Expanding rare disease drug trials based on shared molecular etiology

Nature Biotechnology volume 32, pages515–518(2014)Cite this article

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Current clinical trial approaches in rare disease test one drug on one indication defined by a clinical phenotype. For targeted drugs, grouping patients by molecular etiology would make much more sense.

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Figure 1: Benefits of grouping by molecular etiology.

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Acknowledgements

P.J.B. is also a Program Director in the Division of Metabolism and Health Effects, National Institute on Alcohol Abuse and Alcoholism, National Institutes of Health, Bethesda, Maryland, USA.

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Affiliations

  1. Philip J. Brooks and Steve Groft are at the Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health, Bethesda, Maryland, USA.

    Philip J Brooks & Steve Groft

  2. Danilo A. Tagle is at the Office of Special Initiatives, Office of the Director, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health, Bethesda, Maryland, USA.

    Danilo A Tagle

Authors
  1. Philip J Brooks
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  2. Danilo A Tagle
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  3. Steve Groft
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Correspondence to Philip J Brooks.

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The authors declare no competing financial interests.

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Brooks, P., Tagle, D. & Groft, S. Expanding rare disease drug trials based on shared molecular etiology. Nat Biotechnol 32, 515–518 (2014). https://doi.org/10.1038/nbt.2924

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