Summary of Guidance on Health-Utility Measures by Selected Health Technology Assessment Agencies (Information Updated September 2017)

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1 Summary of Guidance on Health-Utility Measures by Selected Health Technology Assessment Agencies (Information Updated September 2017) Country Agency Guidance Source Australia PBAC No specific utility instrument is favored. The generally preferred method of measuring QALYs is to use quality-of-life or utility data. Australian-based preference weights are preferred for use in the scoring algorithm to calculate the utility weights. Where these weights are not available, outcomes may be valued using preferences that reflect the general population with justification for doing so. Alternatively, scenariobased utility weights could also be used, along with the use of utility weights published in the literature. PBAC Guidelines 2016, version 5.0 Brazil DECIT-CGATS SG, TTO, personal trade-off, or VAS. ISPOR s Canada CADTH Health preferences should reflect the general Canadian population and should be obtained from an indirect method of measurement based on a generic classification system (e.g., EQ-5D, HUI, SF 6D). Researchers must justify where an indirect method is not used. Selection of data sources should be based on their fitness for purpose, credibility, and consistency. China No policygenerating agency France HAS EQ-5D or HUI3 Three direct valuation methods such as SG, TTO, and VAS, are recommended. Four indirect valuation methods such as EQ-5D, SF-6D, HUI, and QWB, are recommended. It is recommended to use a health status classification system for which validated preference-based scores are available in France. In absence of French data, it is possible to use preference-based scores from foreign studies, subject to a critical analysis of their quality. The use of any other method must be duly qualified, and its validity must be demonstrated in France. CADTH Guidelines 2017, 4th edition ISPOR s HAS Methodological Guide (HAS, 2012) Contact Us: info@rtihs.org The power of knowledge. The value of understanding. TM

2 Germany IQWiG/G-BA Note that no cost-utility analysis is required for G-BA submission. Italy l Ufficio Coordinamento OsMed e attività HTA Data should be collected in a clinical setting. No particular utility measure is preferred. For generic index instruments, a validated German version must be used to determine the utility value. A German tariff should be used. Justification for the use of the generic instrument should be stated, and the measure should be comprehensible. Evidence of the instrument s objectivity, reliability, validity, and responsiveness is required. In addition, disease-specific instruments should be included in clinical studies to determine quality of life. The mapping of diseasespecific instruments to generic instruments is discouraged. The EQ-5D is preferred. IQWiG General Methods (IQWiG, 2017) Personal communication; no official written statement exists a Mexico CENETEC No preference. ISPOR s Netherlands ZiN EQ-5D-5L should be used and valued using Dutch reference values. Zorginstituut Nederland, 2016 Additional questionnaires and methods can be used to measure quality of life to identify the effect to its fullest; however, the EQ- 5D-5L should still be administered. Norway NoMA To measure QALY outcomes, MAUIs should be used to evaluate NoMA, 2012 the patients physical, psychological, and social functioning. Stated examples of MAUIs are the EQ-5D, the SF-36, and the 15D. No preference for generic MAUI is specified, but the choice of instrument must be justified. It is required that any data from clinical studies that also includes quality of life data or data that can be translated into quality of life scores using MAUIs is used. Data from studies in which the relevant health states have been valued using TTO or SG can be used if there are no data from relevant MAUIs available. Mapping health-state valuation data over to MAUIs is allowed if data from TTO or SG are not available. If the available QALY-weights are from a non-norwegian population, its transferability to a Norwegian setting must be specified. South Korea HIRA EQ-5D or SF-36. Personal communication Spain Spanish HTA Network EQ-5D or SF-36. Indirect measurements of preference scores are preferred; however, direct measurements can be used when their use and scientific validity is justified for the study in question. Sweden TLV Calculations based on appraisals of persons in the health condition are preferred over weights calculated from an average of a population estimating a condition depicted for it i.e. the social tariff from EQ-5D. Lopez-Bastida et al., 2010 TLV, 2003

3 USA Second Panel on Cost- Effectiveness in Health and Medicine ICER To enhance the comparability of results across studies, the use of a generic preference-based measure is recommended. The measure should be fit for purpose, with measurement properties that allow measurement of differences and changes in health across interventions. No specific, generic, preference-based measure is recommended. No specific measure of health utilities is recommended. Scenario analyses should be performed to examine the influence on costper-qaly findings of lower utilities for individuals with chronic severe conditions. UK NICE For adults, EQ-5D (i.e., the EQ-5D-3L) using UK general population TTO value set is recommended. The EQ-5D-5L descriptive system may be used; currently the 5L valuation set is not recommended for use, referencecase analyses should calculate utility values by mapping the 5L descriptive system data onto the 3L valuation using the mapping function by van Hout et al. (2012). If analyses use data gathered using both EQ- 5D-3L and EQ-5D-5L descriptive systems, the 3L valuation set should be used to derive all utility values, with 5L mapped onto 3L where needed. When EQ-5D data are not available, these data can be estimated by mapping other health-related qualityof-life measures or health-related benefits observed in the relevant clinical trial(s) to the EQ 5D. For children, an alternative health-related quality-of-life measure can be used. A version of the EQ-5D is available for children aged 7-12 years; however, there is no validated UK valuation set. In some circumstances, the EQ-5D may not be the most appropriate measure. To make a case that the EQ-5D is inappropriate, qualitative empirical evidence on the lack of content validity for the EQ-5D should be provided, demonstrating that key dimensions of health are missing. This should be supported by evidence that shows that EQ-5D performs poorly on tests of construct validity and responsiveness in a particular patient population. This evidence should be derived from a synthesis of peer-reviewed literature. Sanders et al., 2016 ICER, 2017 NICE Methods Guide (NICE, 2013); NICE Position Statement (NICE, 2017)

4 UK SMC Preference for EQ-5D in randomized controlled studies of the medicine. Preference for health states to be measured in patients using a generic and validated classification system for which reliable and appropriate population preference values, elicited using a choice-based method such as the time trade-off or standard gamble (but not rating scale), are available. Utilities derived from observational studies of patients would be acceptable as long as it can be shown that the patients and health states adequately match those in the clinical studies used in the submitted economic evaluation. SMC Guidance (SMC, 2017) Alternative, well-designed methods of utility measurement are allowed if generic utility data are not available (mapped from disease-specific quality of life measure; direct measurement in patients or vingettes valued using TTO or SG; or published data). The use of a non-qaly outcome measure also is allowed if the measure is shown to be appropriate and the value of the medicine to National Health Service in Scotland can be demonstrated. 15D = 15 Dimensions; CADTH = Canadian Agency for Drugs and Technologies in Health; CENETEC = Centro Nacional de Excelencia Tecnológica en Salud [National Center for Health Technology Excellence]; DECIT-CGATS = Secretaria de Ciëncia, Tecnologia e Insumos Estratégicos, Departamento de Ciência e Tecnologia Ministério da Saúde [Department of Science and Technology, Health Technology Assessment General Coordination, Brazilian Ministry of Health]; EQ- 5D-3L = EQ-5D 3 Level instrument; EQ-5D-5L = EQ-5D 5 Level instrument; G-BA = Gemeinsamer Bundesausschuss [Federal Joint Committee]; HAS = Haute Autorité de Santé; HIRA = Health Insurance Review and Assessment Service; HTA = health technology assessment; HUI = Health Utilities Index; HUI3 = Health Utilities Index Mark 3; ICER = Institute for Clinical and Economic Review; IQWiG = Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen; ISPOR = International Society for s and Outcomes Research; MAUI = multi-attribute utility instrument; NICE = National Institute for Health and Care Excellence; NoMA = Norwegian Medicines Agency; PBAC = Pharmaceutical Benefits Advisory Committee; QALY = quality-adjusted life-year; QWB = Quality of Well-Being scale; SF-6D = Medical Outcomes Study Short Form 6D; SF-36 = SF-36 Health Survey; SG = standard gamble; SMC = Scottish Medicines Consortium; TLV = Tandvårds-och läkemedelsförmånsverket; TTO = time trade-off; UK = United Kingdom; USA = United States of America; VAS = visual analogue scale; ZiN = Zorginstituut Nederland. a Published phramacoeconomic guidelines (Capri et al., 2001) state that SG and TTO are recommended and suggest to simultaneously apply general instruments such as the SF-36, disease-specific instruments, and instruments for surveying preferences (such as HUI or EQ-5D).

5 Sources: Canadian Agency for Drugs and Technologies in Health (CADTH). Guidelines for the economic evaluation of health technologies: Canada, 4th edition Available at: Accessed March 29, Capri S, Ceci A, Terranova L, Merlo F, Mantovani L. Guidelines for economic evaluations in Italy: recommendations from the Italian Group of Studies. Drug Inform J. 2001;35: Haute Autorité de Santé (HAS). A methodological guide: choices in methods for economic evaluations Available at: portail/upload/docs/application/pdf/ /choices_in_methods_for_economic_evaluation.pdf. Accessed September 5, Institute for Clinical and Economic Review (ICER). Overview of the ICER value framework and proposals for an update for Available at: Accessed September 5, Institute for Quality and Efficiency in Health Care (IQWiG). General methods, Version Available at: Allgemeine-Methoden_Version-5-0.pdf Accessed September 5, International Society for s and Outcomes Research (ISPOR). guidelines around the world: Brazil Available at: Accessed September 5, Referenced to: Brazil Ministry of Health. Methodological guidelines: economic evaluation of health technologies [Portuguese] Available at: Avaliacao_Economica_Brazil2014.pdf. International Society for s and Outcomes Research (ISPOR). guidelines around the world: China Available at: Accessed September 5, Referenced to: China guidelines for pharmacoeconomic evaluations [Chinese] Available at: Evaluations_2011_Chinese.pdf. International Society for s and Outcomes Research (ISPOR). guidelines around the world: Mexico Available at: Accessed September 5, Referenced to: General Health Council. Economic assessment study guideline for updating the National Formulary in Mexico [Spanish] Available at: PEguidelines/source/Mexico_GCEEE_2015.pdf. Kennedy-Martin T, Mitchell BD, Boye KS, Chen W, Curtis BH, Flynn JA, et al. The health technology assessment environment in Mainland China, Japan, South Korea, and Taiwan implications for the evaluation of diabetes mellitus therapies. Value Health Regional Issues. 2014;3: Lopez-Bastida, Oliva J, Antonanza F, Garcia-Altes A, Gisbert R, Mar J, et al. Spanish recommendations on economic evaluations of health technologies. Eur J Health Econ. 2010;11: National Institute for Health and Care Excellence (NICE). Guide to methods of technology appraisal Available at: PMG9/chapter/Foreword. Accessed July 24, National Institute for Health and Care Excellence (NICE). Position statement on use of the EQ-5D-5L valuation set Available at: org.uk/media/default/about/what-we-do/nice-guidance/nice-technology-appraisal-guidance/eq5d5l_nice_position_statement.pdf. Accessed September 5, Norwegian Medicines Agency (NoMA). Guidelines on how to conduct pharmacoeconomic analyses Available at: Documents/English/Price%20and%20reimbursement/Application%20for%20reimbursement/%20guidelines%20-%20Norway. pdf. Accessed September 5, Pharmaceutical Benefits Advisory Committee (PBAC). Guidelines for preparing submissions to the Pharmaceutical Benefits Advisory Committee Available at: Accessed March 21, Sanders GD, Neumann PJ, Basu A, Brock DW, Feeny D, Krahn M, et al. Recommendations for conduct, methodological practices, and reporting of costeffectiveness analyses: second panel on cost-effectiveness in health and medicine. JAMA. 2016;316(10): Scottish Medicines Consortium (SMC). Guidance to manufacturers for completion of new product assessment form Available at: scottishmedicines.org.uk/submission_process/submission_guidance_and_forms/templates-guidance-for-submission. Accessed September 5, Tandvårds-och läkemedelsförmånsverket (TLV). General guidelines for economic evaluations from the Pharmaceutical Benefits Board (LFNAR 2003:2) Available at: Accessed March 20, Van Hout B, Janssen M, Feng Y et al. (2012) Interim scoring for the EQ-5D-5L: Mapping the EQ-5D-5L to EQ-5D-3L value sets. Value in Health, 15: Zorginstituut Nederland (ZiN). Guideline for economic evaluations in healthcare Available at: publications/reports/2016/06/16/guideline-for-economic-evaluations-in-healthcare. Accessed September 5, Research Triangle Park Ann Arbor Barcelona Ljungskile Manchester Waltham