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Two decades of orphan product development

Nature Reviews Drug Discovery volume 1, pages 821–825 (2002)Cite this article

Abstract

Over the past 20 years, incentives of the Orphan Drug Act (ODA), the largest single source of extramural clinical grants at the US Food and Drug Administration, have had a substantial impact on public health. ODA incentives have contributed to the development of many innovative biotechnology products, and as our understanding of the human genome evolves, it is anticipated that pharmacogenomics will result in the identification of more 'orphan diseases'.

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Figure 1: Distribution of orphan products by therapeutic class.

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References

  1. Title 21. United States Code (USC) Section 360ee.

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Author information

Authors and Affiliations

  1. the Office of Orphan Products Development, US Food and Drug Administration, Room 15A08, 5600 Fishers Lane, Rockville, 20857, Maryland, USA

    Marlene E. Haffner, Janet Whitley & Marie Moses

Authors
  1. Marlene E. Haffner
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  2. Janet Whitley
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  3. Marie Moses
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Corresponding author

Correspondence to Marlene E. Haffner.

Related links

Related links

DATABASES

Cancer.gov

breast cancer

hairy-cell leukaemia

ovarian cancer

Medscape DrugInfo

albuterol

alglucerase

caffeine

clonidine

cysteamine

ganciclovir

pegademase

thalidomide

OMIM

cystic fibrosis

nephropathic cystinosis

severe combined immunodeficiency

FURTHER INFORMATION

ClinicalTrials.gov

Federal Food, Drug and Cosmetic Act

National Organization for Rare Diseases

Orphan Drug Act

US Food and Drug Administration

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Haffner, M., Whitley, J. & Moses, M. Two decades of orphan product development. Nat Rev Drug Discov 1, 821–825 (2002). https://doi.org/10.1038/nrd919

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