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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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Mirzayeh Fashami F, Tarride JE, Sadeghirad B, Hariri K, Peyrovinasab A, Levine M. Health Technology Assessment Reports for Non-Oncology Medications in Canada from 2018 to 2022: Methodological Critiques on Manufacturers' Submissions and a Comparison between Manufacturer and Canadian Agency for Drugs and Technologies in Health (CADTH) Analyses. PHARMACOECONOMICS - OPEN 2024; 8:823-836. [PMID: 39103675 PMCID: PMC11499573 DOI: 10.1007/s41669-024-00511-9] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Track Full Text] [Subscribe] [Scholar Register] [Accepted: 07/03/2024] [Indexed: 08/07/2024]
Abstract
INTRODUCTION Identifying key differences between manufacturers' submitted analysis and economic reanalysis by the Canadian Agency for Drugs and Technologies in Health (CADTH) is an important step toward understanding reimbursement recommendations. We compared economic values reported in manufacturers' analysis with the CADTH reanalysis and also assessed methodological critiques. METHODS Two reviewers extracted data from the clinical and economic reports in publicly available CADTH reports from 2018 to 2022. We used the Wilcoxon rank-sum test to assess the difference between mean economic values, and the Chi-square test to assess the association between the CADTH critique final recommendations. RESULTS Of the total submissions, 99.4% included effectiveness critiques, 88.8% included model structure critiques, 69.1% included utility score critiques, and 78.7% included cost critiques. The median incremental cost-utility ratio (ICUR) in the manufacturers' analyses was $138,658/quality-adjusted life-year (QALY), 2.5-fold lower than the CADTH's reanalysis at $380,251/QALY (p < 0.001). The median CADTH reanalysis for 3-year budget impact analysis (BIA) was $4,575,102, which was 27% higher than the manufacturers' submitted 3-year BIA (p < 0.001). CADTH requested a price reduction for 95% of all submissions, and the median price reduction request was 63.5%. In 2021 and 2022, the willingness-to-pay threshold identified in CADTH reports remained constant at $50,000 per QALY gained for all medications. CONCLUSION There was high frequency of CADTH critiques on manufacturers' submissions in all four aspects of economic submissions: effectiveness, cost, utility score and structure. We observed a higher median incremental cost and lower median incremental QALYs in the CADTH reanalysis compared with the manufacturers' submissions. The resulting higher ICUR in the CADTH reanalysis often leads to a recommendation that the manufacturer needs to reduce its price. The 3-year budget impact was higher in the CADTH reanalyses compared with manufacturers' submissions.
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Affiliation(s)
- Fatemeh Mirzayeh Fashami
- Health Research Methodology Graduate Program, McMaster University, 1280 Main St West, Hamilton, ON, Canada.
| | - Jean-Eric Tarride
- Center for Health Economics and Policy Analysis (CHEPA), McMaster University, 1280 Main St West, Hamilton, ON, Canada
- Programs for Assessment of Technology in Health (PATH), The Research Institute of St. Joe's Hamilton, St Joseph's Healthcare Hamilton, 50 Charlton Ave E, Hamilton, ON, Canada
- McMaster Chair in Health Technology Management, McMaster University, 1280 Main St West, Hamilton, ON, Canada
| | - Behnam Sadeghirad
- Department of Health Research Methods, Evidence, and Impact, McMaster University, 1280 Main St West, Hamilton, ON, Canada
- Michael G. DeGroote Institute for Pain Research and Care, McMaster University, Hamilton, ON, Canada
- Department of Anesthesia, McMaster University, Hamilton, ON, Canada
| | - Kimia Hariri
- Faculty of Pharmacy, Mazandaran University of Medical Science, Sari, Iran
| | - Amirreza Peyrovinasab
- Faculty of Pharmacy, Islamic Azad University, Tehran Medical Sciences University (IAUTMU), Tehran, Iran
| | - Mitchell Levine
- Department of Health Research Methods, Evidence, and Impact, McMaster University, 1280 Main St West, Hamilton, ON, Canada
- Center for Health Economics and Policy Analysis (CHEPA), McMaster University, 1280 Main St West, Hamilton, ON, Canada
- Programs for Assessment of Technology in Health (PATH), The Research Institute of St. Joe's Hamilton, St Joseph's Healthcare Hamilton, 50 Charlton Ave E, Hamilton, ON, Canada
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Shupo F, Abrams KR, Ademi Z, Wayi-Wayi G, Zibelnik N, Kirchmann M, Rutherford C, Makarounas-Kirchmann K. Cost-Effectiveness Analysis of Siltuximab for Australian Public Investment in the Rare Condition Idiopathic Multicentric Castleman Disease. PHARMACOECONOMICS - OPEN 2023; 7:777-792. [PMID: 37306929 PMCID: PMC10471559 DOI: 10.1007/s41669-023-00426-x] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Grants] [Track Full Text] [Subscribe] [Scholar Register] [Accepted: 05/21/2023] [Indexed: 06/13/2023]
Abstract
OBJECTIVES This paper presents an Australian model that formed part of the health technology assessment for public investment in siltuximab for the rare condition of idiopathic Multicentric Castleman Disease (iMCD) in Australia. METHODS Two literature reviews were conducted to identify the appropriate comparator and model structure. Survival gain based on available clinical trial data were modelled using an Excel-based model semi-Markov model including time-varying transition probabilities, an adjustment for trial crossover and long-term data. A 20-year horizon was taken, and an Australian healthcare system perspective was adopted, with both benefits and costs discounted at 5%. The model was informed with an inclusive stakeholder approach that included a review of the model by an independent economist, Australian clinical expert opinion and feedback from the Pharmaceutical Benefits Advisory Committee (PBAC). The price used in the economic evaluation reflects a confidential discounted price, which was agreed to with the PBAC. RESULTS An incremental cost-effectiveness ratio of A$84,935 per quality-adjusted life-year (QALY) gained was estimated. At a willingness-to-pay threshold of A$100,000 per QALY, siltuximab has a 72.1% probability of being cost-effective compared with placebo and best supportive care. Sensitivity analyses results were most sensitive to the length of interval between administrations (from 3- to 6-weekly) and crossover adjustments. CONCLUSION Within a collaborative and inclusive stakeholder framework, the model submitted to the Australian PBAC found siltuximab to be cost-effective for the treatment of iMCD.
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Affiliation(s)
- Francis Shupo
- EUSA Pharma UK (LTD.), Breakspear Park, Breakspear Way, Hemel Hempstead, HP2 4TZ, UK
| | - Keith R Abrams
- Visible Analytics Limited, 3 King's Meadows, Oxford, OX2 0DP, UK
| | - Zanfina Ademi
- Faculty of Pharmacy and Pharmaceutical Sciences, Centre for Medicine Use and Safety, School of Public Health and Preventive Medicine, Monash University, Clayton, Australia
| | - Grace Wayi-Wayi
- EUSA Pharma UK (LTD.), Breakspear Park, Breakspear Way, Hemel Hempstead, HP2 4TZ, UK
| | - Natasa Zibelnik
- EUSA Pharma UK (LTD.), Breakspear Park, Breakspear Way, Hemel Hempstead, HP2 4TZ, UK
| | | | | | - Kelly Makarounas-Kirchmann
- KMC Healthcare, Frankston South, VIC, Australia.
- School of Public Health and Preventive Medicine, Monash University, Clayton, Australia.
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Tzanetakos C, Gourzoulidis G. Does a Standard Cost-Effectiveness Threshold Exist? The Case of Greece. Value Health Reg Issues 2023; 36:18-26. [PMID: 37004314 DOI: 10.1016/j.vhri.2023.02.006] [Citation(s) in RCA: 13] [Impact Index Per Article: 6.5] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 11/01/2022] [Revised: 01/26/2023] [Accepted: 02/27/2023] [Indexed: 04/03/2023]
Abstract
OBJECTIVES This study aimed to systematically review the use of cost-effectiveness (CE) threshold for evaluating pharmacological interventions in Greece. METHODS A systematic search of PubMed and ScienceDirect was conducted between January 2009 and June 2022. The data of selected studies were extracted using a relevant form and consequently were synthesized. Qualitative variables were presented with relative frequencies (%) and quantitative variables with median and interquartile range (IQR). RESULTS From the 302 identified studies, 83 satisfied the inclusion criteria. Studies were categorized to oncology (26.5%) and a nononcology related (73.5%) based on drug treatment. The most frequently reported outcome associated with CE threshold was the "per quality-adjusted life-year gained." A total of 32.5% of the studies with a reported threshold did not specify the origin of the threshold. From the rest of studies, the vast majority (92.8%) adopted thresholds equal to 1 to 3 times the gross domestic product (GDP) per capita, whereas the rest similar to National Institute for Health and Care Excellence guidelines. The median CE threshold was differentiated between oncology (€51 000 [IQR €50 000-€60 000]) and nononcology studies (€34 000 [IQR €30 000-€36 000]; P < .001). In both type of studies, the median CE thresholds were not statistically significantly different among GDP, National Institute for Health and Care Excellence, and not specified approaches. CONCLUSIONS Aligned with other countries where there is no standard CE threshold to promote efficient use of healthcare resources, the most prominent practice in Greece was found to be that of 1 to 3 times the GDP per capita irrespective of type of treatment or outcome studied.
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Iglesias-López C, Agustí A, Vallano A, Obach M. Financing and Reimbursement of Approved Advanced Therapies in Several European Countries. VALUE IN HEALTH : THE JOURNAL OF THE INTERNATIONAL SOCIETY FOR PHARMACOECONOMICS AND OUTCOMES RESEARCH 2023; 26:841-853. [PMID: 36646280 DOI: 10.1016/j.jval.2022.12.014] [Citation(s) in RCA: 7] [Impact Index Per Article: 3.5] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Subscribe] [Scholar Register] [Received: 05/28/2022] [Revised: 12/01/2022] [Accepted: 12/27/2022] [Indexed: 06/04/2023]
Abstract
OBJECTIVES The uncertainty in the cost-benefit of advanced therapy medicinal products (ATMPs) is a current challenge for their reimbursement in health systems. This study aimed to provide a comparative analysis of the National Health Authorities (NHAs) reimbursement recommendations issued in different European countries. METHODS The NHA reimbursement recommendations for the approved ATMPs were compared among 8 European Union (EU) Countries (EU8: Ireland, England/Wales, Scotland, The Netherlands, France, Germany, Spain, and Italy). The search was carried out until December 31, 2021. RESULTS A total of 19 approved ATMPs and 76 appraisal reports were analyzed. The majority of the ATMPs were reimbursed, although with uncertainty in added therapeutic value. No relationship between the type of the European Medicines Agency approval and reimbursement was found. Managed entry agreements, such as payment by results, were necessary to ensure market access. The main issue during the evaluation was to base the cost-effectiveness analyses on assumptions because of the limited long-term data. The estimated incremental cost-effectiveness ratio among countries reveals high variability. Overall, the median time to NHA recommendation for the EU8 is in the range of 9 to 17 months. CONCLUSIONS Transparent, harmonized, and systematic assessments across the EU NHAs in terms of cost-effectiveness, added therapeutic value, and grade of innovativeness are needed. This could lead to a more aligned access, increasing the EU market attractiveness and raising public fairness in terms of patient access and pricing.
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Affiliation(s)
- Carolina Iglesias-López
- Department of Pharmacology, Therapeutics and Toxicology, Universitat Autònoma de Barcelona, Barcelona, Spain
| | - Antònia Agustí
- Department of Pharmacology, Therapeutics and Toxicology, Universitat Autònoma de Barcelona, Barcelona, Spain; Clinical Pharmacology Service, Vall d'Hebron University Hospital, Barcelona, Spain
| | - Antoni Vallano
- Department of Pharmacology, Therapeutics and Toxicology, Universitat Autònoma de Barcelona, Barcelona, Spain; Medicines Department, Catalan Healthcare Service, Barcelona, Spain.
| | - Mercè Obach
- Healthcare Planning Department, Catalan Healthcare Service, Barcelona, Spain
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Charlton V. The normative grounds for NICE decision-making: a narrative cross-disciplinary review of empirical studies. HEALTH ECONOMICS, POLICY, AND LAW 2022; 17:444-470. [PMID: 35293306 DOI: 10.1017/s1744133122000032] [Citation(s) in RCA: 1] [Impact Index Per Article: 0.3] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Subscribe] [Scholar Register] [Indexed: 06/14/2023]
Abstract
The National Institute for Health and Care Excellence (NICE) is the UK's primary health care priority-setter, responsible for advising the National Health Service on its adoption of health technologies. The normative basis for NICE's advice has long been the subject of public and academic interest, but the existing literature does not include any comprehensive summary of the factors observed to have substantively shaped NICE's recommendations. The current review addresses this gap by bringing together 29 studies that have explored NICE decision-making from different disciplinary perspectives, using a range of quantitative and qualitative methods. It finds that although cost-effectiveness has historically played a central role in NICE decision-making, 10 other factors (uncertainty, budget impact, clinical need, innovation, rarity, age, cause of disease, wider societal impacts, stakeholder influence and process factors) are also demonstrably influential and interact with one another in ways that are not well understood. The review also highlights an over-representation in the literature of appraisals conducted prior to 2009, according to methods that have since been superseded. It suggests that this may present a misleading view of the importance of allocative efficiency to NICE's current approach and illustrates the need for further up-to-date research into the normative grounds for NICE's decisions.
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Affiliation(s)
- Victoria Charlton
- Department of Global Health & Social Medicine, King's College London, London, UK
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Kennedy C, McCullagh L, Adams R, Trela-Larsen L, Tilson L, Barry M. Estimating the Theoretical Cost Implications of Funding New Drugs Considered Not to Be Cost-Effective. VALUE IN HEALTH : THE JOURNAL OF THE INTERNATIONAL SOCIETY FOR PHARMACOECONOMICS AND OUTCOMES RESEARCH 2021; 24:1402-1406. [PMID: 34593162 DOI: 10.1016/j.jval.2021.03.012] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Subscribe] [Scholar Register] [Received: 03/27/2020] [Revised: 03/22/2021] [Accepted: 03/31/2021] [Indexed: 06/13/2023]
Abstract
This study aims to estimate the theoretical excess expenditure that would be incurred by the Irish state-payer, should drugs be reimbursed at their original asking ("list") price rather than at a price at which the drug is considered cost-effective. In Ireland, all new drugs are evaluated by the National Centre for Pharmacoeconomics. For this study, drugs that were submitted by pharmaceutical companies from 2012 to 2017 and considered not cost-effective at list price were reviewed. A total of 43 such drugs met our inclusion criteria, and their pharmacoeconomic evaluations were further assessed. The price at which the drug could be considered cost-effective (cost-effective price) at the upper cost-effectiveness threshold used in Ireland (€ 45 000/quality adjusted life-year) was estimated for 18 drugs with an available cost-effectiveness model. Then, for each drug, the list price and cost-effective price (both per unit) were both individually applied to 1 year of national real-world drug utilization data. This allowed the estimation of the expected expenditures under the assumptions of list price paid and cost-effective price paid. The resulting theoretical excess expenditure, the expenditure at list price minus the expenditure at the cost-effective price, was estimated to be €108.2 million. This estimate is theoretical because of the confidentiality of actual drug prices. The estimation is calculated using the list price and likely overestimates the actual excess expenditure, which would reduce to zero if cost-effective prices are agreed. Nevertheless, this estimate illustrates the importance of a process to assess the value of new drugs so that potential excess drug expenditure is identified.
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Affiliation(s)
- Cormac Kennedy
- Department of Pharmacology and Therapeutics, Trinity Centre for Health Sciences, Trinity College, Dublin 8, Ireland.
| | - Laura McCullagh
- Department of Pharmacology and Therapeutics, Trinity Centre for Health Sciences, Trinity College, Dublin 8, Ireland; National Centre for Pharmacoeconomics, St James Hospital, Dublin 8, Ireland
| | - Roisin Adams
- Department of Pharmacology and Therapeutics, Trinity Centre for Health Sciences, Trinity College, Dublin 8, Ireland; National Centre for Pharmacoeconomics, St James Hospital, Dublin 8, Ireland
| | - Lea Trela-Larsen
- National Centre for Pharmacoeconomics, St James Hospital, Dublin 8, Ireland
| | - Lesley Tilson
- Department of Pharmacology and Therapeutics, Trinity Centre for Health Sciences, Trinity College, Dublin 8, Ireland; National Centre for Pharmacoeconomics, St James Hospital, Dublin 8, Ireland
| | - Michael Barry
- Department of Pharmacology and Therapeutics, Trinity Centre for Health Sciences, Trinity College, Dublin 8, Ireland; National Centre for Pharmacoeconomics, St James Hospital, Dublin 8, Ireland
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Boriani G, Vitolo M, Wright DJ, Biffi M, Brown B, Tarakji KG, Wilkoff BL. Infections associated with cardiac electronic implantable devices: economic perspectives and impact of the TYRX™ antibacterial envelope. Europace 2021; 23:iv33-iv44. [PMID: 34160600 PMCID: PMC8221050 DOI: 10.1093/europace/euab126] [Citation(s) in RCA: 13] [Impact Index Per Article: 3.3] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 03/15/2021] [Accepted: 04/20/2021] [Indexed: 01/02/2023] Open
Abstract
The occurrence of cardiac implantable electronic devices (CIED) infections and related adverse outcomes have an important financial impact on the healthcare system, with hospitalization length of stay (2-3 weeks on average) being the largest cost driver, including the cost of device system extraction and device replacement accounting for more than half of total costs. In the recent literature, the economic profile of the TYRX™ absorbable antibacterial envelope was analysed taking into account both randomized and non-randomized trial data. Economic analysis found that the envelope is associated with cost-effectiveness ratios below USA and European benchmarks in selected patients at increased risk of infection. Therefore, the TYRX™ envelope, by effectively reducing CIED infections, provides value according to the criteria of affordability currently adopted by USA and European healthcare systems.
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Affiliation(s)
- Giuseppe Boriani
- Cardiology Division, Department of Biomedical, Metabolic and Neural Sciences, University of Modena and Reggio Emilia, Policlinico di Modena, Via del Pozzo, 71, 41124 Modena, Italy
| | - Marco Vitolo
- Cardiology Division, Department of Biomedical, Metabolic and Neural Sciences, University of Modena and Reggio Emilia, Policlinico di Modena, Via del Pozzo, 71, 41124 Modena, Italy
- Clinical and Experimental Medicine PhD Program, University of Modena and Reggio Emilia, Modena, Italy
| | | | - Mauro Biffi
- Department of Experimental, Diagnostic and Specialty Medicine, Institute of Cardiology, University of Bologna, Policlinico Sant'Orsola-Malpighi, Bologna, Italy
| | - Benedict Brown
- Medtronic International Trading Sàrl, Route du Molliau 31, Tolochenaz, Switzerland
| | - Khaldoun G Tarakji
- Department of Cardiovascular Medicine and Heart, Vascular and Thoracic Institute, Cleveland Clinic, OH, USA
| | - Bruce L Wilkoff
- Department of Cardiovascular Medicine and Heart, Vascular and Thoracic Institute, Cleveland Clinic, OH, USA
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Alignment of health technology assessments and price negotiations for new drugs for rare disorders in Canada: Does it lead to improved patient access? JOURNAL OF POPULATION THERAPEUTICS AND CLINICAL PHARMACOLOGY 2020; 27:e48-e64. [PMID: 32124580 DOI: 10.15586/jptcp.v27i1.658] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 11/27/2019] [Accepted: 01/24/2020] [Indexed: 11/18/2022]
Abstract
A previous assessment of submissions for rare disorder drugs made to the Canadian Agency for Drugs and Technologies in Health (CADTH) found that, from 2012, all positive recommendations included criteria advocating a price reduction. Since 2016, CADTH and the pan-Canadian Pharmaceutical Alliance (pCPA), which conducts drug price negotiations with manufacturers for all public drug programs, have aligned their processes. This analysis examined drugs for rare and ultra-rare disorders (DRDs and DURDs)-prevalence of ≤20 to >2 and ≤2 per 100,000, respectively-with a completed pCPA negotiation or no negotiation between 2014 and 2018, together with their reimbursement recommendations and listings in public drug programs. A positive recommendation led to a successful price negotiation for 81.8% and 78.6% of the DRD and DURD submissions and a negative recommendation to no negotiation for 100.0% and 66.7%. Less than half the recommendations for DURDs reported before 2016 mentioned the need for a substantial price reduction, but this increased to 80% in those reported from 2016 onwards. A successful price negotiation led to listing in the majority of the public drug programs and a negative recommendation usually led to no listing. The CADTH-pCPA alignment is working for the governments who own and fund public drug programs but has yet to lead to coverage for all appropriate patients in all provinces. There is still a way to go to ensure that patients with unmet needs can access high-cost innovative medicines that alleviate suffering, prevent premature death, and/or significantly improve their quality of life.
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Kogushi K, Ogawa T, Ikeda S. An impact analysis of the implementation of health technology assessment for new treatment of orphan diseases in Japan. Expert Rev Pharmacoecon Outcomes Res 2019; 20:455-471. [PMID: 31496361 DOI: 10.1080/14737167.2019.1665513] [Citation(s) in RCA: 1] [Impact Index Per Article: 0.2] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 10/26/2022]
Abstract
Objectives: In Japan, a cost-effectiveness evaluation (CEE) for pricing was introduced in April 2019 and potentially covers orphan drugs (ODs) within its scope. The purpose of this study was to explore a reasonable approach to evaluate the utility of health technology assessment (HTA) for ODs in Japan. Methods: We extracted ODs that were approved in Japan from 2009 to 2018, and investigated their appraisals in the United Kingdom, Canada, and Australia, where HTA and different frameworks on ODs have been implemented. Results: Overall, 76 ODs were identified, with high recommendation rates in the three countries (80.6%-90.9%). The major reason for negative recommendation was uncertainty regarding clinical effectiveness, with actual decisions varying across countries. This indicates difficulties in setting an objective evaluation for the uncertainty of clinical effectiveness. The results of the CEE were mainly used to adjust prices. Conclusion: As Japan's CEE is expected to be used only for price adjustment after reimbursement is secured, the approach seems to be similar to the other countries. However, pre-launch clinical data are limited and the peak sales of ODs vary in Japan. Therefore, the careful introduction of CEE and multifaceted measures referring to the policies for ODs in other countries should be considered.
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Affiliation(s)
- Kentaro Kogushi
- Graduate School of Medicine, International University of Health and Welfare , Tokyo, Japan.,Medical Affairs, Otsuka Pharmaceutical Co., Ltd ., Tokyo, Japan
| | - Toshio Ogawa
- Graduate School of Health and Welfare Sciences, International University of Health and Welfare , Tokyo, Japan
| | - Shunya Ikeda
- Graduate School of Medicine, International University of Health and Welfare , Tokyo, Japan
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Godman B, Bucsics A, Vella Bonanno P, Oortwijn W, Rothe CC, Ferrario A, Bosselli S, Hill A, Martin AP, Simoens S, Kurdi A, Gad M, Gulbinovič J, Timoney A, Bochenek T, Salem A, Hoxha I, Sauermann R, Massele A, Guerra AA, Petrova G, Mitkova Z, Achniotou G, Laius O, Sermet C, Selke G, Kourafalos V, Yfantopoulos J, Magnusson E, Joppi R, Oluka M, Kwon HY, Jakupi A, Kalemeera F, Fadare JO, Melien O, Pomorski M, Wladysiuk M, Marković-Peković V, Mardare I, Meshkov D, Novakovic T, Fürst J, Tomek D, Zara C, Diogene E, Meyer JC, Malmström R, Wettermark B, Matsebula Z, Campbell S, Haycox A. Barriers for Access to New Medicines: Searching for the Balance Between Rising Costs and Limited Budgets. Front Public Health 2018; 6:328. [PMID: 30568938 PMCID: PMC6290038 DOI: 10.3389/fpubh.2018.00328] [Citation(s) in RCA: 89] [Impact Index Per Article: 12.7] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 08/15/2018] [Accepted: 10/26/2018] [Indexed: 01/26/2023] Open
Abstract
Introduction: There is continued unmet medical need for new medicines across countries especially for cancer, immunological diseases, and orphan diseases. However, there are growing challenges with funding new medicines at ever increasing prices along with funding increased medicine volumes with the growth in both infectious diseases and non-communicable diseases across countries. This has resulted in the development of new models to better manage the entry of new medicines, new financial models being postulated to finance new medicines as well as strategies to improve prescribing efficiency. However, more needs to be done. Consequently, the primary aim of this paper is to consider potential ways to optimize the use of new medicines balancing rising costs with increasing budgetary pressures to stimulate debate especially from a payer perspective. Methods: A narrative review of pharmaceutical policies and implications, as well as possible developments, based on key publications and initiatives known to the co-authors principally from a health authority perspective. Results: A number of initiatives and approaches have been identified including new models to better manage the entry of new medicines based on three pillars (pre-, peri-, and post-launch activities). Within this, we see the growing role of horizon scanning activities starting up to 36 months before launch, managed entry agreements and post launch follow-up. It is also likely there will be greater scrutiny over the effectiveness and value of new cancer medicines given ever increasing prices. This could include establishing minimum effectiveness targets for premium pricing along with re-evaluating prices as more medicines for cancer lose their patent. There will also be a greater involvement of patients especially with orphan diseases. New initiatives could include a greater role of multicriteria decision analysis, as well as looking at the potential for de-linking research and development from commercial activities to enhance affordability. Conclusion: There are a number of ongoing activities across countries to try and fund new valued medicines whilst attaining or maintaining universal healthcare. Such activities will grow with increasing resource pressures and continued unmet need.
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Affiliation(s)
- Brian Godman
- Strathclyde Institute of Pharmacy and Biomedical Sciences, University of Strathclyde, Glasgow, United Kingdom
- Health Economics Centre, University of Liverpool Management School, Liverpool, United Kingdom
- Division of Clinical Pharmacology, Karolinska Institute, Karolinska University Hospital Huddinge, Stockholm, Sweden
- School of Pharmacy, Sefako Makgatho Health Sciences University, Pretoria, South Africa
| | - Anna Bucsics
- Mechanism of Coordinated Access to Orphan Medicinal Products (MoCA), Brussels, Belgium
| | - Patricia Vella Bonanno
- Strathclyde Institute of Pharmacy and Biomedical Sciences, University of Strathclyde, Glasgow, United Kingdom
| | - Wija Oortwijn
- Ecorys, Rotterdam, Netherlands
- Department for Health Evidence, Radboud University Medical Center, Nijmegen, Netherlands
| | - Celia C. Rothe
- Department of Drug Management, Faculty of Health Sciences, Jagiellonian University Medical College, Krakow, Poland
| | - Alessandra Ferrario
- Division of Health Policy and Insurance Research, Department of Population Medicine, Harvard Medical School and Harvard Pilgrim Health Care Institute, Boston, MA, United States
| | | | - Andrew Hill
- Institute of Translational Medicine, University of Liverpool, Liverpool, United Kingdom
| | - Antony P. Martin
- Health Economics Centre, University of Liverpool Management School, Liverpool, United Kingdom
- HCD Economics, The Innovation Centre, Daresbury, United Kingdom
| | - Steven Simoens
- KU Leuven Department of Pharmaceutical and Pharmacological Sciences, Leuven, Belgium
| | - Amanj Kurdi
- Strathclyde Institute of Pharmacy and Biomedical Sciences, University of Strathclyde, Glasgow, United Kingdom
- Department of Pharmacology, College of Pharmacy, Hawler Medical University, Erbil, Iraq
| | - Mohamed Gad
- Global Health and Development Group, Imperial College, London, United Kingdom
| | - Jolanta Gulbinovič
- Department of Pathology, Forensic Medicine and Pharmacology, Faculty of Medicine, Institute of Biomedical Sciences, Vilnius University, Vilnius, Lithuania
| | - Angela Timoney
- Strathclyde Institute of Pharmacy and Biomedical Sciences, University of Strathclyde, Glasgow, United Kingdom
- NHS Lothian, Edinburgh, United Kingdom
| | - Tomasz Bochenek
- Department of Drug Management, Faculty of Health Sciences, Jagiellonian University Medical College, Krakow, Poland
| | | | - Iris Hoxha
- Department of Pharmacy, Faculty of Medicine, University of Medicine, Tirana, Albania
| | - Robert Sauermann
- Hauptverband der Österreichischen Sozialversicherungsträger, Vienna, Austria
| | - Amos Massele
- Department of Biomedical Sciences, Faculty of Medicine, University of Botswana, Gaborone, Botswana
| | - Augusto Alfonso Guerra
- Department of Social Pharmacy, College of Pharmacy, Federal University of Minas Gerais, Av. Presidente Antônio Carlos, Belo Horizonte, Brazil
- SUS Collaborating Centre – Technology Assessment & Excellence in Health (CCATES/UFMG), College of Pharmacy, Federal University of Minas Gerais. Av. Presidente Antônio Carlos, Belo Horizonte, Brazil
| | - Guenka Petrova
- Department of Social Pharmacy and Pharmacoeconomics, Faculty of Pharmacy, Medical University of Sofia, Sofia, Bulgaria
| | - Zornitsa Mitkova
- Department of Social Pharmacy and Pharmacoeconomics, Faculty of Pharmacy, Medical University of Sofia, Sofia, Bulgaria
| | | | - Ott Laius
- State Agency of Medicines, Tartu, Estonia
| | | | - Gisbert Selke
- Wissenschaftliches Institut der AOK (WIdO), Berlin, Germany
| | - Vasileios Kourafalos
- EOPYY-National Organization for the Provision of Healthcare Services, Athens, Greece
| | - John Yfantopoulos
- School of Economics and Political Science, University of Athens, Athens, Greece
| | - Einar Magnusson
- Department of Health Services, Ministry of Health, Reykjavík, Iceland
| | - Roberta Joppi
- Pharmaceutical Drug Department, Azienda Sanitaria Locale of Verona, Verona, Italy
| | - Margaret Oluka
- Department of Pharmacology and Pharmacognosy, School of Pharmacy, University of Nairobi, Nairobi, Kenya
| | - Hye-Young Kwon
- Division of Biology and Public Health, Mokwon University, Daejeon, South Korea
| | | | - Francis Kalemeera
- Department of Pharmacology and Therapeutics, Faculty of Health Sciences, University of Namibia, Windhoek, Namibia
| | - Joseph O. Fadare
- Department of Pharmacology and Therapeutics, Ekiti State University, Ado-Ekiti, Nigeria
| | | | - Maciej Pomorski
- Agency for Health Technology Assessment and Tariff System (AOTMiT), Warsaw, Poland
| | | | - Vanda Marković-Peković
- Ministry of Health and Social Welfare, Banja Luka, Bosnia and Herzegovina
- Department of Social Pharmacy, Faculty of Medicine, University of Banja Luka, Banja Luka, Bosnia and Herzegovina
| | - Ileana Mardare
- Public Health and Management Department, Faculty of Medicine, “Carol Davila”, University of Medicine and Pharmacy Bucharest, Bucharest, Romania
| | - Dmitry Meshkov
- National Research Institution for Public Health, Moscow, Russia
| | | | - Jurij Fürst
- Health Insurance Institute, Ljubljana, Slovenia
| | - Dominik Tomek
- Faculty of Medicine, Slovak Medical University in Bratislava, Bratislava, Slovakia
| | - Corrine Zara
- Drug Territorial Action Unit, Catalan Health Service, Barcelona, Spain
| | - Eduardo Diogene
- Vall d'Hebron University Hospital, Fundació Institut Català de Farmacologia, Barcelona, Spain
| | - Johanna C. Meyer
- School of Pharmacy, Sefako Makgatho Health Sciences University, Pretoria, South Africa
| | - Rickard Malmström
- Department of Medicine Solna, Karolinska Institutet and Clinical Pharmacology Karolinska University Hospital, Stockholm, Sweden
| | - Björn Wettermark
- Department of Medicine Solna, Karolinska Institutet and Clinical Pharmacology Karolinska University Hospital, Stockholm, Sweden
- Department of Healthcare Development, Stockholm County Council, Stockholm, Sweden
| | | | - Stephen Campbell
- Division of Population Health, Health Services Research and Primary Care, Centre for Primary Care, University of Manchester, Manchester, United Kingdom
- NIHR Greater Manchester Patient Safety Translational Research Centre, School of Health Sciences, University of Manchester, Manchester, United Kingdom
| | - Alan Haycox
- Health Economics Centre, University of Liverpool Management School, Liverpool, United Kingdom
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Diaz M, de Sanjosé S, Bosch FX, Bruni L. Present challenges in cervical cancer prevention: Answers from cost-effectiveness analyses. Rep Pract Oncol Radiother 2018; 23:484-494. [PMID: 30534011 PMCID: PMC6277268 DOI: 10.1016/j.rpor.2018.04.006] [Citation(s) in RCA: 8] [Impact Index Per Article: 1.1] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 01/08/2018] [Accepted: 04/08/2018] [Indexed: 12/20/2022] Open
Abstract
Simulation models are commonly used to address important health policy issues that cannot be explored through experimental studies. These models are especially useful to determine a set of strategies that result in a good value for money (cost-effectiveness). Several mathematical models simulating the natural history of HPV and related diseases, especially cervical cancer, have been developed to calculate a relative effectiveness and cost-effectiveness of HPV vaccination and cervical cancer screening interventions. Virtually all cost-effectiveness analyses identify HPV vaccination programmes for preadolescent girls to be cost-effective, even for relatively low vaccination coverage rates. Routine vaccination of preadolescent girls is the primary target population for HPV vaccination as it shows to provide the greatest health impact. Cost-effectiveness analyses assessing other vaccine target groups are less conclusive. Adding additional age-cohorts would accelerate health benefits in some years, although cost-effectiveness becomes less favourable as age at vaccination increases. Including men in HPV vaccination programmes may be a less efficient strategy if done at the expense of female vaccination coverage for reducing the burden of HPV in the population. However, as the HPV vaccine price decreases, the cost-effectiveness of universal vaccination improves, becoming equally as efficient as female-only vaccination. Vaccine price is a decisive factor in the cost-effectiveness analyses. The lower the price, the greater the likelihood that vaccination groups other than the primary target would be considered cost-effective.
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Affiliation(s)
- Mireia Diaz
- Unit of Infections and Cancer (UNIC-I&I), Cancer Epidemiology Research Programme (CERP), Institut Català d’Oncologia (ICO) – IDIBELL, L’Hospitalet de Llobregat, Barcelona, Spain
- CIBERONC, Barcelona, Spain
| | - Silvia de Sanjosé
- Cancer Epidemiology Research Programme (CERP), Institut Català d’Oncologia (ICO) – IDIBELL, L’Hospitalet de Llobregat, Barcelona, Spain
- CIBERESP, Barcelona, Spain
- Path, Reproductive Health Programme, Geneva, Switzerland
| | - F. Xavier Bosch
- CIBERONC, Barcelona, Spain
- Cancer Epidemiology Research Programme (CERP), Institut Català d’Oncologia (ICO) – IDIBELL, L’Hospitalet de Llobregat, Barcelona, Spain
| | - Laia Bruni
- Unit of Infections and Cancer (UNIC-I&I), Cancer Epidemiology Research Programme (CERP), Institut Català d’Oncologia (ICO) – IDIBELL, L’Hospitalet de Llobregat, Barcelona, Spain
- CIBERONC, Barcelona, Spain
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14
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Thokala P, Ochalek J, Leech AA, Tong T. Cost-Effectiveness Thresholds: the Past, the Present and the Future. PHARMACOECONOMICS 2018; 36:509-522. [PMID: 29427072 DOI: 10.1007/s40273-017-0606-1] [Citation(s) in RCA: 132] [Impact Index Per Article: 18.9] [Reference Citation Analysis] [Abstract] [MESH Headings] [Track Full Text] [Subscribe] [Scholar Register] [Indexed: 05/06/2023]
Abstract
Cost-effectiveness (CE) thresholds are being discussed more frequently and there have been many new developments in this area; however, there is a lack of understanding about what thresholds mean and their implications. This paper provides an overview of the CE threshold literature. First, the meaning of a CE threshold and the key assumptions involved (perfect divisibility, marginal increments in budget, etc.) are highlighted using a hypothetical example, and the use of historic/heuristic estimates of the threshold is noted along with their limitations. Recent endeavours to estimate the empirical value of the thresholds, both from the supply side and the demand side, are then presented. The impact on CE thresholds of future directions for the field, such as thresholds across sectors and the incorporation of multiple criteria beyond quality-adjusted life-years as a measure of 'value', are highlighted. Finally, a number of common issues and misconceptions associated with CE thresholds are addressed.
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Affiliation(s)
- Praveen Thokala
- Health Economics and Decision Science, School of Health and Related Research, University of Sheffield, Regent Court, 30 Regent Street, Sheffield, S1 4DA, UK.
| | - Jessica Ochalek
- Centre for Health Economics, University of York, Heslington, York, YO10 5DD, UK
| | - Ashley A Leech
- Center for the Evaluation of Value and Risk in Health (CEVR), Tufts Medical Center, Boston, MA, 02111, USA
| | - Thaison Tong
- Health Economics and Decision Science, School of Health and Related Research, University of Sheffield, Regent Court, 30 Regent Street, Sheffield, S1 4DA, UK
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15
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Which Lynch syndrome screening programs could be implemented in the "real world"? A systematic review of economic evaluations. Genet Med 2018; 20:1131-1144. [PMID: 29300371 PMCID: PMC8660650 DOI: 10.1038/gim.2017.244] [Citation(s) in RCA: 40] [Impact Index Per Article: 5.7] [Reference Citation Analysis] [Abstract] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 09/13/2017] [Accepted: 11/17/2017] [Indexed: 12/14/2022] Open
Abstract
Purpose Lynch syndrome (LS) screening can significantly reduce cancer morbidity and mortality in mutation carriers. Our aim was to identify cost-effective LS screening programs that can be implemented in the “real world.” Methods We performed a systematic review of full economic evaluations of genetic screening for LS in different target populations; health outcomes were estimated in life-years gained or quality-adjusted life-years. Results Overall, 20 studies were included in the systematic review. Based on the study populations, we identified six categories of LS screening program: colorectal cancer (CRC)–based, endometrial cancer–based, general population–based, LS family registry–based, cascade testing–based, and genetics clinic–based screening programs. We performed an in-depth analysis of CRC-based LS programs, classifying them into three additional subcategories: universal, age-targeted, and selective. In five studies, universal programs based on immunohistochemistry, either alone or in combination with the BRAF test, were cost-effective compared with no screening, while in two studies age-targeted programs with a cutoff of 70 years were cost-effective when compared with age-targeted programs with lower age thresholds. Conclusion Universal or <70 years–age-targeted CRC-based LS screening programs are cost-effective and should be implemented in the “real world.”
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16
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Rawson NS, Adams J. Do reimbursement recommendation processes used by government drug plans in Canada adhere to good governance principles? CLINICOECONOMICS AND OUTCOMES RESEARCH 2017; 9:721-730. [PMID: 29200881 PMCID: PMC5702169 DOI: 10.2147/ceor.s144695] [Citation(s) in RCA: 6] [Impact Index Per Article: 0.8] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 12/24/2022] Open
Abstract
In democratic societies, good governance is the key to assuring the confidence of stakeholders and other citizens in how governments and organizations interact with and relate to them and how decisions are taken. Although defining good governance can be debatable, the United Nations Development Program (UNDP) set of principles is commonly used. The reimbursement recommendation processes of the Canadian Agency for Drugs and Technologies in Health (CADTH), which carries out assessments for all public drug plans outside Quebec, are examined in the light of the UNDP governance principles and compared with the National Institute for Health and Care Excellence system in England. The adherence of CADTH’s processes to the principles of accountability, transparency, participatory, equity, responsiveness and consensus is poor, especially when compared with the English system, due in part to CADTH’s lack of genuine independence. CADTH’s overriding responsibility is toward the governments that “own,” fund and manage it, while the agency’s status as a not-for-profit corporation under federal law protects it from standard government forms of accountability. The recent integration of CADTH’s reimbursement recommendation processes with the provincial public drug plans’ collective system for price negotiation with pharmaceutical companies reinforces CADTH’s role as a nonindependent partner in the pursuit of governments’ cost-containment objectives, which should not be part of its function. Canadians need a national organization for evaluating drugs for reimbursement in the public interest that fully embraces the principles of good governance – one that is publicly accountable, transparent and fair and includes all stakeholders throughout its processes.
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Affiliation(s)
- Nigel Sb Rawson
- Eastlake Research Group, Oakville, ON.,Canadian Health Policy Institute, Toronto, ON.,Fraser Institute, Vancouver, BC
| | - John Adams
- Canadian PKU and Allied Disorders Inc., Toronto, ON, Canada
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17
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Szucs TD, Weiss M, Klaus G. The enigma of value: in search of affordable and accessible health care. THE EUROPEAN JOURNAL OF HEALTH ECONOMICS : HEPAC : HEALTH ECONOMICS IN PREVENTION AND CARE 2017; 18:667-670. [PMID: 27913941 PMCID: PMC5486456 DOI: 10.1007/s10198-016-0857-3] [Citation(s) in RCA: 3] [Impact Index Per Article: 0.4] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Subscribe] [Scholar Register] [Received: 11/16/2016] [Accepted: 11/24/2016] [Indexed: 05/13/2023]
Abstract
In times of shrinking resources and pharmaceutical breakthrough situations, our value-assessing systems are stretched to their very limits. Assessing value is highly complex. Current value-assessment systems risk neglecting important factors, such as therapy duration, budget impact, or the importance of combination therapies. Especially when dealing with breakthrough therapies within high-prevalence indications, these factors play an important role in health care spending. When it comes to assessing value in Switzerland, the system is innovation and access-friendly; the price level of pharmaceutical products, however, is relatively high in comparison to neighboring countries. The Swiss pricing and reimbursement system can still improve in terms of efficiency and transparency.
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Affiliation(s)
- Thomas D Szucs
- European Center of Pharmaceutical Medicine, University of Basel, Klingelbergstrasse 61, 4056, Basel, Switzerland.
| | - Martina Weiss
- Helsana Insurance Group, Postfach, 8081, Zurich, Switzerland
| | - Guido Klaus
- Helsana Insurance Group, Postfach, 8081, Zurich, Switzerland
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18
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Cost and Cost-Effectiveness Assessments of Newborn Screening for Critical Congenital Heart Disease Using Pulse Oximetry: A Review. Int J Neonatal Screen 2017; 3:34. [PMID: 29376140 PMCID: PMC5784211 DOI: 10.3390/ijns3040034] [Citation(s) in RCA: 14] [Impact Index Per Article: 1.8] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Indexed: 12/20/2022] Open
Abstract
Screening newborns for critical congenital heart disease (CCHD) using pulse oximetry is recommended to allow for the prompt diagnosis and prevention of life-threatening crises. The present review summarizes and critiques six previously published estimates of the costs or cost-effectiveness of CCHD screening from the United Kingdom, United States, and China. Several elements that affect CCHD screening costs were assessed in varying numbers of studies, including screening staff time, instrumentation, and consumables, as well as costs of diagnosis and treatment. A previous US study that used conservative assumptions suggested that CCHD screening is likely to be considered cost-effective from the healthcare sector perspective. Newly available estimates of avoided infant CCHD deaths in several US states that implemented mandatory CCHD screening policies during 2011-2013 suggest a substantially larger reduction in deaths than was projected in the previous US cost-effectiveness analysis. Taking into account these new estimates, we estimate that cost per life-year gained could be as low as USD 12,000. However, that estimate does not take into account future costs of health care and education for surviving children with CCHD nor the costs incurred by health departments to support and monitor CCHD screening policies and programs.
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Han MK, Martinez CH, Au DH, Bourbeau J, Boyd CM, Branson R, Criner GJ, Kalhan R, Kallstrom TJ, King A, Krishnan JA, Lareau SC, Lee TA, Lindell K, Mannino DM, Martinez FJ, Meldrum C, Press VG, Thomashow B, Tycon L, Sullivan JL, Walsh J, Wilson KC, Wright J, Yawn B, Zueger PM, Bhatt SP, Dransfield MT. Meeting the challenge of COPD care delivery in the USA: a multiprovider perspective. THE LANCET RESPIRATORY MEDICINE 2016; 4:473-526. [PMID: 27185520 DOI: 10.1016/s2213-2600(16)00094-1] [Citation(s) in RCA: 71] [Impact Index Per Article: 7.9] [Reference Citation Analysis] [Abstract] [Subscribe] [Scholar Register] [Received: 01/12/2016] [Revised: 03/01/2016] [Accepted: 03/01/2016] [Indexed: 12/21/2022]
Abstract
The burden of chronic obstructive pulmonary disease (COPD) in the USA continues to grow. Although progress has been made in the the development of diagnostics, therapeutics, and care guidelines, whether patients' quality of life is improved will ultimately depend on the actual implementation of care and an individual patient's access to that care. In this Commission, we summarise expert opinion from key stakeholders-patients, caregivers, and medical professionals, as well as representatives from health systems, insurance companies, and industry-to understand barriers to care delivery and propose potential solutions. Health care in the USA is delivered through a patchwork of provider networks, with a wide variation in access to care depending on a patient's insurance, geographical location, and socioeconomic status. Furthermore, Medicare's complicated coverage and reimbursement structure pose unique challenges for patients with chronic respiratory disease who might need access to several types of services. Throughout this Commission, recurring themes include poor guideline implementation among health-care providers and poor patient access to key treatments such as affordable maintenance drugs and pulmonary rehabilitation. Although much attention has recently been focused on the reduction of hospital readmissions for COPD exacerbations, health systems in the USA struggle to meet these goals, and methods to reduce readmissions have not been proven. There are no easy solutions, but engaging patients and innovative thinkers in the development of solutions is crucial. Financial incentives might be important in raising engagement of providers and health systems. Lowering co-pays for maintenance drugs could result in improved adherence and, ultimately, decreased overall health-care spending. Given the substantial geographical diversity, health systems will need to find their own solutions to improve care coordination and integration, until better data for interventions that are universally effective become available.
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Affiliation(s)
- MeiLan K Han
- Division of Pulmonary and Critical Care, University of Michigan Health System, Ann Arbor, MI, USA.
| | - Carlos H Martinez
- Division of Pulmonary and Critical Care, University of Michigan Health System, Ann Arbor, MI, USA
| | - David H Au
- Center of Innovation for Veteran-Centered and Value-Driven Care, and VA Puget Sound Health Care System, US Department of Veteran Affairs, Seattle, WA, USA; Division of Pulmonary and Critical Care Medicine, University of Washington, Seattle, WA, USA
| | - Jean Bourbeau
- McGill University Health Centre, McGill University, Montreal, QC, Canada
| | - Cynthia M Boyd
- Division of Geriatric Medicine and Gerontology, Department of Medicine, Johns Hopkins University School of Medicine, Baltimore, MD, USA
| | - Richard Branson
- Department of Surgery, University of Cincinnati, Cincinnati, OH, USA
| | - Gerard J Criner
- Department of Thoracic Medicine and Surgery, Lewis Katz School of Medicine at Temple University, Philadelphia, PA, USA
| | - Ravi Kalhan
- Asthma and COPD Program, Northwestern University Feinberg School of Medicine, Chicago, IL, USA
| | | | | | - Jerry A Krishnan
- University of Illinois Hospital & Health Sciences System, University of Illinois, Chicago, IL, USA
| | - Suzanne C Lareau
- University of Colorado Denver, Anschutz Medical Campus, Aurora, CO, USA
| | - Todd A Lee
- Department of Pharmacy Systems, Outcomes and Policy, University of Illinois, Chicago, IL, USA
| | | | - David M Mannino
- Department of Preventive Medicine and Environmental Health, University of Kentucky, Lexington, KY, USA
| | - Fernando J Martinez
- Department of Internal Medicine, Weill Cornell School of Medicine, New York, NY, USA
| | - Catherine Meldrum
- Division of Pulmonary and Critical Care, University of Michigan Health System, Ann Arbor, MI, USA
| | - Valerie G Press
- Section of Hospital Medicine, University of Chicago Medicine, Chicago, IL, USA
| | - Byron Thomashow
- Division of Pulmonary, Critical Care and Sleep Medicine, Columbia University Medical Center, New York, NY, USA
| | - Laura Tycon
- Palliative and Supportive Institute, Pittsburgh, PA, USA
| | | | | | - Kevin C Wilson
- Boston University School of Medicine, Boston, MA, USA; American Thoracic Society, New York, NY, USA
| | - Jean Wright
- Carolinas HealthCare System, Charlotte, NC, USA
| | - Barbara Yawn
- Family and Community Health, University of Minnesota Medical School, Minneapolis, MN, USA
| | - Patrick M Zueger
- Department of Pharmacy Systems, Outcomes and Policy, College of Pharmacy, University of Illinois, Chicago, IL, USA
| | - Surya P Bhatt
- Division of Pulmonary, Allergy and Critical Care Medicine, and UAB Lung Health Center, University of Alabama at Birmingham, Birmingham, AL, USA
| | - Mark T Dransfield
- Division of Pulmonary, Allergy and Critical Care Medicine, and UAB Lung Health Center, University of Alabama at Birmingham, Birmingham, AL, USA; Birmingham VA Medical Center, Birmingham, AL, USA
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20
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Wallner K, Shapiro AMJ, Senior PA, McCabe C. Cost effectiveness and value of information analyses of islet cell transplantation in the management of 'unstable' type 1 diabetes mellitus. BMC Endocr Disord 2016; 16:17. [PMID: 27061400 PMCID: PMC4826503 DOI: 10.1186/s12902-016-0097-7] [Citation(s) in RCA: 16] [Impact Index Per Article: 1.8] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Received: 12/11/2015] [Accepted: 03/22/2016] [Indexed: 12/16/2022] Open
Abstract
BACKGROUND Islet cell transplantation is a method to stabilize type 1 diabetes patients with hypoglycemia unawareness and unstable blood glucose levels by reducing insulin dependency and protecting against severe hypoglycemia through restoring endogenous insulin secretion. This study analyses the current cost-effectiveness of this technology and estimates the value of further research to reduce uncertainty around cost-effectiveness. METHODS We performed a cost-utility analysis using a Markov cohort model with a mean patient age of 49 to simulate costs and health outcomes over a life-time horizon. Our analysis used intensive insulin therapy (IIT) as comparator and took the provincial healthcare provider perspective. Cost and effectiveness data for up to four transplantations per patient came from the University of Alberta hospital. Costs are expressed in 2012 Canadian dollars and effectiveness in quality-adjusted life-years (QALYs) and life years. To characterize the uncertainty around expected outcomes, we carried out a probabilistic sensitivity analysis within the Bayesian decision-analytic framework. We performed a value-of-information analysis to identify priority areas for future research under various scenarios. We applied a structural sensitivity analysis to assess the dependence of outcomes on model characteristics. RESULTS Compared to IIT, islet cell transplantation using non-generic (generic) immunosuppression had additional costs of $150,006 ($112,023) per additional QALY, an average gain of 3.3 life years, and a probability of being cost-effective of 0.5 % (28.3 %) at a willingness-to-pay threshold of $100,000 per QALY. At this threshold the non-generic technology has an expected value of perfect information (EVPI) of $260,744 for Alberta. This increases substantially in cost-reduction scenarios. The research areas with the highest partial EVPI are costs, followed by natural history, and effectiveness and safety. CONCLUSIONS Current transplantation technology provides substantial improvements in health outcomes over conventional therapy for highly selected patients with 'unstable' type 1 diabetes. However, it is much more costly and so is not cost-effective. The value of further research into the cost-effectiveness is dependent upon treatment costs. Further, we suggest the value of information should not only be derived from current data alone when knowing that this data will most likely change in the future.
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Affiliation(s)
- Klemens Wallner
- />Department of Emergency Medicine, University of Alberta, 736 University Terrace Building, 8303 - 112 Street, Edmonton, AB T6G 2T4 Canada
| | - A. M. James Shapiro
- />Clinical Islet Transplant Program, Alberta Diabetes Institute, University of Alberta, 2000 College Plaza, 8215 - 112 Street, Edmonton, AB T6G 2C8 Canada
- />Department of Surgery, University of Alberta, Edmonton, AB Canada
| | - Peter A. Senior
- />Clinical Islet Transplant Program, Alberta Diabetes Institute, University of Alberta, 2000 College Plaza, 8215 - 112 Street, Edmonton, AB T6G 2C8 Canada
- />Division of Endocrinology and Metabolism, Department of Medicine, University of Alberta, Edmonton, Canada
| | - Christopher McCabe
- />Department of Emergency Medicine, University of Alberta, 736 University Terrace Building, 8303 - 112 Street, Edmonton, AB T6G 2T4 Canada
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