Pharmaceutical pricing, cost containment and new treatments for rare diseases in children

Orphanet Journal of Rare Diseases

Gabrielle Gold-von Simson¹  Peter Stella¹ 

[1] Department of Pediatrics, NYU Langone Hospital, New York, NY, USA

关键词: Medicaid;    340b;    Affordable care act;    Pediatrics;    Drug pricing;    Orphan drugs;   

Others  :  1149465
DOI  :  10.1186/s13023-014-0152-2

 received in 2014-07-08, accepted in 2014-09-24,  发布年份 2014

【 摘 要 】

Cost-containment in healthcare spending has become a central issue in public policy and healthcare reform, especially as the affordable care act adds millions of people to public and private insurance rolls. In this climate, longstanding criticism of pharmaceutical pricing has grown sharper, and many in both policy and medicine have characterized the costs of newly developed drugs as both exorbitant and wasteful of scarce healthcare resources. At the same time, pharmaceutical research and development pipeline costs are increasing exponentially.

Price resistance poses a significant threat to the development of drugs to treat rare pediatric diseases, where exceptionally high prices are a sine qua non of commercial viability. This article examines the trends in public discussion of high cost drugs and the potential consequences for orphan drug development. We conclude that despite growing public hostility towards high unit costs, drugs that treat rare diseases in children are likely to remain well-compensated and commercially viable.

【 授权许可】

   
2014 Stella and Gold-von Simson; licensee BioMed Central Ltd.

【 图 表 】

Figure 1.

【 参考文献 】

• [1]Pharmaceuticals in the USA: ISIC 2423. Euromonitor International, Chicago, IL; 2012.
• [2][http:/ / www.forbes.com/ sites/ edsilverman/ 2013/ 10/ 09/ sticker-shock-will-orphan-drug-reim bursement-remain-stable/ ] webcite Sticker Shock: Will Orphan Drug Reimbursement Remain Stable? Forbes at
• [3]O¿Sullivan BP, Orenstein DM, Milla CE: Pricing for orphan drugs: Will the market bear what society cannot? JAMA 2013, 310:1343-1344.
• [4]Abboud C, Berman E, Cohen A, Cortes J, DeAngelo D, Deininger M, Devine S, Druker B, Fathi A, Jabbour E, Jagasia M, Kantarjian H, Khoury J, Laneuville P, Larson R, Lipton J, Moore JO, Mughal T, O¿Brien S, Pinilla-Ibarz J, Quintas-Cardama A, Radich J, Reddy V, Schiffer C, Shah N, Shami P, Silver RT, Snyder D, Stone R, Talpaz M, et al.: The price of drugs for chronic myeloid leukemia (CML) is a reflection of the unsustainable prices of cancer drugs: from the perspective of a large group of CML experts. Blood 2013, 121:4439-4442.
• [5]Cohen J, Milne CP: Is the increasing cost of treating rare diseases sustainable? Expert Opin Orphan Drugs 2013, 1:581-583.
• [6]Talele SS, Xu K, Pariser AR, Braun MM, Farag-El-Massah S, Phillips MI, Thompson BH, Coté TR: Therapies for inborn errors of metabolism: What has the orphan drug act delivered? Pediatrics 2010, 126:101-106.
• [7]Conti RM, Bach PB: COst consequences of the 340b drug discount program. JAMA 2013, 309:1995-1996.
• [8]Federal Register. Volume 78. 141. Tuesday July 23.
• [9]Kesselheim AS, Myers JA, Solomon DH, Winkelmayer WC, Levin R, Avorn J: The prevalence and cost of unapproved uses of top-selling orphan drugs. PLoS One 2012, 7:e31894.
• [10]Liang BA, Mackey T: Health care policy. Reforming off-label promotion to enhance orphan disease treatment. Science 2010, 327:273-274.
• [11][http://www.managedcaremag.com.ezproxy.med.nyu.edu/linkout/2013/3/10] webcite Managed Care Magazine Online. at