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Orphan drug policies and use in pediatric nephrology

Karpman, Diana LU and Höglund, Peter LU (2017) In Pediatric Nephrology 32(1). p.1-6
Abstract

Orphan drugs designed to treat rare diseases are often overpriced per patient. Novel treatments are sometimes even more expensive for patients with ultra-rare diseases, in part due to the limited number of patients. Pharmaceutical companies that develop a patented life-saving drug are in a position to charge a very high price, which, at best, may enable these companies to further develop drugs for use in rare disease. However, is there a limit to how much a life-saving drug should cost annually per patient? Government interventions and regulations may opt to withhold a life-saving drug solely due to its high price and cost-effectiveness. Processes related to drug pricing, reimbursement, and thereby availability, vary between countries,... (More)

Orphan drugs designed to treat rare diseases are often overpriced per patient. Novel treatments are sometimes even more expensive for patients with ultra-rare diseases, in part due to the limited number of patients. Pharmaceutical companies that develop a patented life-saving drug are in a position to charge a very high price, which, at best, may enable these companies to further develop drugs for use in rare disease. However, is there a limit to how much a life-saving drug should cost annually per patient? Government interventions and regulations may opt to withhold a life-saving drug solely due to its high price and cost-effectiveness. Processes related to drug pricing, reimbursement, and thereby availability, vary between countries, thus having implications on patient care. These processes are discussed, with specific focus on three drugs used in pediatric nephrology: agalsidase beta (for Fabry disease), eculizumab (for atypical hemolytic uremic syndrome), and cysteamine bitartrate (for cystinosis). Access to and costs of orphan drugs have most profound implications for patients, but also for their physicians, hospitals, insurance policies, and society at large, particularly from financial and ethical standpoints.

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author
organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
Atypical hemolytic uremic syndrome, Cystinosis, Fabry disease, Orphan drug, Quality-adjusted life-year, Rare disease
in
Pediatric Nephrology
volume
32
issue
1
pages
1 - 6
publisher
Springer
external identifiers
  • scopus:84991056240
  • wos:000388976400001
ISSN
0931-041X
DOI
10.1007/s00467-016-3520-4
language
English
LU publication?
yes
id
cd2ac90b-1982-4029-9b2b-f224a67aa43d
date added to LUP
2016-10-31 10:32:59
date last changed
2018-04-01 04:26:23
@misc{cd2ac90b-1982-4029-9b2b-f224a67aa43d,
  abstract     = {<p>Orphan drugs designed to treat rare diseases are often overpriced per patient. Novel treatments are sometimes even more expensive for patients with ultra-rare diseases, in part due to the limited number of patients. Pharmaceutical companies that develop a patented life-saving drug are in a position to charge a very high price, which, at best, may enable these companies to further develop drugs for use in rare disease. However, is there a limit to how much a life-saving drug should cost annually per patient? Government interventions and regulations may opt to withhold a life-saving drug solely due to its high price and cost-effectiveness. Processes related to drug pricing, reimbursement, and thereby availability, vary between countries, thus having implications on patient care. These processes are discussed, with specific focus on three drugs used in pediatric nephrology: agalsidase beta (for Fabry disease), eculizumab (for atypical hemolytic uremic syndrome), and cysteamine bitartrate (for cystinosis). Access to and costs of orphan drugs have most profound implications for patients, but also for their physicians, hospitals, insurance policies, and society at large, particularly from financial and ethical standpoints.</p>},
  author       = {Karpman, Diana and Höglund, Peter},
  issn         = {0931-041X},
  keyword      = {Atypical hemolytic uremic syndrome,Cystinosis,Fabry disease,Orphan drug,Quality-adjusted life-year,Rare disease},
  language     = {eng},
  number       = {1},
  pages        = {1--6},
  publisher    = {Springer},
  series       = {Pediatric Nephrology},
  title        = {Orphan drug policies and use in pediatric nephrology},
  url          = {http://dx.doi.org/10.1007/s00467-016-3520-4},
  volume       = {32},
  year         = {2017},
}