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Orphan products: an emerging trend in drug approvals

Nature Reviews Drug Discovery volume 9, page 84 (2010)Cite this article

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As highlighted in a recent News Feature, Rare incentives (Nature Rev. Drug Discov. 7, 190–191; 2008)1, 2008 saw the 25th anniversary of the US Orphan Drug Act (ODA), which was promulgated in recognition of the economic barriers to developing drugs for rare diseases (defined as those that affect fewer than 200,000 persons in the United States)2. The ODA encourages the development of promising new compounds by providing significant fiscal benefits: FDA-enforced marketing exclusivity, tax credits and fee exemptions. These products are designated 'orphan products' by the FDA's Office of Orphan Products Development, and if shown to be safe and effective can be approved for marketing. As of 20 July 2009, 343 orphan products have been marketed to treat rare diseases3. By contrast, only 10 such products were approved in the 10 years preceding the ODA4.

The ODA has been credited not only with offering new hope for those who suffer from rare diseases, but also with nurturing an expanding biotechnology sector and personalizing drug development5,6. In a time of relative contraction in the development of new small-molecule therapeutics, orphan drug development has emerged as a burgeoning sector of the pharmaceutical industry. This correspondence documents its growth.

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Figure 1: Orphan and non-orphan new drugs and biologics approved by the FDA from 1984 to 2008.

References

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Authors and Affiliations

  1. Timothy Coté, Aditya Kelkar, Kui Xu and M. Miles Braun are at the Food and Drug Administration, Office of Orphan Products Development, Rockville, Maryland 20857, USA.,

    Timothy Coté, Aditya Kelkar, Kui Xu & M. Miles Braun

  2. M. Ian Phillips and Aditya Kelkar are at the Keck Graduate Institute, Claremont, California 91711, USA.,

    M. Ian Phillips

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  1. Timothy Coté
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  2. Aditya Kelkar
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  3. Kui Xu
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  4. M. Miles Braun
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  5. M. Ian Phillips
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Corresponding author

Correspondence to Timothy Coté.

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Coté, T., Kelkar, A., Xu, K. et al. Orphan products: an emerging trend in drug approvals. Nat Rev Drug Discov 9, 84 (2010). https://doi.org/10.1038/nrd2546-c1

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