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.
(Less)
- author
- Karpman, Diana
LU
and Höglund, Peter
LU
- organization
- publishing date
- 2017-01
- 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
- pmid:27738765
- 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
- 2024-05-03 12:34:20
@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}},
keywords = {{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}},
doi = {{10.1007/s00467-016-3520-4}},
volume = {{32}},
year = {{2017}},
}