scholarly journals Cost-effectiveness Analysis in R Using a Multi-state Modeling Survival Analysis Framework: A Tutorial

2016 ◽  
Vol 37 (4) ◽  
pp. 340-352 ◽  
Author(s):  
Claire Williams ◽  
James D. Lewsey ◽  
Andrew H. Briggs ◽  
Daniel F. Mackay

This tutorial provides a step-by-step guide to performing cost-effectiveness analysis using a multi-state modeling approach. Alongside the tutorial, we provide easy-to-use functions in the statistics package R. We argue that this multi-state modeling approach using a package such as R has advantages over approaches where models are built in a spreadsheet package. In particular, using a syntax-based approach means there is a written record of what was done and the calculations are transparent. Reproducing the analysis is straightforward as the syntax just needs to be run again. The approach can be thought of as an alternative way to build a Markov decision-analytic model, which also has the option to use a state-arrival extended approach. In the state-arrival extended multi-state model, a covariate that represents patients’ history is included, allowing the Markov property to be tested. We illustrate the building of multi-state survival models, making predictions from the models and assessing fits. We then proceed to perform a cost-effectiveness analysis, including deterministic and probabilistic sensitivity analyses. Finally, we show how to create 2 common methods of visualizing the results—namely, cost-effectiveness planes and cost-effectiveness acceptability curves. The analysis is implemented entirely within R. It is based on adaptions to functions in the existing R package mstate to accommodate parametric multi-state modeling that facilitates extrapolation of survival curves.

2021 ◽  
Vol 42 (Supplement_1) ◽  
Author(s):  
I Tessler ◽  
M Leshno ◽  
A Shmueli ◽  
S Shpitzen ◽  
R Durst ◽  
...  

Abstract Introduction Bicuspid aortic valve (BAV) is the commonest congenital heart valve defect, found in 1% to 2% of the general population and associated with life-threatening complications. Given the high heritability index of BAV, many experts recommend echocardiography for first-degree relatives (FDRs) of an index patient. However, the cost-effectiveness of such cascade screening for BAV has not been fully evaluated. Materials and methods Using a decision-analytic model, we performed a cost-effectiveness analysis of echocardiographic screening of FDRs of BAV index cases. Data on BAV probabilities and BAV complications among FDRs were derived from our institution's BAV familial cohort and from the relevant literature on population-based BAV cohorts with long-term follow-up. Health gain was measured as quality-adjusted life years (QALYs). Cost inputs were based on list prices and literature data. One-way and probabilistic sensitivity analyses were performed to account for uncertainty in the model's variables. Results and disscusion Screening of FDRs was found to be the dominant strategy, being more effective and less costly than no screening, with savings of €208 and gains of 1.6 QALYs. Results were sensitive to the full range of reported BAV rates among FDRs across the literature, with the benefit gradually decreasing from the screening age of 55 years, with trend shifting at the age of 69. Conclusions This economic evaluation model revealed that echocardiographic screening of FDRs of BAV index case is not only clinically important but also highly cost effective and cost-saving. Health gains could be achieved from initiating screening program, along with costs saving. Sensitivity analysis supported the model's robustness, suggesting its generalization. FUNDunding Acknowledgement Type of funding sources: Public Institution(s). Main funding source(s): Center for Interdisciplinary Data Science Research fellowships grant


Author(s):  
Giovanna Bettoli ◽  
Andrew Phillips ◽  
Sudha Sundar ◽  
Carole Cummins ◽  
Anish Bali

Objective To compare current surgical practice for women with AOC to ultra-radical surgery; to assess whether the new approach would be cost-effective under NICE guidelines of approximately £20,000/QALY. Design Cost-effectiveness analysis. Setting NHS, using data from a variety of sources. Population Patients with advanced ovarian cancer (FIGO stages IIIC-IV). Methods A decision analytic model (microsimulation model) was built to examine the Objective; deterministic and probabilistic sensitivity analyses were used to test the susceptibilities of the baseline model and its assumptions. Main Outcome Measures ICER (incremental cost-effectiveness ratio). Results The standard model yielded an ICER of £5325.06; this is in spite of an associated overall decrease in utility due to predicted increase in surgical mortality. The parameters with the most significant impact on the ICER are the cost of ultra-radical surgery, the utility associated with progression-free survival, and the probability of death from ultra-radical surgery. Conclusions Ultra-radical surgery is cost-effective under NICE willingness-to-pay thresholds of £20000; the costs of ultra-radical surgery are bound to decrease as centres specialise further, and its effectiveness is also likely due to increase with development of newer techniques and more surgical training.


2019 ◽  
Vol 37 (01) ◽  
pp. 001-007 ◽  
Author(s):  
Carmen M. Avram ◽  
Leah Yieh ◽  
Dmitry Dukhovny ◽  
Aaron B. Caughey

Abstract Objective Our cost-effectiveness analysis investigated rooming-in versus not rooming-in to determine optimal management of neonates with neonatal opioid withdrawal (NOW). Study Design A decision-analytic model was constructed using TreeAge to compare rooming-in versus not rooming-in in a theoretical cohort of 23,200 newborns, the estimated annual number affected by NOW in the United States. Additional considerations included the effect of breast milk versus formula milk in evaluating the need for pharmacotherapy. Primary outcomes were needed for pharmacotherapy and neurodevelopment. We assumed a societal perspective in evaluating costs and maternal-neonatal quality-adjusted life years (QALYs) using a willingness-to-pay threshold of $100,000/QALY. Model inputs were derived from literature and varied in sensitivity analyses. Results Rooming-in resulted in fewer neonates requiring pharmacotherapy when compared with not rooming-in. The rooming-in group had more neonates with intact/mild neurodevelopmental impairment and fewer cases of moderate to severe impairment. Rooming-in resulted in cost savings of $509,652,728 and 12,333 additional QALYs per annual cohort. When the risk ratio of need for pharmacotherapy in rooming-in was varied across a clinically plausible range, rooming-in remained the cost-effective strategy. Conclusion Maternal rooming-in with newborns affected by NOW leads to reduced costs and increased effectiveness. Management strategies should optimize nonpharmacological interventions as first-line treatment.


2021 ◽  
Vol 39 (15_suppl) ◽  
pp. 8043-8043
Author(s):  
Mavis Obeng-Kusi ◽  
Daniel Arku ◽  
Neda Alrawashdh ◽  
Briana Choi ◽  
Nimer S. Alkhatib ◽  
...  

8043 Background: IXA, CAR, ELO and DARin combination with LEN+DEXhave been found superior in efficacy compared to LEN+DEX in the management of R/R MM. Applying indirect treatment comparisons from a network meta-analysis (NMA), this economic evaluation aimed to estimate the comparative cost-effectiveness and cost-utility of these four triplet regimens in terms of progression-free survival (PFS). Methods: In the absence of direct treatment comparison from a single clinical trial, NMA was used to indirectly estimate the comparative PFS benefit of each regimen. A 2-state Markov model simulating the health outcomes and costs was used to evaluate PFS life years (LY) and quality-adjusted life years (QALY) with the triplet regimens over LEN+DEX and expressed as the incremental cost-effectiveness (ICER) and cost-utility ratios (ICUR). Probability sensitivity analyses were conducted to assess the influence of parameter uncertainty on the model. Results: The NMA revealed that DAR+LEN+DEX was superior to the other triplet therapies, which did not differ statistically amongst them. As detailed in the Table, in our cost-effectiveness analysis, all 4 triplet regimens were associated with increased PFSLY and PFSQALY gained (g) over LEN+DEX at an additional cost. DAR+LEN+DEX emerged the most cost-effective with ICER and ICUR of $667,652/PFSLYg and $813,322/PFSQALYg, respectively. The highest probability of cost-effectiveness occurred at a willingness-to-pay threshold of $1,040,000/QALYg. Conclusions: Our economic analysis shows that all the triplet regimens were more expensive than LEN +DEX only but were also more effective with respect to PFSLY and PFSQALY gained. Relative to the other regimens, the daratumumab regimen was the most cost-effective.[Table: see text]


2021 ◽  
Vol 39 (6_suppl) ◽  
pp. 419-419
Author(s):  
Ali Raza Khaki ◽  
Yong Shan ◽  
Richard Nelson ◽  
Sapna Kaul ◽  
John L. Gore ◽  
...  

419 Background: Multiple single-arm clinical trials have shown promising pathologic complete response (pCR) rates with neoadjuvant ICIs in MIBC. However, ICIs remain costly. We conducted a cost-effectiveness analysis comparing neoadjuvant ICIs with CBC. Methods: We applied a decision analytic simulation model with a health care payer perspective and two-year time horizon to compare neoadjuvant ICIs vs CBC. For the primary analysis we compared pembrolizumab with dose dense methotrexate, vinblastine, doxorubicin, and cisplatin (ddMVAC). We performed a secondary analysis with gemcitabine/cisplatin (GC) as CBC and exploratory analyses with atezolizumab or nivolumab/ipilimumab as ICIs (vs both ddMVAC and GC). We input pCR rates from trials (ICIs) or a weighted average of prior studies (CBC) and costs from average sales price. Outcomes of interest included costs, 2-year recurrence-free survival (RFS), and incremental cost-effectiveness ratio (ICER) of cost per 2-year RFS. A threshold analysis estimated a pCR rate or price reduction for ICI to be cost-effective and one-way and probabilistic sensitivity analyses were performed. Results: Results of the cost effectiveness analysis are shown in the table. The incremental cost of pembrolizumab compared with ddMVAC was $8,042 resulting in an incremental improvement of 0.66% in 2-year RFS for an ICER of $1,218,485 per 2-year RFS. A pCR of 71% or a 26% reduction in cost of pembrolizumab would render it more cost-effective with an ICER of $100,000 per 2-year RFS. GC required a 96% pembrolizumab cost reduction to achieve an ICER of $100,000 per 2-year RFS. Atezolizumab appeared to be more cost-effective than ddMVAC, even though the 2yr RFS was 0.66% worse. Conclusions: ICIs were not cost-effective as neoadjuvant therapies, except when atezolizumab was compared with ddMVAC. Pembrolizumab would approach cost-effective thresholds with 26% or 96% reduction in cost when compared to ddMVAC and GC, respectively. Randomized clinical trials, larger sample sizes and longer follow-up are required to better understand the value of ICIs as neoadjuvant treatments. [Table: see text]


2021 ◽  
Author(s):  
Farzaneh Miri ◽  
Nader Jahanmehr ◽  
Reza Goudarzi

Abstract Aims: This study evaluated and compared the cost-effectiveness of rehabilitation interventions in patients with stroke in the three alternatives of hospitals, units and homes due to the fact that one of the stroke management challenges is how to provide a rehabilitation service to these patients in Iran. Methods: This is a cost-effectiveness analysis from the perspective of a health system. A Markov model with a 20-year time horizon in 3-month cycles was used to analyze the costs and outcomes. Cost data were collected from the 210 patients undergoing rehabilitation in the hospital, home and unit. Utility data were extracted from previously published literature with the same setting. The cost-effectiveness analysis was conducted by calculating ICER using TreeAge Software. Basic and probabilistic sensitivity analyses were also conducted at the end. Results: The average cost of rehabilitation in home strategy ($ 2306) was less than hospital ($2955) and unit ($3485) strategies. Furthermore, the utility of home strategy (26.03) was 8 units higher than hospital utility (17.99) and 19 units higher than utility of the stroke unit (7.03). The Acer values of hospital, stroke unit and home groups were $11424, $33159 and $7233 per utility, respectively. According to the results, the home-based rehabilitation strategy is cost effective compared to hospital and unit rehabilitation strategy. The results of the probabilistic sensitivity analysis also showed that the ICER of home strategy is always cost-effective than the other strategies. Limitation: : limitation of the present study was the reliance on utility values of other studies. Conclusion: Rehabilitation at home is the most cost-effective strategy for stroke patients. Given the high rates of this disease in Iran and the high cost of it, it is suggested that policy makers lay the groundwork for providing these services at home.


PLoS ONE ◽  
2021 ◽  
Vol 16 (12) ◽  
pp. e0260808
Author(s):  
Katherine L. Rosettie ◽  
Jonah N. Joffe ◽  
Gianna W. Sparks ◽  
Aleksandr Aravkin ◽  
Shirley Chen ◽  
...  

Cost-effectiveness analysis (CEA) is a well-known, but resource intensive, method for comparing the costs and health outcomes of health interventions. To build on available evidence, researchers are developing methods to transfer CEA across settings; previous methods do not use all available results nor quantify differences across settings. We conducted a meta-regression analysis of published CEAs of human papillomavirus (HPV) vaccination to quantify the effects of factors at the country, intervention, and method-level, and predict incremental cost-effectiveness ratios (ICERs) for HPV vaccination in 195 countries. We used 613 ICERs reported in 75 studies from the Tufts University’s Cost-Effectiveness Analysis (CEA) Registry and the Global Health CEA Registry, and extracted an additional 1,215 one-way sensitivity analyses. A five-stage, mixed-effects meta-regression framework was used to predict country-specific ICERs. The probability that HPV vaccination is cost-saving in each country was predicted using a logistic regression model. Covariates for both models included methods and intervention characteristics, and each country’s cervical cancer burden and gross domestic product per capita. ICERs are positively related to vaccine cost, and negatively related to cervical cancer burden. The mean predicted ICER for HPV vaccination is 2017 US$4,217 per DALY averted (95% uncertainty interval (UI): US$773–13,448) globally, and below US$800 per DALY averted in 64 countries. Predicted ICERs are lowest in Sub-Saharan Africa and South Asia, with a population-weighted mean ICER across 46 countries of US$706 per DALY averted (95% UI: $130–2,245), and across five countries of US$489 per DALY averted (95% UI: $90–1,557), respectively. Meta-regression analyses can be conducted on CEA, where one-way sensitivity analyses are used to quantify the effects of factors at the intervention and method-level. Building on all published results, our predictions support introducing and expanding HPV vaccination, especially in countries that are eligible for subsidized vaccines from GAVI, the Vaccine Alliance, and Pan American Health Organization.


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