메뉴 건너뛰기




Volumn 100, Issue 1, 2011, Pages 25-34

Priority setting for orphan drugs: An international comparison

Author keywords

Decision making; Orphan drugs; Priority setting

Indexed keywords

AGALSIDASE BETA; IMIGLUCERASE;

EID: 79952606431     PISSN: 01688510     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.healthpol.2010.09.008     Document Type: Article
Times cited : (44)

References (46)
  • 1
    • 79952625319 scopus 로고    scopus 로고
    • The European Union and the FDA working together to create Common Application for Orphan Designation for Medicines [cited June 28]. Available from
    • The European Union and the FDA working together to create Common Application for Orphan Designation for Medicines [cited 2010 June 28]. Available from: http://www.ema.europa.eu/pdfs/general/direct/pr/55739107en.pdf.
    • (2010)
  • 2
    • 31544452135 scopus 로고    scopus 로고
    • Is the current approach to reviewing new drugs condemning the victims of rare diseases to death? A call for a national orphan drug review policy
    • Clarke J.T.R. Is the current approach to reviewing new drugs condemning the victims of rare diseases to death? A call for a national orphan drug review policy. CMAJ 2006, 174(2).
    • (2006) CMAJ , vol.174 , Issue.2
    • Clarke, J.T.R.1
  • 3
    • 41249083323 scopus 로고    scopus 로고
    • Drugs for rare diseases: paying for innovation
    • McGill-Queen's University Press, Montreal, C.M. Beach (Ed.)
    • Hollis A. Drugs for rare diseases: paying for innovation. Health services restructuring in Canada: new evidence and new directions 2006, 155-178. McGill-Queen's University Press, Montreal. C.M. Beach (Ed.).
    • (2006) Health services restructuring in Canada: new evidence and new directions , pp. 155-178
    • Hollis, A.1
  • 4
    • 79952626255 scopus 로고    scopus 로고
    • CORD. Towards a Canadian Orphan Drug Policy; [cited 2008 April 7]. Available from
    • CORD. Towards a Canadian Orphan Drug Policy; 2005 [cited 2008 April 7]. Available from: http://www.fragile-x.ca/advocacy%2520files/Canada's%2520Orphan%2520Drug%2520Polic%2520y%25200805.pdf.
    • (2005)
  • 5
    • 79952626893 scopus 로고    scopus 로고
    • Probability of Breast Cancer in American Women. National Cancer Institute's (NCI) Surveillance, Epidemiology, and End Results (SEER)
    • Program has published its SEER Cancer Statistics Review 1975-2003 2006 [cited December 30]. Available from
    • Ries, Lag, Harkins, D, Krapcho, M, Mariotto, A, Miller, BA, Feuer, EJ, et al. Probability of Breast Cancer in American Women. National Cancer Institute's (NCI) Surveillance, Epidemiology, and End Results (SEER) Program has published its SEER Cancer Statistics Review 1975-2003 2006 [cited 2009 December 30]. Available from: http://www.cancer.gov/cancertopics/factsheet/Detection/probability-breast-cancer.
    • (2009)
    • Ries Lag Harkins, D.1    Krapcho, M.2    Mariotto, A.3    Miller, B.A.4    Feuer, E.J.5
  • 6
    • 79952631645 scopus 로고    scopus 로고
    • Targeted Breast Cancer Drug Shrinks Tumors; [cited 2009 December 30]. Available from
    • Laino C. Targeted Breast Cancer Drug Shrinks Tumors; 2009 [cited 2009 December 30]. Available from: http://www.medicinenet.com/script/main/art.asp?articlekey=109470.
    • (2009)
    • Laino, C.1
  • 7
    • 34848851021 scopus 로고    scopus 로고
    • Priority setting for high cost medications (HCMs) in public hospitals in Australia: a case study
    • Gallego G., Taylor S.J., Brien J.A. Priority setting for high cost medications (HCMs) in public hospitals in Australia: a case study. Health Policy 2007, 84(1):58-66.
    • (2007) Health Policy , vol.84 , Issue.1 , pp. 58-66
    • Gallego, G.1    Taylor, S.J.2    Brien, J.A.3
  • 9
    • 79952629703 scopus 로고    scopus 로고
    • National Gaucher Foundation of Canada [cited July 19]. Available from
    • National Gaucher Foundation of Canada [cited 2010 July 19]. Available from: http://www.gauchercanada.ca/en/gaucher.html.
    • (2010)
  • 10
    • 0031841598 scopus 로고    scopus 로고
    • Low-dose low-frequency imiglucerase as a starting regimen of enzyme replacement therapy for patients with type I Gaucher disease
    • Elstein D., Abrahamov A., Hadas-Halpern I., Meyer A., Zimran A. Low-dose low-frequency imiglucerase as a starting regimen of enzyme replacement therapy for patients with type I Gaucher disease. Quarterly Journal of Medicine 1998, 91:483-488.
    • (1998) Quarterly Journal of Medicine , vol.91 , pp. 483-488
    • Elstein, D.1    Abrahamov, A.2    Hadas-Halpern, I.3    Meyer, A.4    Zimran, A.5
  • 11
    • 0025869216 scopus 로고
    • Replacement therapy for inherited enzyme deficiency-macrophage-targeted glucocerebrosidase for Gaucher's disease
    • Barton N.W., Brady R.O., Dambrosia J.M. Replacement therapy for inherited enzyme deficiency-macrophage-targeted glucocerebrosidase for Gaucher's disease. New England Journal of Medicine 1991, 324(21):1464-1470.
    • (1991) New England Journal of Medicine , vol.324 , Issue.21 , pp. 1464-1470
    • Barton, N.W.1    Brady, R.O.2    Dambrosia, J.M.3
  • 12
    • 0028883136 scopus 로고
    • Enzyme therapy in type 1 Gaucher disease: comparative efficacy of mannose-terminated glucocerebrosidase from natural and recombinant sources
    • Grabowski G.A., Barton N.W., Pastores G., Dambrosia J.M., Banerjee T.K., McKee M.A., et al. Enzyme therapy in type 1 Gaucher disease: comparative efficacy of mannose-terminated glucocerebrosidase from natural and recombinant sources. Annals of Internal Medicine 1995, 122(1):33-39.
    • (1995) Annals of Internal Medicine , vol.122 , Issue.1 , pp. 33-39
    • Grabowski, G.A.1    Barton, N.W.2    Pastores, G.3    Dambrosia, J.M.4    Banerjee, T.K.5    McKee, M.A.6
  • 13
  • 14
    • 79952630959 scopus 로고    scopus 로고
    • Lysosomal Disease Program Fabry Disease Description [cited 2010 July 19]. Available from
    • Lysosomal Disease Program Fabry Disease Description [cited 2010 July 19]. Available from: http://www.mghlysosomal.org/Fabry.html.
  • 17
    • 31544456336 scopus 로고    scopus 로고
    • Long-term therapy with agalsidase alfa for Fabry disease: safety and effects on renal function in a home infusion setting
    • Schiffmann R., Ries M., Timmons M., Flaherty J.T., Brady R.O. Long-term therapy with agalsidase alfa for Fabry disease: safety and effects on renal function in a home infusion setting. Nephrology Dialysis Transplantation 2006, 21:345-354.
    • (2006) Nephrology Dialysis Transplantation , vol.21 , pp. 345-354
    • Schiffmann, R.1    Ries, M.2    Timmons, M.3    Flaherty, J.T.4    Brady, R.O.5
  • 18
    • 3142554529 scopus 로고    scopus 로고
    • Long-term safety and efficacy of enzyme replacement therapy for Fabry disease
    • Wilcox W.R., Banikazemi M. Long-term safety and efficacy of enzyme replacement therapy for Fabry disease. American Journal of Human Genetics 2004, 75:65-74.
    • (2004) American Journal of Human Genetics , vol.75 , pp. 65-74
    • Wilcox, W.R.1    Banikazemi, M.2
  • 19
    • 0035816007 scopus 로고    scopus 로고
    • Enzyme replacement therapy in Fabry disease: a randomized controlled trial
    • Schiffmann R., Kopp J.B., Austin H.A., Sabnis S., Moore D.F., Weibel T., et al. Enzyme replacement therapy in Fabry disease: a randomized controlled trial. JAMA 2001, 285(21):2743-2749.
    • (2001) JAMA , vol.285 , Issue.21 , pp. 2743-2749
    • Schiffmann, R.1    Kopp, J.B.2    Austin, H.A.3    Sabnis, S.4    Moore, D.F.5    Weibel, T.6
  • 20
    • 79952630332 scopus 로고    scopus 로고
    • CEDAC [cited November 17]. Available from
    • CEDAC [cited 2007 November 17]. Available from: http://www.cadth.ca/index.php/en/cdr/committees/cedac.
    • (2007)
  • 21
    • 79952627499 scopus 로고    scopus 로고
    • PBAC [cited December 31]. Available from
    • PBAC [cited 2008 December 31]. Available from: http://www.health.gov.au/internet/main/publishing.nsf/Content/PBAC-Meeting-Agenda-and-Consumer-Comments-lp.
    • (2008)
  • 22
    • 0034542376 scopus 로고    scopus 로고
    • Setting priorities for the adoption of health technologies on a national level-the Israeli Experience
    • Shani S., Siebzhner M.I., Luxenburg O., Shemer J. Setting priorities for the adoption of health technologies on a national level-the Israeli Experience. Health Policy 2000, 54:169-185.
    • (2000) Health Policy , vol.54 , pp. 169-185
    • Shani, S.1    Siebzhner, M.I.2    Luxenburg, O.3    Shemer, J.4
  • 23
    • 79952621278 scopus 로고    scopus 로고
    • Ceredase and cerezyme [cited June 13]. Available from
    • Ceredase and cerezyme [cited 2003 June 13]. Available from: http://www.ghimedicare.com/drafts/ceredase.html.
    • (2003)
  • 24
    • 79952627828 scopus 로고    scopus 로고
    • Rx-List [cited February 18]. Available from
    • Rx-List [cited 2005 February 18]. Available from: http://www.rxlist.com/cgi/generic3/fabrazyme.htm.
    • (2005)
  • 25
    • 0346183687 scopus 로고    scopus 로고
    • Qualitative research design: an interactive approach
    • Sage Publications, London
    • Maxwell J.A. Qualitative research design: an interactive approach. Applied social research methods series 1996, vol. 41. Sage Publications, London.
    • (1996) Applied social research methods series , vol.41
    • Maxwell, J.A.1
  • 27
    • 0003874262 scopus 로고    scopus 로고
    • Sage Publications, London, N.K. Denzin, Y.S. Lincoln (Eds.)
    • The sage handbook of qualitative research 2005, Sage Publications, London. 3rd ed. N.K. Denzin, Y.S. Lincoln (Eds.).
    • (2005) The sage handbook of qualitative research
  • 29
  • 30
    • 0034822074 scopus 로고    scopus 로고
    • Managing public payment for high cost, high-benefit therapy for Gaucher's disease in Ontario
    • Clarke J.T.R., Amato D., Deber R.B. Managing public payment for high cost, high-benefit therapy for Gaucher's disease in Ontario. CMAJ 2001, 165(5):595.
    • (2001) CMAJ , vol.165 , Issue.5 , pp. 595
    • Clarke, J.T.R.1    Amato, D.2    Deber, R.B.3
  • 31
    • 79952616890 scopus 로고    scopus 로고
    • Alternative Arrangements for Medicines: Life Saving Drugs [cited March 12]. Available from
    • Alternative Arrangements for Medicines: Life Saving Drugs [cited 2008 March 12]. Available from: http://www.health.gov.au/internet/main/publishing.nsf/Content/health-pbs-general-supply-othersupply.
    • (2008)
  • 32
    • 79952624604 scopus 로고    scopus 로고
    • Guidelines for the Treatment of Gaucher Disease [cited August 14]. Available from
    • Guidelines for the Treatment of Gaucher Disease [cited 2007 August 14]. Available from: http://www.health.gov.au/internet/wcms/publishing.nsf/Content/71D15BD1EE4A4A8DCA256F85007A7F66/$File/gguidelines.pdf.
    • (2007)
  • 33
    • 0036390495 scopus 로고    scopus 로고
    • Ethics, policy, and rare genetic disorders: the case of Gaucher disease in Israel
    • Gross M. Ethics, policy, and rare genetic disorders: the case of Gaucher disease in Israel. Theoretical Medicine and Bioethics 2002, 23(2):151-170.
    • (2002) Theoretical Medicine and Bioethics , vol.23 , Issue.2 , pp. 151-170
    • Gross, M.1
  • 34
    • 72949102806 scopus 로고    scopus 로고
    • Enzyme therapy for the treatment of type 1 Gaucher disease: clinical outcomes and dose-response relationships
    • Hollak C.E., de Fost M., van Dussen L., Vom Dahl S., Aerts J.M. Enzyme therapy for the treatment of type 1 Gaucher disease: clinical outcomes and dose-response relationships. Expert Opinion on Pharmacotherapy 2009, 10(16):2641-2652.
    • (2009) Expert Opinion on Pharmacotherapy , vol.10 , Issue.16 , pp. 2641-2652
    • Hollak, C.E.1    de Fost, M.2    van Dussen, L.3    Vom Dahl, S.4    Aerts, J.M.5
  • 35
    • 79952624838 scopus 로고    scopus 로고
    • CEDAC. CDR drug database listing on agalsidase beta; [cited 2007 November 17]. Available from
    • CEDAC. CDR drug database listing on agalsidase beta; 2005 [cited 2007 November 17]. Available from: http://www.cadth.ca/index.php/en/cdr/search/%3F%26status=all%26keywords=agalsidase%2520beta.
    • (2005)
  • 36
    • 55249116853 scopus 로고    scopus 로고
    • Public healthcare resource allocation and the Rule of Rescue
    • Cookson R., McCabe C., Tsuchiya A. Public healthcare resource allocation and the Rule of Rescue. Journal of Medical Ethics 2008, 34:540-544.
    • (2008) Journal of Medical Ethics , vol.34 , pp. 540-544
    • Cookson, R.1    McCabe, C.2    Tsuchiya, A.3
  • 37
    • 0026086862 scopus 로고
    • Is your treatment economic, effective efficient?
    • Dean M. Is your treatment economic, effective efficient?. British Medical Journal 1991, 337:480-481.
    • (1991) British Medical Journal , vol.337 , pp. 480-481
    • Dean, M.1
  • 38
    • 0031565415 scopus 로고    scopus 로고
    • The case against
    • Coast J. The case against. British Medical Journal 1997, 314(7087):1118.
    • (1997) British Medical Journal , vol.314 , Issue.7087 , pp. 1118
    • Coast, J.1
  • 39
    • 79952616553 scopus 로고    scopus 로고
    • Fairness as a problem of love and the heart: a clinician's perspective on priority setting in health care
    • Sabin J. Fairness as a problem of love and the heart: a clinician's perspective on priority setting in health care. British Medical Journal 1998, 317(7164):1000-1007.
    • (1998) British Medical Journal , vol.317 , Issue.7164 , pp. 1000-1007
    • Sabin, J.1
  • 40
    • 0027219376 scopus 로고
    • Dimensions of rationing: who should do what?
    • Klein R. Dimensions of rationing: who should do what?. British Medical Journal 1993, (4):309-311.
    • (1993) British Medical Journal , Issue.4 , pp. 309-311
    • Klein, R.1
  • 41
    • 79952628362 scopus 로고    scopus 로고
    • World Development Report Making Services Work For Poor People
    • World Development Report 2004: Making Services Work For Poor People.
    • (2004)
  • 42
    • 0029937215 scopus 로고    scopus 로고
    • Rationing, barbarity and the economist's perspective
    • Loughlin M. Rationing, barbarity and the economist's perspective. Health Care Analysis 1996, 4(2):146-156.
    • (1996) Health Care Analysis , vol.4 , Issue.2 , pp. 146-156
    • Loughlin, M.1
  • 43
    • 0006138876 scopus 로고    scopus 로고
    • Priority setting and health technology assessment: beyond evidence-based medicine and cost-effective analysis
    • Open University Press, Philadelphia, A. Coulter, C. Ham (Eds.)
    • Martin D.K., Peter S. Priority setting and health technology assessment: beyond evidence-based medicine and cost-effective analysis. The global challenge of health care rationing 2000, 133-135. Open University Press, Philadelphia. A. Coulter, C. Ham (Eds.).
    • (2000) The global challenge of health care rationing , pp. 133-135
    • Martin, D.K.1    Peter, S.2
  • 44
    • 27744461277 scopus 로고    scopus 로고
    • Drugs for exceptionally rare diseases: do they deserve special status for funding?
    • Hughes D.A., Tunnage B., Yeo S.T. Drugs for exceptionally rare diseases: do they deserve special status for funding?. QJM 2005, 98(11):829-836.
    • (2005) QJM , vol.98 , Issue.11 , pp. 829-836
    • Hughes, D.A.1    Tunnage, B.2    Yeo, S.T.3
  • 45
    • 0035904788 scopus 로고    scopus 로고
    • Priority-setting decisions for new cancer drugs: a qualitative case study
    • Martin D.K., Pater J.L., Singer P.A. Priority-setting decisions for new cancer drugs: a qualitative case study. Lancet 2001, 358(9294):1676-1681.
    • (2001) Lancet , vol.358 , Issue.9294 , pp. 1676-1681
    • Martin, D.K.1    Pater, J.L.2    Singer, P.A.3
  • 46
    • 78649652812 scopus 로고    scopus 로고
    • Policy alternatives for treatments for rare diseases
    • Panju A., Bell C. Policy alternatives for treatments for rare diseases. CMAJ 2010.
    • (2010) CMAJ
    • Panju, A.1    Bell, C.2


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.