Targeted vs opportunistic screening for viral hepatitis among UK migrant communities: a cost-effectiveness analysis

2021 ◽  
Vol 27 (3) ◽  
pp. 90-98
Author(s):  
Harold A Salmon ◽  
Simon Briscoe ◽  
Graham R Foster ◽  
Martin A Pitt
Keyword(s):  
Cost Effectiveness ◽  
Hepatitis B ◽  
Willingness To Pay ◽  
Cost Effective ◽  
Quality Adjusted Life Year ◽  
Ethnic Composition ◽  
World Health ◽  
Opportunistic Screening ◽  
Targeted Screening ◽  
Quality Adjusted Life

Worldwide eradication of chronic hepatitis B and C viruses by 2030 is a stated goal of the World Health Organization, UK government and the European Union. This study investigated the cost-effectiveness of targeted screening vs opportunistic screening for hepatitis B and C among migrant populations in the UK. Results of a previous clinical trial (HepFREE) carried out in London and Bradford were used to develop a combined decision tree and Markov simulation model. Despite a low response to invitations for vaccination, and a heterogeneous level of response between communities of different ethnic composition, this analysis shows that incentivised targeted screening is cost-effective at willingness-to-pay thresholds over £8540 per incremental quality-adjusted life year over a lifetime. Furthermore, probabilistic analysis of input parameter uncertainty suggests that the intervention has a greater than 95% probability of being cost-effective at willingness-to-pay thresholds under £30 000 per incremental quality-adjusted life year. These results strongly suggest that targeted screening should play a key part in the eradication of the hepatitis B and C viruses.

Cost–effectiveness analysis of ribociclib plus fulvestrant for hormone receptor-positive/human EGF receptor 2-negative breast cancer

Immunotherapy ◽  
2021 ◽  
Author(s):  
Wei Jiang ◽  
Zhichao He ◽  
Tiantian Zhang ◽  
Chongchong Guo ◽  
Jianli Zhao ◽  
...  
Keyword(s):  
Breast Cancer ◽  
Cost Effectiveness ◽  
Hormone Receptor ◽  
Egf Receptor ◽  
Cost Effective ◽  
Quality Adjusted Life Year ◽  
Cost Effectiveness Ratio ◽  
Hormone Receptor Positive ◽  
The Cost ◽  
Quality Adjusted Life

Aim: To evaluate the cost–effectiveness of ribociclib plus fulvestrant versus fulvestrant in hormone receptor-positive/human EGF receptor 2-negative advanced breast cancer. Materials & methods: A three-state Markov model was developed to evaluate the costs and effectiveness over 10 years. Direct costs and utility values were obtained from previously published studies. We calculated incremental cost–effectiveness ratio to evaluate the cost–effectiveness at a willingness-to-pay threshold of $150,000 per additional quality-adjusted life year. Results: The incremental cost–effectiveness ratio was $1,073,526 per quality-adjusted life year of ribociclib plus fulvestrant versus fulvestrant. Conclusions: Ribociclib plus fulvestrant is not cost-effective versus fulvestrant in the treatment of advanced hormone receptor-positive/human EGF receptor 2-negative breast cancer. When ribociclib is at 10% of the full price, ribociclib plus fulvestrant could be cost-effective.

scholarly journals A one-year cost–utility analysis of REBOA versus RTACC for non-compressible torso haemorrhage

Trauma ◽  
2017 ◽  
Vol 21 (1) ◽  
pp. 45-54 ◽  
Author(s):  
Maxwell S Renna ◽  
Cristiano van Zeller ◽  
Farah Abu-Hijleh ◽  
Cherlyn Tong ◽  
Jasmine Gambini ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Cost Effective ◽  
Quality Adjusted Life Year ◽  
Cost Utility ◽  
Incremental Cost Effectiveness Ratio ◽  
Cost Utility Analysis ◽  
Utility Analysis ◽  
Cost Effectiveness Ratio ◽  
One Year ◽  
Quality Adjusted Life

Introduction Major trauma is a leading cause of death and disability in young adults, especially from massive non-compressible torso haemorrhage. The standard technique to control distal haemorrhage and maximise central perfusion is resuscitative thoracotomy with aortic cross-clamping (RTACC). More recently, the minimally invasive technique of resuscitative endovascular balloon occlusion of the aorta (REBOA) has been developed to similarly limit distal haemorrhage without the morbidity of thoracotomy; cost–utility studies on this intervention, however, are still lacking. The aim of this study was to perform a one-year cost–utility analysis of REBOA as an intervention for patients with major traumatic non-compressible abdominal haemorrhage, compared to RTACC within the U.K.’s National Health Service. Methods A retrospective analysis of the outcomes following REBOA and RTACC was conducted based on the published literature of survival and complication rates after intervention. Utility was obtained from studies that used the EQ-5D index and from self-conducted surveys. Costs were calculated using 2016/2017 National Health Service tariff data and supplemented from further literature. A cost–utility analysis was then conducted. Results A total of 12 studies for REBOA and 20 studies for RTACC were included. The mean injury severity scores for RTACC and REBOA were 34 and 39, and mean probability of death was 9.7 and 54%, respectively. The incremental cost-effectiveness ratio of REBOA when compared to RTACC was £44,617.44 per quality-adjusted life year. The incremental cost-effectiveness ratio, by exceeding the National Institute for Health and Clinical Effectiveness’s willingness-to-pay threshold of £30,000/quality-adjusted life year, suggests that this intervention is not cost-effective in comparison to RTACC. However, REBOA yielded a 157% improvement in utility with a comparatively small cost increase of 31.5%. Conclusion Although REBOA has not been found to be cost-effective when compared to RTACC, ultimately, clinical experience and expertise should be the main factor in driving the decision over which intervention to prioritise in the emergency context.

Cost-effectiveness of hydrotherapy versus land-based therapy in patients with musculoskeletal disorders in Singapore

2018 ◽  
Vol 41 (2) ◽  
pp. 391-398
Author(s):  
Monica Teng ◽  
Hui Jun Zhou ◽  
Liang Lin ◽  
Pang Hung Lim ◽  
Doreen Yeo ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Musculoskeletal Disorders ◽  
Cost Effective ◽  
Target Population ◽  
Quality Adjusted Life Year ◽  
Decision Analytic Model ◽  
Published Data ◽  
Total Knee ◽  
The Cost ◽  
Quality Adjusted Life

Abstract Background The study evaluated the cost-effectiveness of hydrotherapy versus land-based therapy in patients with musculoskeletal disorders (MSDs) in Singapore. Methods A decision-analytic model was constructed to compare the cost-effectiveness of hydrotherapy to land-based therapy over 3 months from societal perspective. Target population comprised patients with low back pain (LBP), osteoarthritis (OA), rheumatoid arthritis (RA), total hip replacement (THR) and total knee replacement (TKR). Subgroup analyses were carried out to determine the cost-effectiveness of hydrotherapy in individual MSDs. Relative treatment effects were obtained through a systematic review of published data. Results Compared to land-based therapy, hydrotherapy was associated with an incremental cost-effectiveness ratio (ICER) of SGD 27 471 per quality-adjusted life-year (QALY) gained, which was below the willingness-to-pay threshold of SGD 70 000 per QALY (one gross domestic product per capita in Singapore in 2015). For the respective MSDs, hydrotherapy were dominant (more effective and less costly) in THR and TKR, cost-effective for LBP and RA, and not cost-effective for OA. Treatment adherence and cost of hydrotherapy were key drivers to the ICER values. Conclusions Hydrotherapy was a cost-effective rehabilitation compared to land-based therapy for a population with MSDs in Singapore. However, the benefit of hydrotherapy was not observed in patients with OA.

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 Interventions to reduce the risk of surgically transmitted Creutzfeldt–Jakob disease: a cost-effective modelling review

2020 ◽  
Vol 24 (11) ◽  
pp. 1-150 ◽  
Author(s):  
Matt Stevenson ◽  
Lesley Uttley ◽  
Jeremy E Oakley ◽  
Christopher Carroll ◽  
Stephen E Chick ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Cost Effective ◽  
Health Technology ◽  
Quality Adjusted Life Year ◽  
Expert Elicitation ◽  
Jakob Disease ◽  
Single Use ◽  
Quality Adjusted Life

Background Creutzfeldt–Jakob disease is a fatal neurological disease caused by abnormal infectious proteins called prions. Prions that are present on surgical instruments cannot be completely deactivated; therefore, patients who are subsequently operated on using these instruments may become infected. This can result in surgically transmitted Creutzfeldt–Jakob disease. Objective To update literature reviews, consultation with experts and economic modelling published in 2006, and to provide the cost-effectiveness of strategies to reduce the risk of surgically transmitted Creutzfeldt–Jakob disease. Methods Eight systematic reviews were undertaken for clinical parameters. One review of cost-effectiveness was undertaken. Electronic databases including MEDLINE and EMBASE were searched from 2005 to 2017. Expert elicitation sessions were undertaken. An advisory committee, convened by the National Institute for Health and Care Excellence to produce guidance, provided an additional source of information. A mathematical model was updated focusing on brain and posterior eye surgery and neuroendoscopy. The model simulated both patients and instrument sets. Assuming that there were potentially 15 cases of surgically transmitted Creutzfeldt–Jakob disease between 2005 and 2018, approximate Bayesian computation was used to obtain samples from the posterior distribution of the model parameters to generate results. Heuristics were used to improve computational efficiency. The modelling conformed to the National Institute for Health and Care Excellence reference case. The strategies evaluated included neither keeping instruments moist nor prohibiting set migration; ensuring that instruments were kept moist; prohibiting instrument migration between sets; and employing single-use instruments. Threshold analyses were undertaken to establish prices at which single-use sets or completely effective decontamination solutions would be cost-effective. Results A total of 169 papers were identified for the clinical review. The evidence from published literature was not deemed sufficiently strong to take precedence over the distributions obtained from expert elicitation. Forty-eight papers were identified in the review of cost-effectiveness. The previous modelling structure was revised to add the possibility of misclassifying surgically transmitted Creutzfeldt–Jakob disease as another neurodegenerative disease, and assuming that all patients were susceptible to infection. Keeping instruments moist was estimated to reduce the risk of surgically transmitted Creutzfeldt–Jakob disease cases and associated costs. Based on probabilistic sensitivity analyses, keeping instruments moist was estimated to on average result in 2.36 (range 0–47) surgically transmitted Creutzfeldt–Jakob disease cases (across England) caused by infection occurring between 2019 and 2023. Prohibiting set migration or employing single-use instruments reduced the estimated risk of surgically transmitted Creutzfeldt–Jakob disease cases further, but at considerable cost. The estimated costs per quality-adjusted life-year gained of these strategies in addition to keeping instruments moist were in excess of £1M. It was estimated that single-use instrument sets (currently £350–500) or completely effective cleaning solutions would need to cost approximately £12 per patient to be cost-effective using a £30,000 per quality-adjusted life-year gained value. Limitations As no direct published evidence to implicate surgery as a cause of Creutzfeldt–Jakob disease has been found since 2005, the estimations of potential cases from elicitation are still speculative. A particular source of uncertainty was in the number of potential surgically transmitted Creutzfeldt–Jakob disease cases that may have occurred between 2005 and 2018. Conclusions Keeping instruments moist is estimated to reduce the risk of surgically transmitted Creutzfeldt–Jakob disease cases and associated costs. Further surgical management strategies can reduce the risks of surgically transmitted Creutzfeldt–Jakob disease but have considerable associated costs. Study registration This study is registered as PROSPERO CRD42017071807. 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. 11. See the NIHR Journals Library website for further project information.

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.

scholarly journals A Markov Analysis of Screening for Late-Onset Cytomegalovirus Disease in Cytomegalovirus High-Risk Kidney Transplant Recipients

2017 ◽  
Vol 13 (2) ◽  
pp. 290-298
Author(s):  
Chethan M. Puttarajappa ◽  
Sundaram Hariharan ◽  
Kenneth J. Smith
Keyword(s):  
Cost Effectiveness ◽  
Willingness To Pay ◽  
Cytomegalovirus Infection ◽  
Late Onset ◽  
Quality Adjusted Life Year ◽  
Transplant Recipients ◽  
Kidney Transplant Recipients ◽  
Quality Adjusted Life Years ◽  
Life Years ◽  
Quality Adjusted Life

Background and objectivesManagement strategies are unclear for late-onset cytomegalovirus infection occurring beyond 6 months of antiviral prophylaxis in cytomegalovirus high-risk (cytomegalovirus IgG positive to cytomegalovirus IgG negative) kidney transplant recipients. Hybrid strategies (prophylaxis followed by screening) have been investigated but with inconclusive results. There are clinical and potential cost benefits of preventing cytomegalovirus-related hospitalizations and associated increased risks of patient and graft failure. We used decision analysis to evaluate the utility of postprophylaxis screening for late-onset cytomegalovirus infection.Design, setting, participants, & measurementsWe used the Markov decision analysis model incorporating costs and utilities for various cytomegalovirus clinical states (asymptomatic cytomegalovirus, mild cytomegalovirus infection, and cytomegalovirus infection necessitating hospitalization) to estimate cost-effectiveness of postprophylaxis cytomegalovirus screening strategies. Five strategies were compared: no screening and screening at 1-, 2-, 3-, or 4-week intervals. Progression to severe cytomegalovirus infection was modeled on cytomegalovirus replication kinetics. Incremental cost-effectiveness ratios were calculated as a ratio of cost difference between two strategies to difference in quality-adjusted life-years starting with the low-cost strategy. One-way and probabilistic sensitivity analyses were performed to test model’s robustness.ResultsThere was an incremental gain in quality-adjusted life-years with increasing screening frequency. Incremental cost-effectiveness ratios were $783 per quality-adjusted life-year (every 4 weeks over no screening), $1861 per quality-adjusted life-year (every 3 weeks over every 4 weeks), $10,947 per quality-adjusted life-year (every 2 weeks over every 3 weeks), and $197,086 per quality-adjusted life-year (weekly over every 2 weeks). Findings were sensitive to screening cost, cost of hospitalization, postprophylaxis cytomegalovirus incidence, and graft loss after cytomegalovirus infection. No screening was favored when willingness to pay threshold was <$14,000 per quality-adjusted life-year, whereas screening weekly was favored when willingness to pay threshold was >$185,000 per quality-adjusted life-year. Screening every 2 weeks was the dominant strategy between willingness to pay range of $14,000–$185,000 per quality-adjusted life-year.ConclusionsIn cytomegalovirus high-risk kidney transplant recipients, compared with no screening, screening for postprophylactic cytomegalovirus viremia is associated with gains in quality-adjusted life-years and seems to be cost effective. A strategy of screening every 2 weeks was the most cost-effective strategy across a wide range of willingness to pay thresholds.PodcastThis article contains a podcast at https://www.asn-online.org/media/podcast/CJASN/2017_12_18_CJASNPodcast_18_2_P.mp3

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.

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