Assessing the economic challenges posed by orphan drugs

Historically, patients with rare diseases have been underserved by commercial drug development. In several jurisdictions, specific legislation has been enacted to encourage the development of drugs for rare diseases (orphan drugs), which would otherwise not be commercially viable. However, because o...

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Vydáno v:	International journal of technology assessment in health care Ročník 23; číslo 1; s. 36 - 42
Hlavní autoři:	Drummond, Michael F., Wilson, David A., Kanavos, Panos, Ubel, Peter, Rovira, Joan
Médium:	Journal Article
Jazyk:	angličtina
Vydáno:	New York, USA Cambridge University Press 01.01.2007
Témata:	Cost analysis Cost-Benefit Analysis Cost-effectiveness analysis Disease Drugs Equity Europe FDA approval GENERAL ESSAYS Health care policy Health Policy Health services Health technology assessment Hospitals Humans Legislation Medical ethics Orphan Drug Production - economics Patients Pharmaceutical industry Rare diseases Reimbursement Support groups Technological change Technology Assessment, Biomedical United States United States Europe Equity Cost-effectiveness analysis Health policy
ISSN:	0266-4623, 1471-6348
On-line přístup:	Získat plný text
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Popis
Shrnutí:	Historically, patients with rare diseases have been underserved by commercial drug development. In several jurisdictions, specific legislation has been enacted to encourage the development of drugs for rare diseases (orphan drugs), which would otherwise not be commercially viable. However, because of the small market, these drugs are often very expensive. Under the standard methods of health technology assessment (HTA) incorporating economic evaluation, orphan drugs do not usually prove to be cost-effective and this, coupled with their high cost, means that funding and patient access may be limited. However, these restrictions may not be in line with societal preferences. Therefore, this study discusses whether the standard methods of HTA are adequate for assisting decisions on patient access to and funding of orphan drugs and outlines a research agenda to help understand the societal value of orphan drugs and issues surrounding their development, funding, and use.
Bibliografie:	PII:S0266462307051550 ark:/67375/6GQ-SJFFM4SN-B istex:3E4047C25587198DD1013EB7C2CA0AA1DEC19BC7 PMID:17234015 ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 content type line 14 content type line 23
ISSN:	0266-4623 1471-6348
DOI:	10.1017/S0266462307051550