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Designing clinical trials for rare diseases: unique challenges and opportunities

Nature Reviews Methods Primers volume 2, Article number: 13 (2022) Cite this article

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Orphan drug development is a rapidly expanding field. Nevertheless, clinical trials for rare diseases can present inherent challenges. Optimal study design and partnerships between academia and industry are therefore required for the successful development, delivery and clinical approval of effective therapies in this group of disorders.

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Acknowledgements

C.P. is supported by a Clore Duffield Foundation grant. R.D.S.P. is supported by a Medical Research Council (UK) Clinician Scientist Fellowship (MR/S002065/1). R.D.S.P. and M.G.H. are funded by a Medical Research Council (UK) strategic award to establish an International Centre for Genomic Medicine in Neuromuscular Diseases (ICGNMD) (MR/S005021/1).

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Authors and Affiliations

  1. Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology and The National Hospital for Neurology and Neurosurgery, London, UK

    Chiara Pizzamiglio, Michael G. Hanna & Robert D. S. Pitceathly

  2. Department of Genetics, Johns Hopkins University School of Medicine, Baltimore, MD, USA

    Hilary J. Vernon

  3. Department of Neurogenetics, Kennedy Krieger Institute, Baltimore, MD, USA

    Hilary J. Vernon

Authors
  1. Chiara Pizzamiglio
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  2. Hilary J. Vernon
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  3. Michael G. Hanna
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  4. Robert D. S. Pitceathly
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Correspondence to Robert D. S. Pitceathly.

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The authors declare no competing interests.

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Pizzamiglio, C., Vernon, H.J., Hanna, M.G. et al. Designing clinical trials for rare diseases: unique challenges and opportunities. Nat Rev Methods Primers 2, 13 (2022). https://doi.org/10.1038/s43586-022-00100-2

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