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Orphanet Journal of Rare Diseases

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Open Access 01-12-2023 | Research

Analysis of Incentive Policies and Initiatives on Orphan Drug Development in China: Challenges, Reforms and Implications

Authors: Zhiyao Zhao, Zhongyang Pei, Anxia Hu, Yuhui Zhang, Jing Chen

Published in: Orphanet Journal of Rare Diseases | Issue 1/2023

Abstract

Objectives

Rare diseases are a global public health issue with a more pressing situation in China. Unfortunately, the relevant research and development in this country are still in its infancy, leading to limited drug accessibility. In view of this, the Chinese government has taken a series of countermeasures to promote orphan drug R&D in recent years, which has presented encouraging results. This paper aims to review incentive policies and funding initiatives formulated by the Chinese government and examine their implications on orphan drug R&D.

Methods

Policies targeting orphan drug R&D during 2012–2022 were retrieved from the relevant official websites, categorized into different themes and analyzed for the contents. Data on government funding, drug approval, clinical trial approval and orphan drug designation were collected through internet search to analyze the implications of those incentive policies and initiatives on orphan drug R&D in China.

Results

A total of 20 relevant policy documents were identified and five major themes were revealed through content analysis, including national strategy, expedited approval, safety and efficacy requirements, data protection and technical support. The government input in orphan drug R&D has witnessed a steady annual increase. Driven by those incentives, the numbers of orphan drugs approved for marketing and drug candidates entering clinical studies are increasing year by year, and more domestic pharmaceutical companies are actively involved in the R&D of orphan drugs.

Conclusions

Orphan drug development in China is growing rapidly under the stimulation of incentive regulatory policies and more investment in researches. China is working toward a more standardized and comprehensive rare disease ecosystem. However, there are still some challenges, such as the lack of sufficient financial support and the call for systematic legislation on rare diseases, to be addressed for future success.

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Title: Analysis of Incentive Policies and Initiatives on Orphan Drug Development in China: Challenges, Reforms and Implications
Authors: Zhiyao Zhao
Zhongyang Pei
Anxia Hu
Yuhui Zhang
Jing Chen
Publication date: 01-12-2023
Publisher: BioMed Central
Published in: Orphanet Journal of Rare Diseases / Issue 1/2023
Electronic ISSN: 1750-1172
DOI: https://doi.org/10.1186/s13023-023-02684-8

Keynote webinar | Spotlight on medication adherence

Springer Medicine

Analysis of Incentive Policies and Initiatives on Orphan Drug Development in China: Challenges, Reforms and Implications

Abstract

Objectives

Methods

Results

Conclusions

Keynote webinar | Spotlight on medication adherence

Springer Medicine

Abstract

Objectives

Methods

Results

Conclusions

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