Comparison and enlightenment of orphan drug identification standards between America and Europe

Corresponding author: SHAO Rong, shaorong118@163.com

DOI: 10.12201/bmr.202302.00010

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  Abstract: Objective: Comparative analysis of orphan drug designation and approval standards in Europe and the US, to provide suggestions for improving the designation standards for rare diseases and orphan drugs in China. Methods:. By searching the official websites of FDA, EMA and related literature to compare and analyze the designation standards for orphan drugs in Europe and the US. Results: Epidemiological criteria, expected cost-benefit criteria, precision medicine criteria, and clinical advantages for the same drug exist in both Europe and the United States for orphan drug designation standards. However, the EU has two additional criteria for orphan drug designation compared to the US, namely the additional criticality of the disease and the significant benefit. The attitude towards the assessment of orphan subgroups under precision medicine at the practical level is also more different in Europe and the US. In addition, the orphan drug designation system in Europe and the US, in addition to its positive significance, also has the negative impact of disease segmentation leading to orphan drug policy abuse, market exclusivity leading to high-priced orphan drugs to monopolize the market. Conclusion: It is suggested that the designation standards for rare diseases and orphan drugs in China can be borrowed from the experience of orphan drug identification in Europe and the US, starting from the four criteria of epidemiology, expected cost-benefit, additional criticality and precision medicine.

  Key words: rare disease; orphan drug designations; orphan drug approvals

  Submit time: 28 February 2023

  Copyright: The copyright holder for this preprint is the author/funder, who has granted biomedRxiv a license to display the preprint in perpetuity.
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