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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Veröffentlicht in:	International journal of technology assessment in health care Jg. 23; H. 1; S. 36 - 42
Hauptverfasser:	Drummond, Michael F., Wilson, David A., Kanavos, Panos, Ubel, Peter, Rovira, Joan
Format:	Journal Article
Sprache:	Englisch
Veröffentlicht:	New York, USA Cambridge University Press 01.01.2007
Schlagworte:	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
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Abstract	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.
AbstractList	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. 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.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. 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. [PUBLICATION ABSTRACT]
Author	Kanavos, Panos Wilson, David A. Drummond, Michael F. Ubel, Peter Rovira, Joan
Author_xml	– sequence: 1 givenname: Michael F. surname: Drummond fullname: Drummond, Michael F. organization: University of York – sequence: 2 givenname: David A. surname: Wilson fullname: Wilson, David A. organization: Massachusetts General Hospital and Genzyme Corporation – sequence: 3 givenname: Panos surname: Kanavos fullname: Kanavos, Panos organization: LSE Health, London School of Economics – sequence: 4 givenname: Peter surname: Ubel fullname: Ubel, Peter organization: University of Michigan, Ann Arbor – sequence: 5 givenname: Joan surname: Rovira fullname: Rovira, Joan organization: University of Barcelona
BackLink	https://www.ncbi.nlm.nih.gov/pubmed/17234015$$D View this record in MEDLINE/PubMed
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Cites_doi	10.1016/0168-8510(93)90042-N 10.1016/0048-9697(86)90295-0 10.1016/0277-9536(95)00216-2 10.1002/hec.864 10.1136/bmj.331.7523.1016 10.1136/bmj.320.7243.1165 10.1136/bmj.310.6978.509 10.1017/S0266462306050781 10.1136/bmj.329.7459.224 10.1017/S0266462399154138 10.1056/NEJMp058317 10.2165/00019053-200119110-00004
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Snippet	Historically, patients with rare diseases have been underserved by commercial drug development. In several jurisdictions, specific legislation has been enacted...
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SubjectTerms	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
Title	Assessing the economic challenges posed by orphan drugs
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