scholarly journals Comparing Methods for Handling Missing Cost and Outcome Data in Clinical Trial-based Cost-effectiveness Analysis

2021 ◽  
Author(s):  
Modou Diop ◽  
David Epstein
Keyword(s):  
Clinical Trial ◽  
Cost Effectiveness ◽  
Repeated Measures ◽  
Cost Effectiveness Analysis ◽  
Simulation Studies ◽  
Time Horizons ◽  
Effectiveness Analysis ◽  
Life Years ◽  
Significant Difference ◽  
Better Than

Abstract OBJECTIVES: This study compares methods for handling missing data to conduct cost-effectiveness analysis in the context of a clinical study.METHODS: A long-term clinical trial with staggered recruitment was used as a case study (EVRA, NIHR-HTA project 11/129/197). Patients had between 1 year and 5.5 years (median 3 years) of follow-up under “early” or “deferred” treatment. The methods compared were Complete-Case-Analysis (CCA), multiple imputation using linear regression (MILR) and using predictive mean matching (MIPMM), Bayesian parametric approach using the R package missingHE (BPA) and repeated measures mixed model (RMM). The outcomes were total mean costs and total mean quality-adjusted life years (QALYs) at different time horizons (1 year, 3 years and 5 years). RESULTS: All methods found no statistically significant difference in cost at the 5% level in all years, and all methods found statistically significantly greater mean QALY at year 1. At year 3, BPA showed a statistically significant difference while other method did not at later time horizons. Standard errors differed substantially between the methods employed. CONCLUSION: MIPMM seemed to perform better than MILM, confirming findings from simulation studies. RMM and BPA might be feasible options, though they did not perform better than MIPMM in this dataset. Further simulation studies and applications should continue to compare these methods.

scholarly journals Comparing Methods for Handling Missing Cost and Outcome Data in Clinical Trial-based Cost-effectiveness Analysis

2021 ◽  
Author(s):  
Modou Diop ◽  
David Epstein
Keyword(s):  
Cost Effectiveness ◽  
Repeated Measures ◽  
Cost Effectiveness Analysis ◽  
Sensitivity Analyses ◽  
Venous Ulceration ◽  
Time Horizons ◽  
Effectiveness Analysis ◽  
Life Years ◽  
Significant Difference ◽  
Endovenous Ablation

Abstract OBJECTIVES: This study compares methods for handling missing data to conduct cost-effectiveness analysis in the context of a clinical study.METHODS: Patients in the Early Endovenous Ablation in Venous Ulceration (EVRA) trial had between 1 year and 5.5 years (median 3 years) of follow-up under early or deferred endovenous ablation. This study compares Complete-Case-Analysis (CCA), multiple imputation using linear regression (MILR) and using predictive mean matching (MIPMM), Bayesian parametric approach using the R package missingHE (BPA) and repeated measures mixed model (RMM). The outcomes were total mean costs and total mean quality-adjusted life years (QALYs) at different time horizons (1 year, 3 years and 5 years). RESULTS: All methods found no statistically significant difference in cost at the 5% level in all time horizons, and all methods found statistically significantly greater mean QALY at year 1. By year 3, only BPA showed a statistically significant difference in QALY. Standard errors differed substantially between the methods employed. CONCLUSION: CCA can be biased if data are MAR, and is wasteful of the data. Hence the results for CCA are likely to be inaccurate. Other methods coincide in suggesting that early intervention is cost-effective at a threshold of £20,000 per QALY over all time horizons. However, the variation in the results across the methods does generate some additional methodological uncertainty, underlining the importance of conducting sensitivity analyses using alternative approaches.

Cost-effectiveness Analysis of Submandibular Gland Preservation With Sialendoscopy for the Management of Sialolithiasis

2021 ◽  
pp. 019459982110268
Author(s):  
Joseph R. Acevedo ◽  
Ashley C. Hsu ◽  
Jeffrey C. Yu ◽  
Dale H. Rice ◽  
Daniel I. Kwon ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Submandibular Gland ◽  
Average Cost ◽  
Cost Effective ◽  
Cost Effectiveness Analysis ◽  
Effectiveness Analysis ◽  
Life Years ◽  
Probability Of Success ◽  
Markov Decision Model ◽  
The Cost

Objective To compare the cost-effectiveness of sialendoscopy with gland excision for the management of submandibular gland sialolithiasis. Study Design Cost-effectiveness analysis. Setting Outpatient surgery centers. Methods A Markov decision model compared the cost-effectiveness of sialendoscopy versus gland excision for managing submandibular gland sialolithiasis. Surgical outcome probabilities were found in the primary literature. The quality of life of patients was represented by health utilities, and costs were estimated from a third-party payer’s perspective. The effectiveness of each intervention was measured in quality-adjusted life-years (QALYs). The incremental costs and effectiveness of each intervention were compared, and a willingness-to-pay ratio of $150,000 per QALY was considered cost-effective. One-way, multivariate, and probabilistic sensitivity analyses were performed to challenge model conclusions. Results Over 10 years, sialendoscopy yielded 9.00 QALYs at an average cost of $8306, while gland excision produced 8.94 QALYs at an average cost of $6103. The ICER for sialendoscopy was $36,717 per QALY gained, making sialendoscopy cost-effective by our best estimates. The model was sensitive to the probability of success and the cost of sialendoscopy. Sialendoscopy must meet a probability-of-success threshold of 0.61 (61%) and cost ≤$11,996 to remain cost-effective. A Monte Carlo simulation revealed sialendoscopy to be cost-effective 60% of the time. Conclusion Sialendoscopy appears to be a cost-effective management strategy for sialolithiasis of the submandibular gland when certain thresholds are maintained. Further studies elucidating the clinical factors that determine successful sialendoscopy may be aided by these thresholds as well as future comparisons of novel technology.

scholarly journals Systematic review of cost-effectiveness analysis of behavior change communication apps: Assessment of key methods

2021 ◽  
Vol 7 ◽  
pp. 205520762110005
Author(s):  
Cynthia Afedi Hazel ◽  
Sheana Bull ◽  
Elizabeth Greenwell ◽  
Maya Bunik ◽  
Jini Puma ◽  
...  
Keyword(s):  
Systematic Review ◽  
Cost Effectiveness ◽  
Behavior Change ◽  
Quantitative Measure ◽  
Cost Effectiveness Analysis ◽  
Behavior Change Communication ◽  
Change Communication ◽  
Effectiveness Analysis ◽  
Life Years ◽  
The Cost

Objective Evidence backing the effectiveness of mobile health technology is growing, and behavior change communication applications (apps) are fast becoming a useful platform for behavioral health programs. However, data to support the cost-effectiveness of these interventions are limited. Suggestions for overcoming the low output of economic data include addressing the methodological challenges for conducting cost-effectiveness analysis of behavior change app programs. This study is a systematic review of cost-effectiveness analyses of behavior change communication apps and a documentation of the reported challenges for investigating their cost-effectiveness. Materials and methods Four academic databases: Medline (Ovid), CINAHL, EMBASE and Google Scholar, were searched. Eligibility criteria included original articles that use a cost-effectiveness evaluation method, published between 2008 and 2018, and in the English language. Results Out of the 60 potentially eligible studies, 6 used cost-effectiveness analysis method and met the inclusion criteria. Conclusion The evidence to support the cost-effectiveness of behavior change communication apps is insufficient, with all studies reporting significant study challenges for estimating program costs and outcomes. The main challenges included limited or lack of cost data, inappropriate cost measures, difficulty with identifying and quantifying app effectiveness, representing app effects as Quality-adjusted Life Years, and aggregating cost and effects into a single quantitative measure like Incremental Cost Effectiveness Ratio. These challenges highlight the need for comprehensive economic evaluation methods that balance app data quality issues with practical concerns. This would likely improve the usefulness of cost-effectiveness data for decisions on adoption, implementation, scalability, sustainability, and the benefits of broader healthcare investments.

scholarly journals Cost-effectiveness analysis of nonoperative management versus open and laparoscopic surgery for uncomplicated acute appendicitis in Colombia

2021 ◽  
Vol 19 (1) ◽  
Author(s):  
César Augusto Guevara-Cuellar ◽  
María Paula Rengifo-Mosquera ◽  
Elizabeth Parody-Rúa
Keyword(s):  
Cost Effectiveness ◽  
Nonoperative Management ◽  
Open Appendectomy ◽  
Cost Effectiveness Analysis ◽  
Monetary Benefit ◽  
Effectiveness Analysis ◽  
Payer Perspective ◽  
Life Years ◽  
Uncomplicated Acute Appendicitis ◽  
Net Monetary Benefit

Abstract Background Traditionally, uncomplicated acute appendicitis (AA) has been treated with appendectomy. However, the surgical alternatives might carry out significant complications, impaired quality of life, and higher costs than nonoperative treatment. Consequently, it is necessary to evaluate the different therapeutic alternatives' cost-effectiveness in patients diagnosed with uncomplicated appendicitis. Methods We performed a model-based cost-effectiveness analysis comparing nonoperative management (NOM) with open appendectomy (OA) and laparoscopic appendectomy (LA) in patients otherwise healthy adults aged 18–60 years with a diagnosis of uncomplicated AA from the payer´s perspective at the secondary and tertiary health care level. The time horizon was 5 years. A discount rate of 5% was applied to both costs and outcomes. The health outcomes were quality-adjusted life years (QALYs). Costs were identified, quantified, and valorized from a payer perspective; therefore, only direct health costs were included. An incremental analysis was estimated to determine the incremental cost-effectiveness ratio (ICER). In addition, the net monetary benefit (NMB) was calculated for each alternative using a willingness to pay lower than one gross domestic product. A deterministic and probabilistic sensitivity analysis was performed. Methods We performed a model-based cost-effectiveness analysis comparing nonoperative management (NOM) with open appendectomy (OA) and laparoscopic appendectomy (LA) in patients otherwise healthy adults aged 18–60 years with a diagnosis of uncomplicated AA from the payer’s perspective at the secondary and tertiary health care level. The time horizon was five years. A discount rate of 5% was applied to both costs and outcomes. The health outcomes were quality-adjusted life years (QALYs). Costs were identified, quantified, and valorized from a payer perspective; therefore, only direct health costs were included. An incremental analysis was estimated to determine the incremental cost-effectiveness ratio (ICER). In addition, the net monetary benefit (NMB) was calculated for each alternative using a willingness to pay lower than one gross domestic product. A deterministic and probabilistic sensitivity analysis was performed. Results LA presents a lower cost ($363 ± 35) than OA ($384 ± 41) and NOM ($392 ± 44). NOM exhibited higher QALYs (3.3332 ± 0.0276) in contrast with LA (3.3310 ± 0.057) and OA (3.3261 ± 0.0707). LA dominated the OA. The ICER between LA and NOM was $24,000/QALY. LA has a 52% probability of generating the highest NMB versus its counterparts, followed by NOM (30%) and OA (18%). There is a probability of 0.69 that laparoscopy generates more significant benefit than medical management. The mean value of that incremental NMB would be $93.7 per patient. Conclusions LA is a cost-effectiveness alternative in the management of patients with uncomplicated AA. Besides, LA has a high probability of producing more significant monetary benefits than NOM and OA from the payer’s perspective in the Colombian health system.

Ixazomib (IXA), carfilzomib (CAR), elotuzumab (ELO) or daratumumab (DAR) with lenalidomide and dexamethasone (LEN+DEX) versus LEN+DEX only in relapsed/refractory multiple myeloma (R/R MM): A comparative cost-effectiveness analysis.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. 8043-8043
Author(s):  
Mavis Obeng-Kusi ◽  
Daniel Arku ◽  
Neda Alrawashdh ◽  
Briana Choi ◽  
Nimer S. Alkhatib ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Cost Effective ◽  
Cost Utility ◽  
Cost Effectiveness Analysis ◽  
The Other ◽  
Sensitivity Analyses ◽  
Treatment Comparison ◽  
Comparative Cost ◽  
Effectiveness Analysis ◽  
Life Years

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]

scholarly journals Heterogeneity in Cost-Effectiveness Analysis of Vaccination for Mild and Moderate Alzheimer’s Disease

2019 ◽  
Vol 16 (6) ◽  
pp. 495-504
Author(s):  
Chung-Hsien Lin ◽  
Jean Ching-Yuan Fann ◽  
Sam Li-Sheng Chen ◽  
Hsiu-Hsi Chen ◽  
Kuen-Cheh Yang
Keyword(s):  
Alzheimer’S Disease ◽  
Alzheimer's Disease ◽  
Cost Effectiveness ◽  
Age Groups ◽  
Cost Effectiveness Analysis ◽  
Age And Gender ◽  
Effectiveness Analysis ◽  
Population Average ◽  
Life Years ◽  
And Gender

Background:Immunotherapy for Alzheimer’s disease(AD) has gained momentum in recent years. One of the concerns over its application pertains to Cost-Effectiveness Analysis (CEA) from population average and specific subgroup differences, as such a therapy is imperative for health decisionmakers to allocate limited resources. However, this sort of CEA model considering heterogeneous population with risk factors adjustment has been rarely addressed.Methods:We aimed to show the heterogeneity of CEA in immunotherapy for AD in comparison with the comparator without intervention. Economic evaluation was performed via incremental Cost- Effectiveness Ratio (ICER) and Cost-Effectiveness Acceptability Curve (CEAC) in terms of the Quality- Adjusted Life Years (QALY). First, population-average CEA was performed with and without adjustment for age and gender. Secondly, sub-group CEA was performed with the stratification of gender and age based on Markov process.Results:Given the threshold of $20,000 of willingness to pay, the results of ICER without and with adjustment for age and gender revealed similar results ($14,691/QALY and $17,604/QALY). The subgroup ICER results by different age groups and gender showed substantial differences. The CEAC showed that the probability of being cost-effective was only 48.8%-53.3% in terms of QALY at population level but varied from 83.5% in women aged 50-64 years, following women aged 65-74 years and decreased to 0.2% in men≥ 75 years.Conclusion:There were considerable heterogeneities observed in the CEA of vaccination for AD. As with the development of personalized medicine, the CEA results assessed by health decision-maker should not only be considered by population-average level but also specific sub-group levels.

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