비용-효과 분석의 불확실성: 평가와 대응전략 = Uncertainties in cost-effectiveness analysis

다국어 초록 (Multilingual Abstract)

There are many uncertainties on cost-effectiveness among high-priced new drugs that have recently entered the market. Considering the social concerns about these drugs and the financial burden of national health insurance, evaluating and managing the uncertainty is expected to become a significant policy concern in the future. Uncertainties surrounding incremental cost-effectiveness ratio are divided into stochastic, structural, and parametric uncertainties and heterogeneity. Among them, parametric uncertainty is mainly evaluated through sensitivity analysis, and efforts are being made to evaluate the value of securing additional evidence through the value of information analysis. Structural uncertainty is overlooked compared to parametric uncertainty despite its significant impact on the results. Scenario analysis, model selection, model averaging, and parameterization have been proposed to analyze it. The performance-based risk-sharing scheme, particularly coverage with evidence development, has been proposed as a risk management plan. However, suppose the data collection plan is not based on uncertainty assessment and is not liked to the decision-making stage. In that case, additional data may have little impact on the final decisions.

참고문헌 (Reference)

1 김유진, "의약품 접근성 향상을 위한 방안 고찰" 한국임상약학회 28 (28): 40-50, 2018

2 이태진, "의약품 경제성평가지침 개정방안 마련" 건강보험심사평가원․서울대학교 산학협력단·경상대학교 산학협력단 2020

3 건강보험심사평가원, "의약품 경제성평가지침"

4 박실비아, "근거중심의사결정과 조건부 의사결정의 필요성" 대한의사협회 54 (54): 1319-1329, 2011

5 Fenwick, E., "Value of information analysis for research decisions-an introduction : report 1 of the ISPOR value of information analysis emerging good practices task force" 23 (23): 139-150, 2020

6 Cosetta Minelli, "Value of Information: A Tool to Improve Research Prioritization and Reduce Waste" Public Library of Science (PLoS) 12 (12): e1001882-, 2015

7 Claire Rothery, "Value of Information Analytical Methods: Report 2 of the ISPOR Value of Information Analysis Emerging Good Practices Task Force" Elsevier BV 23 (23): 277-286, 2020

8 Bindels, J., "Use of value of information in healthcare decision making: exploring multiple perspectives" 34 : 315-322, 2016

9 Dony Patel, "Use of External Comparators for Health Technology Assessment Submissions Based on Single-Arm Trials" Elsevier BV 24 (24): 1118-1125, 2021

10 Kim, L. G., "Uncertainty and validation of health economic decision models" 19 (19): 43-55, 2010

11 Pouwels, X.G.L.V., "Uncertainty and coverage with evidence development: does practice meet theory?" 22 (22): 799-807, 2019

12 Salah Ghabri, "Towards a New Framework for Addressing Structural Uncertainty in Health Technology Assessment Guidelines" Springer Science and Business Media LLC 36 (36): 127-130, 2018

13 Davies, C., "The ‘‘hazards’’ of extrapolating survival curves" 33 : 369-380, 2013

14 Frederix, G. W., "The impact of structural uncertainty on cost-effectiveness models for adjuvant endocrine breast cancer treatments : the need for disease-specific model standardization and improved guidance" 32 : 47-61, 2014

15 Sabine Elisabeth Grimm, "The HTA Risk Analysis Chart: Visualising the Need for and Potential Value of Managed Entry Agreements in Health Technology Assessment" Springer Science and Business Media LLC 35 (35): 1287-1296, 2017

16 Department of Health & Social Care, "The 2019 voluntary scheme for branded medicines pricing and access" Department of Health 2018

17 Sabine E. Grimm, "State of the ART? Two New Tools for Risk Communication in Health Technology Assessments" Springer Science and Business Media LLC 39 (39): 1185-1196, 2021

18 Seanthel Delos Santos, "Reassessing the net health benefit (NHB) of FDA approved cancer drugs with evolution of evidence using the American Society of Clinical Oncology Value Framework (ASCO-VF)." American Society of Clinical Oncology (ASCO) 38 (38): 7011-7011, 2020

19 Ting-An Tai, "Prevalence of Immature Survival Data for Anti-Cancer Drugs Presented to the National Institute for Health and Care Excellence and Impact on Decision Making" Elsevier BV 24 (24): 505-512, 2021

20 Garrison, L. P., "Performance-based risk-sharing arrangements-good practices for design, implementation, and evaluation : report of the ISPOR good practices for performance-based risk-sharing arrangements task force" 16 (16): 703-719, 2013

21 Louis Everest, "Parametric Survival Extrapolation of Early Survival Data in Economic Analyses: A Comparison of Projected Versus Observed Updated Survival" Elsevier BV 2021

22 Christopher McCabe, "One-Way Sensitivity Analysis for Probabilistic Cost-Effectiveness Analysis: Conditional Expected Incremental Net Benefit" Springer Science and Business Media LLC 38 (38): 135-141, 2020

23 NHS England, "NHS Commercial framework for medicines"

24 Garattini, L., "Modeling in pharmacoeconomic studies : funding sources and outcomes" 26 (26): 330-333, 2010

25 Briggs, A. H., "Model parameter estimation and uncertainty : a report of the ISPOR-SMDM modeling good research practices task force-6" 15 : 835-842, 2012

26 Simone A. Huygens, "Methodological Challenges in the Economic Evaluation of a Gene Therapy for RPE65-Mediated Inherited Retinal Disease: The Value of Vision" Springer Science and Business Media LLC 39 (39): 383-397, 2021

27 Grimm, S. E., "Implementation barriers to value of information analysis in health technology decision making : results from a process evaluation" 24 (24): 1126-1136, 2021

28 Kearns, B., "How uncertainty is the survival extrapolation? a study of the impact of different parametric survival models on extrapolated uncertainty about hazard functions, lifetime mean survival and cost effectiveness" 38 (38): 193-204, 2020

29 Australian government: Department of health (PBAC), "Guidelines for preparing a submission to the Pharmaceutical Benefits Advisory Committee (Ver 5.0)"

30 Ghabri, S., "Exploring uncertainty in economic evaluations of drugs and medical devices : lessons from the first review of manufacturers’submissions to the French National Authority for Health" 34 (34): 617-624, 2016

31 Claxton, K., "Exploring uncertainty in cost-effectiveness analysis" 26 (26): 781-798, 2008

32 Haji Ali Afzali, H., "Exploring structural uncertainty in model-based economic evaluations" 33 : 435-443, 2015

33 Sabine E. Grimm, "Development and Validation of the TRansparent Uncertainty ASsessmenT (TRUST) Tool for Assessing Uncertainties in Health Economic Decision Models" Springer Science and Business Media LLC 38 (38): 205-216, 2020

34 Afschin Gandjour, "Deterministic Sensitivity Analysis Under Ignorance" Springer Science and Business Media LLC 39 (39): 1197-1198, 2021

35 Bojke, L., "Defining and characterising structural uncertainty in decision analytic models" Centre for Health Economics, University of York 2006

36 Hutton, J., "Coverage with evidence development : an examination of conceptual and policy issues" 23 : 425-432, 2007

37 Jang, S., "Conflict of interest in economic analyses of aromatase inhibitors in breast cancer: a systematic review" 121 (121): 273-279, 2010

38 Bojke, L., "Characterizing structural uncertainty in decision analytic models : a review and application of methods" 12 (12): 739-749, 2009

39 Jönsson, B., "Advanced therapy medicinal products and health technology assessment principles and practices for value-based and sustainable healthcare" 20 (20): 427-438, 2019