ReviewIntractable & rare diseases research2019
Accessibility of drugs for rare diseases in China: Policies and current situation.
Review in Intractable & rare diseases research, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers.
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Who cites it
19 citing papers in PubMed.
- Policy lessons regarding medical security for rare diseases inChina: Insights from Zhejiang Province through the multiplestreams framework.Intractable & rare diseases research · 2026Review
- Patent Analysis Perspectives on China's Orphan Drug R&D: Status and Technology Characteristics.Therapeutic innovation & regulatory science · 2026Article
- Optimizing regional rare disease management systems within China's policy framework: practical insights from the shantou experience.Orphanet journal of rare diseases · 2026Review
- Evolution and synergy of healthcare security policies for rare diseases in China: a quantitative content analysis.Frontiers in public health · 2026Article
- Characteristics of rare diseases cases: A summary analysis of hospitalized patients at a hospital in Western China from 2015 to 2023.Intractable & rare diseases research · 2025Article
- Specific drugs for rare diseases in a province of eastern China under catalog management: from 2021 to 2023.Frontiers in pharmacology · 2025Article
- Budget impact analysis of high-priced orphan medicinal products intended for the treatment of rare diseases in China: evidence from a densely populated metropolis of Chengdu.BMC health services research · 2024Article
- Analysis of affordability differences for rare diseases in China: a comparison across disease types and regions.International journal for equity in health · 2024Article
- Article
- Genomic Sequencing as a First-Tier Screening Test and Outcomes of Newborn Screening.JAMA network open · 2023Article
- The benefits of using atypical presentations and rare diseases in problem-based learning in undergraduate medical education.BMC medical education · 2023Article
- Literature Review on Health Emigration in Rare Diseases-A Machine Learning Perspective.International journal of environmental research and public health · 2023Review
- Policy analysis in the field of rare diseases in China: a combined study of content analysis and Bibliometrics analysis.Frontiers in medicine · 2023Article
- Bridging the new drug access gap between China and the United States and its related policies.Frontiers in pharmacology · 2023Article
- A Cross-Sectional Study of Price and Affordability of Drugs for Rare Diseases in Shandong Province, China.International journal of environmental research and public health · 2022Article
- Economic burden and health related quality of life of ultra-rare Gaucher disease in China.Orphanet journal of rare diseases · 2021Article
- Rare disease awareness and perspectives of physicians in China: a questionnaire-based study.Orphanet journal of rare diseases · 2021Article
- Why is misdiagnosis more likely among some people with rare diseases than others? Insights from a population-based cross-sectional study in China.Orphanet journal of rare diseases · 2020Article
- Marketing of drugs for rare diseases is speeding up in China: Looking at the example of drugs for mucopolysaccharidosis.Intractable & rare diseases research · 2019Review
Corrections and comments
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Authors and funding
7 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
"Poor accessibility to drugs" is the most problematic issue for patients with rare diseases in China. In recent years, China has issued a number of policies, such as prioritizing speeding up the evaluation for rare disease drugs, publishing national rare disease lists and giving priority to treatments for severe diseases like rare diseases during annual adjustments of National Medical Insurance Medicine Catalogue to improve the accessibility of rare disease drugs. From the outcome perspective, the evaluation of rare disease drugs takes 3 months shorter than ordinary drugs, basic research projects have been started and the number of rare disease drugs included in National Medical Insurance Medicine Catalogue has increased to 50. However, the policies' effects on new drug research and development, rare disease diagnosis and treatment as well as drug pricing are limited. It is recommended to learn the tilt policy of research and development for rare disease drugs from foreign countries and the mechanism of medical insurance funding and patient co-payments. Thus it is important to improve the availability, accessibility and affordability of rare diseases drugs based on the Chinese context.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.