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Designing clinical trials for rare diseases: unique challenges and opportunities

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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Correspondence to Robert D. S. Pitceathly.

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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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  • DOI: https://doi.org/10.1038/s43586-022-00100-2

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