scholarly journals A Bayesian Framework for Patient-Level Partitioned Survival Cost-Utility Analysis

2021 ◽  
pp. 0272989X2110123
Author(s):  
Andrea Gabrio
Keyword(s):  
Cost Effectiveness ◽  
Bayesian Framework ◽  
Cost Utility ◽  
Policy Makers ◽  
Patient Level ◽  
Clinical Benefits ◽  
New Treatment ◽  
The Cost ◽  
Evidence For Policy

Patient-level health economic data collected alongside clinical trials are an important component of the process of technology appraisal. For end-of-life treatments, the modeling of cost-effectiveness data may involve some form of partitioned survival analysis, in which measures of quality of life and survival for pre- and postprogression periods are combined to generate aggregate measures of clinical benefits (e.g., quality-adjusted survival). In addition, resource use data are often collected and costs are calculated for each type of health service (e.g., treatment, hospital, or adverse events costs). A critical problem in these analyses is that effectiveness and cost data present some complexities, such as nonnormality, spikes, and missingness, which should be addressed using appropriate methods to avoid biased results. This article proposes a general Bayesian framework that takes into account the complexities of trial-based partitioned survival cost-utility data to provide more adequate evidence for policy makers. Our approach is motivated by, and applied to, a working example based on data from a trial assessing the cost-effectiveness of a new treatment for patients with advanced non–small-cell lung cancer. [Box: see text]

scholarly journals Cost-effectiveness of treatment optimisation with biomarkers for immunotherapy in solid tumours: a systematic review protocol

BMJ Open ◽  
2021 ◽  
Vol 11 (9) ◽  
pp. e048141
Author(s):  
Sara Mucherino ◽  
Valentina Lorenzoni ◽  
Valentina Orlando ◽  
Isotta Triulzi ◽  
Marzia Del Re ◽  
...  
Keyword(s):  
Systematic Review ◽  
Cost Effectiveness ◽  
Immune Checkpoint ◽  
Cost Utility ◽  
Solid Tumours ◽  
Economic Evaluations ◽  
Monetary Benefit ◽  
The Cost ◽  
Net Monetary Benefit

IntroductionThe combination of biomarkers and drugs is the subject of growing interest both from regulators, physicians and companies. This study protocol of a systematic review is aimed to describe available literature evidences about the cost-effectiveness, cost-utility or net-monetary benefit of the use of biomarkers in solid tumour as tools for customising immunotherapy to identify what further research needs.Methods and analysisA systematic review of the literature will be carried out according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses statement guidelines. PubMed and Embase will be queried from June 2010 to June 2021. The PICOS model will be applied: target population (P) will be patients with solid tumours treated with immune checkpoint inhibitors (ICIs); the interventions (I) will be test of the immune checkpoint predictive biomarkers; the comparator (C) will be any other targeted or non-targeted therapy; outcomes (O) evaluated will be health economic and clinical implications assessed in terms of incremental cost-effectiveness ratio, net health benefit, net monetary benefit, life years gained, quality of life, etc; study (S) considered will be economic evaluations reporting cost-effectiveness analysis, cost-utility analysis, net-monetary benefit. The quality of the evidence will be graded according to Grading of Recommendations Assessment, Development and Evaluation.Ethics and disseminationThis systematic review will assess the cost-effectiveness implications of using biomarkers in the immunotherapy with ICIs, which may help to understand whether this approach is widespread in real clinical practice. This research is exempt from ethics approval because the work is carried out on published documents. We will disseminate this protocol in a related peer-reviewed journal.PROSPERO registration numberCRD42020201549.

scholarly journals Economic evaluation of online computerised cognitive–behavioural therapy without support for depression in primary care: randomised trial

2010 ◽  
Vol 196 (4) ◽  
pp. 310-318 ◽  
Author(s):  
S. A. H. Gerhards ◽  
L. E. de Graaf ◽  
L. E. Jacobs ◽  
J. L. Severens ◽  
M. J. H. Huibers ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Cost Effectiveness ◽  
Cognitive Behavioural Therapy ◽  
Behavioural Therapy ◽  
Cost Utility ◽  
Significant Group ◽  
Efficient Treatment ◽  
Cognitive Behavioural ◽  
The Cost

BackgroundEvidence about the cost-effectiveness and cost utility of computerised cognitive–behavioural therapy (CCBT) is still limited. Recently, we compared the clinical effectiveness of unsupported, online CCBT with treatment as usual (TAU) and a combination of CCBT and TAU (CCBT plus TAU) for depression. The study is registered at the Netherlands Trial Register, part of the Dutch Cochrane Centre (ISRCTN47481236).AimsTo assess the cost-effectiveness of CCBT compared with TAU and CCBT plus TAU.MethodCosts, depression severity and quality of life were measured for 12 months. Cost-effectiveness and cost-utility analyses were performed from a societal perspective. Uncertainty was dealt with by bootstrap replications and sensitivity analyses.ResultsCosts were lowest for the CCBT group. There are no significant group differences in effectiveness or quality of life. Cost-utility and cost-effectiveness analyses tend to be in favour of CCBT.ConclusionsOn balance, CCBT constitutes the most efficient treatment strategy, although all treatments showed low adherence rates and modest improvements in depression and quality of life.

A Systematic Review of the Cost-Effectiveness of Recombinant Factor VIIa (rFVIIa) and Activated Prothrombin Complex Concentrate (aPCC) in the Treatment of Mild to Moderate Bleeding Episodes for Haemophilia Patients with Inhibitors

Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 4678-4678
Author(s):  
Chris Knight ◽  
Anne Møller Danø ◽  
Tessa Kennedy-Martin
Keyword(s):  
Systematic Review ◽  
Cost Effectiveness ◽  
Factor Viia ◽  
Cost Effective ◽  
Primary Treatment ◽  
Cost Utility ◽  
Economic Consequences ◽  
Modelling Framework ◽  
The Cost

Abstract Objectives: Although haemophilia patients with inhibitors are rare, the clinical, humanistic and economic consequences associated with this disorder are considerable. The primary treatment for such patients is either rFVIIa or aPCC. The aim of this study was to identify, review and evaluate the quality of the published literature on the relative cost-effectiveness of rFVIIa and aPCC in treating haemophilia patients with inhibitors. Methods: The review concentrates on the model type, the model design, model assumptions, and results. Results: The results of this study suggest that rFVIIa may be the cost-effective alternative to treatment with aPCC due to the superior efficacy of rFVIIa and hence the avoidance of subsequent lines of treatment. In 7 of the 9 studies, rFVIIa had the lower average treatment cost. The adapted modelling framework is similar in all the economic models reviewed, suggesting clinical acceptability of the approach used. The estimates of efficacy varied between the models, especially for aPCC. The efficacy for aPCC derived from retrospective studies was lower than reported in the literature. Sensitivity analysis had been undertaken in the majority of the economic analyses and the results were found to be robust to realistic parameter variations. Only one of the studies was a cost-utility study, showing the lack of measuring health status within this area. The results showed the large impact appropriate treatment can have on the quality of life for haemophilia patients with inhibitors. Conclusions: Ideally, there should be a systematic approach to identifying the relevant data and the lack of data from relevant randomized head-to-head trials is a contributing factor to the variation in efficacy rates and average dosages assumed. However, this systematic review has shown that despite differences in the estimates of efficacy, average dosage required, and unit costs the overall results are robust and appear to favour rFVIIa as the cost-effectiveness treatment for haemophilia patients with inhibitors.

Model to assess the cost-effectiveness of new treatments for depression

2006 ◽  
Vol 22 (4) ◽  
pp. 469-477 ◽  
Author(s):  
Patrik Sobocki ◽  
Mattias Ekman ◽  
Hans Ågren ◽  
Bengt Jönsson ◽  
Clas Rehnberg
Keyword(s):  
Quality Of Life ◽  
Cost Effectiveness ◽  
Standard Care ◽  
Transition Probabilities ◽  
Sensitivity Analyses ◽  
Full Remission ◽  
Life Years ◽  
New Treatment ◽  
The Cost

Objectives: The objective of this study was to develop a model to assess the cost-effectiveness of a new treatment for patients with depression.Methods: A Markov simulation model was constructed to evaluate standard care for depression as performed in clinical practice compared with a new treatment for depression. Costs and effects were estimated for time horizons of 6 months to 5 years. A naturalistic longitudinal observational study provided data on costs, quality of life, and transition probabilities. Data on long-term consequences of depression and mortality risks were collected from the literature. Cost-effectiveness was quantified as quality-adjusted life-years (QALYs) gained from the new treatment compared with standard care, and the societal perspective was taken. Probabilistic analyses were conducted to present the uncertainty in the results, and sensitivity analyses were conducted on key parameters used in the model.Results: Compared with standard care, the new hypothetical therapy was predicted to substantially decrease costs and was also associated with gains in QALYs. With an improved treatment effect of 50 percent on achieving full remission, the net cost savings were 20,000 Swedish kronor over a 5-year follow-up time, given equal costs of treatments. Patients gained .073 QALYs over 5 years. The results are sensitive to changes in assigned treatment effects.Conclusions: The present study provides a new model for assessing the cost-effectiveness of treatments for depression by incorporating full remission as the treatment goal and QALYs as the primary outcome measure. Moreover, we show the usefulness of naturalistic real-life data on costs and quality of life and transition probabilities when modeling the disease over time.

scholarly journals A Systematic Review of Economic Analyses Studying Rituximab in R-CHOP Therapy in Patients with Non-Hodgkin Lymphoma

2009 ◽  
Vol 2 (1) ◽  
pp. 1-9 ◽  
Author(s):  
Jenna L. Yoder ◽  
Khalid M. Kamal
Keyword(s):  
Cost Effectiveness ◽  
Hodgkin Lymphoma ◽  
Cost Utility ◽  
Economic Evaluations ◽  
Non Hodgkin Lymphoma ◽  
Life Years ◽  
The Cost ◽  
Economic Studies ◽  
Chop Therapy

Objectives: To explore the use of pharmacoeconomic principles through examination of economic evaluations pertaining to the combination of the monoclonal antibody rituximab with conventional CHOP (cyclophosphamide/doxorubicin/ vincristine/ prednisone) or CHOP-like chemotherapy regimens in patients with non-Hodgkin lymphoma. Methods: A literature search was conducted using Evidence-Based Medical Reviews (EBMR), International Pharmaceutical Abstracts (IPA), and Medline databases to identify all economic studies relating to rituximab in combination with CHOP or CHOP-like regimens. The systematic evaluation also utilized the Quality of Health Economic Studies instrument to assess the quality of each study that was included in the final review. Results: Initially, eight studies were retrieved which included the use of rituximab in non-Hodgkin lymphoma treatment. Of these, four studies were excluded as rituximab was used as a stand-alone treatment option. The remaining four studies involved conventional CHOP therapy versus the combination with rituximab (R-CHOP) in patients with non-Hodgkin lymphoma. One study employed a cost-effectiveness analysis while the remaining three studies used a cost-utility analysis and reported the outcomes in terms of quality-adjusted life years (QALYs). Conclusions: The cost-effectiveness evaluation illustrated the dominance of R-CHOP over CHOP-alone in terms of both lower costs and increased life years gained. The cost-utility of R-CHOP in terms of costs/QALYs were below the accepted threshold of 50,000 in international monetary units. Through examination of evaluation principles employed, it is found that valid results are highly dependent on the input data, assumptions, and sensitivity analyses. Clinical decisionmakers must take into account specific inclusions of costs relevant to their own practice setting.

Economic evaluation of drug-eluting stents: A systematic literature review and model-based cost–utility analysis

2007 ◽  
Vol 23 (4) ◽  
pp. 473-479 ◽  
Author(s):  
Pekka Kuukasjärvi ◽  
Pirjo Räsänen ◽  
Antti Malmivaara ◽  
Pasi Aronen ◽  
Harri Sintonen
Keyword(s):  
Cost Effectiveness ◽  
Incremental Cost ◽  
Cost Effective ◽  
Cost Utility ◽  
Drug Eluting Stents ◽  
Economic Evaluations ◽  
Model Based ◽  
Drug Eluting ◽  
The Cost

Objectives:The aim of this study was to systematically review economic analyses comparing drug-eluting stents (DES) to bare metal stents (BMS) in patients who undergo percutaneous coronary intervention to form an overall view about cost-effectiveness of DES and to construct a simple decision analysis model to evaluate the cost–utility of DES.Methods:Electronic databases searched from January 2004 to January 2006 were Cochrane Database of Systematic Reviews; DARE, HTA, EED (NHS CRD); MEDLINE(R) In-Process, Other Non-Indexed Citations, MEDLINE(R). References of the papers identified were checked. We included randomized controlled trials (RCT) or model-based cost-effectiveness analyses comparing DES to BMS in patients with coronary artery disease. The methodological quality of the papers was assessed by Drummond's criteria. Baseline characteristics and results of the studies were extracted and data synthesized descriptively. A decision tree model was constructed to evaluate the cost–utility of DES in comparison to BMS, where health-related quality of life was measured by the 15D.Results:We identified thirteen good-quality economic evaluations. In two of these based on RCTs, DES was found cost-effective. In six studies, it was concluded that DES might probably be a cost-effective strategy in some circumstances, but not as a single strategy, and four studies concluded that DES is not cost-effective. One study did not draw a clear conclusion. In our analysis, the overall incremental cost-effectiveness ratio was €98,827 per quality-adjusted life-years gained. Avoiding one revascularization with DES would cost €4,794, when revascularization with BMS costs €3,260.Conclusions:The evidence is inconsistent of whether DES would be a cost-effective treatment compared with BMS in any healthcare system where evaluated. A marked restenosis risk reduction should be achieved before use of DES is justifiable at present prices. When considering adoption of a new health technology with a high incremental cost within a fixed budget, opportunity cost in terms of untreated patients should be seriously considered as a question of collective ethics.

Cost-Utility Analysis of Preoperative Radiotherapy in Patients With Rectal Cancer Undergoing Total Mesorectal Excision: A Study of the Dutch Colorectal Cancer Group

2004 ◽  
Vol 22 (2) ◽  
pp. 244-253 ◽  
Author(s):  
Mandy van den Brink ◽  
Wilbert B. van den Hout ◽  
Anne M. Stiggelbout ◽  
Elma Klein Kranenbarg ◽  
Corrie A.M. Marijnen ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Rectal Cancer ◽  
Cost Effectiveness ◽  
Life Expectancy ◽  
Preoperative Radiotherapy ◽  
Cost Utility ◽  
Cost Effectiveness Ratio ◽  
The Cost ◽  
Quality Adjusted Life

Purpose To compare the societal costs and the (quality-adjusted) life expectancy of patients with rectal cancer undergoing total mesorectal excision (TME) with or without short-term preoperative radiotherapy (5 × 5 Gy). Patients and Methods We used a Markov model to project the clinical and economic outcomes of preoperative radiotherapy. Data on local recurrence rates, quality of life, and costs were obtained from the patients of a multicenter randomized clinical trial. In this trial, 1,861 patients with resectable rectal cancer from 108 hospitals were randomly assigned for TME surgery with or without preoperative radiotherapy. Outcome measures of the model were life expectancy, quality-adjusted life expectancy, lifetime costs per patient, and the incremental cost-effectiveness ratio. Results The base case model estimates that the loss of quality of life due to preoperative radiotherapy is outweighed by the gain in life expectancy. Life expectancy increases by 0.67 years; quality-adjusted life expectancy, by 0.39 years; and costs, by $9,800 per patient. The corresponding cost-effectiveness ratio is $25,100 per quality-adjusted life year. Sensitivity analyses indicate that the cost-effectiveness ratio remains acceptable under a wide range of assumptions. Conclusion Assuming that the reduced local recurrence rate does lead to a survival advantage, the cost-utility analysis estimates that the improved survival outweighs the impaired quality of life and the increased costs. We conclude that short-term preoperative radiotherapy in patients with rectal cancer undergoing TME is both effective and cost-effective.

scholarly journals Influence of Patient Preferences on the Cost-Effectiveness of Screening for Lynch Syndrome

2012 ◽  
Vol 8 (3S) ◽  
pp. e24s-e30s ◽  
Author(s):  
Grace Wang ◽  
Miriam Kuppermann ◽  
Benjamin Kim ◽  
Kathryn A. Phillips ◽  
Uri Ladabaum
Keyword(s):  
Quality Of Life ◽  
Colorectal Cancer ◽  
Cost Effectiveness ◽  
Lynch Syndrome ◽  
Patient Preferences ◽  
Cost Utility ◽  
Cost Utility Analysis ◽  
Utility Analysis ◽  
The Cost

This cost-utility analysis reports on the effect of quality of life on the value of screening all new patients with colorectal cancer for Lynch syndrome.

scholarly journals Assessment of the Quality of Cost Analysis Literature in Physical Therapy

2009 ◽  
Vol 89 (8) ◽  
pp. 733-755 ◽  
Author(s):  
Laura E. Peterson ◽  
Clifford Goodman ◽  
Erin K. Karnes ◽  
Charlene J. Chen ◽  
J. Amanda Schwartz
Keyword(s):  
Cost Effectiveness ◽  
Physical Therapy ◽  
Cost Analysis ◽  
Cost Effective ◽  
Evaluation Tool ◽  
Policy Makers ◽  
The Cost ◽  
Effectiveness Studies ◽  
Economic Studies

Background Policy makers, payers, and other stakeholders increasingly call for greater evidence of the cost-effectiveness of health care interventions. Objective The purposes of this study were to identify and rate the quality of cost analysis literature in physical therapy and to report summary information on the findings from the reviewed studies. Design This study was a targeted literature review and rating of relevant studies published in the last decade using a quality evaluation tool for economic studies. Measurements The Quality of Health Economic Studies (QHES) instrument was used to obtain quality scores. Results Ninety-five in-scope studies were identified and rated using the QHES instrument. The average quality score was 82.2 (SD=15.8), and 81 of the studies received a score of 70 or higher, placing them in the “good” to “excellent” quality range. Investigators in nearly two thirds of the studies found the physical therapy intervention under investigation to be cost-effective. Limitations The small number of studies meeting the inclusion criteria was a limitation of the study. Conclusions The quality of the literature regarding the cost-effectiveness of physical therapy is very good, although the magnitude of this body of literature is small. Greater awareness of the strengths and limitations of cost analyses in physical therapy should provide guidance for conducting high-quality cost-effectiveness studies as demand increases for demonstrations of the value of physical therapy.

Cost-Effectiveness and Cost-Utility of a Home-Based Exercise Program in Geriatric Patients with Cognitive Impairment

Gerontology ◽  
2021 ◽  
pp. 1-13
Author(s):  
Tobias Eckert ◽  
Pamela Wronski ◽  
Martin Bongartz ◽  
Phoebe Ullrich ◽  
Bastian Abel ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Cost Effectiveness ◽  
Physical Performance ◽  
Geriatric Patients ◽  
Exercise Intervention ◽  
Exercise Program ◽  
Cost Utility ◽  
Home Based ◽  
The Cost

<b><i>Introduction:</i></b> There is a substantial lack of home-based exercise programs in the highly vulnerable group of geriatric patients with cognitive impairment (CI) after discharge from ward rehabilitation. Beyond clinical effectiveness, the cost-effectiveness of intervention programs to enhance physical performance is not well investigated in this target group. <b><i>Objective:</i></b> The aim of the study was to determine the cost-effectiveness of a 12-week home-based exercise intervention following discharge from ward rehabilitation compared to unspecified flexibility training for geriatric patients with CI from a societal perspective. <b><i>Methods:</i></b> This cost-effectiveness study was conducted alongside a randomized placebo-controlled trial. A total of 118 geriatric patients with CI (Mini-Mental State Examination score: 17–26) were randomized either to the intervention group (IG, <i>n</i> = 63) or control group (CG, <i>n</i> = 55). Participants in the IG received a home-based individually tailored exercise program to increase physical performance, while participants in the CG received unspecific flexibility training (placebo control). Healthcare service use, physical performance (Short Physical Performance Battery, SPPB), and quality of life (EQ-5D-3L) were measured over 24 weeks. The net monetary benefit (NMB) approach was applied to calculate incremental cost-effectiveness of the exercise intervention compared to the CG with respect to improvement of (a) physical performance on the SPPB and (b) quality-adjusted life years (QALYs). <b><i>Results:</i></b> Physical performance was significantly improved in the IG compared to the CG (mean difference at 24 weeks: 1.3 points; 95% confidence interval [95% CI] = 0.5–2.2; <i>p</i> = 0.003), while health-related quality of life did not significantly differ between the groups at 24 weeks (mean difference: 0.08; 95% CI = −0.05 to 0.21; <i>p</i> = 0.218). Mean costs to implement the home-based exercise intervention were EUR 284 per patient. The probability of a positive incremental NMB of the intervention reached a maximum of 92% at a willingness to pay (WTP) of EUR 500 per point on the SPPB. The probability of cost-utility referring to QALYs was 85% at a WTP of EUR 5,000 per QALY. <b><i>Conclusion:</i></b> The home-based exercise intervention demonstrated high probability of cost-effectiveness in terms of improved physical performance in older adults with CI following discharge from ward rehabilitation, but not in terms of quality of life.

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