Quality-adjusted Life Year Gain Research Articles

The comparative effectiveness and cost-effectiveness of ranibizumab for neovascular macular degeneration revisited

BackgroundTo compare a near decade of follow-up, newer control cohort data, use of both the societal and third party insurer cost perspectives, and integration of unilateral/bilateral therapy on the comparative effectiveness and cost-effectiveness of intravitreal ranibizumab therapy for neovascular, age-related macular degeneration (AMD).MethodsValue-Based Medicine®, 12-year, combined-eye model, cost-utility analysis employing MARINA and HORIZON clinical trial data. Preference-based comparative effectiveness outcomes were quantified in (1) QALY (quality-adjusted life-year) gain, and (2) percent improvement in quality-of-life, while cost-effectiveness outcomes were quantified in (3) the cost-utility ratio (CUR) and financial return-on-investment (ROI) to society.ResultsUsing MARINA and HORIZON trial data and a meta-analysis control cohort after 24 months, ranibizumab therapy conferred a combined-eye patient value (quality-of-life) gain of 16.3%, versus 10.4% found in 2006. The two-year direct ophthalmic medical cost for ranibizumab therapy was $46,450, a 33.8% real dollar decrease from 2006. The societal cost perspective CUR was −$242,920/QALY, indicating a $282,517 financial return-on-investment (ROI), or 12.3%/year to society for direct ophthalmic medical costs expended. The 3rd party insurer CUR ranged from $21,199/QALY utilizing all direct, medical costs, to $69,591/QALY using direct ophthalmic medical costs.ConclusionsRanibizumab therapy for neovascular AMD in 2015, considering treatment of both eyes, conferred greater patient value gain (comparative effectiveness) and improved cost-effectiveness than in 2006, as well as a large monetary return-on-investment to the Gross Domestic Product and nation’s wealth. The model herein integrates important novel features for neovascular age-related macular degeneration, vitreoretinal cost effectiveness analyses, including: (1) treatment of both eyes, (2) a long-term, untreated control cohort, and (3) the use of societal costs.

Unloading knee brace is a cost-effective method to bridge and delay surgery in unicompartmental knee arthritis

BackgroundUnloading knee braces can provide good short-term pain relief for some patients with unicompartmental osteoarthritis (UOA). Their cost is relatively small compared with surgical interventions. However, no previous studies have...

Cost-effectiveness of SQ® HDM SLIT-tablet in addition to pharmacotherapy for the treatment of house dust mite allergic rhinitis in Germany.

BackgroundAllergic rhinitis is a global health problem that burdens society due to associated health care costs and its impact on health. Standardized quality (SQ®) house dust mite (HDM) sublingual immunotherapy (SLIT)-tablet is a sublingually administered allergy immunotherapy tablet for patients with persistent moderate to severe HDM allergic rhinitis despite use of allergy pharmacotherapy.ObjectiveTo assess the cost-effectiveness of SQ HDM SLIT-tablet in Germany for patients suffering from HDM allergic rhinitis.MethodsA pharmacoeconomic analysis, based on data collected in a double-blinded, phase III randomized placebo-controlled trial (n=992), was undertaken to compare SQ HDM SLIT-tablet in addition to allergy pharmacotherapy to placebo plus allergy pharmacotherapy. Quality-adjusted life year (QALY) scores and health care resource use data recorded in the trial were applied to each treatment group and extrapolated over a nine-year time horizon. A series of scenarios were used to investigate the impact of changes on long-term patient health for both treatment groups, which was measured by annual changes in QALY scores. Deterministic and probabilistic sensitivity analyses were also performed.ResultsIn the base case analysis, compared with allergy pharmacotherapy, SQ HDM SLIT-tablet led to a QALY gain of 0.31 at an incremental cost of €2,276 over the nine-year time horizon, equating to an incremental cost-effectiveness ratio of €7,519. The treatment was cost-effective for all scenarios analyzed; however, results were sensitive to changes in individual parameter values during the deterministic sensitivity analysis.ConclusionSQ HDM SLIT-tablet in addition to pharmacotherapy is cost-effective compared with allergy pharmacotherapy plus placebo for the treatment of persistent moderate to severe HDM allergic rhinitis that is not well controlled by allergy pharmacotherapy.

Cost-Effectiveness of Cardiovascular Screening in Patients With Rheumatoid Arthritis.

Early detection and preemptive treatment of patients at risk is of great importance in reducing the excess risk of cardiovascular (CV) disease in rheumatoid arthritis (RA). However, it is unclear how much screening is cost-effective in RA. The objective is to assess whether CV screening in RA proves to be cost-effective from a medical perspective, using different scenarios based on different guidelines. A Markov chain model was used with a time horizon of 10 years. Parameter values were mainly obtained from literature and from RA patients screened for CV diseases at the Radboud University Medical Centre, Nijmegen, The Netherlands. The primary outcome was incremental cost-effectiveness expressed as costs per quality-adjusted life year (QALY) gained. Probabilistic sensitivity analysis was performed and described in willingness-to-pay curves; several scenarios were built. In the base case scenario, in 82% of the simulations, screening proved to be dominant compared to no screening. The mean QALY gain was 0.09 (95% percentile -0.07, 0.27), and the mean cost savings were €-1,057 (95% percentile -€2,825, €333). Different scenarios showed small differences in cost-effectiveness; the probability that screening is dominant remained high with the lowest probability being 50% for a very conservative scenario. Screening for CV events in RA patients was estimated to be cost-effective with high chances of being less expensive and more effective. These results support endorsement of screening for CV risk in patients with RA.

Cost-effectiveness of switching to insulin degludec from other basal insulins: evidence from Swedish real-world data

Objectives: Health economic analysis from a healthcare and societal point of view was conducted to assess the cost-effectiveness of insulin degludec (IDeg) after switching from other basal insulins in people with type 1 diabetes.Material and methods: This was a prospective, open-label, single arm, observational follow-up from August 2013 to October 2015 of 476 consecutive patients at Danderyd Hospital (Stockholm, Sweden) who switched to IDeg from other basal insulins (99% basal insulin analogs). The IMS CORE Diabetes Model (CDM) was used to predict the cost-effectiveness of life-long treatment with IDeg vs. other basal insulins, based on a Swedish setting.Results: Mean (SD) duration of follow-up was 21.7 (6.0) weeks. Mean HbA1c decreased by 2.7 mmol/mol, mean basal insulin dose decreased by 13.1% (p < .0001), and mean bolus insulin dose decreased by 7.5% (p < .0001) after switching. Frequencies of non-severe daytime hypoglycemia and non-severe nocturnal hypoglycemia decreased by 12% (p = .0127) and 53% (p < .0001) respectively and severe hypoglycemia was reduced by 62% (p = .0225). The CDM predicted a gain in life expectancy of 0.33 years, a discounted gain in quality-adjusted life-years (QALYs) of 0.54, and lower estimated direct lifetime healthcare costs of SEK 22,757 for patients switching to IDeg. The incremental cost-effectiveness ratio (ICER) showed IDeg as dominant (i.e. higher effectiveness with a lower cost). Sensitivity analyses confirmed the results.Conclusion: Based on this prospective, real-world, follow-up and using the CDM, it was estimated that switching to IDeg from other basal insulins translated into QALY gains including improved life expectancy and health-related quality of life, as well as dominant ICER, meaning cost-savings for the healthcare system. However, the study is limited by its observational design. Extrapolation into the future is only estimated since the actual treatment effect cannot be projected with certainty.

Cost-effectiveness of structured group psychoeducation versus unstructured group support for bipolar disorder: Results from a multi-centre pragmatic randomised controlled trial

BackgroundBipolar disorder (BD) costs the English economy an estimated £5.2billion/year, largely through incomplete recovery. This analysis estimated the cost-effectiveness of group psychoeducation (PEd), versus group peer support (PS), for treating BD. MethodsA 96-week pragmatic randomised controlled trial (RCT), conducted in NHS primary care. The primary analysis compared PEd with PS, using multiple imputed datasets for missing values. An economic model was used to compare PEd with treatment as usual (TAU). The perspective was Health and Personal Social Services. ResultsParticipants receiving PEd (n=153) used more (costly) health-related resources than PS (n=151) (net cost per person £1098 (95% CI, £252-£1943)), with a quality-adjusted life year (QALY) gain of 0.023 (95% CI, 0.001-0.056). The cost per QALY gained was £47,739. PEd may be cost-effective (versus PS) if decision makers are willing to pay at least £37,500 per QALY gained. PEd costs £10,765 more than PS to avoid one relapse. The economic model indicates that PEd may be cost-effective versus TAU if it reduces the probability of relapse (by 15%) or reduces the probability of and increases time to relapse (by 10%). LimitationsParticipants were generally inconsistent in attending treatment sessions and low numbers had complete cost/QALY data. Factors contributing to pervasive uncertainty of the results are discussed. ConclusionsThis is the first economic evaluation of PEd versus PS in a pragmatic trial. PEd is associated with a modest improvement in health status and higher costs than PS. There is a high level of uncertainty in the data and results.

External Validation of Health Economic Decision Models for Chronic Obstructive Pulmonary Disease (COPD): Report of the Third COPD Modeling Meeting.

To validate outcomes of presently available chronic obstructive pulmonary disease (COPD) cost-effectiveness models against results of two large COPD trials-the 3-year TOwards a Revolution in COPD Health (TORCH) trial and the 4-year Understanding Potential Long-term Impacts on Function with Tiotropium (UPLIFT) trial. Participating COPD modeling groups simulated the outcomes for the placebo-treated groups of the TORCH and UPLIFT trials using baseline characteristics of the trial populations as input. Groups then simulated treatment effectiveness by using relative reductions in annual decline in lung function and exacerbation frequency observed in the most intensively treated group compared with placebo as input for the models. Main outcomes were (change in) total/severe exacerbations and mortality. Furthermore, the absolute differences in total exacerbations and quality-adjusted life-years (QALYs) were used to approximate the cost per exacerbation avoided and the cost per QALY gained. Of the six participating models, three models reported higher total exacerbation rates than observed in the TORCH trial (1.13/patient-year) (models: 1.22-1.48). Four models reported higher rates than observed in the UPLIFT trial (0.85/patient-year) (models: 1.13-1.52). Two models reported higher mortality rates than in the TORCH trial (15.2%) (models: 20.0% and 30.6%) and the UPLIFT trial (16.3%) (models: 24.8% and 36.0%), whereas one model reported lower rates (9.8% and 12.1%, respectively). Simulation of treatment effectiveness showed that the absolute reduction in total exacerbations, the gain in QALYs, and the cost-effectiveness ratios did not differ from the trials, except for one model. Although most of the participating COPD cost-effectiveness models reported higher total exacerbation rates than observed in the trials, estimates of the absolute treatment effect and cost-effectiveness ratios do not seem different from the trials in most models.

Cost-Effectiveness Analysis of Second-Line Chemotherapy Agents for Advanced Gastric Cancer.

Gastric cancer is the fifth most common malignancy and second leading cause of cancer-related mortality. Chemotherapy options for patients who fail first-line treatment are limited. Thus the objective of this study was to assess the cost-effectiveness of second-line treatment options for patients with advanced or metastatic gastric cancer. Cost-effectiveness analysis using a Markov model to compare the cost-effectiveness of six possible second-line treatment options for patients with advanced gastric cancer who have failed previous chemotherapy: irinotecan, docetaxel, paclitaxel, ramucirumab, paclitaxel plus ramucirumab, and palliative care. The model was performed from a third-party payer's perspective to compare lifetime costs and health benefits associated with studied second-line therapies. Costs included only relevant direct medical costs. The model assumed chemotherapy cycle lengths of 30 days and a maximum number of 24 cycles. Systematic review of literature was performed to identify clinical data sources and utility and cost data. Quality-adjusted life years (QALYs) and incremental cost-effectiveness ratios (ICERs) were calculated. The primary outcome measure for this analysis was the ICER between different therapies, where the incremental cost was divided by the number of QALYs saved. The ICER was compared with a willingness-to-pay (WTP) threshold that was set at $50,000/QALY gained, and an exploratory analysis using $160,000/QALY gained was also used. The model's robustness was tested by using 1-way sensitivity analyses and a 10,000 Monte Carlo simulation probabilistic sensitivity analysis (PSA). Irinotecan had the lowest lifetime cost and was associated with a QALY gain of 0.35 year. Docetaxel, ramucirumab alone, and palliative care were dominated strategies. Paclitaxel and the combination of paclitaxel plus ramucirumab led to higher QALYs gained, at an incremental cost of $86,815 and $1,056,125 per QALY gained, respectively. Based on our prespecified WTP threshold, our base case analysis demonstrated that irinotecan alone is the most cost-effective regimen, and both paclitaxel alone and the combination of paclitaxel and ramucirumab were not cost-effective (ICER more than $50,000). Both 1-way sensitivity analyses and PSA demonstrated the model's robustness. PSA illustrated that paclitaxel plus ramucirumab was extremely unlikely to be cost-effective at a WTP threshold less than $400,000/QALY gained. Irinotecan alone appears to be the most cost-effective second-line regimen for patients with gastric cancer. Paclitaxel may be cost-effective if the WTP threshold was set at $160,000/QALY gained.

Dalteparin versus vitamin K antagonists for the prevention of recurrent venous thromboembolism in patients with cancer and renal impairment: a Canadian pharmacoeconomic analysis.

BackgroundPatients with cancer are at increased risk of venous thromboembolism (VTE) and the risk is further elevated after a primary VTE. To reduce the risk of recurrent events, extended prophylaxis with vitamin K antagonists (VKA) is available for use. However, in a large randomized trial (Comparison of Low-Molecular-Weight Heparin versus Oral Anticoagulant Therapy for the Prevention of Recurrent Venous Thromboembolism in Patients with Cancer [CLOT]; Lee et al), extended duration dalteparin reduced the relative risk of recurrent VTE by 52% compared to VKA (p=0.002). A recent subgroup analysis of patients with moderate-to-severe renal impairment also revealed lower absolute VTE rates with dalteparin (3% vs. 17%; p=0.011). To measure the economic value of dalteparin as an alternative to VKA, a patient-level cost utility analysis was conducted from a Canadian perspective.MethodsResource use data captured during the CLOT trial were extracted and linked to 2015 Canadian unit cost estimates. Health state utilities were then measured using the Time-Trade-Off technique in 24 randomly selected members of the general Canadian public to estimate the gains in quality-adjusted life years (QALYs).ResultsFor the entire CLOT trial population (n=676), the dalteparin group had significantly higher mean costs compared to the VKA group ($Can5,771 vs. $Can2,569; p<0.001). However, the utility assessment revealed that 21 of 24 respondents (88%) selected dalteparin over VKA, with an associated gain of 0.14 (95% confidence interval [CI]: 0.10–0.18) QALYs. When the incremental cost of dalteparin was combined with the QALY gain, dalteparin had a cost of $Can23,100 (95% CI: $Can19,200–$Can25,800) per QALY gained. The analysis in patients with renal impairment suggested even better economic value with the cost per QALY gained being <$14,000.ConclusionExtended duration dalteparin is a cost-effective alternative to VKA for the prevention of recurrent VTE in patients with cancer, especially in those with renal impairment.

Cost-effectiveness of eculizumab treatment after kidney transplantation in patients with atypical haemolytic uraemic syndrome.

Kidney transplantation in patients with atypical haemolytic uraemic syndrome (aHUS) is frequently complicated by recurrence of aHUS, often resulting in graft loss. Eculizumab prophylaxis prevents recurrence, improving graft survival. An alternative treatment strategy has been proposed where eculizumab is administered upon recurrence. We combined available evidence and performed a cost-effectiveness analysis of these competing strategies. A cost-effectiveness analysis using a decision analytical approach with Markov chain analyses was used to compare alternatives for aHUS patients with end-stage renal disease (ESRD): (i) dialysis treatment, (ii) kidney transplantation, (iii) kidney transplantation with eculizumab therapy upon recurrence of aHUS, (iv) kidney transplantation with eculizumab induction consisting of 12 months of prophylaxis and (v) kidney transplantation with lifelong eculizumab prophylaxis. We assumed that all patients received a graft from a living donor and that recurrence probability was 28.4% within the first year of transplantation. At 8.34 quality-adjusted life years (QALYs) gained and a cost of €402 412, kidney transplantation without eculizumab was the least costly alternative. By comparison, dialysis was more costly and resulted in fewer QALYs gained. Eculizumab upon recurrence resulted in 9.55 QALYs gained at a cost of €425 097. The incremental cost-effectiveness ratio (ICER) was €18 748 per QALY. Both eculizumab induction and lifelong eculizumab were inferior to eculizumab upon recurrence, as both resulted in fewer QALYs gained and higher costs. Kidney transplantation is more cost effective than dialysis to treat ESRD due to aHUS. Adding eculizumab treatment results in a substantial gain in QALYs. When compared with eculizumab upon recurrence, neither eculizumab induction nor lifelong eculizumab prophylaxis resulted in more QALYs, but did yield far higher costs. Therefore, eculizumab upon recurrence of aHUS is more acceptable.

PP025 Thrombopoietin Receptor Agonist For Treatment Of Adults With Chronic Immune Thrombocytopenic Purpura

INTRODUCTION:This study aims to report the clinical effectiveness and cost-effectiveness of Thrombopoietin (TPO) receptor agonist for the treatment of adults with spontaneous Immune Thrombocytopenic Purpura (ITP) in Taiwan.METHODS:In the clinical effectiveness evaluation section, particularly for the TPO receptor agonist, we searched PubMed, EMBASE, and the Cochrane Central Register of Controlled Trials to identify all randomized trials in chronic ITP. In the economic evaluation section, we performed a long-term cost-effectiveness analysis using a Markov model to evaluate the value of TPO receptor agonist to achieve durable platelet response for chronic ITP patients.RESULTS:Our findings revealed that the National Health Insurance (NHI) in Taiwan covers TPO receptor agonists romiplostim and eltrombopag, which have also been recommended by the Pharmaceutical Benefits Advisory Committee (PBAC) of Australia and the National Institute for Health and Care Excellence (NICE) in the United Kingdom. In addition, a systematic review and meta-analysis combining six trials were included to assess the current evidence on the role of TPO receptor agonist in chronic ITP. The primary outcome of randomized controlled trials (RCTs) showed an improving trend in significant bleeding events; however there was not any significant difference between the TPO receptor agonists group and the control group (placebo). The gain in life years and quality-adjusted life-years (QALYs) from introducing long-term use of TPO receptor agonists over current clinical practice were 1.52 years and 1.44 QALYs, respectively. Most of the sensitivity analysis results show that the ICER values were greater than 3GDP per capita in Taiwan.CONCLUSIONS:Compared to placebo, and despite a significantly increased platelet response, there was no evidence to demonstrate that TPO receptor agonists did improve significant bleeding events in chronic ITP. The effect on overall survival awaits further analysis. Although long-term studies are lacking, current data demonstrated that adverse effects of TPO receptor agonists were similar to that of placebo.

Cost-effectiveness and Budget Effect Analysis of a Population-Based Skin Cancer Screening

Several epidemiological studies show an alarming global increase in incidence of melanoma and nonmelanoma skin cancer. To examine the cost-effectiveness of 2 population-based skin cancer screening methods and to assess their budget effect and the influence on skin cancer epidemiological findings. A Markov model with a latent period of 20 years and a time horizon of 50 years was used to analyze the cost-effectiveness (societal perspective) and budget effect (public health care payer perspective) of 2 population-based skin cancer screening programs in Belgium compared with the absence of a screening program. A health economic analysis was based on a clinical trial performed in 2014 in Belgium. In the economic model, the total Belgian population 18 years or older was assumed to have been invited for the screening program. The influence of the screening program on skin cancer epidemiological findings and the cost per quality-adjusted life-year (QALY) gained, as well as the budget effect, expressed as the net costs for the health care payer over 50 years. All participants (1668 total-body skin examination [TBSE] and 248 lesion-directed screening [LDS]) were screened by a team of 6 dermatologists from March 14 to 18, 2014, for TSBE and April 22 and 25 to 27, 2014, for LDS. Both screening strategies produced a gain in QALYs, resulting in incremental cost-effectiveness ratios of €33 072 (US $35 475) per QALY in men and €18 687 (US $20 044) per QALY in women for TBSE and €34 836 (US $37 365) per QALY in men and €19 470 (US $20 884) per QALY in women for LDS. With a 1-time screening, a 4.0% decrease in the incidence rates of stage III and IV melanoma was predicted at the population level relative to the comparator. The budget effect analysis demonstrated that during 20 years, a 1-time screening would incur a net cost for the health care payer of almost €36 million (US $38.6 million) for TBSE or just over €6 million (US $6.4 million) for LDS (€4.1 [US $4.40] or €0.7 [US $0.80], respectively, per adult). These results can be interpreted as cost-effective at a willingness-to-pay threshold in Belgium of €35 000 (US $37 541) per QALY gained. Based on these findings, a 1-time TBSE in the general adult population 18 years or older is the most cost-effective strategy and is predicted to result in a reduction of skin cancer mortality over 20 years and 50 years. The cost-effectiveness for skin cancer screening is higher in women than in men.

Cost-effectiveness of Prophylactic Moisturization for Atopic Dermatitis

Emerging evidence suggests that the use of moisturizers on newborns and infants (ie, from birth to 6 months of age) is potentially helpful in preventing the development of atopic dermatitis. To investigate the cost-effectiveness of using a daily moisturizer as prevention against atopic dermatitis among high-risk newborns. In a cost-effectiveness analysis, the average cost of total-body moisturization using 7 common moisturizers from birth to 6 months of age was determined for male and female infants. We assumed the same unit of weight per moisturizer used for a given body surface area. Based on previously reported data (relative risk reduction of 50%), the incremental gain in quality-adjusted life-years (QALYs) was determined using a 6-month time window. The cost-effectiveness of each moisturizer was determined by assuming equal efficacy. A sensitivity analysis was conducted by varying the relative risk from 0.28 to 0.90. Use of prophylactic moisturizing compounds. The primary outcomes were the incremental cost-effectiveness values ($/QALY) for each moisturizer in preventing atopic dermatitis during a 6-month time window. The calculated amount of daily all-body moisturizer needed at birth was 3.6 g (0.12 oz) per application, which increased to 6.6 g (0.22 oz) at 6 months of age. Of the 7 products evaluated, the average price was $1.07/oz (range, $0.13/oz-$2.96/oz). For a 6-month time window, the average incremental QALY benefit was 0.021. The sensitivity analysis showed that the incremental gain of QALY ranged from 0.0041 to 0.030. Petrolatum was the most cost-effective ($353/QALY [95% CI, $244-$1769/QALY) moisturizer in the cohort. Even assuming the lowest incremental QALYs for the most expensive moisturizer, the intervention was still less than $45 000/QALY. Overall, atopic dermatitis represents a major health expenditure and has been associated with multiple comorbidities. Daily moisturization may represent a cost-effective, preventative strategy to reduce the burden of atopic dermatitis.

Impact of old age on patient-report outcomes and cost utility for anterior cervical discectomy and fusion surgery for degenerative spine disease.

With growing older population and increasing rates of cervical spinal surgery, it is vital to understand the value of cervical surgery in this population. We set forth to determine the cost utility following anterior cervical decompression and fusion (ACDF) for degenerative disease in older patients. Patients undergoing ACDF for degenerative diseases were enrolled into prospective longitudinal registry. Patient-reported outcomes (PROs) were recorded at baseline, 1-year, and 2-year postoperatively. Two-year medical resource utilization, missed work, and health-state values [quality-adjusted life years (QALYs)] were assessed to compute cost per QALY gained. Patients were dichotomized based on age:<65years (younger) and≥65years (older) to compare the cost utility in these age groups. Total 218 (87%) younger patients and 33 (13%) older patients who underwent ACDF were analyzed. Both the groups demonstrated a significant improvement in PROs 2-year following surgery. The older patients had a lower mean cumulative gain in QALYs compared to younger patients at 1 year (0.141 vs. 0.28, P=0.05) and 2 years (0.211 vs. 0.424, P=0.04). There was no significant difference in the mean total 2-year cost between older [$21,041 (95% CI $18,466-$23,616)] and younger [$22,669 (95% CI $$21,259-$24,079)] patients (P=0.27). Two-year cost per QALY gained in older vs. younger patients was ($99,720/QALYs gained vs. ($53,464/QALYs gained, P=0.68). ACDF surgery provided a significant gain in health-state utility in older patients with degenerative cervical pathology, with a mean cumulative 2-year cost per QALY gained of $99,720/QALY. While older patients have a slightly higher cost utility compared to their younger counterparts, surgery in the older cohort does provide a significant improvement in pain, disability, and quality-of-life outcomes.

Cost-Effectiveness of Saxagliptin versus Acarbose as Second-Line Therapy in Type 2 Diabetes in China.

ObjectiveThis study assessed the long-term cost-effectiveness of saxagliptin+metformin (SAXA+MET) versus acarbose+metformin (ACAR+MET) in Chinese patients with type 2 diabetes mellitus (T2DM) inadequately controlled on MET alone.MethodsSystematic literature reviews were performed to identify studies directly comparing SAXA+MET versus ACAR+MET, and to obtain diabetes-related events costs which were modified by hospital surveys. A Cardiff Diabetes Model was used to estimate the long-term economic and health treatment consequences in patients with T2DM. Costs (2014 Chinese yuan) were calculated from the payer’s perspective and estimated over a patient’s lifetime.ResultsSAXA+MET predicted lower incidences of most cardiovascular events, hypoglycemia events and fatal events, and decreased total costs compared with ACAR+MET. For an individual patient, the quality-adjusted life-years (QALYs) gained with SAXA+MET was 0.48 more than ACAR+MET at a cost saving of ¥18,736, which resulted in a cost saving of ¥38,640 per QALY gained for SAXA+MET versus ACAR+MET. Results were robust across various univariate and probabilistic sensitivity analyses.ConclusionSAXA+MET is a cost-effective treatment alternative compared with ACAR+MET for patients with T2DM in China, with a little QALYs gain and lower costs. SAXA is an effective, well-tolerated drug with a low incidence of adverse events and ease of administration; it is anticipated to be an effective second-line therapy for T2DM treatment.

Estimating Population Health Benefits Associated with Specialty and Traditional Drugs in the Year Following Product Approval.

Compared to traditional drugs, specialty drugs tend to be indicated for lower prevalence diseases. Our objective was to compare the potential population health benefits associated with specialty and traditional drugs in the year following product approval. First, we created a dataset of estimates of incremental quality-adjusted life-year (QALY) gains and incremental life-year (LY) gains for US FDA-approved drugs (1999-2011) compared to standard of care at the time of approval identified from a literature search. Second, we categorized each drug as specialty or traditional. Third, for each drug we identified estimates of US disease prevalence for each pertinent indication. Fourth, in order to conservatively estimate the potential population health gains associated with each new drug in the year following its approval we multiplied the health gain estimate by 10% of the identified prevalence. Fifth, we used Mann-Whitney U tests to compare the population health gains for specialty and traditional drugs. We identified QALY gain estimates for 101 drugs, including 56 specialty drugs, and LY gain estimates for 50 drugs, including 34 specialty drugs. The median estimated population QALY gain in the year following approval for specialty drugs was 4200 (IQR=27,000) and for traditional drugs was 694 (IQR=24,400) (p=0.245). The median estimated population LY gain in the year following approval for specialty drugs was 7250 (IQR=39,200) and for traditional drugs was 2500 (IQR=58,200) (p=0.752). Despite often being indicated for diseases of lower prevalence, we found a trend towards specialty drugs offering larger potential population health gains than traditional drugs, particularly when measured in terms of QALYs.

PND42 - Cost-Effectiveness Of Levofloxacin Inhalation Solution Vs. Aztreonam Inhalation Solution In Cystic Fibrosis Patients In Belgium

To conduct an economic evaluation of levofloxacin inhalation solution (LIS) compared to aztreonam lysine inhalation solution (AZLI) in Belgium for the treatment of chronic Pseudomonas aeruginosa lung infection in adult cystic fibrosis (CF) patients. A 24-week cycle Markov model was developed to estimate the expected costs and quality-adjusted life year (QALY) gains over three- and five-years. The model simulated the disease progression of CF patients as measured by the decrease in forced expiratory volume in 1 second (FEV1) percent predicted as well as lung transplantation and death. The base case analysis was conducted from the Belgian health care perspective at 3 years and sensitivity analysis at 5-years. In the base case LIS was dominant vs. AZLI over two studied time horizons from the health care perspective. LIS was associated with a gain in QALYs of 0.117 and 0.224 over three- and five-year time horizons, respectively, and was cost saving (-€515 and -€952, over three- and five-year time horizons, respectively) compared to AZLI. In addition to the base case analysis, two scenarios were conducted to assess the impact of parameter uncertainty on the model results. In all scenarios, conclusions are similar to the base case analysis: LIS was associated with a QALY gain and was cost-saving compared to AZLI. In general, the sensitivity analyses showed that the incremental cost-effectiveness ratio (ICER) was mainly sensitive to the costs of CF management and the mortality. The probability of ICER being less than €50,000 was 89.5%. This model simulated the disease progression of CF patients as measured by the decrease in FEV1 percent predicted. The base case results showed that the cost-effectiveness analysis of LIS is a dominant treatment option vs. AZLI over three- and five-year time horizons from the Belgian healthcare perspective.

English

The transition to university marks a point where young people may be open to changing health behaviours such as smoking, exercise, diet and alcohol intake. This study aimed to estimate the cost-effectiveness of an updated online health behaviour intervention for new university students in the UK – “U@Uni2”, compared with both a control (measurement only) scenario and with the original intervention (“U@Uni1”). The economic analysis, based on a randomized controlled trial, comprised a detailed costing analysis, a within-trial cost-effectiveness analysis and long-term economic modelling. Cost-effectiveness of the U@Uni2 trial was estimated using 6-month data on costs and health-related quality of life. An individual patient simulation model was adapted for long-term economic analysis of U@Uni2. Probabilistic sensitivity analysis and value of information analysis accounted for uncertainty in model inputs and identified key parameters. The U@Uni2 intervention costs £45.97 per person for full implementation, £10.43 per person for roll-out in a different institution and £3.03 per person for roll-out over five years. The U@Uni2 trial was not cost-effective because marginally fewer quality-adjusted life years (QALYs) were obtained in the intervention arm than the control. However, modelled over a lifetime, U@Uni2 is estimated to produce more QALYs than control but fewer than U@Uni1, primarily due to the effect of the interventions on smoking. Roll-out of U@Uni2 is highly likely to be more cost-effective than doing nothing (ICER = £536 per QALY, 86% probability cost-effective). Decision uncertainty occurs primarily around the effectiveness of the U@Uni2 intervention and is worth up to £3.24 m. The U@Uni2 intervention is highly likely to be cost-effective to roll-out when compared with doing nothing. The results suggest that preventing uptake of smoking is the key driver of QALY gain and should be the primary target of such interventions. Key words: Alcohol, diet, exercise, smoking, health behavior, cost-effectiveness, economic evaluation, students, university.

Cost-effectiveness Analysis of Treatment Sequence Initiating With Etanercept Compared With Leflunomide in Rheumatoid Arthritis: Impact of Reduced Etanercept Cost With Patent Expiration in South Korea

PurposeIn south Korea, the price of biologics has been decreasing owing to patent expiration and the availability of biosimilars. This study evaluated the cost-effectiveness of a treatment strategy initiated with etanercept (ETN) compared with leflunomide (LFN) after a 30% reduction in the medication cost of ETN in patients with active rheumatoid arthritis (RA) with an inadequate response to methotrexate (MTX-IR). MethodsA cohort-based Markov model was designed to evaluate the lifetime cost-effectiveness of treatment sequence initiated with ETN (A) compared with 2 sequences initiated with LFN: LFN-ETN sequence (B) and LFN sequence (C). Patients transited through the treatment sequences, which consisted of sequential biologics and palliative therapy, based on American College of Rheumatology (ACR) responses and the probability of discontinuation. A systematic literature review and a network meta-analysis were conducted to estimate ACR responses to ETN and LFN. Utility was estimated by mapping an equation for converting the Health Assessment Questionnaire-Disability Index score to utility weight. The costs comprised medications, outpatient visits, administration, dispensing, monitoring, palliative therapy, and treatment for adverse events. A subanalysis was conducted to identify the influence of the ETN price reduction compared with the unreduced price, and sensitivity analyses explored the uncertainty of model parameters and assumptions. FindingsThe ETN sequence (A) was associated with higher costs and a gain in quality-adjusted life years (QALYs) compared with both sequences initiated with LFN (B, C) throughout the lifetime of patients with RA and MTX-IR. The incremental cost-effectiveness ratio (ICER) for strategy A versus B was ₩13,965,825 (US$1726) per QALY and that for strategy A versus C was ₩9,587,983 (US$8050) per QALY. The results indicated that strategy A was cost-effective based on the commonly cited ICER threshold of ₩20,000,000 (US$16,793) per QALY in South Korea. The robustness of the base-case analysis was confirmed using sensitivity analyses. When the unreduced medication cost of ETN was applied in a subanalysis, the ICER for strategy A versus B was ₩20,909,572 (US$17,556) per QALY and that for strategy A versus C was ₩22,334,713 (US$18,753) per QALY. ImplicationsThis study indicated that a treatment strategy initiated with ETN was more cost-effective in patients with active RA and MTX-IR than 2 sequences initiated with LFN. The results also indicate that the reduced price of ETN affected the cost-effectiveness associated with its earlier use.

Economic Evaluation of Fluticasone Propionate/Formoterol (Flutiform®) vs. Fluticasone/Salmeterol and Budesonide/Formoterol in Spain

The aim of this economic evaluation was to estimate the cost-effectiveness of fluticasone propionate/formoterol (FP/FORM; Flutiform®) and compare it to those of fluticasone/salmeterol (FS) and budesonide/formoterol (BF) when used in the treatment of adult patients with moderate-to-severe asthma. A Markov model was developed with five asthma health states: successful control, suboptimal control, outpatient-managed exacerbation, inpatient-managed exacerbation, and death. The time horizon was set at 12 months. Transition probabilities and indirect resource utilization were derived from previous international and Spanish publications. Univariate and probabilistic sensitivity analyses (SAs) were applied. FP/FORM was less expensive to acquire than FS or BF (20% lower than FS and 30% lower than BF), while the quality-adjusted life years (QALYs) of the three options compared were very similar. Cost per patient in the FP/FORM cohort was 9326€/year, making it the cheapest option, 1.5% cheaper than FS and 2.6% cheaper than BF. The suboptimal control health state dominated the costs (80% of the total cost) in each of the analyzed options and scenarios. The results of the SAs verified the data obtained from the base case scenario. From a Spanish societal perspective, in 2014, FP/FORM produced a similar gain in QALYs but at a lower cost when compared to FS and BF in a highly meaningful number of replications and scenarios. FP/FORM can therefore be considered a cost-effective option in the treatment of moderate-to-severe asthma in Spain. The cost savings were mainly due to the significantly lower acquisition cost of FP/FORM than the other two options. Mundipharma Pharmaceuticals, S.L., Madrid, Spain.