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Rare Diseases Epidemiology: Update and Overview pp 233–247Cite as

New Therapeutic Uses for Existing Drugs

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Part of the book series: Advances in Experimental Medicine and Biology ((AEMB,volume 1031))

Abstract

Eighty percent of drugs that enter human clinical testing are never approved for use. This means that for every five drugs that make it into the clinic, there are four that failed to show effectiveness for treating the disease or condition the drug was designed to treat.

This high failure rate means there are many existing, partially developed therapeutic candidates with known pharmacology, formulation, and potential toxicity. Finding new uses for existing experimental drugs or biologics “repositioning” builds upon previous research and development efforts, so new candidate therapies can be advanced to clinical trials for a new use more quickly than starting from scratch.

Federal funding initiatives in the U.S. and UK started to support pre-clinical /or early stage trials for repositioning existing experimental drugs or biologics (therapies). This chapter covers some of the process issues that have been solved and the remaining challenges that are still in need of solutions. The chapter is primarily written from a U.S. federal funding perspective. The general concepts could be applied more globally to benefit rare and neglected disease populations. The drug development and process bottlenecks are the same for both rare and common disease.

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Author information

Authors and Affiliations

  1. Drug Development Partnership Programs, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health, Bethesda, MD, USA

    Bobbie Ann Austin

  2. Office of Strategic Alliances, National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, MD, USA

    Ami D. Gadhia

Authors
  1. Bobbie Ann Austin
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  2. Ami D. Gadhia
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Corresponding author

Correspondence to Bobbie Ann Austin .

Editor information

Editors and Affiliations

  1. RDR and CIBERER, Instituto de Salud Carlos III, Madrid, Spain

    Manuel Posada de la Paz

  2. Centro Nazionale Malattie Rare, Istituto Superiore di Sanita, Roma, Italy

    Domenica Taruscio

  3. National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, Maryland, USA

    Stephen C. Groft

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Austin, B.A., Gadhia, A.D. (2017). New Therapeutic Uses for Existing Drugs. In: Posada de la Paz, M., Taruscio, D., Groft, S. (eds) Rare Diseases Epidemiology: Update and Overview. Advances in Experimental Medicine and Biology, vol 1031. Springer, Cham. https://doi.org/10.1007/978-3-319-67144-4_14

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