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Clin Pharmacol Ther. 2016 Oct;100(4):339-41. doi: 10.1002/cpt.422. Epub 2016 Aug 16.

Challenges and Hurdles to Business as Usual in Drug Development for Treatment of Rare Diseases.

Author information

1
Institute for Rare and Neglected Diseases Drug Diseases, Mountain View, California, USA. david.swinney@irnd3.org.

Abstract

Only 10-15 first-in-class new medicines are approved each year by the global pharmaceutical industry for all diseases, of which less than a third is for rare (orphan) diseases. The drug discovery processes to identify rare and common diseases are similar, suggesting it will be impossible to discover new drugs for even a small fraction of the rare diseases using the current paradigm. Different approaches are required to address this large unmet medical need.

PMID:
27393380
DOI:
10.1002/cpt.422
[Indexed for MEDLINE]

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