scholarly journals Mapping clinical outcomes to generic preference-based outcome measures: development and comparison of methods

2020 ◽  
Vol 24 (34) ◽  
pp. 1-68 ◽  
Author(s):  
Mónica Hernández Alava ◽  
Allan Wailoo ◽  
Stephen Pudney ◽  
Laura Gray ◽  
Andrea Manca
Keyword(s):  
Cost Effectiveness ◽  
Linear Regression ◽  
Case Studies ◽  
Health Utility ◽  
Health Technology ◽  
Quality Adjusted Life Years ◽  
Indirect Methods ◽  
Direct Mapping ◽  
Life Years ◽  
Quality Adjusted Life

Background Cost-effectiveness analysis using quality-adjusted life-years as the measure of health benefit is commonly used to aid decision-makers. Clinical studies often do not include preference-based measures that allow the calculation of quality-adjusted life-years, or the data are insufficient. ‘Mapping’ can bridge this evidence gap; it entails estimating the relationship between outcomes measured in clinical studies and the required preference-based measures using a different data set. However, many methods for mapping yield biased results, distorting cost-effectiveness estimates. Objectives Develop existing and new methods for mapping; test their performance in case studies spanning different preference-based measures; and develop methods for mapping between preference-based measures. Data sources Fifteen data sets for mapping from non-preference-based measures to preference-based measures for patients with head injury, breast cancer, asthma, heart disease, knee surgery and varicose veins were used. Four preference-based measures were covered: the EuroQoL-5 Dimensions, three-level version (n = 11), EuroQoL-5 Dimensions, five-level version (n = 2), Short Form questionnaire-6 Dimensions (n = 1) and Health Utility Index Mark 3 (n = 1). Sample sizes ranged from 852 to 136,327. For mapping between generic preference-based measures, data from FORWARD, the National Databank for Rheumatic Diseases (which includes the EuroQoL-5 Dimensions, three-level version, and EuroQoL-5 Dimensions, five-level version, in its 2011 wave), were used. Main methods developed Mixture-model-based approaches for direct mapping, in which the dependent variable is the health utility value, including adaptations of methods developed to model the EuroQoL-5 Dimensions, three-level version, and beta regression mixtures, were developed, as were indirect methods, in which responses to the descriptive systems are modelled, for consistent multidirectional mapping between preference-based measures. A highly flexible approach was designed, using copulas to specify the bivariate distribution of each pair of EuroQoL-5 Dimensions, three-level version, and EuroQoL-5 Dimensions, five-level version, responses. Results A range of criteria for assessing model performance is proposed. Theoretically, linear regression is inappropriate for mapping. Case studies confirm this. Flexible, direct mapping methods, based on different variants of mixture models with appropriate underlying distributions, perform very well for all preference-based measures. The precise form is important. Case studies show that a minimum of three components are required. Covariates representing disease severity are required as predictors of component membership. Beta-based mixtures perform similarly to the bespoke mixture approaches but necessitate detailed consideration of the number and location of probability masses. The flexible, bi-directional indirect approach performs well for testing differences between preference-based measures. Limitations Case studies drew heavily on EuroQoL-5 Dimensions. Indirect methods could not be undertaken for several case studies because of a lack of coverage. These methods will often be unfeasible for preference-based measures with complex descriptive systems. Conclusions Mapping requires appropriate methods to yield reliable results. Evidence shows that widely used methods such as linear regression are inappropriate. More flexible methods developed specifically for mapping show that close-fitting results can be achieved. Approaches based on mixture models are appropriate for all preference-based measures. Some features are universally required (such as the minimum number of components) but others must be assessed on a case-by-case basis (such as the location and number of probability mass points). Future research priorities Further research is recommended on (1) the use of the monotonicity concept, (2) the mismatch of trial and mapping distributions and measurement error and (3) the development of indirect methods drawing on methods developed for mapping between preference-based measures. 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. 34. See the NIHR Journals Library website for further project information. This project was also funded by a Medical Research Council grant (MR/L022575/1).

Lenvatinib versus sorafenib for unresectable hepatocellular carcinoma: a cost–effectiveness analysis

2020 ◽  
Vol 9 (8) ◽  
pp. 553-562
Author(s):  
Hongfu Cai ◽  
Longfeng Zhang ◽  
Na Li ◽  
Bin Zheng ◽  
Maobai Liu
Keyword(s):  
Hepatocellular Carcinoma ◽  
Cost Effectiveness ◽  
Cost Effective ◽  
Quality Adjusted Life Years ◽  
Cost Effectiveness Ratio ◽  
Phase 3 ◽  
Sorafenib Treatment ◽  
Life Years ◽  
The Cost ◽  
Quality Adjusted Life

Aim: To investigate the cost–effectiveness of lenvatinib and sorafenib in the treatment of patients with nonresected hepatocellular carcinoma in China. Materials & methods: Markov model was used to simulate the direct medical cost and quality-adjusted life years (QALY) of patients with hepatocellular carcinoma. Clinical data were derived from the Phase 3 randomized clinical trial in a Chinese population. Results: Sorafenib treatment resulted in 1.794 QALYs at a cost of $43,780.73. Lenvatinib treatment resulted in 2.916 QALYs for patients weighing <60 and ≥60 kg at a cost of $57,049.43 and $75,900.36, The incremental cost–effectiveness ratio to the sorafenib treatment group was $11,825.94/QALY and $28,627.12/QALY, respectively. Conclusion: According to WHO’s triple GDP per capita, the use of lenvatinib by providing drugs is a cost-effective strategy.

scholarly journals Cost-effectiveness of introducing cone-beam computed tomography (CBCT) in the management of complex phalangeal fractures: economic simulation

2020 ◽  
Author(s):  
N. Faccioli ◽  
E. Santi ◽  
G. Foti ◽  
G. Mansueto ◽  
M. Corain
Keyword(s):  
Computed Tomography ◽  
Cost Effectiveness ◽  
Incremental Cost ◽  
Incremental Cost Effectiveness Ratio ◽  
Quality Adjusted Life Years ◽  
Cost Effectiveness Ratio ◽  
Monetary Benefit ◽  
Life Years ◽  
Net Monetary Benefit ◽  
Quality Adjusted Life

Abstract Purpose The purpose of this study was to evaluate the cost-effectiveness of introducing cone-beam computed tomography (CBCT) in the management of the complex finger fractures with articular involvement. Methods We created a decision tree model simulating the diagnostic pathway of complex finger fractures, suggesting the use of CBCT as alternative to multi-slice computed tomography (MSCT), and we compared their clinical outcomes, costs, and cost-effectiveness for a hypothetical cohort of 10,000 patients. Measures of effectiveness are analysed by using quality-adjusted life years, incremental cost-effectiveness ratio, and net monetary benefit. Results Diagnosis of a complex finger fracture performed with CBCT costed 67.33€ per patient, yielded 9.08 quality-adjusted life years, and gained an incremental cost-effectiveness ratio of 29.94€ and a net monetary benefit of 9.07 € at 30,000€ threshold. Using MSCT for diagnosis costed 106.23 €, yielded 8.18 quality-adjusted life years, and gained an incremental cost-effectiveness ratio of 371.15 € and a net monetary benefit of 8.09 €. CBCT strategy dominated the MSCT strategy. The acceptability curve shows that there is 98% probability of CBCT being the optimal strategy at 30,000€ threshold (1 EUR equal to 1.11 USD; updated on 02/02/2020). Conclusion CBCT in complex finger fractures management is cost saving compared with MSCT and may be considered a valuable imaging tool in preoperative assessment, allowing early detection and appropriate treatment. It shortens the time to completion of diagnostic work-up, reduces the number of additional diagnostic procedures, improves quality of life, and may reduce costs in a societal perspective.

scholarly journals Prevention versus early detection for long-term control of melanoma and keratinocyte carcinomas: a cost-effectiveness modelling study

BMJ Open ◽  
2020 ◽  
Vol 10 (2) ◽  
pp. e034388 ◽  
Author(s):  
Louisa Gordon ◽  
Catherine Olsen ◽  
David C Whiteman ◽  
Thomas M Elliott ◽  
Monika Janda ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Primary Prevention ◽  
Early Detection ◽  
Population Level ◽  
Population Based ◽  
Quality Adjusted Life Years ◽  
Sunscreen Use ◽  
Life Years ◽  
Quality Adjusted Life

ObjectiveTo compare the long-term economic impact of melanoma prevention by sun protection, with the corresponding impact of early detection of melanoma to decrease melanoma deaths.DesignCost-effectiveness analysis using Markov cohort model. Data were primarily from two population-based randomised controlled trials, epidemiological and costing reports, and included flow-on effects for keratinocyte cancers (previously non-melanoma skin cancers) and actinic keratoses.SettingQueensland, Australia.ParticipantsMen and women with a mean age 50 years modelled for 30 years.InterventionsDaily sunscreen use (prevention) compared with annual clinical skin examinations (early detection) and comparing these in turn with the status quo.Primary and secondary outcomesCosts, counts of melanoma, melanoma deaths, keratinocyte cancers, life years and quality-adjusted life years.ResultsPer 100 000 individuals, for early detection, primary prevention and without intervention, there were 2446, 1364 and 2419 new melanomas, 556, 341 and 567 melanoma deaths, 64 452, 47 682 and 64 659 keratinocyte cancers and £493.5, £386.4 and £406.1 million in economic costs, respectively. There were small differences between prevention and early detection in life years saved (0.09%) and quality-adjusted life years gained (0.10%).ConclusionsCompared with early detection of melanoma, systematic sunscreen use at a population level will prevent substantial numbers of new skin tumours, melanoma deaths and save healthcare costs. Primary prevention through daily use of sunscreen is a priority for investment in the control of melanoma.

scholarly journals Methods for the economic evaluation of changes to the organisation and delivery of health services: principal challenges and recommendations

2018 ◽  
Vol 14 (1) ◽  
pp. 119-134 ◽  
Author(s):  
Rachel Meacock
Keyword(s):  
Economic Evaluation ◽  
Cost Effectiveness ◽  
Health Services ◽  
Opportunity Costs ◽  
Quality Adjusted Life Years ◽  
Health Technologies ◽  
Life Years ◽  
Ex Ante ◽  
Delivery Of Health Services ◽  
Quality Adjusted Life

AbstractThere is a requirement for economic evaluation of health technologies seeking public funding across Europe. Changes to the organisation and delivery of health services, including changes to health policy, are not covered by such appraisals. These changes also have consequences for National Health Service (NHS) funds, yet undergo no mandatory cost-effectiveness assessment. The focus on health technologies may have occurred because larger-scale service changes pose more complex challenges to evaluators. This paper discusses the principal challenges faced when performing economic evaluations of changes to the organisation and delivery of health services and provides recommendations for overcoming them. The five principal challenges identified are as follows: undertakingex-anteevaluation; evaluating impacts in terms of quality-adjusted life years; assessing costs and opportunity costs; accounting for spillover effects; and generalisability. Of these challenges, methods for estimating the impact on costs and quality-adjusted life years are those most in need of development. Methods are available forex-anteevaluation, assessing opportunity costs and examining generalisability. However, these are rarely applied in practice. The general principles of assessing the cost-effectiveness of interventions should be applied to all NHS spending, not just that involving health technologies. Advancements in this area have the potential to improve the allocation of scarce NHS resources.

Cost-of-illness and economic evaluations in axial spondyloarthritis

2016 ◽  
Author(s):  
Annelies Boonen
Keyword(s):  
Cost Effectiveness ◽  
Cost Of Illness ◽  
Axial Spondyloarthritis ◽  
Economic Evaluations ◽  
Low Back ◽  
Quality Adjusted Life Years ◽  
Life Years ◽  
Methodological Principles ◽  
The Cost ◽  
Quality Adjusted Life

Consideration of costs and budgets plays an increasingly important role in decisions on access to innovative technologies. When clinicians want to influence such decisions, it is essential to understand the information on the burden of the disease and the evidence on cost-effectiveness of technologies. This chapter provides guidance to understanding the key methodological principles of economic evaluations, and describes available evidence on these issues in axial spondyloarthritis (axSpA). In the prebiologics era, the cost-of-illness for society of ankylosing spondylitis was slightly lower than for rheumatoid arthritis, and substantially lower than chronic low back pain. Cost of sick leave and work disability accounted for up to 75% of total cost-of-illness. Treatment with biologics increased cost-of-illness substantially, but the important gain in quality-adjusted life years resulted in acceptable cost-effectiveness in patients with active disease. There remains a gap in knowledge about the cost-effectiveness of diagnosing and treating axSpA earlier.

scholarly journals Using QALYs versus DALYs to measure cost-effectiveness: How much does it matter?

2020 ◽  
Vol 36 (2) ◽  
pp. 96-103 ◽  
Author(s):  
Xue Feng ◽  
David D. Kim ◽  
Joshua T. Cohen ◽  
Peter J. Neumann ◽  
Daniel A. Ollendorf
Keyword(s):  
Cost Effectiveness ◽  
Medical Center ◽  
Disability Adjusted Life Years ◽  
Quality Adjusted Life Years ◽  
Effectiveness Analysis ◽  
Disability Weights ◽  
Disability Adjusted Life ◽  
Life Years ◽  
Relative Differences ◽  
Quality Adjusted Life

ObjectivesQuality-adjusted life-years (QALYs) and disability-adjusted life-years (DALYs) are commonly used in cost-effectiveness analysis (CEA) to measure health benefits. We sought to quantify and explain differences between QALY- and DALY-based cost-effectiveness ratios, and explore whether using one versus the other would materially affect conclusions about an intervention's cost-effectiveness.MethodsWe identified CEAs using both QALYs and DALYs from the Tufts Medical Center CEA Registry and Global Health CEA Registry, with a supplemental search to ensure comprehensive literature coverage. We calculated absolute and relative differences between the QALY- and DALY-based ratios, and compared ratios to common benchmarks (e.g., 1× gross domestic product per capita). We converted reported costs into US dollars.ResultsAmong eleven published CEAs reporting both QALYs and DALYs, seven focused on pharmaceuticals and infectious disease, and five were conducted in high-income countries. Four studies concluded that the intervention was “dominant” (cost-saving). Among the QALY- and DALY-based ratios reported from the remaining seven studies, absolute differences ranged from approximately $2 to $15,000 per unit of benefit, and relative differences from 6–120 percent, but most differences were modest in comparison with the ratio value itself. The values assigned to utility and disability weights explained most observed differences. In comparison with cost-effectiveness thresholds, conclusions were consistent regardless of the ratio type in ten of eleven cases.ConclusionsOur results suggest that although QALY- and DALY-based ratios for the same intervention can differ, differences tend to be modest and do not materially affect comparisons to common cost-effectiveness thresholds.

scholarly journals Evaluation of an Extended Stroke Rehabilitation Service (EXTRAS)

Stroke ◽  
2019 ◽  
Vol 50 (12) ◽  
pp. 3561-3568 ◽  
Author(s):  
Helen Rodgers ◽  
Denise Howel ◽  
Nawaraj Bhattarai ◽  
Robin Cant ◽  
Avril Drummond ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Usual Care ◽  
Resource Utilization ◽  
Daily Living ◽  
Stroke Rehabilitation ◽  
Rehabilitation Service ◽  
Quality Adjusted Life Years ◽  
Early Supported Discharge ◽  
Life Years ◽  
Quality Adjusted Life

Background and Purpose— There is limited evidence to guide rehabilitation to meet the longer term needs of stroke survivors. The clinical effectiveness and cost-effectiveness of an extended stroke rehabilitation service (EXTRAS) provided following early supported discharge were determined. Methods— EXTRAS was a pragmatic parallel-group observer-blind randomized controlled trial involving 19 UK centers. Patients with stroke were individually randomized to receive EXTRAS or usual care at discharge from early supported discharge. Five EXTRAS reviews were provided by an early supported discharge team member between one and 18 months, usually by telephone. Reviews consisted of a semi-structured interview assessing progress, rehabilitation needs, and service provision, with goal setting and action planning. The primary outcome was performance in extended activities of daily living (Nottingham EADL Scale) at 24 months post-randomization. The Nottingham EADL Scale is scored 0 to 66, with higher scores indicating better performance in these activities. Cost-effectiveness was estimated using resource utilization costs and Quality Adjusted Life Years. Analyses were intention to treat. Results— Between January 9, 2013 and October 26, 2015, 573 participants were randomized (EXTRAS, n=285; usual care, n=288). Mean 24 month Nottingham EADL Scale scores were EXTRAS (n=219) 40.0 (SD 18.1) and usual care (n=231) 37.2 (SD 18.5) giving an adjusted mean difference of 1.8 (95% CI, –0.7 to 4.2). 1155/1338 (86%) of expected EXTRAS reviews were undertaken. Over 24 months, the mean cost of resource utilization was lower in the intervention group: –£311 (–$450 [95% CI, −£3292 to £2787; −$4764 to $4033]). EXTRAS provided more Quality Adjusted Life Years (0.07 [95% CI, 0.01 to 0.12]). At current conventional thresholds of willingness to pay (£20 000 [$28 940] per Quality Adjusted Life Years), there was a 90% chance that EXTRAS could be considered cost-effective. Conclusions— EXTRAS did not significantly improve stroke survivors’ performance in extended activities of daily living. However, given the impact on costs and Quality Adjusted Life Years, EXTRAS may be an affordable addition to improve stroke care. Clinical Trial Registration— URL: www.isrctn.com . Unique identifier: ISRCTN45203373.

scholarly journals Incremental cost-effectiveness of adult spinal deformity surgery: observed quality-adjusted life years with surgery compared with predicted quality-adjusted life years without surgery

2014 ◽  
Vol 36 (5) ◽  
pp. E3 ◽  
Author(s):  
Ian McCarthy ◽  
Michael O'Brien ◽  
Christopher Ames ◽  
Chessie Robinson ◽  
Thomas Errico ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Surgical Treatment ◽  
Incremental Cost ◽  
Adult Spinal Deformity ◽  
Quality Adjusted Life Years ◽  
Life Years ◽  
Nonsurgical Patients ◽  
The Cost ◽  
Quality Adjusted Life

Object Incremental cost-effectiveness analysis is critical to the efficient allocation of health care resources; however, the incremental cost-effectiveness ratio (ICER) of surgical versus nonsurgical treatment for adult spinal deformity (ASD) has eluded the literature, due in part to inherent empirical difficulties when comparing surgical and nonsurgical patients. Using observed preoperative health-related quality of life (HRQOL) for patients who later underwent surgery, this study builds a statistical model to predict hypothetical quality-adjusted life years (QALYs) without surgical treatment. The analysis compares predicted QALYs to observed postoperative QALYs and forms the resulting ICER. Methods This was a single-center (Baylor Scoliosis Center) retrospective analysis of consecutive patients undergoing primary surgery for ASD. Total costs (expressed in 2010 dollars) incurred by the hospital for each episode of surgical care were collected from administrative data and QALYs were calculated from the 6-dimensional Short-Form Health Survey, each discounted at 3.5% per year. Regression analysis was used to predict hypothetical QALYs without surgery based on preoperative longitudinal data for 124 crossover surgical patients with similar diagnoses, baseline HRQOL, age, and sex compared with the surgical cohort. Results were projected through 10-year follow-up, and the cost-effectiveness acceptability curve (CEAC) was estimated using nonparametric bootstrap methods. Results Three-year follow-up was available for 120 (66%) of 181 eligible patients, who were predominantly female (89%) with average age of 50. With discounting, total costs averaged $125,407, including readmissions, with average QALYs of 1.93 at 3-year follow-up. Average QALYs without surgery were predicted to be 1.6 after 3 years. At 3- and 5-year follow-up, the ICER was $375,000 and $198,000, respectively. Projecting through 10-year follow-up, the ICER was $80,000. The 10-year CEAC revealed a 40% probability that the ICER was $80,000 or less, a 90% probability that the ICER was $90,000 or less, and a 100% probability that the ICER was less than $100,000. Conclusions Based on the WHO's suggested upper threshold for cost-effectiveness (3 times per capita GDP, or $140,000 in 2010 dollars), the analysis reveals that surgical treatment for ASD is cost-effective after a 10-year period based on predicted deterioration in HRQOL without surgery. The ICER well exceeds the WHO threshold at earlier follow-up intervals, highlighting the importance of the durability of surgical treatment in assessing the value of surgical intervention. Due to the study's methodology, the results are dependent on the predicted deterioration in HRQOL without surgery. As such, the results may not extend to patients whose HRQOL would remain steady without surgery. Future research should therefore pursue a direct comparison of QALYs for surgical and nonsurgical patients to better understand the cost-effectiveness of surgery for the average ASD patient.

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.

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