References
- Cost-effectiveness in health and medicine. Gold MR, Siegel JE, Russell LB, Weinstein MC. editors. Oxford University Press; NY, USA: 1996
- Prevention effectiveness: a guide to decision analysis and economic evaluation. Haddix AC, Teutsch SM, Corso PS. editors. 2nd edition. Oxford University Press; NY, USA: 2003
- Miners AH. Economic evaluations of prophylaxis with clotting factor for people with severe haemophilia: why do the results vary so much? Haemophilia 2013;19:174-80
- Colombo GL, Di Matteo S, Mancuso ME, Santagostino E. Cost-utility analysis of prophylaxis versus treatment on demand in severe hemophilia A. Clinicoecon Outcomes Res 2011;3:55-61
- Farrugia A, Cassar J, Kimber MC, et al. Treatment for life for severe haemophilia A- A cost-utility model for prophylaxis vs. on-demand treatment. Haemophilia 2013;19:e228-38
- Miners A. Revisiting the cost-effectiveness of primary prophylaxis with clotting factor for the treatment of severe haemophilia. Haemophilia 2009;15:881-7
- Miners AH, Sabin CA, Tolley KH, et al. A cost-utility analysis of primary prophylaxis versus treatment on-demand for individuals with severe haemophilia. Pharmacoeconomics 2002;20:759-74
- Risebrough N, Oh P, Blanchette V, et al. Cost-utility analysis of Canadian tailored prophylaxis, primary prophylaxis and on-demand therapy in young children with severe haemophilia A. Haemophilia 2008;14:743-52
- den Uijl I, Biesma D, Grobbee D, Fischer K. Turning severe into moderate haemophilia by prophylaxis: are we reaching our goal? Blood Transfus 2013;11:364-9
- Grosse SD. Assessing cost effectiveness in health care: the history of the $50,000 per QALY threshold. Expert Rev Pharmacoecon Outcomes Res 2008;8:165-78
- Farrugia A, O’Mahony B, Cassar J. Health technology assessment and haemophilia. Haemophilia 2012;18:152-7
- Rawlins MD, Culyer AJ. National Institute for Clinical Excellence and its value judgments. BMJ 2004;329:224-7
- Neumann PJ, Goldie SJ, Weinstein MC. Preference-based measures in economic evaluation in health care. Annu Rev Public Health 2000;21:587-611
- Nord E, Daniels N, Kamlet M. QALYs: some challenges. Value Health 2009;12:S10-15
- Ubel PA, Loewenstein G, Schwarz N, Smith D. Misimagining the unimaginable: the disability paradox and health care decision making. Health Psychol 2005;24:S57-62
- Bullinger M, Globe D, Wasserman J, et al. Challenges of patient-reported outcome assessment in hemophilia care – a state of the art review. Value Health 2009;12:808-20
- Revicki DA, Lenderking WR. Methods and issues associated with the use of quality-adjusted life-years. Expert Rev Pharmacoecon Outcomes Res 2012;12:105-14
- Torrance GW, Feeny D, Furlong W. Visual analog scales: do they have a role in the measurement of preferences for health states? Med Decis Making 2001;21:329-34
- Carroll AE, Downs SM. Improving decision analyses: parent preferences (utility values) for pediatric health outcomes. J Pediatr 2009;155:21-5
- Bleichrodt H. A new explanation for the difference between time trade-off utilities and standard gamble utilities. Health Econ 2002;11:447-56
- van Houtven G, Johnson FR, Kilambi V, Hauber AB. Eliciting benefit-risk preferences and probability-weighted utility using choice-format conjoint analysis. Med Decis Making 2011;31:469-80
- Bansback N, Tsuchiya A, Brazier J, Anis A. Canadian valuation of EQ-5D health states: preliminary value set and considerations for future valuation studies. PLoS One 2012;7(2):e31115
- Craig BM, Pickard AS, Stolk E, Brazier JE. US valuation of the SF-6D. Med Decis Making 2013;33:793-803
- Richardson J, McKie J, Bariola E. Review and critique of health related multi attribute utility instruments. Centre for Health Economics, Monash University. Research Paper 2011. p. 64. Available from: www.buseco.monash.edu.au/centres/che/pubs/researchpaper64.pdf
- Devlin NJ, Krabbe PF. The development of new research methods for the valuation of EQ-5D-5L. Eur J Health Econ 2013;14(Suppl 1):1-3
- Torrance GW, Feeny DH, Furlong WJ, et al. A multi-attribute utility function for a comprehensive health status classification system. Health Utilities Mark 2. Med Care 1996;34:702-22
- Feeny D, Furlong W, Torrance GW, et al. Multiattribute and single-attribute utility functions for the Health Utilities Index Mark 3 system. Med Care 2002;40:113-28
- Feeny D, Furlong W, Saigal S, et al. Comparing directly measured standard gamble scores to HUI2 and HUI3 utility scores: group- and individual-level comparisons. Soc Sci Med 2004;58:799-809
- Stevens KJ, McCabe CJ, Brazier JE. Mapping between visual analogue and standard gamble data; results from the UK Health Utilities Index 2 valuation. Health Econ 2005;15:527-34
- McDonough CM, Tosteson AN. Measuring preferences for cost-utility analysis: how choice of method may influence decision-making. Pharmacoeconomics 2007;25:93-106
- Feeny D. Standardization and regulatory guidelines may inhibit science and reduce the usefulness of analyses based on the application of preference-based measures for policy decisions. Med Decis Making 2013;33:316-29
- Grosse SD, Prosser LA, Asakawa K, Feeny D. QALY weights for neurosensory impairments in pediatric economic evaluations: case studies and a critique. Expert Rev Pharmacoecon Outcomes Res 2010;10:293-308
- Noyes K, Dick AW, Holloway RG. The implications of using US-specific EQ-5D preference weights for cost-effectiveness evaluation. Med Decis Making 2007;27:327-34
- Brazier J, Usherwood T, Harper R, et al. Deriving a preference-based single index from the UK SF-36 Health Survey. J Clin Epidemiol 1998;51:1115-28
- Brazier JE, Roberts J. The estimation of a preference-based measure of health from the SF-12. Med Care 2004;42:851-9
- Barton GR, Bankart J, Davis AC, Summerfield QA. Comparing utility scores before and after hearing-aid provision: results according to the EQ-5D, HUI3 and SF-6D. Appl Health Econ Health Policy 2004;3:103-5
- Teckle P, Peacock S, McTaggart-Cowan H, et al. The ability of cancer-specific and generic preference-based instruments to discriminate across clinical and self-reported measures of cancer severities. Health Qual Life Outcomes 2011;9:106
- Fryback DG, Palta M, Cherepanov D, et al. Comparison of 5 health-related quality-of-life indexes using item response theory analysis. Med Decis Making 2010;30:5-15
- Whitehurst DG, Bryan S, Lewis M. Systematic review and empirical comparison of contemporaneous EQ-5D and SF-6D group mean scores. Med Decis Making 2011;31:E34-44
- Gaujoux-Viala C, Rat AC, Guillemin F, et al. Responsiveness of EQ-5D and SF-6D in patients with early arthritis: results from the ESPOIR cohort. Ann Rheum Dis 2012;71:1478-83
- Noyes J, Edwards RT. EQ-5D for the assessment of health-related quality of life and resource allocation in children: a systematic methodological review. Value Health 2011;14:1117-29
- Ungar WJ. Challenges in health state valuation in paediatric economic evaluation: are QALYs contraindicated? Pharmacoeconomics 2011;29:641-52
- Payakachat N, Tilford JM, Kovacs E, Kuhlthau K. Autism spectrum disorders: a review of measures for clinical, health services and cost-effectiveness applications. Expert Rev Pharmacoecon Outcomes Res 2012;12:485-503
- Prosser LA, Grosse SD, Wittenberg E. Health utility elicitation: is there still a role for direct methods? Pharmacoeconomics 2012;30:83-6
- Valuing health for regulatory cost-effectiveness analysis. Institute of Medicine, Miller W, Robinson LA, Lawrence RS. editors. National Academies Press; Washington, DC, USA: 2006
- Naraine VS, Risebrough NA, Oh P, et al. Health-related quality-of-life treatments for severe haemophilia: utility measurements using the Standard Gamble technique. Haemophilia 2002;8:112-20
- Wasserman J, Aday LA, Begley CE, et al. Measuring health state preferences for hemophilia: development of a disease-specific utility instrument. Haemophilia 2005;11:49-57
- Miners AH, Sabin CA, Tolley KH, et al. Assessing health-related quality-of-life in individuals with haemophilia. Haemophilia 1999;5:378-85
- Trippoli S, Vaiani M, Linari S, et al. Multivariate analysis of factors influencing quality of life and utility in patients with haemophilia. Haematologica 2001;86:722-8
- Gringeri A, Mantovani LG, Scalone L, Mannucci PM. Cost of care and quality of life for patients with hemophilia complicated by inhibitors: the COCIS Study Group. Blood 2003;102:2356-63
- Hoots WK, Ebbesen LS, Konkle BA, et al. Secondary prophylaxis with recombinant activated factor VII improves health-related quality of life of haemophilia patients with inhibitors. Haemophilia 2008;14:466-75
- Stasyshyn O, Antunes S, Mamonov V, et al. Prophylaxis with anti-inhibitor coagulant complex improves health-related quality of life in haemophilia patients with inhibitors: results from FEIBA NF Prophylaxis Study. Haemophilia 2014;20:644-50
- Neufeld EJ, Recht M, Sabio H, et al. Effect of acute bleeding on daily quality of life assessments in patients with congenital hemophilia with inhibitors and their families: observations from the dosing observational study in hemophilia. Value Health 2012;15:916-25
- de Kleijn P, Sluiter D, Vogely HCh, et al. Long-term outcome of multiple joint procedures in haemophilia. Haemophilia 2014;20:276-81
- Noone D, O’Mahony B, Prihodova L. A survey of the outcome of prophylaxis, on-demand or combined treatment in 20-35 year old men with severe haemophilia in four European countries. Haemophilia 2011;17:e831-48
- Noone D, O’Mahony B, van Dijk JP, Prihodova L. A survey of the outcome of prophylaxis, on-demand treatment or combined treatment in 18-35-year old men with severe haemophilia in six countries. Haemophilia 2013;19:44-50
- Fischer K, Steen Carlsson K, Petrini P, et al. Intermediate-dose versus high-dose prophylaxis for severe hemophilia: comparing outcome and costs since the 1970s. Blood 2013;122:1129-36
- Szucs TD, Offner A, Schramm W. Socioeconomic impact of haemophilia care: results of a pilot study. Haemophilia 1996;2:211-21
- Barr RD, Saleh M, Furlong W, et al. Health status and health-related quality of life associated with hemophilia. Am J Hematol 2002;71:152-60
- Lippert B, Berger K, Berntorp E, et al. Cost effectiveness of haemophilia treatment: a cross-national assessment. Blood Coagul Fibrinolysis 2005;16:477-85
- Carvalhosa AM, Henrard S, Lambert C, Hermans C. Physical and mental quality of life in adult patients with haemophilia in Belgium: the impact of financial issues. Haemophilia 2014;20:479-85
- Gringeri A, Leissinger C, Cortesi PA, et al. Health-related quality of life in patients with haemophilia and inhibitors on prophylaxis with anti-inhibitor complex concentrate: results from the Pro-FEIBA study. Haemophilia 2013;19:736-43
- Henrard S, Devleesschauwer B, Beutels P, et al. The health and economic burden of haemophilia in Belgium: a rare, expensive and challenging disease. Orphanet J Rare Dis 2014;9:39
- Duncan N, Shapiro A, Ye X, et al. Treatment patterns, health-related quality of life and adherence to prophylaxis among haemophilia A patients in the United States. Haemophilia 2012;18:760-5
- Poon JL, Zhou ZY, Doctor JN, et al. Quality of life in haemophilia A. Hemophilia Utilization Group Study Va (HUGS- Va). Haemophilia 2012;18:699-707
- Royal S, Schramm W, Berntorp E, et al. Quality-of-life differences between prophylactic and on-demand factor replacement therapy in European haemophilia patients. Haemophilia 2002;8:44-50
- Szende A, Schramm W, Flood E, et al. Health-related quality of life assessment in adult haemophilia patients: a systematic review and evaluation of instruments. Haemophilia 2003;9:678-87
- Posthouwer D, Plug I, van der Bom JG, et al. Hepatitis C and health-related quality of life among patients with hemophilia. Haematologica 2005;90:846-50
- Elander J, Robinson G, Mitchell K, Morris J. An assessment of the relative influence of pain coping, negative thoughts about pain, and pain acceptance on health-related quality of life among people with hemophilia. Pain 2009;145:169-75
- Molho P, Rolland N, Lebrun T, et al. Epidemiological survey of the orthopaedic status of severe haemophilia A and B patients in France. Haemophilia 2000;6:23-32
- Lindvall K, Astermark J, Bjorkman S, et al. Daily dosing prophylaxis for haemophilia: a randomized crossover pilot study evaluating feasibility and efficacy. Haemophilia 2012;18:855-9
- Knight C, Paisley S, Wight J, Lloyd-Jones M. Economic modelling of different treatment strategies for haemophilia A with high-responding inhibitors. Haemophilia 2003;9:521-40
- Lubetkin E, Jia H, Franks P, Gold M. Relationship among sociodemographic factors, clinical conditions, and health-related quality of life: examining the EQ-5D in the U.S. general population. Qual Life Res 2005;14:2187-96
- Walters S, Brazier J. Comparison of the minimally important difference for two health state utility measures: EQ-5D and SF-6D. Qual Life Res 2005;4:1523-2532
- Siddiqi AE, Ebrahim SH, Soucie JM, et al. Burden of disease resulting from hemophilia in the U.S. Am J Prev Med 2010;38:S482-8
- Buchbinder D, Ragni V. What is the role of prophylaxis in the improvement of health-related quality of life of patients with hemophilia? Hematology Am Soc Hematol Educ Program 2013;2013:52-5
- Tagliaferri A, Franchini M, Coppola A, et al. Effects of secondary prophylaxis started in adolescent and adult haemophiliacs. Haemophilia 2008;14:945-51
- Pollmann H, Klamroth R, Vidovic N, et al. Prophylaxis and quality of life in patients with hemophilia A during routine treatment with ADVATE [antihemophilic factor (recombinant), plasma/albumin-free method] in Germany: a subgroup analysis of the ADVATE PASS post-approval, non-interventional study. Ann Hematol 2013;92:689-98
- Wiebe S, Guyatt G, Weaver B, et al. Comparative responsiveness of generic and specific quality-of-life instruments. J Clin Epidemiol 2003;56:52-60
- Marra CA, Woolcott JC, Kopec JA, et al. A comparison of generic, indirect utility measures (the HUI2, HUI3, SF-6D, and the EQ-5D) and disease-specific instruments (the RAQoL and the HAQ) in rheumatoid arthritis. Soc Sci Med 2005;60:1571-82
- Lloyd A, Kerr C, Breheny K, et al. Economic evaluation in short bowel syndrome (SBS): an algorithm to estimate utility scores for a patient-reported SBS-specific quality of life scale (SBS-QoL™). Qual Life Res 2014;23:449-58
- Versteegh MM, Leunis A, Uyl-de Groot CA, Stolk EA. Condition-specific preference-based measures: benefit or burden? Value Health 2012;15:504-13
- Lin FJ, Longworth L, Pickard AS. Evaluation of content on EQ-5D as compared to disease-specific utility measures. Qual Life Res 2013;22:853-74
- Davis EM, Lynd LD, Grubisic M, et al. Responsiveness of health state utility values in knee osteoarthritis. J Rheumatol 2013;40:2075-82
- von Mackensen S, Bullinger M; Haemo-QoL Group. Development and testing of an instrument to assess the Quality of Life of Children with Haemophilia in Europe (Haemo-QoL). Haemophilia 2004;1:17-25
- von Mackensen S, Campos IG, Acquadro C, Strandberg-Larsen M. Cross-cultural adaptation and linguistic validation of age-group-specific haemophilia patient-reported outcome (PRO) instruments for patients and parents. Haemophilia 2013;19:e73-83
- Rentz A, Flood E, Altisent C, et al. Cross-cultural development and psychometric evaluation of a patient-reported health-related quality of life questionnaire for adults with haemophilia. Haemophilia 2008;14:1023-34
- Young NL, Bradley CS, Blanchette V, et al. Development of a health-related quality of life measure for boys with haemophilia: the Canadian Haemophilia Outcomes–Kids Life Assessment Tool (CHO-KLAT). Haemophilia 2004;10:34-43
- Young NL, Bradley CS, Wakefield CD, et al. How well does the Canadian Haemophilia Outcomes-Kids’ Life Assessment Tool (CHO-KLAT) measure the quality of life of boys with haemophilia? Pediatr Blood Cancer 2006;47:305-11
- Fischer K, Pouw ME, Lewandowski D, et al. A modeling approach to evaluate long-term outcome of prophylactic and on demand treatment strategies for severe hemophilia A. Haematologica 2011;96:738-43
- Blome C, Beikert FC, Rustenbach SJ, Augustin M. Mapping DLQI on EQ-5D in psoriasis: transformation of skin-specific health-related quality of life into utilities. Arch Dermatol Res 2013;305:197-204
- Mulhern B, Rowen D, Brazier J, et al. Development of DEMQOL-U and DEMQOL-PROXY-U: generation of preference-based indices from DEMQOL and DEMQOL-PROXY for use in economic evaluation. Health Technol Assess 2013;17(5):1-140
- Groot Koerkamp B, Hunink MG, Stijnen T, et al. Limitations of acceptability curves for presenting uncertainty in cost-effectiveness analysis. Med Decis Making 2007;27:101-11
- Hay JW, Zhou ZY. Systematic literature review of economics analysis on treatment of mild-to-moderate bleeds with aPCC versus rFVIIa. J Med Econ 2011;14:516-25
- Rasekh HR, Imani A, Karimi M, Golestani M. Cost-utility analysis of immune tolerance induction therapy versus on-demand treatment with recombinant factor VII for hemophilia A with high titer inhibitors in Iran. Clinicoecon Outcomes Res 2011;3:207-12
- Konkle BA, Ebbesen LS, Erhardtsen E, et al. Randomized, prospective clinical trial of recombinant factor VIIa for secondary prophylaxis in hemophilia patients with inhibitors. J Thromb Haemost 2007;5:1904-13