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Beresniak A, Bremond-Gignac D, Dupont D, Duru G. Reevaluating health metrics: Unraveling the limitations of disability-adjusted life years as an indicator in disease burden assessment. World J Methodol 2025; 15:95796. [PMID: 40115408 PMCID: PMC11525889 DOI: 10.5662/wjm.v15.i1.95796] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Received: 04/18/2024] [Revised: 09/12/2024] [Accepted: 09/23/2024] [Indexed: 09/29/2024] Open
Abstract
In 1993, the World Bank released a global report on the efficacy of health promotion, introducing the disability-adjusted life years (DALY) as a novel indicator. The DALY, a composite metric incorporating temporal and qualitative data, is grounded in preferences regarding disability status. This review delineates the algorithm used to calculate the value of the proposed DALY synthetic indicator and elucidates key methodological challenges associated with its application. In contrast to the quality-adjusted life years approach, derived from multi-attribute utility theory, the DALY stands as an independent synthetic indicator that adopts the assumptions of the Time Trade Off utility technique to define Disability Weights. Claiming to rely on no mathematical or economic theory, DALY users appear to have exempted themselves from verifying whether this indicator meets the classical properties required of all indicators, notably content validity, reliability, specificity, and sensitivity. The DALY concept emerged primarily to facilitate comparisons of the health impacts of various diseases globally within the framework of the Global Burden of Disease initiative, leading to numerous publications in international literature. Despite widespread adoption, the DALY synthetic indicator has prompted significant methodological concerns since its inception, manifesting in inconsistent and non-reproducible results. Given the substantial diffusion of the DALY indicator and its critical role in health impact assessments, a reassessment is warranted. This reconsideration is imperative for enhancing the robustness and reliability of public health decision-making processes.
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Affiliation(s)
| | - Dominique Bremond-Gignac
- Department of Ophtalmology, Necker-Enfants Malades University Hospital, AP-HP, Paris-Cité University, INSERM, UMRS1138, Team 17, From Physiopathology of Ocular Diseases to Clinical Development, Sorbonne Paris Cité University, Centre de Recherche des Cordeliers, Paris 75015, France
| | | | - Gerard Duru
- Data Mining International, Geneva 1216, Switzerland
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Jessop J. The UK lockdown and the economic value of human life. ECONOMIC AFFAIRS 2020; 40. [PMCID: PMC7361259 DOI: 10.1111/ecaf.12417] [Citation(s) in RCA: 5] [Impact Index Per Article: 1.0] [Reference Citation Analysis] [Track Full Text] [Subscribe] [Scholar Register] [Indexed: 05/03/2023]
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Almutairi AR, Alkhatib NS, Oh M, Curiel-Lewandrowski C, Babiker HM, Cranmer LD, McBride A, Abraham I. Economic Evaluation of Talimogene Laherparepvec Plus Ipilimumab Combination Therapy vs Ipilimumab Monotherapy in Patients With Advanced Unresectable Melanoma. JAMA Dermatol 2019; 155:22-28. [PMID: 30477000 PMCID: PMC6439581 DOI: 10.1001/jamadermatol.2018.3958] [Citation(s) in RCA: 23] [Impact Index Per Article: 3.8] [Reference Citation Analysis] [Abstract] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 06/01/2018] [Accepted: 09/11/2018] [Indexed: 12/11/2022]
Abstract
Importance A phase 2 trial comparing talimogene laherparepvec plus ipilimumab vs ipilimumab monotherapy in patients with advanced unresectable melanoma found no differential benefit in progression-free survival (PFS) but noted objective response rates (ORRs) of 38.8% (38 of 98 patients) vs 18.0% (18 of 100 patients), respectively. Objective To perform an economic evaluation of talimogene laherparepvec plus ipilimumab combination therapy vs ipilimumab monotherapy. Design, Setting, and Participants For PFS, cost-effectiveness and cost-utility analyses using a 2-state Markov model (PFS vs progression or death) was performed. For ORRs, cost-effectiveness analysis of the incremental cost of 1 additional patient achieving objective response was performed. In this setting based on a US payer perspective (2017 US dollars), participants were patients with advanced unresectable melanoma. Main Outcomes and Measures The PFS life-years and PFS quality-adjusted life-years were determined, and the associated incremental cost-effectiveness ratios (ICERs) and incremental cost-utility ratios (ICURs) were estimated. Also estimated was the ICER per 1 additional patient (out of 100 treated patients) achieving objective response. Base-case analyses were validated by sensitivity analyses. Results In PFS analyses, the cost of talimogene laherparepvec plus ipilimumab ($494 983) exceeded the cost of ipilimumab monotherapy ($132 950) by $362 033. The ICER was $2 129 606 per PFS life-years, and the ICUR was $2 262 706 per PFS quality-adjusted life-year gained. Probabilistic sensitivity analyses yielded an ICER of $1 481 208 per PFS life-year gained and an ICUR of $1 683 191 per PFS quality-adjusted life-year gained. In 1-way sensitivity analyses, the PFS hazard ratio and the utility of response were the most influential parameters. Talimogene laherparepvec plus ipilimumab has a 50% likelihood of being cost-effective at a willingness-to-pay threshold of $1 683 191 per PFS quality-adjusted life-year gained. In ORR analyses, talimogene laherparepvec plus ipilimumab ($474 904) vs ipilimumab alone ($132 810), a $342 094 difference, yielded an ICER of $1 629 019 per additional patient achieving objective response. In subgroup analyses by disease stage and BRAFV600E mutation status, ICERs ranged from $1 069 044 to $17 104 700 per 1 additional patient achieving objective response. Conclusions and Relevance The cost to gain 1 additional progression-free quality-adjusted life-year, 1 additional progression-free life-year, or to have 1 additional patient attain objective response is about $1.6 million. This amount may be beyond what payers typically are willing to pay. Combination therapy of talimogene laherparepvec plus ipilimumab does not offer an economically beneficial treatment option relative to ipilimumab monotherapy at the population level. This should not preclude treatment for individual patients for whom this regimen may be indicated.
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Affiliation(s)
- Abdulaali R. Almutairi
- Center for Health Outcomes and PharmacoEconomic Research, College of Pharmacy, The University of Arizona, Tucson
- Department of Pharmacy Practice and Science, College of Pharmacy, The University of Arizona, Tucson
| | - Nimer S. Alkhatib
- Center for Health Outcomes and PharmacoEconomic Research, College of Pharmacy, The University of Arizona, Tucson
- Department of Pharmacy Practice and Science, College of Pharmacy, The University of Arizona, Tucson
| | - Mok Oh
- Center for Health Outcomes and PharmacoEconomic Research, College of Pharmacy, The University of Arizona, Tucson
- Department of Pharmacy Practice and Science, College of Pharmacy, The University of Arizona, Tucson
| | - Clara Curiel-Lewandrowski
- Division of Dermatology, Department of Medicine, College of Medicine, The University of Arizona, Tucson
- The University of Arizona Cancer Center, Tucson
| | - Hani M. Babiker
- The University of Arizona Cancer Center, Tucson
- Division of Hematology-Oncology, Department of Medicine, College of Medicine, The University of Arizona, Tucson
| | - Lee D. Cranmer
- Division of Medical Oncology, Department of Medicine, University of Washington, Seattle
- Seattle Cancer Care Alliance, Seattle, Washington
| | - Ali McBride
- Department of Pharmacy Practice and Science, College of Pharmacy, The University of Arizona, Tucson
- The University of Arizona Cancer Center, Tucson
| | - Ivo Abraham
- Center for Health Outcomes and PharmacoEconomic Research, College of Pharmacy, The University of Arizona, Tucson
- Department of Pharmacy Practice and Science, College of Pharmacy, The University of Arizona, Tucson
- The University of Arizona Cancer Center, Tucson
- Department of Family and Community Medicine, College of Medicine, The University of Arizona, Tucson
- Matrix45, Tucson, Arizona
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Wang T, Kim HY, Wu CW, Rausei S, Sun H, Pergolizzi FP, Dionigi G. Analyzing cost-effectiveness of neural-monitoring in recurrent laryngeal nerve recovery course in thyroid surgery. Int J Surg 2017; 48:180-188. [PMID: 29030215 DOI: 10.1016/j.ijsu.2017.10.003] [Citation(s) in RCA: 15] [Impact Index Per Article: 1.9] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 09/15/2017] [Revised: 09/21/2017] [Accepted: 10/02/2017] [Indexed: 12/25/2022]
Abstract
PURPOSE The increasing use of intraoperative neural monitoring (IONM) of the recurrent laryngeal nerve (RLN) during thyroid surgery imposes an evaluation of cost-effectiveness. METHODS The analysis estimated the cost versus utility of different alternatives that simulate nerve injury course and the consequences for the following cohorts of patients: (1) no RLN injury, or vocal fold palsy (VCP) recovery within 1 month (2), 2 months (3), 6 months (4), and after 12 months (5). In the model applied, the average simulated cohort consisted of a young female patient, 40 years old, employed, daily voice user, who underwent elective, conventional total thyroidectomy via cervical incision using a standardized intermittent IONM technique, for an operable benign, bilateral, diffuse, multinodular, non-toxic, non-retrosternal goiter. RESULTS IONM was cost-ineffective when parameters such as the rates of transient vocal fold palsy (VCP) reached 38.5%. IONM was cost-effective if the rate of VCP was 33.6% at 1 month, 22.9% at 2 months, 9.8% at 6 months, and 3.8% at 12 months, independent of phono-surgery. The described scenario is cost-effective only in a high-volume setting. CONCLUSIONS This study used simulation economic modeling to assess clinical and cost-effectiveness utility of IONM implementation. In light of the limitations of a simulation-based study, we conclusively assumed that IONM is cost-effective for permeant RLN injuries.
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Affiliation(s)
- Tie Wang
- Jilin Provincial Key Laboratory of Surgical Translational Medicine, China-Japan Union Hospital of Jilin University, Division of Thyroid Surgery, Changchun City, Jilin Province, China
| | - Hoon Yub Kim
- Department of Surgery, Korea University College of Medicine, Seoul, Republic of Korea
| | - Che-Wei Wu
- Department of Otolaryngology, Kaohsiung Municipal Hsiao-Kang Hospital, Kaohsiung Medical University Hospital, Faculty of Medicine, College of Medicine, Kaohsiung Medical University, Kaohsiung City, Taiwan
| | | | - Hui Sun
- Jilin Provincial Key Laboratory of Surgical Translational Medicine, China-Japan Union Hospital of Jilin University, Division of Thyroid Surgery, Changchun City, Jilin Province, China.
| | - Francesca Pia Pergolizzi
- Division for Endocrine and Minimally Invasive Surgery, Department of Human Pathology in Adulthood and Childhood "G. Barresi", University Hospital G. Martino, University of Messina, Via C. Valeria 1, 98125, Messina, Italy
| | - Gianlorenzo Dionigi
- Division for Endocrine and Minimally Invasive Surgery, Department of Human Pathology in Adulthood and Childhood "G. Barresi", University Hospital G. Martino, University of Messina, Via C. Valeria 1, 98125, Messina, Italy.
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Beresniak A, Dupont D. Is there an alternative to quality-adjusted life years for supporting healthcare decision making? Expert Rev Pharmacoecon Outcomes Res 2016; 16:351-7. [DOI: 10.1080/14737167.2016.1184975] [Citation(s) in RCA: 5] [Impact Index Per Article: 0.6] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 10/21/2022]
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Beresniak A, Caruba T, Sabatier B, Juillière Y, Dubourg O, Danchin N. Cost-effectiveness modelling of percutaneous coronary interventions in stable coronary artery disease. World J Cardiol 2015; 7:594-602. [PMID: 26516413 PMCID: PMC4620070 DOI: 10.4330/wjc.v7.i10.594] [Citation(s) in RCA: 2] [Impact Index Per Article: 0.2] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Received: 01/27/2015] [Revised: 03/06/2015] [Accepted: 09/30/2015] [Indexed: 02/06/2023] Open
Abstract
The objective of this study is to develop a cost-effectiveness model comparing drug eluting stents (DES) vs bare metal stent (BMS) in patients suffering of stable coronary artery disease. Using a 2-years time horizon, two simulation models have been developed: BMS first line strategy and DES first line strategy. Direct medical costs were estimated considering ambulatory and hospital costs. The effectiveness endpoint was defined as treatment success, which is the absence of major adverse cardiac events. Probabilistic sensitivity analyses were carried out using 10000 Monte-Carlo simulations. DES appeared slightly more efficacious over 2 years (60% of success) when compared to BMS (58% of success). Total costs over 2 years were estimated at 9303 € for the DES and at 8926 € for bare metal stent. Hence, corresponding mean cost-effectiveness ratios showed slightly lower costs (P < 0.05) per success for the BMS strategy (15520 €/success), as compared to the DES strategy (15588 €/success). Incremental cost-effectiveness ratio is 18850 € for one additional percent of success. The sequential strategy including BMS as the first option appears to be slightly less efficacious but more cost-effective compared to the strategy including DES as first option. Future modelling approaches should confirm these results as further comparative data in stable coronary artery disease and long-term evidence become available.
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Abstract
In a Guest Editorial, Cosetta Minelli and Gianluca Baio explain how VOI analysis can prioritize research projects by identifying uncertainty in existing knowledge and then estimating expected benefits from reducing that uncertainty.
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Abstract
Introduction: The heath care system in the United States is in the midst of a transition, in large part to help accommodate an older and more medically complex population. Central to the current evolution is the reassessment of value based on the cost utility of a particular procedure compared to alternatives. The existing contribution of geriatric orthopedics to the societal burden of disease is substantial, and literature focusing on the economic value of treating elderly populations with musculoskeletal injuries is growing. Materials and Methods: A literature review of peer-reviewed publications and abstracts related to the cost-effectiveness of treating geriatric patients with orthopedic injuries was carried out. Results: In our review, we demonstrate that while cost-utility studies generally demonstrate net society savings for most orthopedic procedures, geriatric populations often contribute to negative net society savings due to decreased working years and lower salaries while in the workforce. However, the incremental cost-effective ratio for operative intervention has been shown to be below the financial willingness to treat threshold for common procedures including joint replacement surgery of the knee (ICER US$8551), hip (ICER US$17 115), and shoulder (CE US$957) as well as for spinal procedures and repair of torn rotator cuffs (ICER US$12 024). We also discuss the current trends directed toward improving institutional value and highlight important complementary next steps to help overcome the growing demands of an older, more active society. Conclusion: The geriatric population places a significant burden on the health care system. However, studies have shown that treating this demographic for orthopedic-related injuries is cost effective and profitable for providers under certain scenarios.
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Affiliation(s)
- Jeremy Truntzer
- Department of Orthopaedic Surgery, The Warren Alpert Medical School of Brown University and Rhode Island Hospital, Providence, RI, USA
| | - Christopher Nacca
- Department of Orthopaedic Surgery, The Warren Alpert Medical School of Brown University and Rhode Island Hospital, Providence, RI, USA
| | - David Paller
- Department of Orthopaedic Surgery, The Warren Alpert Medical School of Brown University and Rhode Island Hospital, Providence, RI, USA
| | - Alan H Daniels
- Department of Orthopaedic Surgery, The Warren Alpert Medical School of Brown University and Rhode Island Hospital, Providence, RI, USA
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Custer B, Janssen MP. Health economics and outcomes methods in risk-based decision-making for blood safety. Transfusion 2015; 55:2039-47. [DOI: 10.1111/trf.13080] [Citation(s) in RCA: 26] [Impact Index Per Article: 2.6] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 08/29/2014] [Revised: 02/05/2015] [Accepted: 02/10/2015] [Indexed: 01/05/2023]
Affiliation(s)
- Brian Custer
- Blood Systems Research Institute and
- Department of Laboratory Medicine; University of California; San Francisco California
| | - Mart P. Janssen
- Transfusion Technology Assessment Unit; Sanquin Research; Amsterdam the Netherlands
- Julius Center for Health Sciences and Primary Care; University Medical Centre Utrecht; Utrecht the Netherlands
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Gharaibeh M, McBride A, Bootman JL, Abraham I. Economic evaluation for the UK of nab-paclitaxel plus gemcitabine in the treatment of metastatic pancreas cancer. Br J Cancer 2015; 112:1301-5. [PMID: 25791875 PMCID: PMC4402455 DOI: 10.1038/bjc.2015.65] [Citation(s) in RCA: 19] [Impact Index Per Article: 1.9] [Reference Citation Analysis] [Abstract] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 08/27/2014] [Revised: 01/14/2015] [Accepted: 01/27/2015] [Indexed: 12/16/2022] Open
Abstract
BACKGROUND The combination of nab-paclitaxel plus gemcitabine (NAB-P+GEM) has shown superior efficacy over GEM monotherapy in metastatic pancreas cancer (MPC). Independent cost-effectiveness/utility analyses of NAB-P+GEM from the payer perspective have not been conducted for the UK. METHODS A Markov model simulating the health outcomes and total costs was developed to estimate the life years gained (LYG) and quality-adjusted life years gained (QALY) and incremental cost-effectiveness (ICER) and cost-utility ratios (ICUR) for patients with MPC in a base case and in a probabilistic (PSA) sensitivity analysis. Total cost included the cost of supportive care medications, administration, chemotherapy, disease monitoring, and adverse reactions; and was discounted at 3.5% per year. A full lifetime horizon and third party payer perspective was chosen. RESULTS The total cost of NAB-P+GEM was £5466 higher than the cost for GEM. Respectively, LYGs were 0.97 vs 0.79 and QALYs were 0.52 vs 0.45, with ICER of £30 367/LYG and ICUR of £78 086/QALY, confirmed by PSA. CONCLUSIONS The superior survival efficacy of NAB-P+GEM over GEM in the management of MPC is associated with positive cost-effectiveness and cost-utility.
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Affiliation(s)
- M Gharaibeh
- Center for Health Outcomes and PharmacoEconomic Research, College of Pharmacy, University of Arizona, Tucson, AZ 85721, USA
| | - A McBride
- Arizona Cancer Center, University of Arizona, Tucson, AZ 85721, USA
| | - J L Bootman
- Center for Health Outcomes and PharmacoEconomic Research, College of Pharmacy, University of Arizona, Tucson, AZ 85721, USA
| | - I Abraham
- 1] Center for Health Outcomes and PharmacoEconomic Research, College of Pharmacy, University of Arizona, Tucson, AZ 85721, USA [2] Arizona Cancer Center, University of Arizona, Tucson, AZ 85721, USA
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Beresniak A, Medina-Lara A, Auray JP, De Wever A, Praet JC, Tarricone R, Torbica A, Dupont D, Lamure M, Duru G. Validation of the underlying assumptions of the quality-adjusted life-years outcome: results from the ECHOUTCOME European project. PHARMACOECONOMICS 2015; 33:61-9. [PMID: 25230587 DOI: 10.1007/s40273-014-0216-0] [Citation(s) in RCA: 34] [Impact Index Per Article: 3.4] [Reference Citation Analysis] [Abstract] [MESH Headings] [Track Full Text] [Subscribe] [Scholar Register] [Indexed: 05/20/2023]
Abstract
BACKGROUND Quality-adjusted life-years (QALYs) have been used since the 1980s as a standard health outcome measure for conducting cost-utility analyses, which are often inadequately labeled as 'cost-effectiveness analyses'. This synthetic outcome, which combines the quantity of life lived with its quality expressed as a preference score, is currently recommended as reference case by some health technology assessment (HTA) agencies. While critics of the QALY approach have expressed concerns about equity and ethical issues, surprisingly, very few have tested the basic methodological assumptions supporting the QALY equation so as to establish its scientific validity. OBJECTIVES The main objective of the ECHOUTCOME European project was to test the validity of the underlying assumptions of the QALY outcome and its relevance in health decision making. METHODS An experiment has been conducted with 1,361 subjects from Belgium, France, Italy, and the UK. The subjects were asked to express their preferences regarding various hypothetical health states derived from combining different health states with time durations in order to compare observed utility values of the couples (health state, time) and calculated utility values using the QALY formula. RESULTS Observed and calculated utility values of the couples (health state, time) were significantly different, confirming that preferences expressed by the respondents were not consistent with the QALY theoretical assumptions. CONCLUSIONS This European study contributes to establishing that the QALY multiplicative model is an invalid measure. This explains why costs/QALY estimates may vary greatly, leading to inconsistent recommendations relevant to providing access to innovative medicines and health technologies. HTA agencies should consider other more robust methodological approaches to guide reimbursement decisions.
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Affiliation(s)
- Ariel Beresniak
- Data Mining International, Route de l'Aéroport, 29-31, CP 221, 1215, Geneva 15, Switzerland,
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Evans RW, Pagani FD. Economic evaluation in health care: a modern day quagmire. J Heart Lung Transplant 2014; 33:344-6. [PMID: 24661452 DOI: 10.1016/j.healun.2014.02.003] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 02/05/2014] [Accepted: 02/07/2014] [Indexed: 10/25/2022] Open
Affiliation(s)
- Roger W Evans
- United Network for the Recruitment of Transplantation Professionals, Rochester, Minnesota
| | - Francis D Pagani
- Center for Circulatory Support and the Cardiovascular Center, University of Michigan, Ann Arbor, Michigan
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