Abstract
Rare disease drug development could benefit substantially from increased patient engagement and input to enhance understanding of the key aspects of disease impact, ways to measure these impacts and patients' perspectives on the benefit–risk profile of potential therapies.
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E.K. is an employee and stockholder of Ultragenyx Pharmaceutical Inc. and is the President of the EveryLife Foundation for Rare Diseases, a non-profit advocacy organization that promotes incorporating patient input in the drug development process. M.G.B. is Senior Director of Advocacy and Science Policy at the EveryLife Foundation for Rare Diseases.
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Bronstein, M., Kakkis, E. Patients as key partners in rare disease drug development. Nat Rev Drug Discov 15, 731–732 (2016). https://doi.org/10.1038/nrd.2016.133
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DOI: https://doi.org/10.1038/nrd.2016.133
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