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Flexibility in the FDA approach to orphan drug development

Nature Reviews Drug Discovery volume 16, pages 737–738 (2017)Cite this article

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Scientific advances, in combination with government incentives and commercial opportunity, have fuelled strong investment in orphan drugs, resulting in many innovative therapies. Here, we discuss the approach of the FDA to a range of issues that remain crucial to maintaining this momentum, such as the use of the totality of evidence in evaluating orphan drugs.

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References

  1. Sherman, R.E., Davies, K. M., Robb, M.A., Hunter, N.L, & Califf, R. M. Accelerating development of scientific evidence for medical products within the existing US regulatory framework. Nat. Rev. Drug Discov. 16, 297–298 (2017).

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Acknowledgements

The authors would like to thank D. Lewis (at the FDA) and R. Sher (formerly at the FDA) for their valuable assistance in facilitating the development of the paper.

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  1. Office of the Commissioner at the US Food and Drug Administration, 10903 New Hampshire Avenue, Silver Spring, 20993, Maryland, USA

    Nina L. Hunter, Gayatri R. Rao & Rachel E. Sherman

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  1. Nina L. Hunter
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  2. Gayatri R. Rao
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  3. Rachel E. Sherman
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Correspondence to Nina L. Hunter.

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The authors declare no competing financial interests.

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FURTHER INFORMATION

U.S. Food and Drug Administration, Developing Products for Rare Diseases & Conditions

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Hunter, N., Rao, G. & Sherman, R. Flexibility in the FDA approach to orphan drug development. Nat Rev Drug Discov 16, 737–738 (2017). https://doi.org/10.1038/nrd.2017.151

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