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Journal of Market Access & Health Policy Volume 2, 2014 - Issue 1
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Original Research Articles

Are payers treating orphan drugs differently?

Joshua P. Cohen Tufts Center for the Study of Drug Development, Boston, MA, USACorrespondence[email protected]
, PhD &
Abigail Felix Tufts Center for the Study of Drug Development, Boston, MA, USA
, BA
Article: 23513 | Accepted 12 May 2013, Published online: 15 Jan 2014

References

  • Orphan Drug Regulations (57 FR 62076), December 29, 1992 and 21 CFR Part 316 et seq..
  • Blankart CR , Stargardt T , Schreyogg J . Availability of and access to orphan drugs: An international comparison of pharmaceutical treatments for pulmonary arterial hypertension, Fabry disease, hereditary angioedema and chronic myeloid leukemia. Pharmacoeconomics. 2011; 29: 63–82.
  • Kaitin KI . U.S. orphan product designations more than doubled from 2000–02 to 2006–08: Tufts Center for the Study of Drug Development Impact Report. 2010; Boston, MA: Tufts Center for the Study of Drug Development. 1–5.
  • European Parliament. Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products. Official J Eur Comm . 2000. 22 1. 2000, L 18/1.
  • Seoane-Vazquez E , Rodriguez-Monguio R , Szeinbach SL , Visaria J . Incentives for orphan drug research and development in the United States. Orphanet J Rare Dis. 2008; 3: 33.
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  • Cohen JP , Malins A , Shahpurwala Z . Compared to US practice, evidence-based reviews in Europe appear to lead to lower prices for some drugs. Health Aff. 2013; 32: 762–70.
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  • Denis A , Simoens S , Fostier C , Mergaert L , Cleemput I . KCE reports 112C; Policies for orphan diseases and orphan drugs. Belgian Health Care Knowledge Centre. 2009. Available from: http://ec.europa.eu/health/ph_threats/non_com/docs/policies_orphan_en.pdf [cited 1 November 2013]..
  • Faden L , Huskamp H . Field M , Boat T . Medicare Part D coverage and reimbursement of orphan drugs. Institute of Medicine Committee on Accelerating Rare Diseases Research and Orphan Product Development. Rare diseases and orphan products: Accelerating research and development. 2010; Washington DC: National Academies Press. 309–343.
  • Cohen J , Young B , Rochon S , Faden L . Are Medicare plans complying with CMS regulation?. Expert Rev Pharmacoecon Outcomes Res. 2008; 8(2): 133–8.
  • Cohen J , Faden L , Predaris S , Young B . Patient access to pharmaceuticals: An international comparison. Eur J Health Econ. 2007; 8(3): 253–66.
  • Hargrave E , Hoadley J , Thompson J . Coverage and pricing of drugs that can be covered under Part B and Part D. 2007; Washington DC: MedPAC.
  • Medicare Plan Finder. Available from: https://www.medicare.gov/find-a-plan/questions/home.aspx?AspxAutoDetectCookieSupport=1 [cited 23 October 2013]..
  • Forgotten conditions: Misdiagnosed and unsupported, how patients are being let down. Available from: http://www.2020health.org/2020health/Publication-2012/Wellbeing-and-Public-Health/forgotten-conditions.html [cited 20 September 2013]..
  • Dupont A , van Wilder P . Access to orphan drugs despite poor quality of clinical evidence. Br J Clin Pharmacol. 2011; 71(4): 488–96.
  • Herper M . Inside the pricing of a $300,000-a-year drug. Forbes Magazine. 1 January 2013. Available from: http://www.forbes.com/sites/matthewherper/2013/01/03/inside-the-pricing-of-a-300000-a-year-drug/ [cited 1 November 2013]..
  • Tilles S , Borish L , Cohen J . Management of hereditary angioedema in 2012: Scientific and pharmacoeconomic perspectives. Ann Allergy Asthma Immunol. 2013; 110(2): 70–4.
  • Schey C , Milanova T , Hutchings A . Estimating the budget impact of orphan medicines in Europe: 2010–2020. Orphanet J Rare Dis. 2011; 6: 62.

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