Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years

Braun, M. Miles; Farag-El-Massah, Sheiren; Xu, Kui; Coté, Timothy R.

doi:10.1038/nrd3160

Cited by 149Pharmaceutical Economics and PolicyPharmaceutical studies and practicesPharmaceutical industry and healthcare

Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years

Braun, M. Miles, Farag-El-Massah, Sheiren, Xu, Kui, Coté, Timothy R.·2010·Nature Reviews Drug Discovery

DOI 10.1038/nrd3160

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Abstract

The 1983 US Orphan Drug Act has stimulated the development of new therapies for rare diseases. To provide the first comprehensive overview of orphan-designated products and their indications, this article quantitatively analyses the characteristics and distribution of orphan designations and approvals by the US Food and Drug Administration from 1983 to August 2008. Of the 1,892 orphan-designated products, 326 received marketing approval, representing 247 different drugs and more than 200 different diseases. About half of the approvals had occurred by 4 years after designation was granted. The most common patient population size for orphan designations and approvals was fewer than 10,000 patients, and cancer was the most common disease area. The implications of such findings for future development and marketing of therapies for rare diseases are discussed.

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Drug discovery for neuropsychiatric diseases could involve developing a catalogue of drugs targeting a variety of orphan diseases.
“Hence, drug discovery for neuropsychiatric diseases could involve developing a catalogue of drugs targeting a variety of orphan diseases (Braun et al., 2010).”
From CNVs: Harbingers of a Rare Variant Revolution in Psychiatric Genetics (2012) · cites this paper as Braun et al., 2010

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Authors: Braun, M. M., Farag-El-Massah, S., Xu, K., & Coté, T. R.
Affiliations: United States Food and Drug Administration (US)
Corresponding author: Coté, Timothy R.
ORCID: Xu, Kui
Venue: Nature Reviews Drug Discovery
Publisher: Springer Science and Business Media LLC
Published: 2010-06-07
Volume: 9
Issue: 7
Pages: 519-522
ISSN: 1474-1776 (ISSN-L)
1474-1784
DOI: 10.1038/nrd3160
OpenAlex: W2024559544
Cited by: 148
References: 3
Citations by year: 2026: 1 · 2025: 3 · 2024: 2 · 2023: 11 · 2022: 4 · 2021: 6 · 2020: 4 · 2019: 6 · 2018: 9 · 2017: 9 · 2016: 12 · 2015: 11 · 2014: 14 · 2013: 19 · 2012: 18
FWCI: 42.07
Type: journal article
Language: en
Open access: closed
Licence: www.springer.com/tdm (tdm)
Locations: Nature Reviews Drug Discovery (publishedVersion)
PubMed (publishedVersion)
Topics: Social Sciences › Economics, Econometrics and Finance › Economics and Econometrics › Pharmaceutical Economics and Policy
Health Sciences › Medicine › Pediatrics, Perinatology and Child Health › Pharmaceutical studies and practices
Life Sciences › Pharmacology, Toxicology and Pharmaceutics › Pharmacology › Pharmaceutical industry and healthcare
Keywords: orphan drugs, rare diseases, Orphan Drug Act, orphan drug designation, drug approval, FDA, drug development incentives, cancer
MeSH terms: Humans, Orphan Drug Production, United States, United States Food and Drug Administration, Drug Approval, History, 20th Century, History, 21st Century

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Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years

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