• 国家药监局综合司 国家卫生健康委办公厅
  • 国家药监局综合司 国家卫生健康委办公厅

Implications on the Model of Drug Guarantee for Rare Diseases at Domestic and Abroad

Corresponding author: PAN JAY, panjie.jay@scu.edu.cn
DOI: 10.12201/bmr.202009.00016
Statement: This article is a preprint and has not been peer-reviewed. It reports new research that has yet to be evaluated and so should not be used to guide clinical practice.
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    Abstract: Objective: By comparing the characteristics and emphases of overseas and domestic security models for orphan drugs, we summarized the current experience and deficiencies in China, so as to provide suggestions for choosing appropriate models and promoting policy improvements. Methods: We sorted out the measures for rare diseases issued by the United States, the European Union, Singapore, Australia and other countries, as well as the relevant experiences of various regions in China, and analyzed the differences between these modes of different countries and regions. Results: At present, the typical security models for rare diseases include medical insurance reimbursement, financial fund subsidies, special fund subsides, medical assistance fund and so on. They are different in terms of financing scale, assistance level, and sustainability. Conclusions: China needs to comprehensively consider its own economic strength, the epidemiological characteristics of rare diseases and other factors. Then, it adopts a multi-level security model to accurately fund the drugs of patients with rare diseases and share the cost together.

    Key words: Rare diseases; Orphan drugs; Security model

    Submit time: 15 December 2020

    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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    1 2020-09-23

    bmr.202009.00016V1

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Zhang Xiaoxing, Zhou Tingting, PAN JAY. Implications on the Model of Drug Guarantee for Rare Diseases at Domestic and Abroad. 2020. biomedRxiv.202009.00016

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