Analysis of the European Unions Orphan Drug Designation Policy and Its Enlightenment to China

Corresponding author: xiejinping, 15151870834@163.com

DOI: 10.12201/bmr.202207.00031

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  Abstract: objective：To provide reference for the designation and management of rare diseases and orphan drugs and the follow-up incentive policies in China. Methods: Combined with literature research and data analysis (2000-2020 EU orphan drug designation and approval data), the EU orphan drug designation process and policy implementation effects were analyzed and studied. Result and Conclusions: The EU orphan drug designation process is roughly composed of four stages: the pre-submission meeting stage, the submission stage, the validation stage, as well as the evaluation and opinion stage. In the 20 years from 2000 to 2020, the EU accepted a total of 3678 cases of the applications for orphan drug designation, 2,399 were approved by the Orphan Drug Committee (COMP), 1,047 were withdrawn, and 2,379 were identified by the European Commission (EC). Finally, a total of 190 orphan drugs were approved for marketing. It is suggested that on the basis of defining the concept of rare diseases and orphan drugs，we should establish designation mechanism for orphan drugs, and realize the dual catalog management of rare diseases and orphan drugs, to provide legal basis for the medication of patients with rare diseases; establish a database related to rare diseases, and cooperate with international rare disease network, assisting the research and development of orphan drugs, and forming a global rare disease diagnosis and orphan drugs security community.

  Key words: Rare diseases; Orphan drugs; Designation; Policy analysis; Enlightenment

  Submit time: 15 July 2022

  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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