scholarly journals Cost-Effectiveness of Tafamidis Therapy for Transthyretin Amyloid Cardiomyopathy

Circulation ◽  
2020 ◽  
Vol 141 (15) ◽  
pp. 1214-1224 ◽  
Author(s):  
Dhruv S. Kazi ◽  
Brandon K. Bellows ◽  
Suzanne J. Baron ◽  
Changyu Shen ◽  
David J. Cohen ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Incremental Cost ◽  
Cost Effective ◽  
The United States ◽  
Quality Adjusted Life Year ◽  
Life Years ◽  
The Us ◽  
Amyloid Cardiomyopathy ◽  
Quality Adjusted Life

Background: In patients with transthyretin amyloid cardiomyopathy, tafamidis reduces all-cause mortality and cardiovascular hospitalizations and slows decline in quality of life compared with placebo. In May 2019, tafamidis received expedited approval from the US Food and Drug Administration as a breakthrough drug for a rare disease. However, at $225 000 per year, it is the most expensive cardiovascular drug ever launched in the United States, and its long-term cost-effectiveness and budget impact are uncertain. We therefore aimed to estimate the cost-effectiveness of tafamidis and its potential effect on US health care spending. Methods: We developed a Markov model of patients with wild-type or variant transthyretin amyloid cardiomyopathy and heart failure (mean age, 74.5 years) using inputs from the ATTR-ACT trial (Transthyretin Amyloidosis Cardiomyopathy Clinical Trial), published literature, US Food and Drug Administration review documents, healthcare claims, and national survey data. We compared no disease–specific treatment (“usual care”) with tafamidis therapy. The model reproduced 30-month survival, quality of life, and cardiovascular hospitalization rates observed in ATTR-ACT; future projections used a parametric survival model in the control arm, with constant hazards reduction in the tafamidis arm. We discounted future costs and quality-adjusted life-years by 3% annually and examined key parameter uncertainty using deterministic and probabilistic sensitivity analyses. The main outcomes were lifetime incremental cost-effectiveness ratio and annual budget impact, assessed from the US healthcare sector perspective. This study was independent of the ATTR-ACT trial sponsor. Results: Compared with usual care, tafamidis was projected to add 1.29 (95% uncertainty interval, 0.47–1.75) quality-adjusted life-years at an incremental cost of $1 135 000 (872 000–1 377 000), resulting in an incremental cost-effectiveness ratio of $880 000 (697 000–1 564 000) per quality-adjusted life-year gained. Assuming a threshold of $100 000 per quality-adjusted life-year gained and current drug price, tafamidis was cost-effective in 0% of 10 000 probabilistic simulations. A 92.6% price reduction from $225 000 to $16 563 would be necessary to make tafamidis cost-effective at $100 000/quality-adjusted life-year. Results were sensitive to assumptions related to long-term effectiveness of tafamidis. Treating all eligible patients with transthyretin amyloid cardiomyopathy in the United States with tafamidis (n=120 000) was estimated to increase annual healthcare spending by $32.3 billion. Conclusions: Treatment with tafamidis is projected to produce substantial clinical benefit but would greatly exceed conventional cost-effectiveness thresholds at the current US list price. On the basis of recent US experience with high-cost cardiovascular medications, access to and uptake of this effective therapy may be limited unless there is a large reduction in drug costs.

scholarly journals Comparative clinical effects and cost–effectiveness of maximum androgen blockade, docetaxel with androgen deprivation therapy and ADT alone for the treatment of mHSPC in China

2019 ◽  
Vol 8 (11) ◽  
pp. 865-877 ◽  
Author(s):  
Maobai Liu ◽  
Shuli Qu ◽  
Yanjun Liu ◽  
Xingxing Yao ◽  
Wei Jiang
Keyword(s):  
Cost Effectiveness ◽  
Incremental Cost ◽  
Androgen Deprivation Therapy ◽  
Cost Effective ◽  
Quality Adjusted Life Year ◽  
Incremental Cost Effectiveness Ratio ◽  
Clinical Effects ◽  
Cost Effectiveness Ratio ◽  
Quality Adjusted Life ◽  
Androgen Blockade

Aim: To compare the clinical effects and cost–effectiveness of maximum androgen blockade (MAB), docetaxel to androgen deprivation therapy (Doc-ADT) and ADT alone for the treatment of patients with metastatic hormone-sensitive prostate cancer in China. Methods: A network meta-analysis and a Markov model were adopted for effectiveness and economic evaluation. Results: The hazard ratios of overall survival and progression-free survival were 0.782 and 0.628 for Doc-ADT versus ADT alone; 0.897 and 0.824 for MAB versus ADT alone. Doc-ADT was cost-effective compared with MAB and ADT alone, with an incremental cost–effectiveness ratio of CNY 96,848 and CNY 67,758 per quality-adjusted life year, respectively. MAB was cost-effective compared with ADT alone, with an incremental cost–effectiveness ratio of CNY 137,487 per quality-adjusted life year. Conclusion: Doc-ADT is likely the optimal option from the perspective of both clinical outcomes and economic considerations.

scholarly journals Cost-Effectiveness Evaluation of Etoricoxib versus Celecoxib and Nonselective NSAIDs in the Treatment of Ankylosing Spondylitis in Norway

2011 ◽  
Vol 2011 ◽  
pp. 1-14 ◽  
Author(s):  
Jeroen P. Jansen ◽  
Stephanie D. Taylor
Keyword(s):  
Cost Effectiveness ◽  
Ankylosing Spondylitis ◽  
Time Horizon ◽  
Cost Effective ◽  
Quality Adjusted Life Year ◽  
Quality Adjusted Life Years ◽  
Life Years ◽  
Cost Effective Treatment ◽  
Ceiling Ratio ◽  
Quality Adjusted Life

Objectives. To evaluate the cost-effectiveness of etoricoxib (90 mg) relative to celecoxib (200/400 mg), and the nonselective NSAIDs naproxen (1000 mg) and diclofenac (150 mg) in the initial treatment of ankylosing spondylitis in Norway.Methods. A previously developed Markov state-transition model was used to estimate costs and benefits associated with initiating treatment with the different competing NSAIDs. Efficacy, gastrointestinal and cardiovascular safety, and resource use data were obtained from the literature. Data from different studies were synthesized and translated into direct costs and quality adjusted life years by means of a Bayesian comprehensive decision modeling approach.Results. Over a 30-year time horizon, etoricoxib is associated with about 0.4 more quality adjusted life years than the other interventions. At 1 year, naproxen is the most cost-saving strategy. However, etoricoxib is cost and quality adjusted life year saving relative to celecoxib, as well as diclofenac and naproxen after 5 years of follow-up. For a willingness-to-pay ceiling ratio of 200,000 Norwegian krones per quality adjusted life year, there is a >95% probability that etoricoxib is the most-cost-effective treatment when a time horizon of 5 or more years is considered.Conclusions. Etoricoxib is the most cost-effective NSAID for initiating treatment of ankylosing spondylitis in Norway.

scholarly journals Economic Evaluation of CYP2C19 Genotype-Guided Antiplatelet Therapy Compared to Universal use of Ticagrelor or Clopidogrel in Qatar

2020 ◽  
Author(s):  
Sawsan Ibrahim AlMukdad ◽  
Hazem Elewa ◽  
Daoud Al-Badriyeh
Keyword(s):  
Cost Effectiveness ◽  
Incremental Cost ◽  
Antiplatelet Therapy ◽  
Cost Effective ◽  
Cyp2c19 Genotype ◽  
Life Years ◽  
One Year ◽  
The One ◽  
Guided Therapy

Background: Patients having CYP2C19 loss-of-function alleles and receiving clopidogrel are at higher risk of adverse cardiovascular outcomes. Ticagrelor is a more effective and expensive antiplatelet that is unaffected by the CYP2C19 polymorphism. The main aim of the current research is to evaluate the cost-effectiveness among CYP2C19 genotype-guided therapy, universal ticagrelor, and universal clopidogrel after a percutaneous coronary intervention (PCI). Methods: A two-part simulation model, including a one-year decision-analytic model and a 20-year followup Markov model, was created to follow the use of (i) universal clopidogrel, (ii) universal ticagrelor, and (iii) genotype-guided antiplatelet therapy. Outcome measures were the incremental cost-effectiveness ratio (ICER, cost/success) and incremental cost-utility ratio (ICUR, cost/qualityadjusted life years [QALY]). Therapy success was defined as survival without myocardial infarction, stroke, cardiovascular death, stent thrombosis, and no therapy discontinuation because of adverse events, i.e. major bleeding and dyspnea. The model was based on a multivariate analysis, and a sensitivity analysis confirmed the robustness of the model outcomes. Results: Against universal clopidogrel, genotype-guided therapy was cost-effective over the one-year duration (ICER, USD 6,102 /success), and dominant over the long-term. Genotype-guided therapy was dominant over universal ticagrelor over the one-year duration and cost-effective over the long term (ICUR, USD 1,383 /QALY). Universal clopidogrel was dominant over ticagrelor over the short term, and cost-effective over the long-term (ICUR, 10,616 /QALY). Conclusion: CYP2C19 genotype-guided therapy appears to be the preferred antiplatelet strategy, followed by universal clopidogrel, and then universal ticagrelor for post-PCI patients in Qatar.

scholarly journals Interventions based on early intensive applied behaviour analysis for autistic children: a systematic review and cost-effectiveness analysis

2020 ◽  
Vol 24 (35) ◽  
pp. 1-306
Author(s):  
Mark Rodgers ◽  
David Marshall ◽  
Mark Simmonds ◽  
Ann Le Couteur ◽  
Mousumi Biswas ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Confidence Interval ◽  
Cost Effective ◽  
Quality Adjusted Life Year ◽  
Mean Difference ◽  
Autistic Children ◽  
Behaviour Analysis ◽  
Applied Behaviour Analysis ◽  
Quality Adjusted Life

Background Early intensive applied behaviour analysis-based interventions are intensive interventions for autistic children that are often delivered on a one-to-one basis for 20–50 hours per week. Objectives To evaluate the clinical effectiveness and cost-effectiveness of early intensive applied behaviour analysis-based interventions for autistic children, based on current evidence. Methods A systematic review and individual participant data meta-analysis were conducted to evaluate the clinical effectiveness of an early intensive applied behaviour analysis-based intervention for autistic children. An economic analysis included a review of existing analyses and the development of a new model. Results Twenty studies were included in the clinical review. Individual participant data were retrieved from 15 of these studies. Results favoured the interventions when assessing adaptive behaviour after 2 years compared with treatment as usual/eclectic interventions (mean difference 7.00, 95% confidence interval 1.95 to 12.06). In analyses of cognitive ability (intelligence quotient), results favoured the interventions by approximately 10 points after 1 year (mean difference 9.16, 95% confidence interval 4.38 to 13.93) and 2 years (mean difference 14.13, 95% confidence interval 9.16 to 19.10). Evidence for other outcomes was limited and meta-analyses were generally inconclusive. There was no evidence that the effect of the interventions varied with characteristics of the children, but data were limited. Adopting a £30,000 per quality-adjusted life-year threshold, the results of the cost-effectiveness analysis indicate that early intensive applied behaviour analysis-based interventions would need to generate larger benefits or cost savings to be cost-effective. Adopting a public sector perspective and making pessimistic assumptions about long-term effects, the incremental cost-effectiveness ratio for early intensive applied behaviour analysis-based therapy compared with treatment as usual is £189,122 per quality-adjusted life-year. When optimistic assumptions are made, the incremental cost-effectiveness ratio is £46,768 per quality-adjusted life-year. Scenario analyses indicated that these interventions can potentially be cost-effective if long-term improvements persist into adulthood, or if they have significant impact on educational placement. Care should be taken when interpreting these scenarios owing to the limited data. Limitations All included studies were at risk of bias, there was substantial heterogeneity and effects varied considerably across studies. The effect of intervention on autism symptom severity, language development and school placement remains uncertain because of the limited data. The long-term effects are unclear owing to a lack of follow-up data. Conclusions This review found limited evidence that early intensive applied behaviour analysis-based interventions may improve cognitive ability and adaptive behaviour, but the long-term impact of the interventions remains unknown. The economic analysis is constrained by the limited effectiveness evidence, but suggests that these interventions are unlikely to be cost-effective unless clear long-term benefits, or a substantial change in which schools children attend, can be identified. Future work Further studies into the effectiveness of early intensive applied behaviour analysis-based interventions may be warranted if they include well-defined, alternative interventions as comparators and collect relevant outcomes. Consideration should be given to future studies that not only address whether or not early intensive applied behaviour analysis-based interventions are clinically effective, but also aim to identify which components of early intensive applied behaviour analysis-based interventions might drive effectiveness. Study registration This study is registered as PROSPERO CRD42017068303. Funding This project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 24, No. 35. See the NIHR Journals Library website for further project information.

scholarly journals Cost-Effectiveness of Ipilimumab Plus Anti-PD-1 Therapy Versus Ipilimumab Alone in Patients With Metastatic Melanoma Resistant to Anti-PD-(L)1 Monotherapy

2021 ◽  
Vol 11 ◽  
Author(s):  
Ye Peng ◽  
Xiaohui Zeng ◽  
Liubao Peng ◽  
Qiao Liu ◽  
Lidan Yi ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Metastatic Melanoma ◽  
Subgroup Analysis ◽  
Cost Effective ◽  
Quality Adjusted Life Year ◽  
Cost Information ◽  
Payer Perspective ◽  
The Us ◽  
The Cost ◽  
Quality Adjusted Life

ObjectiveThe use of ipilimumab plus anti-PD-1 has recently been shown to significantly improve the survival of patients with metastatic melanoma resistant to anti-PD-(L)1 monotherapy. The study assessed the cost-effectiveness of ipilimumab plus anti-PD-1 therapy in this population from the US payer perspective.Materials and MethodsA Markov model was created based on a retrospective analysis of patients with metastatic melanoma who were resistant to anti-PD-(L)1. Cost information was obtained from the Centers for Medicare and Medicaid Services and literature-based costs. The utility value was derived from the published literature. The results of the model was the total cost, quality-adjusted life-year (QALY), and incremental cost-effectiveness ratio (ICER). The uncertainty of the model was addressed through sensitivity analysis. In addition, we also conducted subgroup analysis.ResultsIpilimumab plus anti-PD-1 provided an improvement of 1.39 QALYs and 2.48 LYs, at a ICER of $73,163 per QALY. The HR of OS was the variable that had the greatest impact on ICER. Compared to ipilimumab, the probability of ipilimumab plus anti-PD-1 being cost-effective was 94% at the WTP of $150,000/QALY. The results of the subgroup analysis showed that the ICER in the majority of the subgroups was less than $150,000/QALY.ConclusionsIpilimumab plus anti-PD-1 was likely to be cost-effective compared to ipilimumab for patients with metastatic melanoma who are resistant to anti-PD-(L)1 at a WTP threshold of 150,000/QALY.

CyberKnife for prostate cancer: Is it cost-effective?

2011 ◽  
Vol 29 (7_suppl) ◽  
pp. 87-87 ◽  
Author(s):  
A. Parthan ◽  
N. Pruttivarasin ◽  
D. Taylor ◽  
D. Davies ◽  
G. Yang ◽  
...  
Keyword(s):  
Prostate Cancer ◽  
Radiation Therapy ◽  
Cost Effectiveness ◽  
Incremental Cost ◽  
Societal Perspective ◽  
Cost Effective ◽  
Sensitivity Analyses ◽  
Life Years ◽  
Lifetime Costs

87 Background: The study assessed the cost-effectiveness of CyberKnife (CK) compared to surgery and radiation therapy for the treatment of prostate cancer (PC) from a third-party and societal perspective. Methods: For patients > 65 yrs with localized PC, a Markov model compared treatment with CK, intensity modulated radiation therapy (IMRT), surgery or proton therapy (PT). Following treatment, patients were at risk of long-term toxicity: genitourinary (GU); gastrointestinal (GI); and sexual dysfunction (SD). Long-term toxicity was defined as adverse events >grade 2 on Radiation Therapy Oncology Group scale occurring at least 12 months following treatment. Markov states included all possible combinations of GI, GU, and SD long-term toxicities, no toxicity, and death. During each year patients remained in the same Markov state or died. Costs and utilities were assigned using published sources. Toxicity probabilities were derived using meta-analytical techniques to pool results from multiple studies. It was assumed that long-term disease control would not differ across treatments. The model projected expected lifetime costs and quality adjusted life years (QALYs) for each treatment and incremental cost-effectiveness of CK vs comparators as cost per QALY gained. Costs from societal perspective included lost productivity. Extensive sensitivity analyses were conducted. Results: Surgery was the least expensive treatment option followed by CK. CK patients had higher expected QALYs (8.11) than other treatment options (7.72- 8.06). From a payer perspective, total lifetime costs were $25,904, $22,295, $38,915, and $58,100 for CK, surgery, IMRT and PT, respectively. Incremental cost per QALY gained for CK versus Surgery was $9,200/QALY. Compared to IMRT and PT, CK was less costly and resulted in higher QALYs (dominance). At a threshold of $50,000/QALY, CK was cost effective in 86%, 79%, and 91% of simulations compared to surgery, IMRT, and PT, respectively. From a societal perspective, CK costs $4,200/QALY compared to surgery and remained dominant vs IMRT and PT. Results were most sensitive to costs of surgery and CK. Conclusions: Initial CK costs are higher than surgery, but CK patients have better quality of life. CK patients have lower lifetime costs and higher QALYs than IMRT and PT patients. [Table: see text]

Cost-Effectiveness of Posaconazole vs Fluconazole in the Prevention of Invasive Fungal Infections among Patients with Graft-Versus-Host Disease (GVHD) in the US.

Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 3336-3336 ◽  
Author(s):  
Amy K. O’Sullivan ◽  
Milton C. Weinstein ◽  
Ankur Pandya ◽  
David Thompson ◽  
Amelia Langston ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Graft Versus Host Disease ◽  
Fungal Infections ◽  
Cost Effective ◽  
Graft Versus Host ◽  
Life Years ◽  
The Us ◽  
The Cost ◽  
Long Term Mortality

Abstract Trial data suggest that posaconazole is similar to fluconazole in preventing invasive fungal infections (IFIs) among allogeneic progenitor cell transplant recipients with graft-versus-host disease (GVHD). We estimated the cost-effectiveness of posaconazole versus fluconazole in this population in the US. A decision-analytic model was developed to estimate the average per patient treatment costs, IFIs avoided, life-years gained, and incremental cost per life-year gained of prophylaxis (2006 US$). The model extrapolates the trial results to a lifetime horizon to include long-term mortality due to GVHD. In the model, patients are assumed to receive posaconazole or fluconazole; efficacy data were obtained from the clinical trial. Long-term mortality and prophylaxis drug and IFI treatment costs were estimated from secondary sources. One-way and probabilistic sensitivity analyses were conducted. Posaconazole is associated with fewer IFIs (0.05 vs. 0.09), increased life years (7.87 vs. 7.66), and higher IFI-related costs (prophylaxis and IFI treatment) ($8,750 vs. $5,530) per patient relative to fluconazole. Costs for treatment of IFIs comprised 95% of the total cost for fluconazole and 35% for posaconazole. The incremental cost-effectiveness of posaconazole versus fluconazole is estimated to be $15,700 per life-year saved. Results are most sensitive to changes in the cost of treating an IFI and the efficacy of prophylaxis. Results from the probabilistic analysis indicate that there is an 88% probability that posaconazole is cost-effective at a $50,000 per life year saved threshold. We conclude that posaconazole is a cost-effective strategy for the prevention of IFIs in patients with GVHD.

scholarly journals The impact and cost-effectiveness of 9-valent human papillomavirus vaccine in adolescent females in Hong Kong

2021 ◽  
Vol 19 (1) ◽  
Author(s):  
Tak Hong Cheung ◽  
Sally Shuk Yee Cheng ◽  
Danny C. Hsu ◽  
Queenie Wing-Lei Wong ◽  
Andrew Pavelyev ◽  
...  
Keyword(s):  
Hong Kong ◽  
Human Papillomavirus ◽  
Cost Effectiveness ◽  
Incremental Cost ◽  
Cost Effective ◽  
Health Impact ◽  
Quality Adjusted Life Year ◽  
Catch Up ◽  
Coverage Rates ◽  
Quality Adjusted Life

Abstract Introduction In Hong Kong (HK), a single-cohort vaccination program for 10–12-year-old girls with the 9-valent human papillomavirus (HPV) vaccine (9vHPV; types 6/11/16/18/31/33/45/52/58) has been launched. This study assessed the public health impact and cost-effectiveness of implementing routine 9vHPV vaccination (12-year-olds) with or without catch-up 9vHPV vaccination (13–18-year-olds) in HK. Methods The health impact and costs of implementing routine 9vHPV vaccination with or without catch-up vaccination over a 100-year time horizon were evaluated using a validated HPV-type transmission dynamic model adapted to the HK population; analyses were performed from a healthcare payer perspective. Routine vaccination (12-year-old girls) and catch-up vaccination (13–18 years) assumed vaccine coverage rates of 70% (base case) and 30%, respectively. The model also assumed herd immunity, lifelong vaccine protection, a discount rate of 3%, and a cost per dose of HK dollars (HKD) 858 [United States dollars (USD) 110] and HKD 1390 (USD 179) for the 2-valent HPV (2vHPV) and 9vHPV vaccines, respectively. HPV disease-related incidence and the incremental cost-effectiveness ratio (ICER) per quality-adjusted-life-year (QALY) were estimated. Cost-effectiveness was determined at a ceiling threshold of HK dollars (HKD) 382,046 (USD 49,142) or 1.0 times the gross domestic product per capita of HK. Results Compared with routine 9vHPV alone, routine plus catch-up 9vHPV is projected to reduce cervical cancer incidence by 3.4%. Routine plus catch-up 9vHPV will also reduce genital warts incident cases for males/females by 2.6%/5.4%. The incremental cost-effectiveness ratios were HKD 29,911 (USD 3847)/quality-adjusted life-year (QALY) for routine plus catch-up 9vHPV versus routine 9vHPV alone and HKD 25,524 (USD 3283)/QALY for routine 9vHPV alone versus screening only. Sensitivity analyses indicated that routine plus catch-up 9vHPV compared with routine 9vHPV alone remained cost-effective at coverage rates of 30% and 90%. Conclusions This analysis predicts that the current HK vaccination strategy can be considered cost-effective and will provide maximum health benefit. These results support addition of the routine 9vHPV vaccine with or without catch-up 9vHPV vaccination to the regional vaccination program in HK.

Economic rationale on the use of m-Health in uncontrolled hypertensive outpatients: from three months and beyond

2020 ◽  
Vol 41 (Supplement_2) ◽  
Author(s):  
M Ionov ◽  
O.V Zhukova ◽  
N.E Zvartau ◽  
A.O Konradi
Keyword(s):  
Cost Effectiveness ◽  
Incremental Cost ◽  
Usual Care ◽  
Cost Effective ◽  
Cost Utility ◽  
Scientific Data ◽  
Funding Source ◽  
Life Years ◽  
Cardiovascular Morbidity And Mortality

Abstract Background/Introduction Current scientific data show that blood pressure (BP) telemonitoring with/without additional counseling is rather effective in hypertension (HTN) management. However, cost-effectiveness and long-term social sequelae are lacking. This is because of diverse technologies and economic climate which make results highly heterogeneous across countries. Purpose To construct predictive model of long-term outcomes and to conduct the cost-effectiveness analysis of BP telemonitoring and remote counseling (BPTM) using m-Health in Russian population of outpatients with HTN. Methods Total of 240 patients were randomized (2:1) to either BPTM (n=160, mean age 47 y.o.) and to usual care (UC, n=80, mean age 49 y.o.) with baseline, three-month follow-up clinic visits combined with ambulatory BP measurement (ABPM). BPTM consisted of m-Health tool for patients, desktop module for clinicians. It enables BP data transfer and analysis, secure web chatting to support and counsel. Main outcomes were change in office, ambulatory systolic (S) BP and rate of BP control. A Markov cohort-based (1000 patients per study arm) model was developed and adopted a 10-year time horizon with 12-month time cycles. All patients started at a non-complicated HTN “well” state with a certain possibility of disease progression in a number of health states over a discrete time period. BPTM was compared with usual care in terms of 10-year healthcare costs, quality adjusted life years (QALY) using a Ministry of Health of Russian Federation perspective. Incremental cost-effectiveness ratio (ICER), incremental cost-utility ratio (ICUR) represented economic analysis. Results BPTM was associated with steeper decrease in office, ambulatory SBP (−16,8 mm Hg and −8,9 mm Hg, respectively; p<0,05) with the same treatment intensity (2,4 drugs per patient). There were 102 (64%) and 11 (14%) patients with fully controlled HTN in BPTM and UC groups, respectively (OR 11,03 95% CI [5,4–22,5]). An ICER of BPTM resulted in additional 11,1 EUR/1 mm Hg/year. It is expected that BPTM will be at least 76% cost-effective as per relevant Russian willingness-to-pay threshold. In a modelled 10-year period BPTM was life-saving (9,71 vs 9,6 life years gained) and cheap (cost of illness 1,5 mln vs 2,1 mln EUR). BPTM was also more valuable (8,31 versus 7,82 QALYs gained) so the ICUR was 3601,47 EUR/QALY gained. Cost-effectiveness was further confirmed by one-way deterministic sensitivity analysis. Conclusion BPTM seems to be clinically and economically effective when implemented into clinical practice. It provides greater BP reduction, improves BP control short-term. In a long-term it is likely to reduce cardiovascular morbidity and mortality in a cost-effective way. Larger randomized studies are needed to confirm these pilot results. Cost-effectiveness acceptability curve Funding Acknowledgement Type of funding source: Foundation. Main funding source(s): The Russian Scientific Foundation

Cost-Effectiveness Analysis of Ofatumumab As a Treatment for Relapsed Chronic Lymphocytic Leukemia in the United States

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 2366-2366
Author(s):  
Gabriel Tremblay ◽  
Anna Forsythe ◽  
Vasudha Bal ◽  
Snigdha Santra ◽  
Andrew Briggs
Keyword(s):  
Adverse Event ◽  
Cost Effectiveness ◽  
Chronic Lymphocytic Leukemia ◽  
Cost Effective ◽  
The United States ◽  
Lymphocytic Leukemia ◽  
Phase Iii ◽  
Life Years ◽  
Quality Adjusted Life

Abstract Background In a Phase III COMPLEMENT 2 study,ofatumumab(OFA) plusfludarabine(F) and cyclophosphamide (C) demonstrated significantly improved median progression-free survival (PFS) by 54% compared to FC treatment alone (HR=0.67, p=0.0032) in patients with relapsed chronic lymphocytic leukemia (rCLL). However, the relative value of OFA in rCLL has not been formally assessed. The objective of this study was to estimate the incremental cost per (quality-adjusted) life-year of utilizing OFA+FC vs. FC for rCLL in the US. Methods A partition survival model was developed to estimate the expected outcomes and costs of treatment of OFA+FC vs FC forrCLLover a lifetime horizon. The model includes 4 health states: PFS on treatment, PFS off-treatment, post-progression and death. Time during PFS following protocol-defined treatment duration of 6 months, was considered a treatment-free period in the model. Data on PFS, OS and frequencies of adverse events (AEs)were obtained from the Phase III clinical trial for OFA (COMPLEMENT 2). For the extrapolation of OS and PFS a piecewise approach was used, where the efficacy was based on the patient-level data (Kaplan-Meier Survivor Function) until the trial cut-off and a tail extrapolation thereafter (gamma distribution). Health state utilities and dis-utilities for AEs were obtained from previously published vignette studies. Costs incorporated in the model included drug and administration for primary and follow-up therapies, adverse event treatments, medical costs for hospitalizations and physician visits; and end of life costs. The costs were derived from databases (AnalySourceOnline, AHRQ, CMS). Results Treatment with OFA+FC led to an increase of 0.803 life years and 0.543 quality-adjusted life years (QALYs) relative to FC. The total cost of OFA+FC was higher by $6,693 per patient relative to FC. Although addition of AFA to FC lead to higher drug and adverse event costs, these were partially offset by lower follow-up costs compared to FC. The ICER per LY and per QALY gained with OFA+FC vs. FC was $8,333 and $12,322, respectively. Based on probabilistic sensitivity analyses, there wasa85% probability that OFA+FC was cost-effective compared to FC at a societal willingness-to-pay threshold of $100,000 per QALY saved. Conclusions Our analysis suggests treatment with OFA+FC compared to FC is highly cost-effective based Phase 3 within-trial analysis. These results are driven by the improved PFS and OS of OFA+FC vs. FC, as well as the treatment-free period, during which patients experienced PFS without the burden of treatment AEs or costs. Future direct comparisons of OFA+FC versus other treatment options will further clarify the cost-effectiveness of OFA+FC to inform coverage and reimbursement policy decisions. Disclosures Tremblay: Novartis Pharmaceuticals Corporation: Consultancy. Forsythe:Novartis Pharmaceuticals Corporation: Consultancy. Bal:Novartis Pharmaceuticals: Employment. Santra:Novartis Pharmaceuticals Corporation: Employment. Briggs:Novartis Pharmaceuticals Corporation: Consultancy.

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