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  • FROM THE ANALYST'S COUCH

Trends in rare disease drug development

  1. Rui Chen

    1. Clinical Pharmacology Research Center, Peking Union Medical College Hospital, Beijing, China; State Key Laboratory of Complex Severe and Rare Diseases, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China. Rui Chen, Sen Liu and Jiashu Han contributed equally to this work.

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  2. Sen Liu

    1. Tsinghua Clinical Research Institute, School of Medicine, Tsinghua University, Beijing, China. Rui Chen, Sen Liu and Jiashu Han contributed equally to this work.

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  3. Jiashu Han

    1. State Key Laboratory of Complex Severe and Rare Diseases, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China. Rui Chen, Sen Liu and Jiashu Han contributed equally to this work.

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  4. Shuhua Zhou

    1. Citeline, London, UK.

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  5. Yang Liu

    1. Tsinghua Clinical Research Institute, School of Medicine, Tsinghua University, Beijing, China.

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  6. Xiaoyuan Chen

    1. Tsinghua Clinical Research Institute, School of Medicine, Tsinghua University, Beijing, China; Office of Clinical Trial Institute, Beijing Tsinghua Changgung Hospital, Beijing, China.

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  7. Shuyang Zhang

    1. State Key Laboratory of Complex Severe and Rare Diseases, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China; Department of Cardiology, Peking Union Medical College Hospital, Beijing, China.

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Advances in the understanding of the biology underlying rare diseases, as well as progress with therapeutic platforms such as nucleic-acid agents, have enabled increased research and development (R&D) on drugs for rare diseases. Governmental incentives and regulatory initiatives have played a key role too, such as the Orphan Drug Act of 1983 in the USA.

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Nature Reviews Drug Discovery 23, 168-169 (2024)

doi: https://doi.org/10.1038/d41573-023-00177-8

Acknowledgements

The authors gratefully acknowledge X. Wang and S. Ji from the Center for Drug Evaluation, National Medical Products Administration in China, as well as Z. Yan, Y. Li, C. Ge and J. An from the Tsinghua Clinical Research Institute, Tsinghua University, for their contribution to the work.

Supplementary Information

  1. Supplementary information
  2. Supplementary Table 1
  3. Supplementary Table 2

Competing Interests

S. Zhou is an employee of Citeline. The other authors declare no competing interests.

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