 94 percent for a willingness-to-pay threshold value of $0 for one-unit gain in ASKp score and exceeded 82 percent for all threshold values.
In this RCT, splint management was cost-effective compared with casting in children with acceptably angulated distal radius greenstick or transverse fractures. This study challenges the existing standard of care for children with this type of fracture and provides justification on clinical and economic grounds for a change in routine practice.
Cost-effectiveness analysis of elective endovascular repair compared with open surgical repair of abdominal aortic aneurysms for patients at a high surgical risk: A 1-year patient-level analysis conducted in Ontario, Canada.
Abdominal aortic aneurysm (AAA) is a prevalent health condition affecting up to 14% of men and 6% of women. The objective of this study was to estimate the cost-effectiveness and cost-utility of elective endovascular aneurysm repair (EVAR) compared with open surgical repair (OSR) in patients at a high risk of surgical complications.
Patient-level cost and outcome data from a 1-year prospective observational study conducted at London Health Sciences Centre, London, Ontario, Canada, was used to determine the incremental cost per life-year gained and the incremental cost per quality-adjusted life year (QALY) gained of EVAR compared with OSR in patients with an AAA >5.5 cm and a high risk of surgical complications. The analysis was taken from a societal perspective and the time horizon was 1 year. To measure sampling uncertainty on costs and effects, nonparametric bootstrap techniques were applied. Uncertainty results were expressed using cost-effectiveness acceptability curves. Extrapolations of the 1-year results to a 5-year time horizon were conducted in sensitivity analyses.
Between August 11, 2003, and April 3, 2005, 192 patients at a high risk of surgical complications were enrolled: 140 received EVAR and 52 OSR. Point estimates during a 1-year period showed that EVAR dominated OSR for high-risk patients in terms of incremental cost per life-year gained and incremental cost per QALYs. However, bootstrap estimates for the two cost-effectiveness measures indicated there was a great deal of uncertainty regarding the costs and the QALYs and less uncertainty regarding life-years gained. If society was willing to pay $50,000 per life-year gained or per QALY gained, the probability of EVAR being cost-effective was found to be 0.76 and 0.55, respectively. Five-year extrapolations indicated that EVAR was cost-effective compared with OSR.
According to this 1-year observational study, EVAR may be a cost-effective strategy compared with OSR for high-risk patients. Longer-term data are needed to decrease the uncertainty associated with the results.
To compare the expected costs and outcomes of seven alternative long-term primary care strategies for the management of patients with moderate-to-severe heartburn over a 1-year period.
A decision-analytic model was developed to estimate costs and effects (weeks with heartburn symptoms and quality adjusted life years [QALYs]) for each strategy. Meta-analyses were used to synthesize acute treatment and maintenance studies and physician surveys to collect information on patient management. The impact of uncertainty on the base case results was assessed using probabilistic sensitivity analysis. Probability distributions were defined for key model parameters and techniques of Monte Carlo simulation were used to draw values from these distributions. Cost-effectiveness acceptability curves (CEACs) conditional on the monetary value decision makers are willing to pay for a symptom-free day or QALY were created for each strategy.
In the base case, no strategy was strictly dominated by any other strategy. However, two strategies (maintenance H2-receptor antagonists H2RA] and step-down proton pump inhibitor PPI]) were dominated through principles of extended dominance. The least costly and least effective strategy was intermittent H2RA, while maintenance PPI was the most costly and most effective.
This analysis showed that the best way of managing patients with heartburn depends on how much society is willing to pay to achieve health improvements. Based on the commonly quoted threshold of US 50,000 dollars per QALY, the optimal primary care strategy for managing patients with moderate-to-severe heartburn symptoms is to treat the symptoms with a PPI followed by maintenance therapy with an H2RA to prevent symptomatic recurrence.
The primary risk of abdominal aortic aneurysms (AAAs) is rupture, which is associated with a high mortality rate. Elective surgical options for AAA include open repair (OR) and endovascular aneurysm repair (EVAR). EVAR is less invasive than OR, and therefore may have less surgical risk than OR. However, the graft used for EVAR is much more expensive then the graft used for OR.
A decision model with a 10-year time horizon was used to assess the cost-effectiveness of EVAR versus OR. The primary outcome measure was quality-adjusted life-years (QALYs). The model incorporated the costs and benefits of both perioperative outcomes and postoperative outcomes. A systematic review was conducted to derive clinical outcome rates. Cost and utility model variables were based on various literature sources and data from a recent Canadian observational study. Parameter uncertainty was assessed using probabilistic sensitivity analysis.
In the base-case model, the incremental cost per QALY of EVAR was estimated to be $268,337, whereas the incremental cost per life-year was found to be $444,129. The incremental cost per QALY of EVAR remained above $295,715 under different assumptions of cohort age and model time horizon.
Based on commonly quoted willingness-to-pay thresholds, EVAR was not found to be cost-effective compared to OR.
Conventional coronary angiography (CCA) is the standard diagnostic for coronary artery disease (CAD), but multi-detector computed tomography coronary angiography (CTCA) is a non-invasive alternative.
A multi-center coverage with evidence development study was undertaken and combined with an economic model to estimate the cost-effectiveness of CTCA followed by CCA vs CCA alone. Alternative assumptions were tested in patient scenario and sensitivity analyses.
CCA was found to dominate CTCA, however, CTCA was relatively more cost-effective in females, in advancing age, in patients with lower pre-test probabilities of CAD, the higher the sensitivity of CTCA and the lower the probability of undergoing a confirmatory CCA following a positive CTCA.
RESULTS were very sensitive to alternative patient populations and modeling assumptions. Careful consideration of patient characteristics, procedures to improve the diagnostic yield of CTCA and selective use of CCA following CTCA will impact whether CTCA is cost-effective or dominates CCA.