ArticleOrphanet journal of rare diseases2023
Analysis of Incentive Policies and Initiatives on Orphan Drug Development in China: Challenges, Reforms and Implications.
Article in Orphanet journal of rare diseases, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.
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Who cites it
17 citing papers in PubMed.
- The impact of China's drug regulatory reform on access to orphan drugs: a cross-sectional study of diseases listed in the rare disease catalog.Frontiers in pharmacology · 2026Article
- Research on the innovation of medical expenses protection mechanism for rare disease patients in China.Frontiers in public health · 2026Article
- Trends, lag and characteristics of rare disease drug approval in the USA and China, 1983-2022.Orphanet journal of rare diseases · 2025Article
- Advances in Cell and Gene Therapy for Rare Disease Treatment.International journal of stem cells · 2025Review
- Capability wellbeing of parents caring for children with rare diseases: a national cross-sectional study.BMC public health · 2025Article
- Multi-criteria decision analysis of value evaluation of rare disease medical insurance drugs in China.BMC public health · 2025Article
- Insights into Support Systems for Orphan Drug Development: A Comparative Study.Therapeutic innovation & regulatory science · 2025Review
- Digital health technology use in clinical trials of rare diseases: a systematic review.Communications medicine · 2025Article
- Reforming China's Rare Disease Security System: Risk Management Perspectives and a Dedicated Insurance Innovation.Healthcare (Basel, Switzerland) · 2025Article
- Changes in Clinical Trials of Dermatological Drugs in Mainland China Between 2016 and 2022: A Narrative Review.Therapeutic innovation & regulatory science · 2025Review
- Regulatory efforts to address the access gap for foreign new drugs in China: the priority review program and related policies.Global health research and policy · 2025Article
- How does a preference-based generic health-related quality of life measure perform in patients with a rare disease? Measurement properties of the EQ-5D-Y proxy version among underage patients with neurofibromatosis type 1.Orphanet journal of rare diseases · 2025Article
- Specific drugs for rare diseases in a province of eastern China under catalog management: from 2021 to 2023.Frontiers in pharmacology · 2025Article
- Drug lag and associated factors of orphan drugs approved by the U.S. in China.Frontiers in pharmacology · 2025Article
- Technological and industrial trends in China's pharmaceutical sector.Frontiers in pharmacology · 2025Review
- Promoting the development and approval of new traditional Chinese medicines in China: a pooled analysis of data from 2013 to 2024.Frontiers in medicine · 2025Article
- Patterns of the Health and Economic Burden of 33 Rare Diseases in China: Nationwide Web-Based Study.JMIR public health and surveillance · 2024Article
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Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectivesRare 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.
methodsPolicies 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.
resultsA 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.
conclusionsOrphan 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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