Monthly dosing with ranibizumab (RBZ) is needed to achieve maximal visual gains in patients with neovascular ('wet') age-related macular degeneration (wAMD). In Sweden, dosing is performed as needed (RBZ PRN), resulting in suboptimal efficacy. Intravitreal aflibercept (IVT-AFL) every 2 months after three initial monthly doses was clinically equivalent to RBZ monthly dosing (RBZ q4) in wAMD clinical trials. We assessed the cost-effectiveness of IVT-AFL versus RBZ q4 and RBZ PRN in Sweden.
A Markov model compared IVT-AFL to RBZ q4 or RBZ PRN over 2 years. Health states were based on visual acuity in better-seeing eye; a proportion discontinued treatment monthly or upon visual acuity
OBJECTIVE: To assess the cost effectiveness of screening men aged 65 for abdominal aortic aneurysm. DESIGN: Cost effectiveness analysis based on a probabilistic, enhanced economic decision analytical model from screening to death. POPULATION AND SETTING: Hypothetical population of men aged 65 invited (or not invited) for ultrasound screening in the Danish healthcare system. DATA SOURCES: Published results from randomised trials and observational epidemiological studies retrieved from electronic bibliographic databases, and supplementary data obtained from the Danish Vascular Registry. DATA SYNTHESIS: A hybrid decision tree and Markov model was developed to simulate the short term and long term effects of screening for abdominal aortic aneurysm compared with no systematic screening on clinical and cost effectiveness outcomes. Probabilistic sensitivity analyses using Monte Carlo simulation were carried out. Results were presented in a cost effectiveness acceptability curve, an expected value of perfect information curve, and a curve showing the expected (net) number of avoided deaths from abdominal aortic aneurysm over time after the introduction of screening. The model was validated by calibrating base case health outcomes and expected activity levels against evidence from the recent Cochrane review of screening for abdominal aortic aneurysm. RESULTS: The estimated costs per quality adjusted life year (QALY) gained discounted at 3% per year over a lifetime for costs and QALYs was pound43 485 (euro54,852; $71,160). At a willingness to pay threshold of pound30,000 the probability of screening for abdominal aortic aneurysm being cost effective was less than 30%. One way sensitivity analyses showed the incremental cost effectiveness ratio varying from pound32,640 to pound66,001 per QALY. CONCLUSION: Screening for abdominal aortic aneurysm does not seem to be cost effective. Further research is needed on long term quality of life outcomes and costs.
INTRODUCTION: Strontium ranelate is a new therapy for the treatment and prevention of osteoporosis that has been shown in two phase III clinical trials (the Spinal Osteoporosis Therapeutic Intervention [SOTI] and the Treatment Of Peripheral OSteoporosis Study [TROPOS] trials) to reduce the risk of osteoporotic fractures at the vertebral, non-vertebral and hip level in postmenopausal women. The aim of this study was to estimate the potential cost-effectiveness of strontium ranelate in the treatment of osteoporosis in postmenopausal Swedish patients. METHODS: A Markov cohort model was adapted to fit patients corresponding to the patients in the SOTI and TROPOS clinical trials. The model was populated with Swedish cost and epidemiological data. In the base case, the cost-effectiveness was estimated for 69-year old women with low bone mineral density (BMD) and prevalent vertebral fractures (SOTI) and for 77-year old women with low BMD (TROPOS). The cost-effectiveness analysis had a societal perspective. RESULTS: In the base case analysis, the cost per quality-adjusted life years (QALY) gained of strontium ranelate patients compared to no treatment patients was estimated at SEK 472,586 and SEK 259,643, including costs in added life years, based on the SOTI and the TROPOS trials, respectively. Excluding cost in added life years, the cost per QALY gained was estimated at SEK 336,420 (SOTI) and SEK 165,680 (TROPOS). In subgroup analyses, in patients 74 years and older with a T-score lower than -2.4 and patients older than 80 years of age, strontium ranelate was found to be cost saving compared to no treatment. CONCLUSIONS: The results in the base case analyses and the sensitivity analyses of this study indicate that, compared to no treatment, strontium ranelate is cost-effective in the treatment of postmenopausal women with low BMD.
The use of Computer Assisted Surgery (CAS) for knee replacements is intended to improve the alignment of knee prostheses in order to reduce the number of revision operations. Is the cost effectiveness of computer assisted surgery influenced by patient volume and age?
By employing a Markov model, we analysed the cost effectiveness of computer assisted surgery versus conventional arthroplasty with respect to implant survival and operation volume in two theoretical Norwegian age cohorts. We obtained mortality and hospital cost data over a 20-year period from Norwegian registers. We presumed that the cost of an intervention would need to be below NOK 500,000 per QALY (Quality Adjusted Life Year) gained, to be considered cost effective.
The added cost of computer assisted surgery, provided this has no impact on implant survival, is NOK 1037 and NOK 1414 respectively for 60 and 75-year-olds per quality-adjusted life year at a volume of 25 prostheses per year, and NOK 128 and NOK 175 respectively at a volume of 250 prostheses per year. Sensitivity analyses showed that the 10-year implant survival in cohort 1 needs to rise from 89.8% to 90.6% at 25 prostheses per year, and from 89.8 to 89.9% at 250 prostheses per year for computer assisted surgery to be considered cost effective. In cohort 2, the required improvement is a rise from 95.1% to 95.4% at 25 prostheses per year, and from 95.10% to 95.14% at 250 prostheses per year.
The cost of using computer navigation for total knee replacements may be acceptable for 60-year-old as well as 75-year-old patients if the technique increases the implant survival rate just marginally, and the department has a high operation volume. A low volume department might not achieve cost-effectiveness unless computer navigation has a more significant impact on implant survival, thus may defer the investments until such data are available.
Escalating medical costs have made it increasingly important to carry out economic evaluations of drug therapy. In the area of acid-related diseases, much of the current interest is focused on comparisons between omeprazole and H2 receptor antagonists. After having discussed the basic methodology used in these analyses, viz. the decision-tree analysis, this paper presents an extension of this methodology, the Markov chain approach, which is more appropriate for analyses of longer time periods. Thereafter, this methodology is used to analyze the cost-effectiveness of omeprazole in intermittent versus maintenance treatment of reflux esophagitis. The cost data are from Sweden and the time period studied is one year. It is found that maintenance treatment provides 63 more healthy days per year at an extra direct cost of SEK 40 per day. From a sensitivity analysis it is concluded that the cost-effectiveness of intermittent versus maintenance treatment is mainly determined by the probability of relapse when off treatment, the severity of the symptoms in the case of a relapse and the value to the patient of a healthy day, i.e. a day free from reflux esophagitis.
To estimate the cost effectiveness of solifenacin 5?mg/day compared to oxybutynin immediate-release (IR) 15?mg/day in patients with overactive bladder, from the perspective of the Canadian healthcare (payer) system.
A Markov model was adapted to estimate the incremental cost per quality-adjusted life-year (QALY) of solifenacin and oxybutynin IR over a 1-year time horizon, based on efficacy and discontinuation data from the Canadian VECTOR (VEsicare in Comparison To Oxybutynin for oveRactive bladder patients) study. In the model, patients who discontinued treatment were offered tolterodine extended release 4?mg/day as second-line. Model robustness was tested using various sensitivity analyses. Utility values were derived from published literature; incontinence pads were included in a secondary analysis.
In the base-case analysis, total costs over 1 year were CAN$695 and CAN$550 in the solifenacin and oxybutynin IR groups, respectively. When including incontinence pad costs, there was an incremental saving of CAN$1,831 per patient with solifenacin. Solifenacin was associated with an incremental QALY gain of 0.01 over 1 year. In the base-case analysis without incontinence pads, the incremental cost-utility ratio for solifenacin was CAN$14,092. Probabilistic analyses showed no overlap in the 95% confidence intervals for total costs or QALYs with or without incontinence pads. Solifenacin was cost effective in >90% of cases, based on a willingness-to-pay threshold of CAN$50,000 per additional QALY, irrespective of whether pad costs were included in the model. The most influential variables were the discontinuation rates and the cost of incontinence pads. Limitations of the analysis relate mainly to the fact that data in the VECTOR study were collected using a direct questioning approach, which might have increased the reporting of dry mouth.
Solifenacin 5?mg/day was a cost-effective treatment compared with oxybutynin IR 15?mg/day.
NCT00431041 (of the VECTOR study, upon which the analysis in this paper was based).
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
Occurrence of a venous thromboembolism (VTE) in patients undergoing major orthopedic surgery who are not given thromboprophylactic therapy presents considerable danger to patient medical outcomes and a significant economic burden to the health care system at large. Apixaban is a direct factor Xa inhibitor that has been shown in clinical trial use to safely reduce the composite of VTE and mortality rates in patients undergoing total hip arthroplasty (THA) and total knee arthroplasty (TKA); however, the cost-effectiveness of apixaban treatment in Canadian settings has not been studied. Our study evaluated the cost-effectiveness of apixaban compared with enoxaparin as VTE preventive therapy in patients undergoing elective THA or TKA in Canada.
An economic model, including both a decision-tree component and a Markov model, was created. The decision tree considered VTE, bleeding, and mortality incidence that occurred in patients within 90 days post-surgery using data from the Apixaban Versus Enoxaparin for Thromboprophylaxis After Knee or Hip Replacement (ADVANCE) trials, which compared apixaban therapy with 30-mg twice daily and 40-mg daily enoxaparin treatment. The Markov model provided the option to simulate events that may occur over the long term, such as recurrent VTE and post-thrombotic syndrome. Outcomes during the short-term phase directly impact the risk of events occurring during the long-term phase (5 years post-surgery).
The results of our analysis indicated that apixaban is dominant (ie, more effective and less expensive) than enoxaparin in treating patients undergoing THA and TKA. There were fewer occurrences of VTEs, bleeding events, recurrent VTEs, and post-thrombotic syndrome events in the TKA population with apixaban therapy. Similar results were seen in patients undergoing THA, with the exception of bleeding events, which were more common with apixaban treatment. Savings of $180 to $270 per patient are expected with apixaban treatment compared with enoxaparin treatment, and health outcomes in general are better with apixaban use. Sensitivity analyses yielded consistent results across the THA and TKA populations.
: This is the first economic evaluation of apixaban use for VTE thromboprophylaxis in the Canadian setting, and our study results show apixaban to be a cost-effective treatment alternative to preventive treatment with enoxaparin.
This paper examines the challenge of conducting economic evaluations to support patient access to cancer therapies when the cost-effectiveness estimation is hampered by crossover trial design. To demonstrate these limitations, we present the submission to the Canadian Drug Review (CDR) of a cost-effectiveness evaluation of sunitinib versus best supportive care (BSC) for the treatment of gastrointestinal stromal tumour in patients intolerant or resistant to imatinib. The economic model generated an incremental cost-effectiveness ratio for sunitinib versus BSC of dollars 79,884/quality-adjusted life-year gained. Eight months after initial submission, CDR granted a final recommendation to fund sunitinib following the manufacturer's appeal against their first recommendation. Although cost-effectiveness is an important consideration in reimbursement decisions, there is a need for improved decision-making processes for cancer drugs, as well as a better understanding of the limitations of clinical trial design.