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What impact does ‘conventional’ economic evaluation have on patient access to new orphan medicines? A comparative study of their reimbursement in Australia (2005–2012)

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References

  • Drummond MF, Wilson DA, Kanavos P, et al. Assessing the economic challenges posed by orphan drugs. Int J Technol Assess Health Care 2007;23(1):36-42
  • Haffner ME, Torrent-Farnell J, Maher PD. Does orphan drug legislation really answer the needs of patients? Lancet 2008;371:2041-4
  • Sasinowski F. Quantum of effectiveness evidence in FDA’a approval of orphan drugs. Cataloguing FDA’s flexibility in regulating therapies for persons with rare disorders. National Organization for Rare Disorders, Chairman of the Board. 2011. Available from: http://nordphysicianguides.org/wp-content/uploads/2012/02/NORD-study-of-FDA-approval-of-orphan-drugs.pdf
  • Denis A, Mergaert L, Fostier C, et al. Critical assessment of Belgian reimbursement dossiers of orphan drugs. Pharmacoeconomics 2011;29(10):883-93
  • Drummond M, Towse A. Orphan drugs policies: a suitable case for treatment. Eur J Health Econ 2014;15:335-40
  • Simoens S. Health technologies for rare diseases: does conventional HTA still apply? Expert Rev Pharmacoecon Outcomes Res 2014;14(3):315-17
  • McCabe C, Claxton K, Tsuchiya A. Orphan drugs and the NHS: should we value rarity? BMJ 2005;331:1016-19
  • Australian Government. Department of Health. PBAC outcomes. Available from: www.pbs.gov.au/info/industry/listing/elements/pbac-meetings/pbac-outcomes
  • Australian Government. Department of health. Therapeutic Goods Administration. eBusiness services. Available from: www.ebs.tga.gov.au/
  • Australian Government. Department of Health. Public Summary Documents. Available from: www.pbs.gov.au/info/industry/listing/elements/pbac-meetings/psd
  • Australian Government. Department of Health. Therapeutic Goods Administration. Industry. Prescription Medicines. Orphan Drugs. Available from: www.tga.gov.au/industry/pm-orphan-drugs.htm
  • Australian Government. Department of Health. Media centre. Departmental media release. Available from: www.health.gov.au/internet/main/publishing.nsf/Content/Departmental+Media+Releases-1
  • Australian Government. Past Budgets. Available from: www.budget.gov.au/past_budgets.htm
  • SAS Institute Inc., Cary, NC, USA
  • Dupont AG, Van Wilder PB. Access to orphan drugs despite poor quality of clinical evidence. Br J Clin Pharmacol 2011;71(4):488-96
  • Cote A, Keating B. What is wrong with orphan drug policies? Value Health 2012;15(8):1184-90
  • Australian Government. Department of Health. Therapeutic Goods Administration. TGA Basics. Available from: www.tga.gov.au/about/tga.htm
  • Australian Government. Department of Health. About the PBS. Available from: www.pbs.gov.au/info/about-the-pbs
  • Australian Government. Department of Health. Pharmaceutical Benefits Advisory Committee. Available from: www.pbs.gov.au/info/industry/listing/participants/pbac
  • Australian Government. Department of Health. Other supply arrangements outside the Pharmaceutical Benefits Scheme. Available from: www.health.gov.au/lsdp

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