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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Bibliographic Details
Published in:International journal of technology assessment in health care Vol. 23; no. 1; pp. 36 - 42
Main Authors: Drummond, Michael F., Wilson, David A., Kanavos, Panos, Ubel, Peter, Rovira, Joan
Format: Journal Article
Language:English
Published: New York, USA Cambridge University Press 01.01.2007
Subjects:
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
Online Access:Get full text
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Summary: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.
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ISSN:0266-4623
1471-6348
DOI:10.1017/S0266462307051550