Given the high attrition rates, substantial costs and slow pace of new drug discovery and development, repurposing of 'old' drugs to treat both common and rare diseases is increasingly becoming an attractive proposition because it involves the use of de-risked compounds, with potentially lower overall development costs and shorter development timelines. Various data-driven and experimental approaches have been suggested for the identification of repurposable drug candidates; however, there are also major technological and regulatory challenges that need to be addressed. In this Review, we present approaches used for drug repurposing (also known as drug repositioning), discuss the challenges faced by the repurposing community and recommend innovative ways by which these challenges could be addressed to help realize the full potential of drug repurposing.
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This paper stems from a workshop hosted by the Medical Research Council (MRC) Centre for Drug Safety Science (CDSS; http://www.liv.ac.uk/drug-safety), University of Liverpool, in conjunction with the UK Pharmacogenetics and Stratified Medicine Network (http://www.uk-pgx-stratmed.co.uk) in November 2015 to discuss the current status of drug repurposing and evaluate various challenges faced by this field. The workshop was attended by representatives from pharmaceutical and biotechnology companies, including small-and-medium-sized enterprises focusing on drug repurposing, contract research organizations, regulatory agencies, research funding charities and academia. The content of this review has been expanded through literature search and further discussions of the authors and their research networks since the workshop.
S.H. is an author on this manuscript and works for the Medicines and Healthcare Products Regulatory Agency (MHRA), UK; the opinions expressed in this review are her own and should not be attributed to the MHRA/European Medicines Agency (EMA). A.D. is a director of PharmaKure Ltd.
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Pushpakom, S., Iorio, F., Eyers, P. et al. Drug repurposing: progress, challenges and recommendations. Nat Rev Drug Discov 18, 41–58 (2019). https://doi.org/10.1038/nrd.2018.168
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