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Volumn 34, Issue 3, 2016, Pages 285-301

Can the EVIDEM Framework Tackle Issues Raised by Evaluating Treatments for Rare Diseases: Analysis of Issues and Policies, and Context-Specific Adaptation

Author keywords

[No Author keywords available]

Indexed keywords

ARTICLE; CONCEPTUAL FRAMEWORK; DISEASE SEVERITY; EVALUATION STUDY; HEALTH CARE POLICY; MEDICAL ETHICS; OUTCOME ASSESSMENT; PRIORITY JOURNAL; RARE DISEASE; RATING SCALE; SCORING SYSTEM; DECISION SUPPORT SYSTEM; ECONOMICS; HUMAN; RARE DISEASES; SEVERITY OF ILLNESS INDEX; TREATMENT OUTCOME;

EID: 84959106689     PISSN: 11707690     EISSN: 11792027     Source Type: Journal    
DOI: 10.1007/s40273-015-0340-5     Document Type: Article
Times cited : (57)

References (96)
  • 1
    • 85031888624 scopus 로고    scopus 로고
    • Rare diseases: understanding this public health priority
    • EURORDIS. Rare diseases: understanding this public health priority. EURORDIS. 2005. Available at: http://www.eurordis.org/publication/rare-diseases-understanding-public-health-priority. Accessed 21 Oct 2013.
    • (2005) EURORDIS
  • 2
    • 79952044620 scopus 로고    scopus 로고
    • Ethical aspects on rare diseases
    • PID: 20824462
    • Barrera LA, Galindo GC. Ethical aspects on rare diseases. Adv Exp Med Biol. 2010;686:493–511.
    • (2010) Adv Exp Med Biol , vol.686 , pp. 493-511
    • Barrera, L.A.1    Galindo, G.C.2
  • 3
    • 79952050684 scopus 로고    scopus 로고
    • Rare diseases social epidemiology: analysis of inequalities
    • PID: 20824449
    • Kole A, Faurisson F. Rare diseases social epidemiology: analysis of inequalities. Adv Exp Med Biol. 2010;686:223–50.
    • (2010) Adv Exp Med Biol , vol.686 , pp. 223-250
    • Kole, A.1    Faurisson, F.2
  • 4
    • 33745506072 scopus 로고    scopus 로고
    • Lysosomal storage diseases: natural history and ethical and economic aspects
    • COI: 1:CAS:528:DC%2BD28Xms1agu7g%3D, PID: 16515872
    • Beutler E. Lysosomal storage diseases: natural history and ethical and economic aspects. Mol Genet Metab. 2006;88(3):208–15.
    • (2006) Mol Genet Metab , vol.88 , Issue.3 , pp. 208-215
    • Beutler, E.1
  • 5
    • 79952051195 scopus 로고    scopus 로고
    • Statistical methods for the geographical analysis of rare diseases
    • PID: 20824445
    • Gomez-Rubio V, Lopez-Quilez A. Statistical methods for the geographical analysis of rare diseases. Adv Exp Med Biol. 2010;686:151–71.
    • (2010) Adv Exp Med Biol , vol.686 , pp. 151-171
    • Gomez-Rubio, V.1    Lopez-Quilez, A.2
  • 7
    • 79952060054 scopus 로고    scopus 로고
    • Rare diseases—avoiding misperceptions and establishing realities: the need for reliable epidemiological data
    • PID: 20824436
    • Groft SC, de la Paz MP. Rare diseases—avoiding misperceptions and establishing realities: the need for reliable epidemiological data. Adv Exp Med Biol. 2010;686:3–14.
    • (2010) Adv Exp Med Biol , vol.686 , pp. 3-14
    • Groft, S.C.1    de la Paz, M.P.2
  • 8
    • 79961046765 scopus 로고    scopus 로고
    • Epidemiology of lysosomal storage diseases: an overview
    • Mehta A, Beck M, Sunder-Plassmann G, (eds), Oxford PharmaGenesis, Oxford
    • Fuller M, Meikle PJ, Hopwood JJ. Epidemiology of lysosomal storage diseases: an overview. In: Mehta A, Beck M, Sunder-Plassmann G, editors. Fabry disease: perspectives from 5 years of FOS. Oxford: Oxford PharmaGenesis; 2006.
    • (2006) Fabry disease: perspectives from 5 years of FOS
    • Fuller, M.1    Meikle, P.J.2    Hopwood, J.J.3
  • 9
    • 27744461277 scopus 로고    scopus 로고
    • Drugs for exceptionally rare diseases: do they deserve special status for funding?
    • COI: 1:STN:280:DC%2BD2MrlvVKrsg%3D%3D, PID: 16203824
    • Hughes DA, Tunnage B, Yeo ST. Drugs for exceptionally rare diseases: do they deserve special status for funding? QJM. 2005;98(11):829–36.
    • (2005) QJM , vol.98 , Issue.11 , pp. 829-836
    • Hughes, D.A.1    Tunnage, B.2    Yeo, S.T.3
  • 11
    • 78649889087 scopus 로고    scopus 로고
    • A cross-national comparative study of orphan drug policies in the United States, the European Union, and Japan: towards a made-in-China orphan drug policy
    • PID: 21119648
    • Liu BC, He L, He G, He Y. A cross-national comparative study of orphan drug policies in the United States, the European Union, and Japan: towards a made-in-China orphan drug policy. J Public Health Policy. 2010;31(4):407–20.
    • (2010) J Public Health Policy , vol.31 , Issue.4 , pp. 407-420
    • Liu, B.C.1    He, L.2    He, G.3    He, Y.4
  • 12
    • 85031880280 scopus 로고    scopus 로고
    • Drugs for rare diseases: evolving trends in regulatory and health technology assessment perspectives
    • Loorand-Stiver L. Drugs for rare diseases: evolving trends in regulatory and health technology assessment perspectives. Environ Scan. 2013;2013(42):1–40.
    • (2013) Environ Scan. , vol.2013 , Issue.42 , pp. 1-40
    • Loorand-Stiver, L.1
  • 13
    • 80052509315 scopus 로고    scopus 로고
    • Authorization and reimbursement of orphan drugs in an international comparison [in German]
    • COI: 1:STN:280:DC%2BC3MfgtlSrsA%3D%3D, PID: 20848380
    • Roll K, Stargardt T, Schreyogg J. Authorization and reimbursement of orphan drugs in an international comparison [in German]. Gesundheitswesen. 2011;73(8–9):504–14.
    • (2011) Gesundheitswesen. , vol.73 , Issue.8-9 , pp. 504-514
    • Roll, K.1    Stargardt, T.2    Schreyogg, J.3
  • 14
    • 78649700786 scopus 로고    scopus 로고
    • Rare diseases, orphan drugs and their regulation: questions and misconceptions
    • COI: 1:CAS:528:DC%2BC3cXhtl2itbvO, PID: 21060315
    • Tambuyzer E. Rare diseases, orphan drugs and their regulation: questions and misconceptions. Nat Rev Drug Discov. 2010;9(12):921–9.
    • (2010) Nat Rev Drug Discov. , vol.9 , Issue.12 , pp. 921-929
    • Tambuyzer, E.1
  • 15
    • 84880034374 scopus 로고    scopus 로고
    • When everyone is an orphan: against adopting a U.S.-styled orphan drug policy in Canada
    • Herder M. When everyone is an orphan: against adopting a U.S.-styled orphan drug policy in Canada. Acc Res. 2013;20(4):227–69.
    • (2013) Acc Res. , vol.20 , Issue.4 , pp. 227-269
    • Herder, M.1
  • 16
    • 84959161471 scopus 로고    scopus 로고
    • National Institute for Health and Care Excellence. Interim process and methods of the Highly Specialised Technologies programme. 2013. Accessed 30 Oct 2013
    • National Institute for Health and Care Excellence. Interim process and methods of the Highly Specialised Technologies programme. 2013. Available at: http://www.nice.org.uk/aboutnice/howwework/devhst/DevelopingNICEHighlySpecialisedTechnologies.jsp. Accessed 30 Oct 2013.
  • 18
    • 84866564048 scopus 로고    scopus 로고
    • Paying for the Orphan Drug System: break or bend? Is it time for a new evaluation system for payers in Europe to take account of new rare disease treatments?
    • PID: 23013790
    • Hughes-Wilson W, Palma A, Schuurman A, Simoens S. Paying for the Orphan Drug System: break or bend? Is it time for a new evaluation system for payers in Europe to take account of new rare disease treatments? Orphanet J Rare Dis. 2012;7:74.
    • (2012) Orphanet J Rare Dis. , vol.7 , pp. 74
    • Hughes-Wilson, W.1    Palma, A.2    Schuurman, A.3    Simoens, S.4
  • 19
    • 84925513451 scopus 로고    scopus 로고
    • Value-based reimbursement decisions for orphan drugs: a scoping review and decision framework
    • Paulden M, Stafinski T, Menon D, McCabe C. Value-based reimbursement decisions for orphan drugs: a scoping review and decision framework. Pharmacoeconomics. 2014;33(3):255–69.
    • (2014) Pharmacoeconomics. , vol.33 , Issue.3 , pp. 255-269
    • Paulden, M.1    Stafinski, T.2    Menon, D.3    McCabe, C.4
  • 20
    • 84890312186 scopus 로고    scopus 로고
    • A pilot study of multicriteria decision analysis for valuing orphan medicines
    • PID: 24326170
    • Sussex J, Rollet P, Garau M, Schmitt C, Kent A, Hutchings A. A pilot study of multicriteria decision analysis for valuing orphan medicines. Value Health. 2013;16(8):1163–9.
    • (2013) Value Health. , vol.16 , Issue.8 , pp. 1163-1169
    • Sussex, J.1    Rollet, P.2    Garau, M.3    Schmitt, C.4    Kent, A.5    Hutchings, A.6
  • 21
    • 84861325032 scopus 로고    scopus 로고
    • Social values in health priority setting: a conceptual framework
    • PID: 22852453
    • Clark S, Weale A. Social values in health priority setting: a conceptual framework. J Health Organ Manag. 2012;26(3):293–316.
    • (2012) J Health Organ Manag. , vol.26 , Issue.3 , pp. 293-316
    • Clark, S.1    Weale, A.2
  • 22
    • 84861308184 scopus 로고    scopus 로고
    • Social value judgments in healthcare: a philosophical critique
    • PID: 22852454
    • Biron L, Rumbold B, Faden R. Social value judgments in healthcare: a philosophical critique. J Health Organ Manag. 2012;26(3):317–30.
    • (2012) J Health Organ Manag. , vol.26 , Issue.3 , pp. 317-330
    • Biron, L.1    Rumbold, B.2    Faden, R.3
  • 23
    • 0031217084 scopus 로고    scopus 로고
    • Limits to health care: fair procedures, democratic deliberation, and the legitimacy problem for insurers
    • PID: 11660435
    • Daniels N, Sabin J. Limits to health care: fair procedures, democratic deliberation, and the legitimacy problem for insurers. Philos Public Aff. 1997;26(4):303–50.
    • (1997) Philos Public Aff , vol.26 , Issue.4 , pp. 303-350
    • Daniels, N.1    Sabin, J.2
  • 24
    • 0032782604 scopus 로고    scopus 로고
    • Decisions about access to health care and accountability for reasonableness
    • COI: 1:STN:280:DC%2BD3cvgt1yrtw%3D%3D, PID: 10924028
    • Daniels N. Decisions about access to health care and accountability for reasonableness. J Urban Health. 1999;76(2):176–91.
    • (1999) J Urban Health. , vol.76 , Issue.2 , pp. 176-191
    • Daniels, N.1
  • 26
    • 77950631288 scopus 로고    scopus 로고
    • Combining multicriteria decision analysis, ethics and health technology assessment: applying the EVIDEM decision making framework to growth hormone for Turner syndrome patients
    • PID: 20377888
    • Goetghebeur M, Wagner M, Khoury H, Rindress D, Gregoire JP, Deal C. Combining multicriteria decision analysis, ethics and health technology assessment: applying the EVIDEM decision making framework to growth hormone for Turner syndrome patients. Cost Eff Resour Alloc. 2010;8(1):4.
    • (2010) Cost Eff Resour Alloc. , vol.8 , Issue.1 , pp. 4
    • Goetghebeur, M.1    Wagner, M.2    Khoury, H.3    Rindress, D.4    Gregoire, J.P.5    Deal, C.6
  • 27
    • 84859342157 scopus 로고    scopus 로고
    • Bridging health technology assessment (HTA) and efficient health care decision making with multicriteria decision analysis (MCDA): applying the EVIDEM framework to medicines appraisal
    • Goetghebeur MM, Wagner M, Khoury H, Levitt RJ, Erickson LJ, Rindress D. Bridging health technology assessment (HTA) and efficient health care decision making with multicriteria decision analysis (MCDA): applying the EVIDEM framework to medicines appraisal. Med Decis Mak. 2012;32(2):376–88.
    • (2012) Med Decis Mak , vol.32 , Issue.2 , pp. 376-388
    • Goetghebeur, M.M.1    Wagner, M.2    Khoury, H.3    Levitt, R.J.4    Erickson, L.J.5    Rindress, D.6
  • 28
    • 84889562526 scopus 로고    scopus 로고
    • Which criteria are considered in healthcare decisions? Insights from an international survey of policy and clinical decision makers
    • PID: 24290340
    • Tanios N, Wagner M, Tony M, Baltussen R, van Til J, Rindress D, et al. Which criteria are considered in healthcare decisions? Insights from an international survey of policy and clinical decision makers. Int J Technol Assess Health Care. 2013;29(4):456–65.
    • (2013) Int J Technol Assess Health Care , vol.29 , Issue.4 , pp. 456-465
    • Tanios, N.1    Wagner, M.2    Tony, M.3    Baltussen, R.4    van Til, J.5    Rindress, D.6
  • 29
    • 84868109036 scopus 로고    scopus 로고
    • From efficacy to equity: review of decision criteria used in resource allocation and healthcare decisionmaking
    • PID: 22808944
    • Guindo LA, Wagner M, Baltussen R, Rindress D, van Til J, Kind P, et al. From efficacy to equity: review of decision criteria used in resource allocation and healthcare decisionmaking. Cost Eff Resour Alloc. 2012;10(1):9.
    • (2012) Cost Eff Resour Alloc. , vol.10 , Issue.1 , pp. 9
    • Guindo, L.A.1    Wagner, M.2    Baltussen, R.3    Rindress, D.4    van Til, J.5    Kind, P.6
  • 30
    • 84928107668 scopus 로고    scopus 로고
    • Does technique matter: a pilot study exploring weighting techniques for a multi-criteria decision support framework
    • PID: 25904823
    • van Til J, Groothuis-Oudshoorn C, Lieferink M, Dolan J, Goetghebeur M. Does technique matter: a pilot study exploring weighting techniques for a multi-criteria decision support framework. Cost Eff Resour Alloc. 2014;12:22.
    • (2014) Cost Eff Resour Alloc. , vol.12 , pp. 22
    • van Til, J.1    Groothuis-Oudshoorn, C.2    Lieferink, M.3    Dolan, J.4    Goetghebeur, M.5
  • 31
    • 84959091051 scopus 로고    scopus 로고
    • EVIDEM Collaboration website. EVIDEM Collaboration. 2015. Accessed 12 June 2015
    • EVIDEM Collaboration website. EVIDEM Collaboration. 2015. Available at: http://www.evidem.org. Accessed 12 June 2015.
  • 33
    • 82255167655 scopus 로고    scopus 로고
    • Bridging health technology assessment (HTA) with multicriteria decision analyses (MCDA): field testing of the EVIDEM framework for coverage decisions by a public payer in Canada
    • PID: 22129247
    • Tony M, Wagner M, Khoury H, Rindress D, Papastavros T, Oh P, et al. Bridging health technology assessment (HTA) with multicriteria decision analyses (MCDA): field testing of the EVIDEM framework for coverage decisions by a public payer in Canada. BMC Health Serv Res. 2011;11:329.
    • (2011) BMC Health Serv Res. , vol.11 , pp. 329
    • Tony, M.1    Wagner, M.2    Khoury, H.3    Rindress, D.4    Papastavros, T.5    Oh, P.6
  • 35
    • 84959155225 scopus 로고    scopus 로고
    • MCDA for HTA in action: leveraging experience from Europe and Latin America. Panel presented at the Health Technology Assessment International (HTAi) 12th Annual Conference, 15–17 June 2015: Oslo; 2015
    • MCDA for HTA in action: leveraging experience from Europe and Latin America. Panel presented at the Health Technology Assessment International (HTAi) 12th Annual Conference, 15–17 June 2015: Oslo; 2015.
  • 36
    • 84959174271 scopus 로고    scopus 로고
    • Defining value: the ethical foundations of MCDA in healthcare decision making. Manuscript in preparation
    • Healthcare decisions supported by multi-criteria decision analysis. Cham: Springer International Publishing AG
    • Goetghebeur M, Wagner M. Defining value: the ethical foundations of MCDA in healthcare decision making. Manuscript in preparation. In: Marsh K, Thokala P, Goetghebeur M, Baltussen R, editors. Healthcare decisions supported by multi-criteria decision analysis. Cham: Springer International Publishing AG; 2015.
    • (2015) Marsh K
    • Goetghebeur, M.1    Wagner, M.2    Thokala, P.3    Goetghebeur, M.4    Baltussen, R.5
  • 37
    • 85031892273 scopus 로고    scopus 로고
    • Decision criteria of the framework: conceptual background, definitions, design & instructions
    • EVIDEM Collaboration. Decision criteria of the framework: conceptual background, definitions, design & instructions. EVIDEM Collaboration. 2015. Available at: https://www.evidem.org/components-decision.php. Accessed 29 July 2015.
    • (2015) EVIDEM Collaboration
  • 38
    • 84959175523 scopus 로고    scopus 로고
    • Alexander L, Moore M. Deontological ethics. In: Zalta EN, editor. The Stanford Encyclopedia of Philosophy (Spring 2015 edition)
    • Alexander L, Moore M. Deontological ethics. In: Zalta EN, editor. The Stanford Encyclopedia of Philosophy (Spring 2015 edition). Available at: http://plato.stanford.edu/archives/spr2015/entries/ethics-deontological/.
  • 40
    • 0002336739 scopus 로고
    • The ‘four principles’ approach. Principles of health care ethics
    • Beauchamp TL. The ‘four principles’ approach. Principles of health care ethics. Chichester: Wiley; 1994. p. 3–12.
    • (1994) Chichester: Wiley , pp. 3-12
    • Beauchamp, T.L.1
  • 41
    • 84923227647 scopus 로고    scopus 로고
    • The four principles of biomedical ethics: a foundation for current bioethical debate
    • Lawrence DJ. The four principles of biomedical ethics: a foundation for current bioethical debate. J Chiropr Humanit. 2007;14:34–40.
    • (2007) J Chiropr Humanit. , vol.14 , pp. 34-40
    • Lawrence, D.J.1
  • 42
    • 85031890335 scopus 로고    scopus 로고
    • Driver J. The history of utilitarianism. In: Zalta EN, editor. The Stanford Encyclopedia of Philosophy (Winter 2014 edition)
    • Driver J. The history of utilitarianism. In: Zalta EN, editor. The Stanford Encyclopedia of Philosophy (Winter 2014 edition). Available at: http://plato.stanford.edu/archives/win2014/entries/utilitarianism-history/.
  • 43
    • 85031887872 scopus 로고    scopus 로고
    • Guidance on ethics and equitable access to HIV treatment and care
    • World Health Organization. Guidance on ethics and equitable access to HIV treatment and care. World Health Organization. 2004. Available at: http://www.who.int/ethics/Guidance%20on%20Ethics%20and%20HIV.pdf. Accessed 6 Feb 2009.
    • (2004) World Health Organization
  • 44
    • 1642579637 scopus 로고    scopus 로고
    • Justice and solidarity in priority setting in health care
    • PID: 14769014
    • Hoedemaekers R, Dekkers W. Justice and solidarity in priority setting in health care. Health Care Anal. 2003;11(4):325–43.
    • (2003) Health Care Anal , vol.11 , Issue.4 , pp. 325-343
    • Hoedemaekers, R.1    Dekkers, W.2
  • 45
    • 84959153752 scopus 로고    scopus 로고
    • Hursthouse R. Virtue ethics. In: Zalta EN, editor. The Stanford Encyclopedia of Philosophy (Fall 2013 edition)
    • Hursthouse R. Virtue ethics. In: Zalta EN, editor. The Stanford Encyclopedia of Philosophy (Fall 2013 edition). Available at: http://plato.stanford.edu/archives/fall2013/entries/ethics-virtue.
  • 47
    • 79953681954 scopus 로고    scopus 로고
    • The impact of parent advocacy groups, the Internet, and social networking on rare diseases: the IDEA League and IDEA League United Kingdom example
    • PID: 21463291
    • Black AP, Baker M. The impact of parent advocacy groups, the Internet, and social networking on rare diseases: the IDEA League and IDEA League United Kingdom example. Epilepsia. 2011;52(Suppl 2):102–4.
    • (2011) Epilepsia. , vol.52 , pp. 102-104
    • Black, A.P.1    Baker, M.2
  • 48
    • 75149164701 scopus 로고    scopus 로고
    • The role of patient advocacy organisations in neuromuscular disease R&D: the case of the Dutch neuromuscular disease association VSN
    • PID: 20106662
    • Boon W, Broekgaarden R. The role of patient advocacy organisations in neuromuscular disease R&D: the case of the Dutch neuromuscular disease association VSN. Neuromuscul Disord. 2010;20(2):148–51.
    • (2010) Neuromuscul Disord , vol.20 , Issue.2 , pp. 148-151
    • Boon, W.1    Broekgaarden, R.2
  • 49
  • 50
    • 77953354292 scopus 로고    scopus 로고
    • Budget impact analysis of orphan drugs in Belgium: estimates from 2008 to 2013
    • PID: 20482245
    • Denis A, Mergaert L, Fostier C, Cleemput I, Simoens S. Budget impact analysis of orphan drugs in Belgium: estimates from 2008 to 2013. J Med Econ. 2010;13(2):295–301.
    • (2010) J Med Econ. , vol.13 , Issue.2 , pp. 295-301
    • Denis, A.1    Mergaert, L.2    Fostier, C.3    Cleemput, I.4    Simoens, S.5
  • 51
    • 77957171034 scopus 로고    scopus 로고
    • Societal views on orphan drugs: cross sectional survey of Norwegians aged 40 to 67
    • PID: 20861122
    • Desser AS, Gyrd-Hansen D, Olsen JA, Grepperud S, Kristiansen IS. Societal views on orphan drugs: cross sectional survey of Norwegians aged 40 to 67. BMJ. 2010;341:c4715.
    • (2010) BMJ , vol.341
    • Desser, A.S.1    Gyrd-Hansen, D.2    Olsen, J.A.3    Grepperud, S.4    Kristiansen, I.S.5
  • 52
    • 84881344116 scopus 로고    scopus 로고
    • Prioritizing treatment of rare diseases: a survey of preferences of Norwegian doctors
    • PID: 23931945
    • Desser AS. Prioritizing treatment of rare diseases: a survey of preferences of Norwegian doctors. Soc Sci Med. 2013;94:56–62.
    • (2013) Soc Sci Med , vol.94 , pp. 56-62
    • Desser, A.S.1
  • 53
    • 84862889106 scopus 로고    scopus 로고
    • Clinical trial design in haemophilia
    • PID: 22726077
    • Dimichele DM, Blanchette V, Berntorp E. Clinical trial design in haemophilia. Haemophilia. 2012;18(Suppl. 4):18–23.
    • (2012) Haemophilia , vol.18 , pp. 18-23
    • Dimichele, D.M.1    Blanchette, V.2    Berntorp, E.3
  • 54
    • 79952060881 scopus 로고    scopus 로고
    • Advocacy groups and their role in rare diseases research
    • PID: 20824463
    • Dunkle M, Pines W, Saltonstall PL. Advocacy groups and their role in rare diseases research. Adv Exp Med Biol. 2010;686:515–25.
    • (2010) Adv Exp Med Biol , vol.686 , pp. 515-525
    • Dunkle, M.1    Pines, W.2    Saltonstall, P.L.3
  • 55
    • 85031884815 scopus 로고    scopus 로고
    • Maánahmen zur Verbesserung der gesundheitlichen Situation von Menschen mit Seltenen Erkrankungen in Deutschland
    • Eidt D, Frank M, Reimann A, Wagner TOF, Mittendorf T. Maánahmen zur Verbesserung der gesundheitlichen Situation von Menschen mit Seltenen Erkrankungen in Deutschland. Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz. 2009. Available at: http://www.bmg.bund.de/uploads/publications/BMG-G-09050-Bericht-Massnahmen-seltene-Krankheiten_200908.pdf. Accessed 28 Nov 2013.
    • (2009) Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz
    • Eidt, D.1    Frank, M.2    Reimann, A.3    Wagner, T.O.F.4    Mittendorf, T.5
  • 56
    • 15044363337 scopus 로고    scopus 로고
    • Ethical issues in funding orphan drug research and development
    • COI: 1:STN:280:DC%2BD2M7gsl2nug%3D%3D, PID: 15738438
    • Gericke CA, Riesberg A, Busse R. Ethical issues in funding orphan drug research and development. J Med Ethics. 2005;31(3):164–8.
    • (2005) J Med Ethics , vol.31 , Issue.3 , pp. 164-168
    • Gericke, C.A.1    Riesberg, A.2    Busse, R.3
  • 57
    • 70449372647 scopus 로고    scopus 로고
    • Psychosocial aspects of patients with Niemann-Pick disease, type B
    • PID: 19877061
    • Henderson SL, Packman W, Packman S. Psychosocial aspects of patients with Niemann-Pick disease, type B. Am J Med Genet A. 2009;149A(11):2430–6.
    • (2009) Am J Med Genet A. , vol.149A , Issue.11 , pp. 2430-2436
    • Henderson, S.L.1    Packman, W.2    Packman, S.3
  • 58
    • 79955119883 scopus 로고    scopus 로고
    • Limitations of drug registries to evaluate orphan medicinal products for the treatment of lysosomal storage disorders
    • PID: 21496291
    • Hollak CE, Aerts JM, Ayme S, Manuel J. Limitations of drug registries to evaluate orphan medicinal products for the treatment of lysosomal storage disorders. Orphanet J Rare Dis. 2011;6:16.
    • (2011) Orphanet J Rare Dis. , vol.6 , pp. 16
    • Hollak, C.E.1    Aerts, J.M.2    Ayme, S.3    Manuel, J.4
  • 59
    • 84876743099 scopus 로고    scopus 로고
    • Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU
    • PID: 23090701
    • Joppi R, Bertele’ V, Garattini S. Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU. Eur J Clin Pharmacol. 2013;69(4):1009–24.
    • (2013) Eur J Clin Pharmacol , vol.69 , Issue.4 , pp. 1009-1024
    • Joppi, R.1    Bertele’, V.2    Garattini, S.3
  • 60
    • 52349112701 scopus 로고    scopus 로고
    • Monitoring clinical quality in rare disease services: experience in England
    • PID: 18793435
    • Kenny TD, Jessop EG, Gutteridge WH. Monitoring clinical quality in rare disease services: experience in England. Orphanet J Rare Dis. 2008;3:23.
    • (2008) Orphanet J Rare Dis. , vol.3 , pp. 23
    • Kenny, T.D.1    Jessop, E.G.2    Gutteridge, W.H.3
  • 61
    • 79958036418 scopus 로고    scopus 로고
    • Characteristics of clinical trials to support approval of orphan vs nonorphan drugs for cancer
    • COI: 1:CAS:528:DC%2BC3MXntlOht70%3D, PID: 21642684
    • Kesselheim AS, Myers JA, Avorn J. Characteristics of clinical trials to support approval of orphan vs nonorphan drugs for cancer. JAMA. 2011;305(22):2320–6.
    • (2011) JAMA , vol.305 , Issue.22 , pp. 2320-2326
    • Kesselheim, A.S.1    Myers, J.A.2    Avorn, J.3
  • 62
    • 79960934199 scopus 로고    scopus 로고
    • Role of patient support groups in lysosomal storage diseases
    • Mehta A, Beck M, Sunder-Plassmann G, (eds), Oxford PharmaGenesis, Oxford
    • Lavery C. Role of patient support groups in lysosomal storage diseases. In: Mehta A, Beck M, Sunder-Plassmann G, editors. Fabry disease: perspectives from 5 years of FOS. Oxford: Oxford PharmaGenesis; 2006.
    • (2006) Fabry disease: perspectives from 5 years of FOS
    • Lavery, C.1
  • 63
    • 84880132926 scopus 로고    scopus 로고
    • Societal views on NICE, cancer drugs fund and value-based pricing criteria for prioritising medicines: a cross-sectional survey of 4118 adults in Great Britain
    • PID: 22961976
    • Linley WG, Hughes DA. Societal views on NICE, cancer drugs fund and value-based pricing criteria for prioritising medicines: a cross-sectional survey of 4118 adults in Great Britain. Health Econ. 2013;22(8):948–64.
    • (2013) Health Econ , vol.22 , Issue.8 , pp. 948-964
    • Linley, W.G.1    Hughes, D.A.2
  • 64
    • 48249130052 scopus 로고    scopus 로고
    • Optimal therapy for rare disorders and genetic diseases: ethical and political challenges
    • PID: 18605224
    • MacLeod S. Optimal therapy for rare disorders and genetic diseases: ethical and political challenges. Proc West Pharmacol Soc. 2007;50:21–3.
    • (2007) Proc West Pharmacol Soc , vol.50 , pp. 21-23
    • MacLeod, S.1
  • 65
    • 84870778464 scopus 로고    scopus 로고
    • Involvement of patient organisations in research and development of orphan drugs for rare diseases in europe
    • COI: 1:STN:280:DC%2BC3s3ntFyntA%3D%3D, PID: 23293582
    • Mavris M, Le CY. Involvement of patient organisations in research and development of orphan drugs for rare diseases in europe. Mol Syndromol. 2012;3(5):237–43.
    • (2012) Mol Syndromol , vol.3 , Issue.5 , pp. 237-243
    • Mavris, M.1    Le, C.Y.2
  • 66
    • 79960136544 scopus 로고    scopus 로고
    • A discrete choice experiment investigating preferences for funding drugs used to treat orphan diseases: an exploratory study
    • PID: 21205401
    • Mentzakis E, Stefanowska P, Hurley J. A discrete choice experiment investigating preferences for funding drugs used to treat orphan diseases: an exploratory study. Health Econ Policy Law. 2011;6(3):405–33.
    • (2011) Health Econ Policy Law. , vol.6 , Issue.3 , pp. 405-433
    • Mentzakis, E.1    Stefanowska, P.2    Hurley, J.3
  • 67
    • 77956289514 scopus 로고    scopus 로고
    • Global spending on orphan drugs in France, Germany, the UK, Italy and Spain during 2007
    • PID: 20804223
    • Orofino J, Soto J, Casado MA, Oyaguez I. Global spending on orphan drugs in France, Germany, the UK, Italy and Spain during 2007. Appl Health Econ Health Policy. 2010;8(5):301–15.
    • (2010) Appl Health Econ Health Policy. , vol.8 , Issue.5 , pp. 301-315
    • Orofino, J.1    Soto, J.2    Casado, M.A.3    Oyaguez, I.4
  • 68
    • 84860431228 scopus 로고    scopus 로고
    • A fair share for the orphans: ethical guidelines for a fair distribution of resources within the bounds of the 10-year-old European Orphan Drug Regulation
    • PID: 21947805
    • Pinxten W, Denier Y, Dooms M, Cassiman JJ, Dierickx K. A fair share for the orphans: ethical guidelines for a fair distribution of resources within the bounds of the 10-year-old European Orphan Drug Regulation. J Med Ethics. 2012;38(3):148–53.
    • (2012) J Med Ethics , vol.38 , Issue.3 , pp. 148-153
    • Pinxten, W.1    Denier, Y.2    Dooms, M.3    Cassiman, J.J.4    Dierickx, K.5
  • 70
    • 84870350793 scopus 로고    scopus 로고
    • Ethical and economic considerations of rare diseases in ethnic minorities: the case of mucopolysaccharidosis VI in Colombia
    • PID: 22550061
    • Rosselli D, Rueda JD, Solano M. Ethical and economic considerations of rare diseases in ethnic minorities: the case of mucopolysaccharidosis VI in Colombia. J Med Ethics. 2012;38(11):699–700.
    • (2012) J Med Ethics , vol.38 , Issue.11 , pp. 699-700
    • Rosselli, D.1    Rueda, J.D.2    Solano, M.3
  • 71
    • 80053379101 scopus 로고    scopus 로고
    • Estimating the budget impact of orphan medicines in Europe: 2
    • PID: 21951518
    • Schey C, Milanova T, Hutchings A. Estimating the budget impact of orphan medicines in Europe: 2. Orphanet J Rare Dis. 2011;6:62.
    • (2011) Orphanet J Rare Dis. , vol.6 , pp. 62
    • Schey, C.1    Milanova, T.2    Hutchings, A.3
  • 72
    • 77956478781 scopus 로고    scopus 로고
    • The psychological burden of an initially unexplained illness: patients with sternocostoclavicular hyperostosis before and after delayed diagnosis
    • PID: 20828391
    • van der Kloot WA, Hamdy NA, Hafkemeijer LC, den Dulk FM, Chotkan SA, van Emmerik AA, et al. The psychological burden of an initially unexplained illness: patients with sternocostoclavicular hyperostosis before and after delayed diagnosis. Health Qual Life Outcomes. 2010;8:97.
    • (2010) Health Qual Life Outcomes. , vol.8 , pp. 97
    • van der Kloot, W.A.1    Hamdy, N.A.2    Hafkemeijer, L.C.3    den Dulk, F.M.4    Chotkan, S.A.5    van Emmerik, A.A.6
  • 73
    • 84868272640 scopus 로고    scopus 로고
    • The effectiveness and cost-effectiveness of enzyme and substrate replacement therapies: a longitudinal cohort study of people with lysosomal storage disorders
    • COI: 1:STN:280:DC%2BC3s%2Fnt1Cltg%3D%3D, PID: 23089251
    • Wyatt K, Henley W, Anderson L, Anderson R, Nikolaou V, Stein K, et al. The effectiveness and cost-effectiveness of enzyme and substrate replacement therapies: a longitudinal cohort study of people with lysosomal storage disorders. Health Technol Assess. 2012;16(39):1–543.
    • (2012) Health Technol Assess , vol.16 , Issue.39 , pp. 1-543
    • Wyatt, K.1    Henley, W.2    Anderson, L.3    Anderson, R.4    Nikolaou, V.5    Stein, K.6
  • 74
    • 84902168946 scopus 로고    scopus 로고
    • Orphan drugs policies: a suitable case for treatment
    • PID: 24435513
    • Drummond M, Towse A. Orphan drugs policies: a suitable case for treatment. Eur J Health Econ. 2014;15(4):335–40.
    • (2014) Eur J Health Econ. , vol.15 , Issue.4 , pp. 335-340
    • Drummond, M.1    Towse, A.2
  • 75
    • 77956512548 scopus 로고    scopus 로고
    • Multi-criteria clinical decision support. A primer on the use of multiple-criteria decision-making methods to promote evidence-based, patient-centered healthcare
    • Dolan JG. Multi-criteria clinical decision support. A primer on the use of multiple-criteria decision-making methods to promote evidence-based, patient-centered healthcare. Patient. 2010;3(4):229–248.
    • (2010) Patient , vol.3 , Issue.4 , pp. 229-248
    • Dolan, J.G.1
  • 76
    • 85031885767 scopus 로고    scopus 로고
    • Orphanet Report Series. Prevalence of rare diseases: bibliographic data. 2014
    • Orphanet Report Series. Prevalence of rare diseases: bibliographic data. 2014. Available at: http://www.orpha.net/consor/cgi-bin/Education_Home.php?lng=EN#REPORT_RARE_DISEASES.
  • 77
    • 84879883725 scopus 로고    scopus 로고
    • Which health technologies should be funded? A prioritization framework based explicitly on value for money
    • PID: 23181391
    • Golan OG, Hansen P. Which health technologies should be funded? A prioritization framework based explicitly on value for money. Isr J Health Policy Res. 2012;1(1):44.
    • (2012) Isr J Health Policy Res. , vol.1 , Issue.1 , pp. 44
    • Golan, O.G.1    Hansen, P.2
  • 78
    • 77954608997 scopus 로고    scopus 로고
    • AGREE II: advancing guideline development, reporting and evaluation in health care
    • PID: 20603348
    • Brouwers MC, Kho ME, Browman GP, Burgers JS, Cluzeau F, Feder G, et al. AGREE II: advancing guideline development, reporting and evaluation in health care. CMAJ. 2010;182(18):E839–42.
    • (2010) CMAJ , vol.182 , Issue.18 , pp. E839-E842
    • Brouwers, M.C.1    Kho, M.E.2    Browman, G.P.3    Burgers, J.S.4    Cluzeau, F.5    Feder, G.6
  • 79
    • 37349079944 scopus 로고    scopus 로고
    • Teaching medical students about fair distribution of healthcare resources
    • COI: 1:STN:280:DC%2BD2sjisFGhsQ%3D%3D, PID: 18055907
    • Leget C, Hoedemaekers R. Teaching medical students about fair distribution of healthcare resources. J Med Ethics. 2007;33(12):737–41.
    • (2007) J Med Ethics , vol.33 , Issue.12 , pp. 737-741
    • Leget, C.1    Hoedemaekers, R.2
  • 80
    • 85031887516 scopus 로고    scopus 로고
    • EUROPLAN 2012-2015
    • EUROPLAN Project. EUROPLAN 2012-2015. EUROPLAN Project. 2015. http://www.europlanproject.eu/Content?folder=1&content=1. Accessed 23 Oct 2015.
    • (2015) EUROPLAN Project
    • Project, E.U.R.O.P.L.A.N.1
  • 81
    • 79952050558 scopus 로고    scopus 로고
    • National plans and strategies on rare diseases in Europe
    • PID: 20824461
    • Taruscio D, Vittozzi L, Stefanov R. National plans and strategies on rare diseases in Europe. Adv Exp Med Biol. 2010;686:475–91.
    • (2010) Adv Exp Med Biol , vol.686 , pp. 475-491
    • Taruscio, D.1    Vittozzi, L.2    Stefanov, R.3
  • 82
    • 85031889141 scopus 로고    scopus 로고
    • EUCERD Joint Action: working for rare diseases
    • EUCERD. EUCERD Joint Action: working for rare diseases. EUCERD. 2014. http://www.eucerd.eu/?page_id=54. Accessed 23 Oct 2015.
    • (2014) EUCERD
  • 83
    • 84864819821 scopus 로고    scopus 로고
    • Key outcomes from stakeholder workshops at a symposium to inform the development of an Australian national plan for rare diseases
    • PID: 22883422
    • Molster C, Youngs L, Hammond E, Dawkins H. Key outcomes from stakeholder workshops at a symposium to inform the development of an Australian national plan for rare diseases. Orphanet J Rare Dis. 2012;7:50.
    • (2012) Orphanet J Rare Dis. , vol.7 , pp. 50
    • Molster, C.1    Youngs, L.2    Hammond, E.3    Dawkins, H.4
  • 84
    • 84959097304 scopus 로고    scopus 로고
    • NICE’s equality objectives and equality programme
    • National Institute for Health and Care Excellence. NICE’s equality objectives and equality programme 2013–2016. 2013. Available at: http://www.nice.org.uk/about/nice-communities/public-involvement/equality-scheme#. Accessed 28 Jan 2015.
    • (2013) National Institute for Health and Care Excellence , vol.2013-2016
  • 85
    • 85031881687 scopus 로고    scopus 로고
    • ® (efalizumab). European Medicines Agency. 2015. v.2.6:1–99. Accessed 23 Oct 2015
    • ® (efalizumab). European Medicines Agency. 2015. v.2.6:1–99.http://www.imi-protect.eu/. Accessed 23 Oct 2015.
  • 86
    • 84881356074 scopus 로고    scopus 로고
    • Multi-criteria decision analysis tools for prioritising emerging or re-emerging infectious diseases associated with climate change in Canada
    • COI: 1:CAS:528:DC%2BC3sXhtlWgt7bM, PID: 23950868
    • Cox R, Sanchez J, Revie CW. Multi-criteria decision analysis tools for prioritising emerging or re-emerging infectious diseases associated with climate change in Canada. PLoS One. 2013;8(8):e68338.
    • (2013) PLoS One , vol.8 , Issue.8
    • Cox, R.1    Sanchez, J.2    Revie, C.W.3
  • 87
    • 48249153186 scopus 로고
    • Intraclass correlations: uses in assessing rater reliability
    • COI: 1:STN:280:DC%2BD1cnkslSnsg%3D%3D, PID: 18839484
    • Shrout PE, Fleiss JL. Intraclass correlations: uses in assessing rater reliability. Psychol Bull. 1979;86(2):420–8.
    • (1979) Psychol Bull , vol.86 , Issue.2 , pp. 420-428
    • Shrout, P.E.1    Fleiss, J.L.2
  • 89
    • 66349107417 scopus 로고    scopus 로고
    • Evidence and values: requirements for public reimbursement of drugs for rare diseases: a case study in oncology
    • PID: 19439771
    • Drummond M, Evans B, LeLorier J, Karakiewicz P, Martin D, Tugwell P, et al. Evidence and values: requirements for public reimbursement of drugs for rare diseases: a case study in oncology. Can J Clin Pharmacol. 2009;16(2):e273–81.
    • (2009) Can J Clin Pharmacol. , vol.16 , Issue.2 , pp. e273-e281
    • Drummond, M.1    Evans, B.2    LeLorier, J.3    Karakiewicz, P.4    Martin, D.5    Tugwell, P.6
  • 90
    • 33847416589 scopus 로고    scopus 로고
    • Schizophrenia: multi-attribute utility theory approach to selection of atypical antipsychotics
    • COI: 1:CAS:528:DC%2BD2sXjsF2ktLo%3D, PID: 17311836
    • Bettinger TL, Shuler G, Jones DR, Wilson JP. Schizophrenia: multi-attribute utility theory approach to selection of atypical antipsychotics. Ann Pharmacother. 2007;41(2):201–7.
    • (2007) Ann Pharmacother , vol.41 , Issue.2 , pp. 201-207
    • Bettinger, T.L.1    Shuler, G.2    Jones, D.R.3    Wilson, J.P.4
  • 91
    • 33645562677 scopus 로고    scopus 로고
    • Optimal management of adults with pharyngitis: a multi-criteria decision analysis
    • PID: 16533386
    • Singh S, Dolan JG, Centor RM. Optimal management of adults with pharyngitis: a multi-criteria decision analysis. BMC Med Inform Decis Mak. 2006;6:14.
    • (2006) BMC Med Inform Decis Mak , vol.6 , pp. 14
    • Singh, S.1    Dolan, J.G.2    Centor, R.M.3
  • 92
    • 84885368180 scopus 로고    scopus 로고
    • Multi-criteria decision analysis as an innovative approach to managing zoonoses: results from a study on Lyme disease in Canada
    • PID: 24079303
    • Aenishaenslin C, Hongoh V, Cisse HD, Hoen AG, Samoura K, Michel P, et al. Multi-criteria decision analysis as an innovative approach to managing zoonoses: results from a study on Lyme disease in Canada. BMC Public Health. 2013;13(1):897.
    • (2013) BMC Public Health. , vol.13 , Issue.1 , pp. 897
    • Aenishaenslin, C.1    Hongoh, V.2    Cisse, H.D.3    Hoen, A.G.4    Samoura, K.5    Michel, P.6
  • 93
    • 84870950627 scopus 로고    scopus 로고
    • Mapping of multiple criteria for priority setting of health interventions: an aid for decision makers
    • PID: 23234463
    • Tromp N, Baltussen R. Mapping of multiple criteria for priority setting of health interventions: an aid for decision makers. BMC Health Serv Res. 2012;12:454.
    • (2012) BMC Health Serv Res. , vol.12 , pp. 454
    • Tromp, N.1    Baltussen, R.2
  • 94
    • 85031887411 scopus 로고    scopus 로고
    • Principles for the development of NICE guidance
    • National Institute for Health and Care Excellence. Social value judgements. Principles for the development of NICE guidance. 2nd ed. 2013. Available at: http://www.nice.org.uk/proxy/?sourceurl=http://www.nice.org.uk/aboutnice/howwework/socialvaluejudgements/socialvaluejudgements.jsp. Accessed 28 Jan 2015.
    • (2013) 2nd ed
  • 95
    • 85031887521 scopus 로고    scopus 로고
    • Therapeutic goods regulations 1990: statutory rules No. 394, 1990 as amended
    • Australian Government. Therapeutic goods regulations 1990: statutory rules No. 394, 1990 as amended. Australian Government. 2013. https://www.comlaw.gov.au/Details/F2013C00670. Accessed 23 Oct 2015.
    • (2013) Australian Government
  • 96
    • 85031890022 scopus 로고    scopus 로고
    • Initial draft discussion document for a Canadian orphan drug regulatory framework
    • Office of Legislative and Regulatory Modernization. Initial draft discussion document for a Canadian orphan drug regulatory framework. Orphanet. 2012. 1-25. http://www.orpha.net/national/data/CA-EN/www/uploads/Initial-Draft-Discussion-Document-for-A-Canadian-Orphan-Drug--Regulatory-Framework.doc. Accessed 30 Oct 2013.
    • (2012) Orphanet , pp. 1-25


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