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Oxford University Press, New York",{"doi":284},false,{"id":330,"createTime":331,"updateTime":332,"relativeEntities":333,"slug":334,"properties":335,"entityType":214,"verifyStatus":215,"verifyTime":332,"verifyNote":217,"syncStatus":19,"languages":20,"translateLanguages":20,"viewCount":21,"primaryUrl":346,"fullTextUrl":20,"authors":347,"publicationType":239,"publisherRelationship":381,"citationCount":20,"citationInfo":20,"publishDate":410,"publishYear":411,"citationAnalyzeStatus":19,"lastCitationAnalyze":20,"indexDatabases":20,"openAccess":20,"references":20,"isForceReanalyzing":328},"491e2d3a-63b2-4f8e-99c3-341331a48905","2024-04-06T07:30:40.332+00:00","2025-01-11T23:56:53.729+00:00",[],"Valuation-of-preference-based-measures-can-existing-preference-data-be-used-to-select-a-smaller-sample-of-health-states-",{"references":336,"keywords":338,"abstract":340,"title":342,"doi":344},{"VOID":337},"Drummond, M.F., Sculpher, M., O’Brien, B., Stoddart, G.L., Torrance, G.W.: Methods for the economic evaluation of health care programmes. Oxford Medical Publications: Oxford (2005)\nBrooks, R.: EuroQol: the current state of play. Health Pol. 37, 53(72 (1996). https:\u002F\u002Fdoi.org\u002F10.1016\u002F0168-8510(96)00822-6\nTorrance, G.W., Feeny, D.H., Furlong, W.J., et al.: Multi-attribute utility function for a comprehensive health status classification system: health utilities index mark 2. Med. Care. 34(7), 702–722 (1996). https:\u002F\u002Fdoi.org\u002F10.1097\u002F00005650-199607000-00004\nFeeny, D.H., Furlong, W.J., Torrance, G.W., et al.: Multi-attribute and single-attribute utility function for the health utility index mark 3 system. Medical care 40(20), 113(128 (2002)\nHawthorne, G., Richardson, G., Atherton_Day, N.: A comparison of the assessment of quality of life (AQoL) with four other generic utility instruments. Ann. Med. 33, 358–370 (2001). https:\u002F\u002Fdoi.org\u002F10.3109\u002F07853890109002090\nKaplan, R.M., Anderson, JP: A general health policy model: update and application. Health Serv. Res. 23, 203–235 (1988)\nBrazier, J.E., Roberts, J., Deverill, M.: The estimation of a preference based measure of health from the SF-36. J. Health Econ. 21, 271(292 (2002). https:\u002F\u002Fdoi.org\u002F10.1016\u002FS0167-6296(01)00130-8\nRowen, D.L., Brazier, J., Ara, R., Azzabi Zouraq, I.: The role of condition-specific preference-based measures in health technology assessment. PharmacoEconomics 35(Suppl 1), 33–41 (2017)\nBadia, X., Roset, M., Herdman, M., et al.: A comparison of United Kingdom and Spanish general population time trade-off values for EQ-5D health states. Med. Decis. Making. 20, 7–16 (2001). https:\u002F\u002Fdoi.org\u002F10.1177\u002F0272989X0102100102\nJohnson, J.A., Luo, N., Shaw, J.W., et al.: Valuations of EQ-5D Health States: are the United States and United Kingdom different. Med. Care. 43, 221–228 (2005). https:\u002F\u002Fdoi.org\u002F10.1097\u002F00005650-200503000-00004\nRowen, D.L., Azzabi Zouraq, I., Chevrou-Severac, H., van Hout, B.: International regulations and recommendations for utility data for health technology assessment. PharmacoEconomics 35(Suppl 1), 11–19 (2017)\nKharroubi, S.A., O’Hagan, A., Brazier, J.E.: A comparison of United States and United Kingdom EQ-5D health state valuations using a non-parametric Bayesian method. Stat. Med. 29, 1622–1634 (2010). https:\u002F\u002Fdoi.org\u002F10.1002\u002Fsim.3874\nKharroubi, S.A., Brazier, J., McGhee, S.: A comparison of Hong Kong and United Kingdom SF-6D health states valuations using a non-parametric Bayesian method. Value Health. 17(4), 397–405 (2014). https:\u002F\u002Fdoi.org\u002F10.1016\u002Fj.jval.2014.02.011 (PMid: 24969000).\nKharroubi, S.A.: A comparison of Japan and United Kingdom SF-6D health states valuations using a non-parametric Bayesian method. Appl. Health Econ. Pol. 13, 409–420 (2015). https:\u002F\u002Fdoi.org\u002F10.1007\u002Fs40258-015-0171-8. (PMid: 25896874)\nThe EuroQol Group (1990) EuroQol—a new facility for the measurement of health-related quality of life. Health Policy 16(3), 199–208\nBrazier, J.E., Ratcliffe, J., Tsuchiya, A., Solomon, J.: Measuring and valuing health for economic evaluation. Oxford University Press, Oxford (2007)\nDolan, P.: Modeling valuation for Euroqol health states. Med. Care 35, 351–363 (1997)\nShaw, J.W., Johnson, J.A., Coons, S.J.: US valuation of the EQ-5D health states: development and testing of the D1 valuation model. Med. Care 43(3), 203–220 (2005)\nPatrick, D.L., Starks, H.E., Cain, K.C., Uhlmann, R.F., Pearlman, R.A.: Measuring preferences for health states worse than death. Med. Decis. Making 14, 9–18 (1994)\nKharroubi, S.A., O’Hagan, A., Brazier, J.E.: Estimating utilities from individual health state preference data: a nonparametric Bayesian approach. Appl. Stat. 54, 879–895 (2005). https:\u002F\u002Fdoi.org\u002F10.1111\u002Fj.1467-9876.2005.00511.x",{"EN":339},"",{"EN":341},"Different countries have different preferences regarding health, and there are different value sets for popular preference-based measures across different countries. However, the cost of collecting data to generate country-specific value sets can be prohibitive for countries with smaller population size or low- and middle-income countries (LMIC). This paper explores whether existing preference weights could be modelled alongside a small own country valuation study to generate representative estimates. This is explored using a case study modelling UK data alongside smaller US samples to generate US estimates. We analyse EQ-5D valuation data derived from representative samples of the US and UK populations using time trade-off to value 42 health states. A nonparametric Bayesian model was applied to estimate a US value set using the full UK dataset and subsets of the US dataset for 10, 15, 20 and 25 health states. Estimates are compared to a US value set estimated using US values alone using mean predictions and root mean square error. The results suggest that using US data elicited for 20 health states alongside the existing UK data produces similar predicted mean valuations and RMSE as the US value set, while 25 health states produce the exact features. The promising results suggest that existing preference data could be combined with a small valuation study in a new country to generate preference weights, making own country value sets more achievable for LMIC. Further research is encouraged.",{"EN":343},"Valuation of preference-based measures: can existing preference data be used to select a smaller sample of health states?",{"VOID":345},"10.1007\u002Fs10198-018-0991-1","https:\u002F\u002Flink.springer.com\u002Farticle\u002F10.1007\u002Fs10198-018-0991-1",[348,364],{"id":349,"sortIndex":350,"researcher":20,"roles":351,"affiliations":352,"properties":361},"afcbd1ca-d0ba-425b-a492-0a670b5bb782",1,[223],[353],{"id":20,"sortIndex":21,"affiliation":354,"properties":20},{"id":355,"createTime":356,"updateTime":356,"relativeEntities":357,"slug":20,"properties":358,"entityType":48,"verifyStatus":19,"verifyTime":20,"verifyNote":20,"syncStatus":19,"languages":20,"translateLanguages":20,"viewCount":21},"8252422b-3211-4af8-bbff-61e1549f4975","2023-11-30T06:25:49.937+00:00",[],{"title":359},{"VI":360},"Health Economics and Decision Science, School of Health and Related Research, The University of Sheffield, Sheffield, UK",{"title":362},{"VI":363},"Donna Rowen",{"id":365,"sortIndex":21,"researcher":20,"roles":366,"affiliations":367,"properties":378},"87576c06-80a2-48d2-a440-8fb1ee197d2a",[223],[368],{"id":20,"sortIndex":21,"affiliation":369,"properties":20},{"id":370,"createTime":371,"updateTime":372,"relativeEntities":373,"slug":374,"properties":375,"entityType":48,"verifyStatus":19,"verifyTime":20,"verifyNote":20,"syncStatus":19,"languages":20,"translateLanguages":20,"viewCount":21},"57e2437c-f1f5-4e20-95f3-091a7897f477","2024-01-25T08:32:39.657+00:00","2025-01-27T06:06:10.616+00:00",[],"Department-of-Nutrition-and-Food-Sciences-Faculty-of-Agricultural-and-Food-Sciences-American-University-of-Beirut-Beirut-Lebanon",{"title":376},{"VI":377},"Department of Nutrition and Food Sciences, Faculty of Agricultural and Food Sciences, American University of Beirut, Beirut, Lebanon",{"title":379},{"VI":380},"Samer A. Kharroubi",{"url":20,"publisher":382,"properties":20},{"id":6,"createTime":7,"updateTime":8,"relativeEntities":383,"slug":10,"properties":384,"entityType":18,"verifyStatus":19,"verifyTime":20,"verifyNote":20,"syncStatus":19,"languages":20,"translateLanguages":20,"viewCount":21,"subjectFields":388,"manageAffiliations":389,"indexDatabases":390,"url":103,"thumbnailPath":20,"statistic":405,"gsStatistic":20,"type":196,"analyzePriority":20},[],{"issn":385,"eissn":386,"title":387},{"VOID":13},{"VOID":15},{"EN":17},[],[],[391,398],{"id":64,"indexDatabase":392,"url":77,"indexYears":78,"academicFieldIds":397,"indexDatabaseRanking":82},{"id":66,"createTime":67,"updateTime":68,"relativeEntities":393,"label":394,"description":395,"key":74,"publicationTags":396,"standard":20},[],{"EN":71,"VI":71},{"EN":71,"VI":73},[76],[80,81],{"id":84,"indexDatabase":399,"url":99,"indexYears":20,"academicFieldIds":404,"indexDatabaseRanking":20},{"id":86,"createTime":87,"updateTime":88,"relativeEntities":400,"label":401,"description":402,"key":95,"publicationTags":403,"standard":20},[],{"EN":91,"VI":91},{"VI":93,"EN":94},[97,98],[101,102],{"impactFactor":21,"impactFactorByYear":406,"i10Index":120,"i10IndexLast5Year":121,"totalPublication":122,"totalPublicationByYear":407,"totalCitation":147,"totalCitationByYear":408,"totalCitationPerPublication":171,"totalCitationPerPublicationByYear":409,"hindexLast5Year":131,"hindex":131},{"2007":106,"2008":107,"2012":108,"2013":109,"2014":110,"2015":111,"2016":112,"2017":113,"2018":114,"2019":115,"2020":116,"2021":117,"2022":118,"2023":119},{"2001":124,"2002":125,"2003":126,"2004":127,"2005":128,"2006":129,"2007":130,"2008":131,"2009":127,"2010":132,"2011":133,"2012":134,"2013":135,"2014":136,"2015":137,"2016":138,"2017":139,"2018":140,"2019":141,"2020":142,"2021":143,"2022":144,"2023":145,"2024":146},{"2001":125,"2002":149,"2003":150,"2004":151,"2005":152,"2006":153,"2007":120,"2008":154,"2009":155,"2010":156,"2011":157,"2012":158,"2013":159,"2014":160,"2015":161,"2016":162,"2017":163,"2018":164,"2019":165,"2020":166,"2021":167,"2022":168,"2023":169,"2024":170},{"2001":173,"2002":174,"2003":175,"2004":176,"2005":177,"2006":178,"2007":179,"2008":180,"2009":181,"2010":182,"2011":183,"2012":184,"2013":185,"2014":186,"2015":187,"2016":188,"2017":189,"2018":190,"2019":191,"2020":192,"2021":193,"2022":194,"2023":113,"2024":195},"2018-07-06",2018,{"id":413,"createTime":414,"updateTime":415,"relativeEntities":416,"slug":417,"properties":418,"entityType":214,"verifyStatus":215,"verifyTime":415,"verifyNote":217,"syncStatus":19,"languages":20,"translateLanguages":20,"viewCount":21,"primaryUrl":427,"fullTextUrl":20,"authors":428,"publicationType":239,"publisherRelationship":454,"citationCount":20,"citationInfo":20,"publishDate":488,"publishYear":489,"citationAnalyzeStatus":19,"lastCitationAnalyze":20,"indexDatabases":20,"openAccess":20,"references":20,"isForceReanalyzing":328},"ad96f458-801a-4287-9634-47b45ca087c4","2024-02-14T02:38:32.067+00:00","2025-01-05T23:54:43.700+00:00",[],"A-method-for-cross-sector-priority-setting",{"references":419,"abstract":421,"title":423,"doi":425},{"VOID":420},"Alston JM, Norton G, Pardey P (1995) Science under scarcity: principles and practice for agricultural research evaluation and priority setting. Ithaca: Cornell University Press\nArrow JK (1986) The value of and demand for information. In: McGuire CB, Radner R (eds), Decision and organization, 2nd edn. Minnesota: University of Minnesota Press\nClyde DF (1987) Recent trends in the epidemiology and control of malaria. Epidemiol Rev 9:21–243\nFreeman LC (2000) Using available graph theoretic or molecular modeling programs in social network analysis (unpublished)\nGallup JL, Sachs JD (2001) The economic burden of malaria. Am J Trop Med Hyg 64:85–96\nHudson JA (1992) Rock engineering systems: theory and practice. London: Horwood\nJanssen W (1995) Priority setting as a practical tool for research management. In: Bosch M, Preuss H-JA (eds) Management issues in national agricultural research systems: concepts, instruments, experiences. Hamburg: LIT\nKrier JP, Baker JR (eds) (1980) Parasitic protozoa, vol 7, 2nd edn. New York, Academic\nMurota K (1987) Systems analysis by graphs and matroids: structural solvability and controlability. Springer, Berlin Heidelberg New York\nPanvisavas S (2001) Poverty and malaria: a study in a Thai-Myanmar border area. Southeast Asian J Trop Med Public Health 32:608–614\nSaaty T, Vargas L (1994) Decision making in economic, political, social and technological environments with the AHP. Pittsburgh: University of Pittsburgh\nSachs J, Malaney P (2002) The economic and social burden of malaria. Nature 416:581\nSharma SK, Pradhan P, Padhi DM (2001) Socio-economic factors associated with malaria in a tribal area of Orissa. Indian J Public Health 45:93–98\nTemel T (2003) Proceedings of workshop: Tanzania. A joint ISNAR-SIMA Report. The Hague, Netherlands: ISNAR\nTemel T, Maru A (2004) A systems approach to malaria control: institutional perspective. Forthcoming in Health Policy\nTemel T (2004) Malaria from the gap: need for cross-sectoral cooperation. Acta Tropica 89:249–259\nTheodorakopoulou I, Kalaitzandonakes N (2001) Structure of public-private knowledge networks in plant biotechnology: an EU-US comparison. In: Wolf S, Zilberman D (eds) Knowledge generation and technical change: institutional innovation in agriculture. Boston: Kluwer\nWolf S, Zilberman D, Wu S, Just D (2001) Institutional relations in agricultural information systems. In: Wolf S, Zilberman D (eds) Knowledge generation and technical change: institutional innovation in agriculture. Boston: Kluwer",{"EN":422},"This study proposes a new priority setting method for identifying critical information gaps in a multisector system. The method uses graph-theoretical concepts and principles of systems theory in characterizing the underlying information structure of the system. Its application is illustrated in the context of malaria control in Tanzania, drawing on the findings of a workshop held in October 2003 in Dar es Salaam. The workshop identified two critical pathways, EVHA and EVHPA, which warrant better understanding. The first suggests that the malaria research should generate information on the effects of environmental changes (E) on vector ecology (V) and then on the effects of V on human health (H) and then on the effects of H on agriculture (A). Interpreted likewise, the second pathway additionally points to the need for information on the effects of socioeconomic conditions (P) on A.",{"EN":424},"A method for cross-sector priority setting",{"VOID":426},"10.1007\u002Fs10198-004-0243-4","https:\u002F\u002Flink.springer.com\u002Farticle\u002F10.1007\u002Fs10198-004-0243-4",[429],{"id":430,"sortIndex":21,"researcher":20,"roles":431,"affiliations":432,"properties":451},"e652d942-84a4-44b1-b113-89e6f92bc340",[223],[433,441],{"id":20,"sortIndex":21,"affiliation":434,"properties":20},{"id":435,"createTime":436,"updateTime":436,"relativeEntities":437,"slug":20,"properties":438,"entityType":48,"verifyStatus":19,"verifyTime":20,"verifyNote":20,"syncStatus":19,"languages":20,"translateLanguages":20,"viewCount":21},"27a60207-4efd-4958-9da8-53fae0135fa5","2024-01-15T07:21:00.607+00:00",[],{"title":439},{"VI":440},"Agricultural Economics and Rural Policy Group, Wageningen University, The Netherlands",{"id":442,"sortIndex":350,"affiliation":443,"properties":450},"ec9ccaf0-6a85-4b27-a1a6-22a61af94bcd",{"id":444,"createTime":445,"updateTime":445,"relativeEntities":446,"slug":20,"properties":447,"entityType":48,"verifyStatus":19,"verifyTime":20,"verifyNote":20,"syncStatus":19,"languages":20,"translateLanguages":20,"viewCount":21},"ee27c034-ffad-4f08-8a6e-1671faa66406","2023-12-06T03:57:02.752+00:00",[],{"title":448},{"VI":449},"Agricultural Economics and Rural Policy Group, Wageningen University, Wageningen, The Netherlands",{},{"title":452},{"VI":453},"Tugrul Temel",{"url":427,"publisher":455,"properties":483},{"id":6,"createTime":7,"updateTime":8,"relativeEntities":456,"slug":10,"properties":457,"entityType":18,"verifyStatus":19,"verifyTime":20,"verifyNote":20,"syncStatus":19,"languages":20,"translateLanguages":20,"viewCount":21,"subjectFields":461,"manageAffiliations":462,"indexDatabases":463,"url":103,"thumbnailPath":20,"statistic":478,"gsStatistic":20,"type":196,"analyzePriority":20},[],{"issn":458,"eissn":459,"title":460},{"VOID":13},{"VOID":15},{"EN":17},[],[],[464,471],{"id":64,"indexDatabase":465,"url":77,"indexYears":78,"academicFieldIds":470,"indexDatabaseRanking":82},{"id":66,"createTime":67,"updateTime":68,"relativeEntities":466,"label":467,"description":468,"key":74,"publicationTags":469,"standard":20},[],{"EN":71,"VI":71},{"EN":71,"VI":73},[76],[80,81],{"id":84,"indexDatabase":472,"url":99,"indexYears":20,"academicFieldIds":477,"indexDatabaseRanking":20},{"id":86,"createTime":87,"updateTime":88,"relativeEntities":473,"label":474,"description":475,"key":95,"publicationTags":476,"standard":20},[],{"EN":91,"VI":91},{"VI":93,"EN":94},[97,98],[101,102],{"impactFactor":21,"impactFactorByYear":479,"i10Index":120,"i10IndexLast5Year":121,"totalPublication":122,"totalPublicationByYear":480,"totalCitation":147,"totalCitationByYear":481,"totalCitationPerPublication":171,"totalCitationPerPublicationByYear":482,"hindexLast5Year":131,"hindex":131},{"2007":106,"2008":107,"2012":108,"2013":109,"2014":110,"2015":111,"2016":112,"2017":113,"2018":114,"2019":115,"2020":116,"2021":117,"2022":118,"2023":119},{"2001":124,"2002":125,"2003":126,"2004":127,"2005":128,"2006":129,"2007":130,"2008":131,"2009":127,"2010":132,"2011":133,"2012":134,"2013":135,"2014":136,"2015":137,"2016":138,"2017":139,"2018":140,"2019":141,"2020":142,"2021":143,"2022":144,"2023":145,"2024":146},{"2001":125,"2002":149,"2003":150,"2004":151,"2005":152,"2006":153,"2007":120,"2008":154,"2009":155,"2010":156,"2011":157,"2012":158,"2013":159,"2014":160,"2015":161,"2016":162,"2017":163,"2018":164,"2019":165,"2020":166,"2021":167,"2022":168,"2023":169,"2024":170},{"2001":173,"2002":174,"2003":175,"2004":176,"2005":177,"2006":178,"2007":179,"2008":180,"2009":181,"2010":182,"2011":183,"2012":184,"2013":185,"2014":186,"2015":187,"2016":188,"2017":189,"2018":190,"2019":191,"2020":192,"2021":193,"2022":194,"2023":113,"2024":195},{"volume":484,"pages":486},{"VOID":485},"5",{"VOID":487},"317-323","2004-11-01",2004,{"id":491,"createTime":492,"updateTime":493,"relativeEntities":494,"slug":495,"properties":496,"entityType":214,"verifyStatus":215,"verifyTime":493,"verifyNote":217,"syncStatus":19,"languages":20,"translateLanguages":20,"viewCount":21,"primaryUrl":505,"fullTextUrl":20,"authors":506,"publicationType":239,"publisherRelationship":628,"citationCount":20,"citationInfo":20,"publishDate":662,"publishYear":663,"citationAnalyzeStatus":19,"lastCitationAnalyze":20,"indexDatabases":20,"openAccess":20,"references":20,"isForceReanalyzing":328},"c0828dd1-244e-4651-8354-0b82ec2911d7","2023-12-07T15:24:46.519+00:00","2024-12-05T23:54:30.902+00:00",[],"Comparing-EQ-5D-3L-and-EQ-5D-5L-in-measuring-the-HRQoL-burden-of-4-health-conditions-in-China",{"references":497,"abstract":499,"title":501,"doi":503},{"VOID":498},"Devlin, N.J., Brooks, R.: EQ-5D and the EuroQol Group: past, present and future. Appl. Health Econ. Health Policy. 15(2), 127–137 (2017). https:\u002F\u002Fdoi.org\u002F10.1007\u002Fs40258-017-0310-5\nSzende, A., Janssen, B.: Socio-demographic indicators based on EQ-5D. In: Szende, A., Janssen, B., Cabases, J. (eds.) Self-Reported Population Health: An International Perspective Based on EQ-5D, pp. 37–46. Springer, Dordrecht (2014)\nDevlin, N.J., Krabbe, P.F.: The development of new research methods for the valuation of EQ-5D-5L. Eur. J. Health Econ. 14(Suppl 1), S1-3 (2013). https:\u002F\u002Fdoi.org\u002F10.1007\u002Fs10198-013-0502-3\nJanssen, M.F., Pickard, A.S., Golicki, D., Gudex, C., Niewada, M., Scalone, L., et al.: Measurement properties of the EQ-5D-5L compared to the EQ-5D-3L across eight patient groups: a multi-country study. Qual. Life Res. 22(7), 1717–1727 (2013). https:\u002F\u002Fdoi.org\u002F10.1007\u002Fs11136-012-0322-4\nHerdman, M., Gudex, C., Lloyd, A., Janssen, M.F., Kind, P., Parkin, D., et al.: Development and preliminary testing of the new five-level version of EQ-5D (EQ-5D-5L). Qual. Life Res. 20(10), 1727–1736 (2011). https:\u002F\u002Fdoi.org\u002F10.1007\u002Fs11136-011-9903-x\nHan, Y., Wu, J., Cong, H., Zhou, J., Xu, C.: Validity and sensitivity of the SF-6D and EQ-5D in Chinese patients with stable angina pectoris. Chin. J. Health Stat. 30(06), 829–832 (2013). (in Chinese)\nCheung, P.W.H., Wong, C.K.H., Samartzis, D., Luk, K.D.K., Lam, C.L.K., Cheung, K.M.C., et al.: Psychometric validation of the EuroQoL 5-Dimension 5-Level (EQ-5D-5L) in Chinese patients with adolescent idiopathic scoliosis. Scoliosis Spinal Disord. 11, 19 (2016). https:\u002F\u002Fdoi.org\u002F10.1186\u002Fs13013-016-0083-x\nWang, S.L., Wu, B., Zhu, L.A., Leng, L., Bucala, R., Lu, L.J.: Construct and criterion validity of the Euro Qol-5D in patients with systemic lupus erythematosus. PLoS One 9(6), e98883 (2014). https:\u002F\u002Fdoi.org\u002F10.1371\u002Fjournal.pone.0098883\nYao, J., Liang, Y., Xu, Y., Guo, L., Yang, L., Pan, J., et al.: Analysis of the measurement characteristics of Chinese version of EQ-5D-3L applied in population with chronic disease: Rasch Model. Chin. J. Health Stat. 35(05), 681–683 (2018). (in Chinese)\nJia, Y., Cui, F., Zhang, D., Zhang, G., Gong, X., Wang, F., et al.: Investigation on the applicability of EQ-5D-5L in Chinese Patients with chronic hepatitis B. Chin. J. Vaccines Immun. 19(06), 492–497 (2013). (in Chinese)\nTsang, H.H.L., Cheung, J.P.Y., Wong, C.K.H., Cheung, P.W.H., Lau, C.S., Chung, H.Y.: Psychometric validation of the EuroQoL 5-dimension (EQ-5D) questionnaire in patients with spondyloarthritis. Arthritis Res. Ther. 21(1), 41 (2019). https:\u002F\u002Fdoi.org\u002F10.1186\u002Fs13075-019-1826-x\nXu, R., Dong, D., Luo, N., Wong, E., Wu, Y., Yu, S., et al.: Evaluating the psychometric properties of the EQ-5D-5L and SF-6D among patients with haemophilia. Eur. J. Health Econ. 22(4), 547–557 (2021). https:\u002F\u002Fdoi.org\u002F10.1007\u002Fs10198-021-01273-5\nZhu, J., Yan, X., Liu, C., Wang, H., Wang, L., Cao, S., et al.: Comparing EQ-5D-3L and EQ-5D-5L performance in common cancers: suggestions for instrument choosing. Qual. Life Res. 30(3), 841–854 (2021). https:\u002F\u002Fdoi.org\u002F10.1007\u002Fs11136-020-02636-w\nJia, Y., Cui, F., Li, L., Zhang, D., Zhang, G., Wang, F., et al.: Comparison between the EQ-5D-5L and the EQ-5D-3L in patients with hepatitis B. Qual. Life Res. 23(8), 2355–2363 (2014). https:\u002F\u002Fdoi.org\u002F10.1007\u002Fs11136-014-0670-3\nZeng, X., Sui, M., Liu, B., Yang, H., Liu, R., Tan, R., et al.: Measurement properties of the EQ-5D-5L and EQ-5D-3L in six commonly diagnosed cancers. Patient 14(2), 209–222 (2021). https:\u002F\u002Fdoi.org\u002F10.1007\u002Fs40271-020-00466-z\nYou, R., Liu, J., Yang, Z., Pan, C., Ma, Q., Luo, N.: Comparing the performance of the EQ-5D-3 L and the EQ-5D-5 L in an elderly Chinese population. Health Qual. Life Outcomes 18(1), 97 (2020). https:\u002F\u002Fdoi.org\u002F10.1186\u002Fs12955-020-01324-0\nYu, H., Zeng, X., Sui, M., Liu, R., Tan, R., Yang, J., et al.: A head-to-head comparison of measurement properties of the EQ-5D-3L and EQ-5D-5L in acute myeloid leukemia patients. Qual. Life Res. 30(3), 855–866 (2021). https:\u002F\u002Fdoi.org\u002F10.1007\u002Fs11136-020-02644-w\nJiang, J., Hong, Y., Zhang, T., Yang, Z., Lin, T., Liang, Z., et al.: Comparing the measurement properties of the EQ-5D-5L and the EQ-5D-3L in hypertensive patients living in rural China. Qual. Life Res. 30(7), 2045–2060 (2021). https:\u002F\u002Fdoi.org\u002F10.1007\u002Fs11136-021-02786-5\nPan, C., Sun, H., Wang, X., Ma, Q., Xu, Y., Luo, N., et al.: The EQ-5D-5L index score is more discriminative than the EQ-5D-3L index score in diabetes patients. Qual. Life Res. 24(7), 1767–1774 (2015). https:\u002F\u002Fdoi.org\u002F10.1007\u002Fs11136-014-0902-6\nZhou, T., Guan, H., Liu, G., Ma, A.: Comparison analysis on the EQ-5D-5L and the EQ-5D-3L for general population in China. Chin. Health Econ. 35(03), 17–20 (2016). (in Chinese)\nCai, Y., Wu, H., Yang, Z.: Comparison analysis of European quality of life-5 dimensions with three-level and five-level based on four types of people in China. Chin. J. Dis. Control Prev. 24(10), 1190–1194 (2020). ((in Chinese))\nZhao, Z., Xu, S., Wang, X., Zhang, P., Bi, Y., Li, D., et al.: Scientific evaluation of the European Five-dimensional Health Scale inpatients with chronic heart failure. Chin. J. Tradit. Chin. Med. Pharm. 34(04), 1715–1718 (2019). (in Chinese)\nTsang, H.H.L., Wong, C.K.H., Cheung, P.W.H., Lau, C.S., Chung, H.Y., Cheung, J.P.Y.: Responsiveness of the EuroQoL 5-Dimension (EQ-5D) questionnaire in patients with spondyloarthritis. BMC Musculoskelet. Disord. 22(1), 439 (2021). https:\u002F\u002Fdoi.org\u002F10.1186\u002Fs12891-021-04315-4\nCheung, P.W.H., Wong, C.K.H., Lau, S.T., Cheung, J.P.Y.: Responsiveness of the EuroQoL 5-dimension (EQ-5D) in adolescent idiopathic scoliosis. Eur. Spine J. 27(2), 278–285 (2018). https:\u002F\u002Fdoi.org\u002F10.1007\u002Fs00586-017-5330-1\nXing, Y., Ma, A.: An empirical study comparing the Chinese versions of EQ-5D-3L and EQ-5D-5L. Shanghai Med. Pharma. J. 34(07), 27–31 (2013). (in Chinese)\nHu, H., Shen, W., Jia, M., Yu, G., Mei, X., Bian, Q., et al.: Reliability and validity of the Chinese Version of the EQ-5D-3L in patients of chronic kidney disease. Chin. J. Integr. Tradit. Western Nephrol. 13(08), 690–693 (2012). (in Chinese)\nHu, X., Jing, M., Zhang, M., Yan, X., Huang, Y.: The reliability and validity of EQ-5D-3L in Uygur cervical cancer patients. J. Shihezi Univ. (Nat. Sci.). 37(06), 780–785 (2019). (in Chinese)\nQiu, Y.: Reliability and validity of the Chinese Version of EQ-5D in the knee osteoarthritis [Master, Capital Institute of Physical Education]. (2015). (in Chinese)\nXia, J., Wu, N., Ma, T., Yu, C., Li, N.: Evaluation of reliability and validity of EQ-5D-5L based on residents in Southwest China. J. Sichuan Univ. (Med. Sci. Ed.) 51(05), 691–694 (2020). (in Chinese)\nXing, Y., Ma, A.: Study on reliability and validity of Chinese version of EQ-5D-5L. Shanghai Med. Pharma. J. 34(09), 40–43 (2013). (in Chinese)\nFang, H., Farooq, U., Wang, D., Yu, F., Younus, M.I., Guo, X.: Reliability and validity of the EQ-5D-3L for Kashin-Beck disease in China. Springerplus 5(1), 1924 (2016). https:\u002F\u002Fdoi.org\u002F10.1186\u002Fs40064-016-3613-3\nLi, L., Liu, C., Cai, X., Yu, H., Zeng, X., Sui, M., et al.: Validity and reliability of the EQ-5D-5L in family caregivers of leukemia patients. BMC Cancer 19(1), 522 (2019). https:\u002F\u002Fdoi.org\u002F10.1186\u002Fs12885-019-5721-2\nYang, F., Jiang, S., He, X., Li, H., Wu, H., Zhang, T., et al.: Do rural residents in China understand EQ-5D-5L as intended? Evidence from a qualitative study. Pharmacoecon. Open 5(1), 101–109 (2021). https:\u002F\u002Fdoi.org\u002F10.1007\u002Fs41669-020-00212-z\nDai, L., Wen, Y., Lu, T., Cai, J., Wang, L., Qin, M., et al.: Measurement characteristics evaluation of SF-HSTCM and EQ-5D-3L among patients with stroke. J. Cap. Med. Univ. 40(04), 627–634 (2019). (in Chinese)\nPeasgood, T., Mukuria, C., Brazier, J., Marten, O., Kreimeier, S., Luo, N., et al.: Developing a new generic health and wellbeing measure: psychometric survey results for the EQ health and wellbeing. Value Health. (2022). https:\u002F\u002Fdoi.org\u002F10.1016\u002Fj.jval.2021.11.1361\nYfantopoulos, J., Chantzaras, A., Kontodimas, S.: Assessment of the psychometric properties of the EQ-5D-3L and EQ-5D-5L instruments in psoriasis. Arch. Dermatol. Res. 309(5), 357–370 (2017). https:\u002F\u002Fdoi.org\u002F10.1007\u002Fs00403-017-1743-2\nBhadhuri, A., Kind, P., Salari, P., Jungo, K.T., Boland, B., Byrne, S., et al.: Measurement properties of EQ-5D-3L and EQ-5D-5L in recording self-reported health status in older patients with substantial multimorbidity and polypharmacy. Health Qual. Life Outcomes 18(1), 317 (2020). https:\u002F\u002Fdoi.org\u002F10.1186\u002Fs12955-020-01564-0\nPoor, A.K., Rencz, F., Brodszky, V., Gulacsi, L., Beretzky, Z., Hidvegi, B., et al.: Measurement properties of the EQ-5D-5L compared to the EQ-5D-3L in psoriasis patients. Qual. Life Res. 26(12), 3409–3419 (2017). https:\u002F\u002Fdoi.org\u002F10.1007\u002Fs11136-017-1699-x\nGandhi, M., Ang, M., Teo, K., Wong, C.W., Wei, Y.C.H., Tan, R.L.Y., et al.: EQ-5D-5L is more responsive than EQ-5D-3L to treatment benefit of cataract surgery. Patient-Patient Cent. Outcomes Res. 12(4), 383–392 (2019)\nLiu, G.G., Wu, H., Li, M., Gao, C., Luo, N.: Chinese time trade-off values for EQ-5D health states. Value Health 17(5), 597–604 (2014). https:\u002F\u002Fdoi.org\u002F10.1016\u002Fj.jval.2014.05.007\nLuo, N., Liu, G., Li, M., Guan, H., Jin, X., Rand-Hendriksen, K.: Estimating an EQ-5D-5L value set for China. Value Health 20(4), 662–669 (2017). https:\u002F\u002Fdoi.org\u002F10.1016\u002Fj.jval.2016.11.016\nParkin, D., Devlin, N., Feng, Y.: What determines the shape of an EQ-5D index distribution? Med. Decis. Making 36(8), 941–951 (2016). https:\u002F\u002Fdoi.org\u002F10.1177\u002F0272989X16645581\nTerwee, C.B., Bot, S.D.M., de Boer, M.R., van der Windt, D.A.W.M., Knol, D.L., Dekker, J., Bouter, L.A., et al.: Quality criteria were proposed for measurement properties of health status questionnaires. J. Clin. Epidemiol. 60(1), 34–42 (2007)\nVickrey, B.G., Hays, R.D., Genovese, B.J., Myers, L.W., Ellison, G.W.: Comparison of a generic to disease-targeted health-related quality-of-life measures for multiple sclerosis. J. Clin. Epidemiol. 50(5), 557–569 (1997). https:\u002F\u002Fdoi.org\u002F10.1016\u002Fs0895-4356(97)00001-2\nLuo, N., Johnson, J.A., Shaw, J.W., Coons, S.J.: Relative efficiency of the EQ-5D, HUI2, and HUI3 Index Scores in measuring health burden of chronic medical conditions in a population health survey in the United States. Med. Care 47(1), 53–60 (2009)\nKennedy-Martin, M., Slaap, B., Herdman, M., van Reenen, M., Kennedy-Martin, T., Greiner, W., et al.: Which multi-attribute utility instruments are recommended for use in cost-utility analysis? A review of national health technology assessment (HTA) guidelines. Eur. J. Health Econ. 21(8), 1245–1257 (2020). https:\u002F\u002Fdoi.org\u002F10.1007\u002Fs10198-020-01195-8\nBuchholz, I., Janssen, M.F., Kohlmann, T., Feng, Y.S.: A systematic review of studies comparing the measurement properties of the three-level and five-level versions of the EQ-5D. Pharmacoeconomics 36(6), 645–661 (2018). https:\u002F\u002Fdoi.org\u002F10.1007\u002Fs40273-018-0642-5\nJanssen, M.F., Bonsel, G.J., Luo, N.: Is EQ-5D-5L better than EQ-5D-3L? A head-to-head comparison of descriptive systems and value sets from seven countries. Pharmacoeconomics 36(6), 675–697 (2018). https:\u002F\u002Fdoi.org\u002F10.1007\u002Fs40273-018-0623-8\nFeng, Y., Devlin, N., Bateman, A., Zamora, B., Parkin, D.: distribution of the EQ-5D-5L profiles and values in three patient groups. Value Health. 22(3), 355–361 (2019). https:\u002F\u002Fdoi.org\u002F10.1016\u002Fj.jval.2018.08.012",{"EN":500},"EQ-5D-3L has been used in the National Health Services Survey of China since 2008 to monitor population health. The five-level version of EQ-5D was developed, but there lacks evidence to support the use of five-level version of EQ-5D in China. This study was conducted to compare the measurement properties of both the EQ-5D-3L and EQ-5D-5L in quantifying health-related quality of life (HRQoL) burden for 4 different health conditions in China. Participants from China were recruited to complete the 3L and 5L questionnaire via Internet. Quota was set to recruit five groups of individuals, with one group of individuals without any health condition and one group of generalized anxiety disorder (GAD), HIV\u002FAIDS, chronic Hepatitis B (CHB), or depression, respectively. The 3L and 5L were compared in terms of response distributions, percentages of reporting ‘no problems’, index value distributions, known-group validity and their relative efficiency. In total, 500 individuals completed the online survey, including 140 healthy individuals, 122 individuals with hepatitis B, 107 with depression, 90 individuals with GAD and 101 with HIV\u002FAIDS. 5L also had smoother and less clustered index value distributions. Healthy group showed different response distributions to the four condition groups. The percentage of reporting ‘no problems’ decreased significantly in the 5L in all domains (P \u003C 0.01), especially in the pain\u002Fdiscomfort dimension (relative difference: 43.10%). Relative efficiency suggested that 5L had a higher absolute discriminatory power than the 3L version between healthy participant and the other 4 condition groups, especially for the HIV\u002FAIDS group when the 3L results was not significant. The 5L version may be preferable to the 3L, as it demonstrated superior performance with respect to higher sensitivity to mild health problems, better relative efficiency and responses and index value distributions.",{"EN":502},"Comparing EQ-5D-3L and EQ-5D-5L in measuring the HRQoL burden of 4 health conditions in 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Weng",{"id":542,"sortIndex":350,"researcher":20,"roles":543,"affiliations":544,"properties":550},"4efd240d-96c4-4517-a119-8bd976767c7e",[223],[545],{"id":20,"sortIndex":21,"affiliation":546,"properties":20},{"id":531,"createTime":532,"updateTime":532,"relativeEntities":547,"slug":534,"properties":548,"entityType":48,"verifyStatus":19,"verifyTime":20,"verifyNote":20,"syncStatus":19,"languages":20,"translateLanguages":20,"viewCount":21},[],{"title":549},{"EN":537},{"title":551},{"VI":552},"Yanming Hong",{"id":554,"sortIndex":555,"researcher":20,"roles":556,"affiliations":557,"properties":573},"c98bda55-6189-4c9c-afdf-fe5d920051ec",4,[223],[558,563],{"id":20,"sortIndex":21,"affiliation":559,"properties":20},{"id":531,"createTime":532,"updateTime":532,"relativeEntities":560,"slug":534,"properties":561,"entityType":48,"verifyStatus":19,"verifyTime":20,"verifyNote":20,"syncStatus":19,"languages":20,"translateLanguages":20,"viewCount":21},[],{"title":562},{"EN":537},{"id":564,"sortIndex":350,"affiliation":565,"properties":572},"1c195148-ab7e-4a99-8d54-f5ce80a4a7ed",{"id":566,"createTime":567,"updateTime":567,"relativeEntities":568,"slug":20,"properties":569,"entityType":48,"verifyStatus":19,"verifyTime":20,"verifyNote":20,"syncStatus":19,"languages":20,"translateLanguages":20,"viewCount":21},"574f1d28-7e89-42d3-afa6-5fb514615ddb","2023-12-07T15:24:46.591+00:00",[],{"title":570},{"VI":571},"Dongguan Institute of Jinan University, Dongguan, People’s Republic of China",{},{"title":574},{"VI":575},"Jie Jiang",{"id":577,"sortIndex":179,"researcher":20,"roles":578,"affiliations":579,"properties":590},"37daebdc-625a-454d-93a4-c55fda0cc01e",[223],[580],{"id":20,"sortIndex":21,"affiliation":581,"properties":20},{"id":582,"createTime":583,"updateTime":584,"relativeEntities":585,"slug":586,"properties":587,"entityType":48,"verifyStatus":19,"verifyTime":20,"verifyNote":20,"syncStatus":19,"languages":20,"translateLanguages":20,"viewCount":21},"186d98e0-191a-4009-988d-d2375be8dcd3","2023-12-06T07:16:36.675+00:00","2025-02-02T21:49:31.978+00:00",[],"School-of-Health-and-Related-Research-University-of-Sheffield-Sheffield-UK",{"title":588},{"VI":589},"School of Health and Related Research, University of Sheffield, Sheffield, UK",{"title":591},{"VI":592},"Clara Mukuria",{"id":594,"sortIndex":595,"researcher":20,"roles":596,"affiliations":597,"properties":603},"418f3209-9616-495c-9dde-5620f6b49fad",6,[223],[598],{"id":20,"sortIndex":21,"affiliation":599,"properties":20},{"id":531,"createTime":532,"updateTime":532,"relativeEntities":600,"slug":534,"properties":601,"entityType":48,"verifyStatus":19,"verifyTime":20,"verifyNote":20,"syncStatus":19,"languages":20,"translateLanguages":20,"viewCount":21},[],{"title":602},{"EN":537},{"title":604},{"VI":605},"Sha Li",{"id":607,"sortIndex":170,"researcher":20,"roles":608,"affiliations":609,"properties":625},"91adb9a1-ebc6-4f9c-9eda-c0bebd381d6b",[223],[610,615],{"id":20,"sortIndex":21,"affiliation":611,"properties":20},{"id":531,"createTime":532,"updateTime":532,"relativeEntities":612,"slug":534,"properties":613,"entityType":48,"verifyStatus":19,"verifyTime":20,"verifyNote":20,"syncStatus":19,"languages":20,"translateLanguages":20,"viewCount":21},[],{"title":614},{"EN":537},{"id":616,"sortIndex":350,"affiliation":617,"properties":624},"86aaeb2f-bee9-42bf-a40d-7df19066bdf2",{"id":618,"createTime":619,"updateTime":619,"relativeEntities":620,"slug":20,"properties":621,"entityType":48,"verifyStatus":19,"verifyTime":20,"verifyNote":20,"syncStatus":19,"languages":20,"translateLanguages":20,"viewCount":21},"ca983b9f-83f4-4141-a8f8-69a83a5c7010","2023-12-07T15:24:46.608+00:00",[],{"title":622},{"VI":623},"Health Services Management Department, Guizhou Medical University, Guiyang, People’s Republic of China",{},{"title":626},{"VI":627},"Zhihao Yang",{"url":505,"publisher":629,"properties":657},{"id":6,"createTime":7,"updateTime":8,"relativeEntities":630,"slug":10,"properties":631,"entityType":18,"verifyStatus":19,"verifyTime":20,"verifyNote":20,"syncStatus":19,"languages":20,"translateLanguages":20,"viewCount":21,"subjectFields":635,"manageAffiliations":636,"indexDatabases":637,"url":103,"thumbnailPath":20,"statistic":652,"gsStatistic":20,"type":196,"analyzePriority":20},[],{"issn":632,"eissn":633,"title":634},{"VOID":13},{"VOID":15},{"EN":17},[],[],[638,645],{"id":64,"indexDatabase":639,"url":77,"indexYears":78,"academicFieldIds":644,"indexDatabaseRanking":82},{"id":66,"createTime":67,"updateTime":68,"relativeEntities":640,"label":641,"description":642,"key":74,"publicationTags":643,"standard":20},[],{"EN":71,"VI":71},{"EN":71,"VI":73},[76],[80,81],{"id":84,"indexDatabase":646,"url":99,"indexYears":20,"academicFieldIds":651,"indexDatabaseRanking":20},{"id":86,"createTime":87,"updateTime":88,"relativeEntities":647,"label":648,"description":649,"key":95,"publicationTags":650,"standard":20},[],{"EN":91,"VI":91},{"VI":93,"EN":94},[97,98],[101,102],{"impactFactor":21,"impactFactorByYear":653,"i10Index":120,"i10IndexLast5Year":121,"totalPublication":122,"totalPublicationByYear":654,"totalCitation":147,"totalCitationByYear":655,"totalCitationPerPublication":171,"totalCitationPerPublicationByYear":656,"hindexLast5Year":131,"hindex":131},{"2007":106,"2008":107,"2012":108,"2013":109,"2014":110,"2015":111,"2016":112,"2017":113,"2018":114,"2019":115,"2020":116,"2021":117,"2022":118,"2023":119},{"2001":124,"2002":125,"2003":126,"2004":127,"2005":128,"2006":129,"2007":130,"2008":131,"2009":127,"2010":132,"2011":133,"2012":134,"2013":135,"2014":136,"2015":137,"2016":138,"2017":139,"2018":140,"2019":141,"2020":142,"2021":143,"2022":144,"2023":145,"2024":146},{"2001":125,"2002":149,"2003":150,"2004":151,"2005":152,"2006":153,"2007":120,"2008":154,"2009":155,"2010":156,"2011":157,"2012":158,"2013":159,"2014":160,"2015":161,"2016":162,"2017":163,"2018":164,"2019":165,"2020":166,"2021":167,"2022":168,"2023":169,"2024":170},{"2001":173,"2002":174,"2003":175,"2004":176,"2005":177,"2006":178,"2007":179,"2008":180,"2009":181,"2010":182,"2011":183,"2012":184,"2013":185,"2014":186,"2015":187,"2016":188,"2017":189,"2018":190,"2019":191,"2020":192,"2021":193,"2022":194,"2023":113,"2024":195},{"volume":658,"pages":660},{"VOID":659},"24",{"VOID":661},"197-207","2022-05-10",2022,{"id":665,"createTime":666,"updateTime":667,"relativeEntities":668,"slug":669,"properties":670,"entityType":214,"verifyStatus":215,"verifyTime":667,"verifyNote":217,"syncStatus":19,"languages":20,"translateLanguages":20,"viewCount":21,"primaryUrl":679,"fullTextUrl":20,"authors":680,"publicationType":239,"publisherRelationship":725,"citationCount":20,"citationInfo":20,"publishDate":758,"publishYear":663,"citationAnalyzeStatus":19,"lastCitationAnalyze":20,"indexDatabases":20,"openAccess":20,"references":20,"isForceReanalyzing":328},"de81244b-b5c1-45be-9f23-384955f473e0","2023-12-20T18:44:51.321+00:00","2025-01-20T23:54:03.547+00:00",[],"The-estimation-of-health-state-utility-values-in-rare-diseases-do-the-approaches-in-submissions-for-NICE-technology-appraisals-reflect-the-existing-literature-A-scoping-review",{"references":671,"abstract":673,"title":675,"doi":677},{"VOID":672},"Bogart, K.R., Irvin, V.L.: Health-related quality of life among adults with diverse rare disorders. Orphanet J. Rare Dis. 12(1), 177 (2017)\nU.S. Food and Drug Administration (FDA): Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims (2009). https:\u002F\u002Fwww.fda.gov\u002Fregulatory-information\u002Fsearch-fda-guidance-documents\u002Fpatient-reported-outcome-measures-use-medical-product-development-support-labeling-claims. Accessed 16 Mar 2021\nWeldring, T., Smith, S.M.: Patient-reported outcomes (PROs) and patient-reported outcome measures (PROMs). Health Serv. Insights 6, 61–68 (2013)\nBansback, N., Harrison, M., Brazier, J., Davies, L., Kopec, J., Marra, C., et al.: Health state utility values: a description of their development and application for rheumatic diseases. Arthritis Rheum. 59(7), 1018–1026 (2008)\nLamers, L.M.: The transformation of utilities for health states worse than death: consequences for the estimation of EQ-5D value sets. Med. Care 45(3), 238–244 (2007)\nArnold, D., Girling, A., Stevens, A., Lilford, R.: Comparison of direct and indirect methods of estimating health state utilities for resource allocation: review and empirical analysis. Br. Med. J. 339, b2688 (2009)\nBahrampour, M., Byrnes, J., Norman, R., Scuffham, P.A., Downes, M.: Discrete choice experiments to generate utility values for multi-attribute utility instruments: a systematic review of methods. Eur. J. Health Econ. 21(7), 983–992 (2020)\nBrazier, J., Ara, R., Rowen, D., Chevrou-Severac, H.: A review of generic preference-based measures for use in cost-effectiveness models. Pharmacoeconomics 35(Suppl 1), 21–31 (2017)\nNational Institute for Health and Care Excellence (NICE): guide to the methods of technology appraisal 2013 (2013). https:\u002F\u002Fwww.nice.org.uk\u002Fprocess\u002Fpmg9\u002Fresources\u002Fguide-to-the-methods-of-technology-appraisal-2013-pdf-2007975843781. Accessed 4 Jul 2021\nNational Institute for Health and Care Excellence (NICE): Position statement on use of the EQ-5D-5L value set for England (updated October 2019). https:\u002F\u002Fwww.nice.org.uk\u002Fabout\u002Fwhat-we-do\u002Four-programmes\u002Fnice-guidance\u002Ftechnology-appraisal-guidance\u002Feq-5d-5l. Accessed 19 Apr 2022\nLongworth, L., Rowen, D.: Mapping to obtain EQ-5D utility values for use in NICE health technology assessment. Value Health 16(1), 202–210 (2013)\nMeregaglia, M., Nicod, E., Drummond, M.: The estimation of health state utility values in rare diseases: overview of existing techniques. Int. J. Technol. Assess. Health Care 36(5), 469–473 (2020)\nMeregaglia, M., Whittal, A., Nicod, E., Drummond, M.: “Mapping” health state utility values from non-preference-based measures: a systematic literature review in rare diseases. Pharmacoeconomics 38(6), 557–574 (2020)\nRowen, D., Brazier, J., Ara, R., Azzabi, Z.I.: The role of condition-specific preference-based measures in health technology assessment. Pharmacoeconomics 35(Suppl 1), 33–41 (2017)\nMcLoughlin, C., Goranitis, I., Al-Janabi, H.: Validity and responsiveness of preference-based quality-of-life measures in informal carers: a comparison of 5 measures across 4 conditions. Value Health 23(6), 782–790 (2020)\nSlade, A., Isa, F., Kyte, D., Pankhurst, T., Kerecuk, L., Ferguson, J., et al.: Patient reported outcome measures in rare diseases: a narrative review. Orphanet J. Rare Dis. 13(1), 61 (2018)\nGoodrich, K., Kaambwa, B., Al-Janabi, H.: The inclusion of informal care in applied economic evaluation: a review. Value Health 15(6), 975–981 (2012)\nNicod, E., Meregaglia, M., Whittal, A., Upadhyaya, S., Facey, K., Drummond, M.: Consideration of quality of life in the health technology assessments of rare disease treatments. Eur. J. Health Econ. 23(4), 645–669 (2022)\nWhittal, A., Meregaglia, M., Nicod, E.: The use of patient reported outcome measures in rare diseases and implications for health technology assessment. Patient 14(5), 485–503 (2021)\nPowell T., O'Donnell M.: NICE appraisals of rare diseases (2019). https:\u002F\u002Fresearchbriefings.files.parliament.uk\u002Fdocuments\u002FCDP-2019-0022\u002FCDP-2019-0022.pdf. Accessed 5 Nov 2021\nOrphanet: Prevalence and incidence of rare diseases: bibliographic data. Orphanet Report Series. 1 (2022) https:\u002F\u002Fwww.orpha.net\u002Forphacom\u002Fcahiers\u002Fdocs\u002FGB\u002FPrevalence_of_rare_diseases_by_alphabetical_list.pd. Accessed 13 Apr 2022\nTricco, A.C., Lillie, E., Zarin, W., O’Brien, K.K., Colquhoun, H., Levac, D., et al.: PRISMA extension for scoping reviews (PRISMA-ScR): checklist and explanation. Ann. Intern. Med. 169(7), 467–473 (2018)\nMunn, Z., Peters, M.D., Stern, C., Tufanaru, C., McArthur, A., Aromataris, E.: Systematic review or scoping review? Guidance for authors when choosing between a systematic or scoping review approach. BMC Med. Res. Methodol. 18(1), 143 (2018)\nNational Institute for Health and Care Excellence (NICE): Burosumab for treating X-linked hypophosphataemia in children and young people. Highly specialised technologies guidance (HST8). 10 October 2018. https:\u002F\u002Fwww.nice.org.uk\u002Fguidance\u002FHST8. Accessed 16 Jun 2020\nNational Institute for Health and Care Excellence (NICE): Asfotase alfa for treating paediatric onset hypophosphatasia. Highly Specialised Technology Guidance (HST6). 2 August 2017. https:\u002F\u002Fwww.nice.org.uk\u002Fguidance\u002Fhst6. Accessed 16 Jun 2020\nNational Institute for Health and Care Excellence (NICE): Eculizumab for treating atypical haemolytic uraemic syndrome. Highly specialised technologies guidance (HST1). 28 January 2015. https:\u002F\u002Fwww.nice.org.uk\u002Fguidance\u002Fhst1. Accessed 16 Jun 2020\nNational Institute for Health and Care Excellence (NICE): Migalastat for treating Fabry disease. Highly specialised technologies guidance (HST4). 22 February 2017. https:\u002F\u002Fwww.nice.org.uk\u002Fguidance\u002Fhst4. Accessed 16 Jun 2020\nNational Institute for Health and Care Excellence (NICE): Inotersen for treating hereditary transthyretin amyloidosis. Highly specialised technology guidance (HST9). 22 May 2019. https:\u002F\u002Fwww.nice.org.uk\u002Fguidance\u002FHST9. Accessed 16 Jun 2020\nNational Institute for Health and Care Excellence (NICE): Patisiran for treating hereditary transthyretin amyloidosis. Highly specialised technology guidance (HST10). 14 August 2019. https:\u002F\u002Fwww.nice.org.uk\u002Fguidance\u002FHST10. Accessed 16 Jun 2020\nNational Institute for Health and Care Excellence (NICE): Voretigene neparvovec for treating inherited retinal dystrophies caused by RPE65 gene mutations. Highly specialised technologies guidance (HST11). 9 October 2019. https:\u002F\u002Fwww.nice.org.uk\u002Fguidance\u002FHST11. Accessed 16 Jun 2020\nNational Institute for Health and Care Excellence (NICE): Mannitol dry powder for inhalation for treating cystic fibrosis. Technology Appraisal Guidance (TA266). 28 November 2012. https:\u002F\u002Fwww.nice.org.uk\u002FGuidance\u002FTA266. Accessed 16 Jun 2020\nNational Institute for Health and Care Excellence (NICE): Lumacaftor–ivacaftor for treating cystic fibrosis homozygous for the F508del mutation. Technology Appraisal Guidance (TA398). 27 July 2016. https:\u002F\u002Fwww.nice.org.uk\u002Fguidance\u002Fta398. Accessed 16 Jun 2020\nNational Institute for Health and Care Excellence (NICE): Colistimethate sodium and tobramycin dry powders for inhalation for treating pseudomonas lung infection in cystic fibrosis. Technology appraisal guidance (TA276). 27 March 2013. https:\u002F\u002Fwww.nice.org.uk\u002Fguidance\u002Fta276. Accessed 16 Jun 2020\nNational Institute for Health and Care Excellence (NICE): Elosulfase alfa for treating mucopolysaccharidosis type IVa. Highly specialised technologies guidance (HST2). 16 December 2015. https:\u002F\u002Fwww.nice.org.uk\u002Fguidance\u002Fhst2. Accessed 16 Jun 2020\nNational Institute for Health and Care Excellence (NICE): Strimvelis for treating adenosine deaminase deficiency - severe combined immunodeficiency. Highly specialised technology guidance (HST7). 7 February 2018. https:\u002F\u002Fwww.nice.org.uk\u002Fguidance\u002Fhst7. 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Chest 108(6), 1594–1601 (1995)\nRice, S., Albani, V., Minos, D., Fattakhova, G., Mells, G.F., Carbone, M., et al.: Effects of primary biliary cholangitis on quality of life and health care costs in the United Kingdom. Clin. Gastroenterol. Hepatol. 19(4), 768-776.e10 (2021)\nRombach, S.M., Hollak, C.E.M., Linthorst, G.E., Dijkgraaf, M.G.W.: Cost-effectiveness of enzyme replacement therapy for Fabry disease. Orphanet J. Rare Dis. 8, 29 (2013)\nSalisbury, M.L., Conoscenti, C.S., Culver, D.A., Yow, E., Neely, M.L., Bender, S., et al.: Antifibrotic drug use in patients with idiopathic pulmonary fibrosis. Data from the IPF-PRO registry. Ann. Am. Thorac. Soc. 17(11), 1413–1423 (2020)\nSantana, M.J., Feeny, D., Ghosh, S., Lien, D.C.: Patient-reported outcome 2 years after lung transplantation: does the underlying diagnosis matter? Patient Relat. Outcome Meas. 3, 79–84 (2012)\nSelvadurai, H.C., Blimkie, C.J., Meyers, N., Mellis, C.M., Cooper, P.J., Van Asperen, P.P.: Randomized controlled study of in-hospital exercise training programs in children with cystic fibrosis. Pediatr. Pulmonol. 33(3), 194–200 (2002)\nShulgina, L., Cahn, A.P., Chilvers, E.R., Parfrey, H., Clark, A.B., Wilson, E.C.F., et al.: Treating idiopathic pulmonary fibrosis with the addition of co-trimoxazole: a randomised controlled trial. Thorax 68(2), 155–162 (2013)\nSinger, J.P., Katz, P.P., Soong, A., Shrestha, P., Huang, D., Ho, J., et al.: The effect of lung transplantation on health related quality of life in the era of the lung allocation score: a US prospective cohort study. Am. J. Transplant. 17(5), 1334–1345 (2017)\nSinger, L.G., Chowdhury, N.A., Faughnan, M.E., Granton, J., Keshavjee, S., Marras, T.K., et al.: Effects of recipient age and diagnosis on health-related quality-of-life benefit of lung transplantation. Am. J. Respir. Crit. Care Med. 192(8), 965–973 (2015)\nSmith, A.B., Retzler, J., Taylor, M.J.: Standard gamble to derive utility health states for limbal stem cell deficiency. Clinicoecon. Outcomes Res. 12, 535–546 (2020)\nSolem, C.T., Vera-Llonch, M., Liu, S., Botteman, M., Castiglione, B.: Impact of pulmonary exacerbations and lung function on generic health-related quality of life in patients with cystic fibrosis. Health Qual. Life Outcomes 14, 63 (2016)\nSundh, J., Bornefalk, H., Sköld, C.M., Janson, C., Blomberg, A., Sandberg, J., et al.: Clinical validation of the Swedish version of Dyspnoea-12 instrument in outpatients with cardiorespiratory disease. BMJ Open Resp. Res. 6(1), e000418 (2019)\nSuri, R., Metcalfe, C., Lees, B., Grieve, R., Flather, M., Normand, C., et al.: Comparison of hypertonic saline and alternate-day or daily recombinant human deoxyribonuclease in children with cystic fibrosis: a randomised trial. Lancet 358(9290), 1316–1321 (2001)\nSzentes, B.L., Kreuter, M., Bahmer, T., Birring, S.S., Claussen, M., Waelscher, J., et al.: Quality of life assessment in interstitial lung diseases: a comparison of the disease specific K-BILD with the generic EQ-5D-5L. Respir. Res. 19(1), 101 (2018)\nTsai, A.P.Y., Hur, S.A., Wong, A., Safavi, M., Assayag, D., Johannson, K.A., et al.: Minimum important difference of the EQ-5D-5L and EQ-VAS in fibrotic interstitial lung disease. Thorax 76(1), 37–43 (2021)\nTsuboi, K., Yamamoto, H.: Clinical observation of patients with Fabry disease after switching from agalsidase beta (Fabrazyme) to agalsidase alfa (Replagal). Genet. Med. 14(9), 779–786 (2012)\nTzanakis, N., Samiou, M., Lambiri, I., Antoniou, K., Siafakas, N., Bouros, D.: Evaluation of health-related quality-of-life and dyspnea scales in patients with idiopathic pulmonary fibrosis. Correlation with pulmonary function tests. Eur. J. Intern. Med. 16(2), 105–112 (2005)\nVan der Ploeg, C.P.B., Blom, M., Bredius, R.G.M., van der Burg, M., Schielen, P.C.J.I., Verkerk, P.H., et al.: Cost-effectiveness of newborn screening for severe combined immunodeficiency. Eur. J. Pediatr. 178(5), 721–729 (2019)\nvan Dussen, L., Biegstraaten, M., Hollak, C.E.M., Dijkgraaf, M.G.W.: Cost-effectiveness of enzyme replacement therapy for type 1 Gaucher disease. Orphanet J. Rare Dis. 9, 51 (2014)\nVasiliadis, H.M., Collet, J.P., Penrod, J.R., Ferraro, P., Poirier, C.: A cost-effectiveness and cost-utility study of lung transplantation. J. Heart Lung Transplant. 24(9), 1275–1283 (2005)\nWapenaar, M., Patel, A.S., Birring, S.S., van Domburg, R.T., Wp, B.E., Vindigni, V., et al.: Translation and validation of the king’s brief interstitial lung disease (K-BILD) questionnaire in French, Italian, Swedish, and Dutch. Chron. Respir. Dis. 14(2), 140–150 (2017)\nWhiting, P., Al, M., Burgers, L., Westwood, M., Ryder, S., Hoogendoorn, M., et al.: Ivacaftor for the treatment of patients with cystic fibrosis and the G551D mutation: a systematic review and cost-effectiveness analysis. Health Technol. Assess. 18(18), 1–106 (2014)\nWixner, J., Mundayat, R., Karayal, O.N., Anan, I., Karling, P., Suhr, O.B., THAOS investigators: THAOS: gastrointestinal manifestations of transthyretin amyloidosis - common complications of a rare disease. Orphanet J. Rare Dis. 9, 61 (2014)\nWyatt, K., Henley, W., Anderson, L., Anderson, R., Nikolaou, V., Stein, K.: The effectiveness and cost-effectiveness of enzyme and substrate replacement therapies: a longitudinal cohort study of people with lysosomal storage disorders. Health Technol. Assess. 16(39), 1–543 (2012)\nYi, M.S., Britto, M.T., Wilmott, R.W., Kotagal, U.R., Eckman, M.H., Nielson, D.W., et al.: Health values of adolescents with cystic fibrosis. J. Pediatr. 142(2), 133–140 (2003)\nYounossi, Z.M., Boparai, N., McCormick, M., Price, L.L., Guyatt, G.: Assessment of utilities and health-related quality of life in patients with chronic liver disease. Am. J. Gastroenterol. 96(2), 579–583 (2001)\nZisman, D.A., Schwarz, M., Anstrom, K.J., Collard, H.R., Flaherty, K.R., Hunninghake, G.W., Idiopathic Pulmonary Fibrosis Clinical Research: A controlled trial of sildenafil in advanced idiopathic pulmonary fibrosis. N Engl J Med. 363(7), 620–628 (2010)\nZuluaga-Sanchez, S., Teynor, M., Knight, C., Thompson, R., Lundqvist, T., Ekelund, M., et al.: Cost effectiveness of nusinersen in the treatment of patients with infantile onset and later onset spinal muscular atrophy in Sweden. Pharmacoeconomics 37(6), 845–865 (2019)\nBeusterien, K.M., Davies, J., Leach, M., Meiklejohn, D., Grinspan, J.L., O’Toole, A., et al.: Population preference values for treatment outcomes in chronic lymphocytic leukaemia: a cross-sectional utility study. Health Qual. Life Outcomes 8, 50 (2010)\nTolley, K., Goad, C., Yi, Y., Maroudas, P., Haiderali, A., Thompson, G.: Utility elicitation study in the UK general public for late-stage chronic lymphocytic leukaemia. Eur. J. Health Econ. 14(5), 749–759 (2013)\nBüchter, R.B., Weise, A., Pieper, D.: Development, testing and use of data extraction forms in systematic reviews: a review of methodological guidance. BMC Med. Res. Methodol. 20(1), 259 (2020)\nKennedy-Martin, M., Slaap, B., Herdman, M., van Reenen, M., Kennedy-Martin, T., Greiner, W., et al.: Which multi-attribute utility instruments are recommended for use in cost-utility analysis? A review of national health technology assessment (HTA) guidelines. Eur. J. Health Econ. 21(8), 1245–1257 (2020)",{"EN":674},"Rare diseases negatively impact patients’ quality of life, but the estimation of health state utility values (HSUVs) in research studies and cost–utility models for health technology assessment is challenging. This study compared the methods for estimating the HSUVs included in manufacturers’ submissions of orphan drugs to the National Institute for Health and Care Excellence (NICE) with those of published studies addressing the same rare diseases to understand whether manufacturers fully exploited the existing literature in developing their economic models. All NICE Technology Appraisal (TA) and Highly Specialized Technologies (HST) guidance documents of non-cancer European Medicines Agency (EMA) orphan medicinal products were reviewed and compared with any published primary studies, retrieved via PubMed until November 2020, and estimating HSUVs for the same conditions addressed in manufacturers’ submissions. We identified 22 NICE TA\u002FHST appraisal reports addressing 19 different rare diseases. Sixteen reports presented original HSUVs estimated using EQ-5D or Health Utility Index (n = 12), direct methods (n = 2) or mapping (n = 2), while the other six included values obtained from the literature only. In parallel, we identified 111 published studies: 86.6% used preference-based measures (mainly EQ-5D, 60.7%), 12.5% direct techniques, and 2.7% mapping. The collection of values from non-patient populations (using ‘vignettes’) was more frequent in manufacturers’ submissions than in the literature (22.7% vs. 8.0%). The agreement on methodological choices between manufacturers’ submissions and published literature was only partial. More efforts should be made by manufacturers to accurately reflect the academic literature and its methodological recommendations in orphan drugs submissions.",{"EN":676},"The estimation of health state utility values in rare diseases: do the approaches in submissions for NICE technology appraisals reflect the existing literature? 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International Diabetes Federation, Brussels (2015)",{},{"id":20,"text":886,"url":20,"identifiers":887},"World Health Organization.: Diagnostic Criteria and Classification of Hyperglycaemia First Detected in Pregnancy. WHO, Geneva (2013)",{},{"id":20,"text":889,"url":20,"identifiers":890},"Linnenkamp, U., Guariguata, L., Beagley, J., Whiting, D.R., Cho, N.H.: The IDF diabetes atlas methodology for estimating global prevalence of hyperglycaemia in pregnancy. Diabetes Res. Clin. Pract. 103(2), 186–196 (2014)",{"doi":891},"10.1016\u002Fj.diabres.2013.11.004",{"id":20,"text":893,"url":20,"identifiers":894},"Kanguru, L., Bezawada, N., Hussein, J., Bell, J.: The burden of diabetes mellitus during pregnancy in low- and middle-income countries: a systematic review. Glob. Health Action 7, 23987 (2014)",{"doi":895},"10.3402\u002Fgha.v7.23987",{"id":20,"text":897,"url":20,"identifiers":898},"Modder, J.F.J.: CMACE\u002FRCOG joint guideline. Management of women with obesity in pregnancy. 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Adherence 12, 2593–2603 (2018)",{"doi":1539},"10.2147\u002FPPA.S182172",{"id":20,"text":1541,"url":20,"identifiers":1542},"Eisele, M., Kaduszkiewicz, H., König, H.-H., et al.: Determinants of health-related quality of life in older primary care patients: results of the longitudinal observational AgeCoDe Study. Br. J. Gen. Pract. 65, e716 (2015). https:\u002F\u002Fdoi.org\u002F10.3399\u002Fbjgp15X687337",{"doi":1543},"10.3399\u002Fbjgp15X687337",{"id":20,"text":1545,"url":20,"identifiers":1546},"Saccò, M., Meschi, M., Regolisti, G., et al.: The Relationship between blood pressure and pain. J. Clin. Hypertens. 15, 600–605 (2013). https:\u002F\u002Fdoi.org\u002F10.1111\u002Fjch.12145",{"doi":1547},"10.1111\u002Fjch.12145",{"id":20,"text":1549,"url":20,"identifiers":1550},"Prazeres, F., Santiago, L.: Relationship between health-related quality of life, perceived family support and unmet health needs in adult patients with multimorbidity attending primary care in Portugal: a multicentre cross-sectional study (Survey). Health Qual. Life Outcomes (2016). https:\u002F\u002Fdoi.org\u002F10.1186\u002Fs12955-016-0559-7",{"doi":1551},"10.1186\u002Fs12955-016-0559-7",{"id":20,"text":1553,"url":20,"identifiers":1554},"Wang, L., Palmer, A., Cocker, F., et al.: Multimorbidity and health-related quality of life (HRQoL) in a nationally representative population sample: implications of count versus cluster method for defining multimorbidity on HRQoL. Health Qual. Life Outcomes (2017). https:\u002F\u002Fdoi.org\u002F10.1186\u002Fs12955-016-0580-x",{"doi":1555},"10.1186\u002Fs12955-016-0580-x",{"id":20,"text":1557,"url":20,"identifiers":1558},"Park, B., Ock, M., Lee, H.A., et al.: Multimorbidity and health-related quality of life in Koreans aged 50 or older using KNHANES 2013–2014. Health Qual. Life Outcomes (2018). https:\u002F\u002Fdoi.org\u002F10.1186\u002Fs12955-018-1016-6",{"doi":1559},"10.1186\u002Fs12955-018-1016-6",{"id":20,"text":1561,"url":20,"identifiers":1562},"Herdman, M., Gudex, C., Lloyd, A., et al.: Development and preliminary testing of the new five-level version of EQ-5D (EQ-5D-5L). An. Int. J. Qual. Life Asp. Treat Care Rehabil. 20, 1727–1736 (2011). https:\u002F\u002Fdoi.org\u002F10.1007\u002Fs11136-011-9903-x",{"doi":1563},"10.1007\u002Fs11136-011-9903-x",{"id":20,"text":1565,"url":20,"identifiers":1566},"Hajian-Tilaki, K., Heidari, B., Hajian-Tilaki, A.: Are gender differences in health-related quality of life attributable to sociodemographic characteristics and chronic disease conditions in elderly people? Int. J. Prev. Med. 8, 95 (2017). https:\u002F\u002Fdoi.org\u002F10.4103\u002Fijpvm.IJPVM_197_16",{"doi":1567},"10.4103\u002Fijpvm.IJPVM_197_16",{"id":20,"text":1569,"url":20,"identifiers":1570},"Szende, A., Janssen, B., Cabases, J.: Self-reported population health: an international perspective based on EQ-5D [electronic resource]. (2014)",{"doi":1571},"10.1007\u002F978-94-007-7596-1",{"id":20,"text":1573,"url":20,"identifiers":1574},"Revicki, D., Hays, R.D., Cella, D., et al.: Recommended methods for determining responsiveness and minimally important differences for patient-reported outcomes. J. Clin. Epidemiol. 61, 102–109 (2008). https:\u002F\u002Fdoi.org\u002F10.1016\u002Fj.jclinepi.2007.03.012",{"doi":1575},"10.1016\u002Fj.jclinepi.2007.03.012",{"id":20,"text":1577,"url":20,"identifiers":1578},"Jaeschke, R., Singer, J., Guyatt, G.H.: Measurement of health status: ascertaining the minimal clinically important difference. Control Clin. Trials 10, 407–415 (1989). https:\u002F\u002Fdoi.org\u002F10.1016\u002F0197-2456(89)90005-6",{"doi":1579},"10.1016\u002F0197-2456(89)90005-6",{"id":1581,"createTime":1582,"updateTime":1583,"relativeEntities":1584,"slug":1585,"properties":1586,"entityType":214,"verifyStatus":215,"verifyTime":1583,"verifyNote":217,"syncStatus":19,"languages":20,"translateLanguages":20,"viewCount":21,"primaryUrl":1595,"fullTextUrl":20,"authors":1596,"publicationType":239,"publisherRelationship":1660,"citationCount":20,"citationInfo":20,"publishDate":1694,"publishYear":1695,"citationAnalyzeStatus":19,"lastCitationAnalyze":20,"indexDatabases":20,"openAccess":20,"references":20,"isForceReanalyzing":328},"10bdf2c5-76b9-41f3-b2dd-634d44d4eb56","2024-01-16T22:07:55.408+00:00","2025-02-22T23:49:33.889+00:00",[],"Predicting-hospital-costs-for-patients-receiving-renal-replacement-therapy-to-inform-an-economic-evaluation",{"references":1587,"abstract":1589,"title":1591,"doi":1593},{"VOID":1588},"Tonelli, M., Wiebe, N., Knoll, G., Bello, A., Browne, S., Jadhav, D., Klarenbach, S., Gill, J.: Systematic review: kidney transplantation compared with dialysis in clinically relevant outcomes. Am. J. Transplant. 11(10), 2093–2109 (2011)\nDepartment of Health. The National Service Framework for renal services. Part one: dialysis and transplantation (2004), https:\u002F\u002Fwww.gov.uk\u002Fgovernment\u002Fuploads\u002Fsystem\u002Fuploads\u002Fattachment_data\u002Ffile\u002F199001\u002FNational_Service_Framework_for_Renal_Services_Part_One_-_Dialysis_and_Transplantation.pdf. Accessed 16 July 2014\nRao, A., Steenkamp, R., Caskey, F.: UK Renal Registry 16th annual report. Chapter 5. Comorbidities and current smoking status amongst patients starting renal replacement therapy in England, Wales and Northern Ireland from 2011 to 2012. Nephron. Clin. Pract 125, 99–110 (2013)\nMetcalfe, W., Khan, I.H., Prescott, G.J., Simpson, K., Macleod, A.M.: Hospitalization in the first year of renal replacement therapy for end-stage renal disease. QJM 96(12), 899–909 (2003)\nHospital episode statistics, http:\u002F\u002Fwww.hscic.gov.uk\u002Fhes. Accessed 2 April 2015\nFotheringham, J., Fogarty, D., Jacques, R., ElNahas, M., Campbell, M.: Chapter 13. The linkage of incident renal replacement therapy patients in England (2002–2006) to hospital episodes and national mortality data: improved demography and hospitalisation data in patients undergoing renal replacement therapy. Nephron. Clin. Pract 120(Suppl 1), c247–c260 (2012)\nUK Renal Registry, https:\u002F\u002Fwww.renalreg.org\u002Fdatasets\u002Fthe-uk-renal-registry-dataset\u002F. Accessed 2 April 2015\nDepartment of Health. Confirmation of payment by results (PbR) arrangements for 2011-12, 2011-12 tariff information spreadsheet. http:\u002F\u002Fwebarchive.nationalarchives.gov.uk\u002F20130507170152\u002Fhttps:\u002Fwww.gov.uk\u002Fgovernment\u002Fpublications\u002Fconfirmation-of-payment-by-results-pbr-arrangements-for-2011-12. Accessed 21 January 2014\nMullahy, J.: Much ado about two: reconsidering retransformation and the two-part model in health econometrics. J. Health. Econ. 17(3), 247–281 (1998)\nDunn, G., Mirandola, M., Amaddeo, F., Tansella, M.: Describing, explaining or predicting mental health care costs: a guide to regression models. Methodological review. Br. J. Psychiatry 183, 398–404 (2003)\nBuntin, M.B., Zaslavsky, A.M.: Too much ado about two-part models and transformation? Comparing methods of modeling Medicare expenditures. J. Health. Econ. 23(3), 525–542 (2004)\nBarber, J., Thompson, S.: Multiple regression of cost data: use of generalised linear models. J. Health Serv. Res. Policy 9(4), 197–204 (2004)\nHardin, J.W.H., J.M.: Generalized linear models and extensions, 2nd ed. Stata Press, Lakeway Drive, Texas (2007)\nKirkwood, B.R., Sterne, J.A.C.: Essential medical statistics, 2nd ed. Blackwell Publishing Oxford (2003)\nHaller, M., Gutjahr, G., Kramar, R., Harnoncourt, F., Oberbauer, R.: Cost-effectiveness analysis of renal replacement therapy in Austria. Nephrol. Dial. Transplant. 26(9), 2988–2995 (2011)\nHoward, K., Salkeld, G., White, S., McDonald, S., Chadban, S., Craig, J.C., Cass, A.: The cost-effectiveness of increasing kidney transplantation and home-based dialysis. Nephrology 14(1), 123–132 (2009)\nJassal, S.V., Krahn, M.D., Naglie, G., Zaltzman, J.S., Roscoe, J.M., Cole, E.H., Redelmeier, D.A.: Kidney transplantation in the elderly: a decision analysis. J. Am. Soc. Nephrol. 14(1), 187–196 (2003)\nKontodimopoulos, N., Niakas, D.: An estimate of lifelong costs and QALYs in renal replacement therapy based on patientsʼ life expectancy. Health Policy 86, 85–96 (2008)\nde Wit, G.A., Ramsteijn, P.G., de Charro, F.T.: Economic evaluation of end stage renal disease treatment. Health Policy 44, 215–232 (1998)\nWong, G., Howard, K., Chapman, J.R., Chadban, S., Cross, N., Tong, A., Webster, A.C., Craig, J.C.: Comparative survival and economic benefits of deceased donor kidney transplantation and dialysis in people with varying ages and co-morbidities. PLoS one 7(1) (2012)\nBriggs, A., Clark, T., Wolstenholme, J., Clarke, P.: Missing… presumed at random: cost-analysis of incomplete data. Health Econ. 12(5), 377–392 (2003)\nSterne, J.A., White, I.R., Carlin, J.B., Spratt, M., Royston, P., Kenward, M.G., Wood, A.M., Carpenter, J.R.: Multiple imputation for missing data in epidemiological and clinical research: potential and pitfalls. BMJ 338, b2393 (2009)\nBang, H.T., Tsiatis, A.A.: Estimating medical costs with censored data. Biometrika 87, 329–343 (2000)\nCarides, G.W., Heyse, J.F., Iglewicz, B.: A regression-based method for estimating mean treatment cost in the presence of right-censoring. Biostatistics 1(3), 299–313 (2000)\nLin, D.Y., Feuer, E.J., Etzioni, R., Wax, Y.: Estimating medical costs from incomplete follow-up data. Biometrics 53(2), 419–434 (1997)\nO’Hagan, A., Stevens, J.W.: On estimators of medical costs with censored data. J. Health Econ. 23(3), 615–625 (2004)\nYoung, T.A.: Estimating mean total costs in the presence of censoring: a comparative assessment of methods. Pharmacoeconomics 23(12), 1229–1242 (2005)",{"EN":1590},"To develop a model to predict annual hospital costs for patients with established renal failure, taking into account the effect of patient and treatment characteristics of potential relevance for conducting an economic evaluation, such as age, comorbidities and time on treatment. The analysis focuses on factors leading to variations in inpatient and outpatient costs and excludes fixed costs associated with dialysis, transplant surgery and high cost drugs. Annual costs of inpatient and outpatient hospital episodes for patients starting renal replacement therapy in England were obtained from a large retrospective dataset. Multiple imputation was performed to estimate missing costs due to administrative censoring. Two-part models were developed using logistic regression to first predict the probability of incurring any hospital costs before fitting generalised linear models to estimate the level of cost in patients with positive costs. Separate models were developed to predict inpatient and outpatient costs for each treatment modality. Data on hospital costs were available for 15,869 incident dialysis patients and 4511 incident transplant patients. The two-part models showed a decreasing trend in costs with increasing number of years on treatment, with the exception of dialysis outpatient costs. Age did not have a consistent effect on hospital costs; however, comorbidities such as diabetes and peripheral vascular disease were strong predictors of higher hospital costs in all four models. Analysis of patient-level data can result in a deeper understanding of factors associated with variations in hospital costs and can improve the accuracy with which costs are estimated in the context of economic evaluations.",{"EN":1592},"Predicting hospital costs for patients receiving renal replacement therapy to inform an economic 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UK",{"title":1612},{"VI":1613},"Bernadette Li",{"id":1615,"sortIndex":509,"researcher":20,"roles":1616,"affiliations":1617,"properties":1628},"df75ad48-705c-49c5-80e7-2529a2a795b2",[223],[1618],{"id":20,"sortIndex":21,"affiliation":1619,"properties":20},{"id":1620,"createTime":1621,"updateTime":1622,"relativeEntities":1623,"slug":1624,"properties":1625,"entityType":48,"verifyStatus":19,"verifyTime":20,"verifyNote":20,"syncStatus":19,"languages":20,"translateLanguages":20,"viewCount":21},"e532ef4d-d1d5-4baa-bd11-cde7be08b792","2024-02-09T00:39:54.003+00:00","2024-10-08T23:38:17.157+00:00",[],"Sheffield-Kidney-Institute-Sheffield-UK",{"title":1626},{"VI":1627},"Sheffield Kidney Institute, Sheffield, UK",{"title":1629},{"VI":1630},"James Fotheringham",{"id":1632,"sortIndex":179,"researcher":20,"roles":1633,"affiliations":1634,"properties":1645},"c69b1736-e448-4f09-9d2b-a959375667d8",[223],[1635],{"id":20,"sortIndex":21,"affiliation":1636,"properties":20},{"id":1637,"createTime":1638,"updateTime":1639,"relativeEntities":1640,"slug":1641,"properties":1642,"entityType":48,"verifyStatus":19,"verifyTime":20,"verifyNote":20,"syncStatus":19,"languages":20,"translateLanguages":20,"viewCount":21},"08d93011-880f-41e5-9e22-bedf9ac73b35","2023-11-30T04:33:08.712+00:00","2025-06-11T21:49:22.453+00:00",[],"Richard-Bright-Renal-Unit-Southmead-Hospital-Bristol-UK",{"title":1643},{"VI":1644},"Richard Bright Renal Unit, Southmead Hospital, Bristol, UK",{"title":1646},{"VI":1647},"Rommel Ravanan",{"id":1649,"sortIndex":350,"researcher":20,"roles":1650,"affiliations":1651,"properties":1657},"f43845bd-b8f0-4ff3-9d74-510aaae79742",[223],[1652],{"id":20,"sortIndex":21,"affiliation":1653,"properties":20},{"id":1603,"createTime":1604,"updateTime":1605,"relativeEntities":1654,"slug":1607,"properties":1655,"entityType":48,"verifyStatus":19,"verifyTime":20,"verifyNote":20,"syncStatus":19,"languages":20,"translateLanguages":20,"viewCount":21},[],{"title":1656},{"VI":1610},{"title":1658},{"VI":1659},"John 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E.A.: Respiratory syncytial virus infection. Lancet 354(9181), 847–852 (1999)\nCanfield, S.D., Simoes, E.A.: Prevention of respiratory syncytial virus (RSV) infection: RSV immune globulin intravenous and palivizumab. Pediatr. Ann. 28(8), 507–514 (1999)\nClark, S., Beresford, M., Subhedar, N., Shaw, N.: Respiratory syncytial virus infection in high risk infants and the potential impact of prophylaxis in a United Kingdom cohort. Arch. Dis. Child. 83, 313–316 (2000)\nGroothuis, J.R., Simoes, E.A., Levin, M.J., et al.: Prophylactic administration of respiratory syncytial virus immune globulin to high-risk infants and young children. The Respiratory Syncytial Virus Immune Globulin Study Group. N. Engl. J. Med. 329, 1524–1530 (1993)\nThe PREVENT Study Group: Reduction of RSV hospitalisation among preterm infants and infants with bronchopulmonary dysplasia using respiratory syncytial virus immune globulin prophylaxis. 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J. 19(7), 592–597 (2000)\nOrden 234\u002F2005, de 23 de febrero, del Consejero de Sanidad y Consumo, por la que sefijan los precios públicos por la prestación de los servicios y actividades de naturaleza sanitaria de la Red de Centros de la Comunidad de Madrid. Boletín Oficial de la Comunidad de Madrid (BOCM) núm. 56, 8 de Marzo de, 2005\nBont, L., Aalderen, W.M., Kimpen, J.L.: Long-term consequences of respiratory syncytial virus (RSV) bronchiolitis. Paediatr. Respir. Rev. 1(3), 221–227 (2000)\nGreenough, A., Cox, S., Alexander, J., et al.: Health care utilisation of infants with chronic lung disease, related to hospitalisation for RSV infection. Arch. Dis. Child. 85(6), 463–468 (2004)\nInstituto Nacional de Estadística (INE).: Contabilidad Regional de España. Available at http:\u002F\u002Fine.es. (Consulted in January 2006)\nLaufer, F.: Thresholds in cost-effectiveness analysis—more of the story. Value Health 8(1), 86–87 (2005)",{"EN":1704},"To assess the cost effectiveness of palivizumab for prevention of severe respiratory syncytial virus (RSV) disease in high-risk infants in Spain, incorporating country-specific observational hospitalisation data. An existing decision tree model, designed using data from a large international clinical trial of palivizumab versus no prophylaxis, was updated to include Spanish observational hospitalisation data. The analysis was performed for preterm children born at or before 32 weeks gestational age, who are at high risk of developing severe RSV disease requiring hospitalisation. Data sources included published literature, official price\u002Ftariff lists and national population statistics. The primary perspective of the study was that of the Spanish National Health Service in 2006. The base-case analysis included the direct medical costs associated with palivizumab prophylaxis and hospital care for RSV infections. Use of palivizumab produces an undiscounted incremental cost-effectiveness ratio (ICER) of €6,142 per quality-adjusted life-year (QALY), and a discounted ICER of €12,814\u002FQALY. Palivizumab provides a cost-effective method of prophylaxis against severe RSV disease requiring hospitalisation among preterm infants in Spain.",{"EN":1706},"Cost effectiveness of palivizumab in Spain: an analysis using observational data",{"VOID":1708},"10.1007\u002Fs10198-009-0206-x","https:\u002F\u002Flink.springer.com\u002Farticle\u002F10.1007\u002Fs10198-009-0206-x",[1711,1739],{"id":1712,"sortIndex":21,"researcher":20,"roles":1713,"affiliations":1714,"properties":1736},"2472ccf9-2f0a-4f4a-a065-30ebe3d4c36c",[223],[1715,1725],{"id":20,"sortIndex":21,"affiliation":1716,"properties":20},{"id":1717,"createTime":1718,"updateTime":1719,"relativeEntities":1720,"slug":1721,"properties":1722,"entityType":48,"verifyStatus":19,"verifyTime":20,"verifyNote":20,"syncStatus":19,"languages":20,"translateLanguages":20,"viewCount":21},"09ce1a57-eefe-44a9-b6e8-2a9a96a1eb4b","2024-01-05T17:08:43.318+00:00","2025-06-11T22:44:16.978+00:00",[],"iMTA-Erasmus-University-Rotterdam-The-Netherlands",{"title":1723},{"VI":1724},"iMTA, Erasmus University, Rotterdam, The Netherlands",{"id":1726,"sortIndex":350,"affiliation":1727,"properties":1735},"875bb270-6d53-4d11-bd31-55c9d01a2859",{"id":1728,"createTime":1729,"updateTime":1729,"relativeEntities":1730,"slug":1731,"properties":1732,"entityType":48,"verifyStatus":19,"verifyTime":20,"verifyNote":20,"syncStatus":19,"languages":20,"translateLanguages":20,"viewCount":21},"c662f886-8b07-4101-ac1e-debf90a77ad3","2024-04-15T09:40:47.601+00:00",[],"Ars-Accessus-Medica-Amsterdam-The-Netherlands",{"title":1733},{"EN":1734},"Ars Accessus Medica, Amsterdam, The Netherlands",{},{"title":1737},{"VI":1738},"Mark J. 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