ArticleOrphanet journal of rare diseases2020
The urgent need to empower rare disease organizations in China: an interview-based study.
Article in Orphanet journal of rare diseases, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.
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14 citing papers in PubMed, 29 citations in OpenAlex.
- Optimizing regional rare disease management systems within China's policy framework: practical insights from the shantou experience.Orphanet journal of rare diseases · 2026Review
- How to evaluate rare disease policy effectiveness based on policy modeling consistency (PMC) index model: A quantitative assessment of policy implementation in China.Intractable & rare diseases research · 2026Article
- From co-creation to compounding value: A new model of rare disease science communication in China.Intractable & rare diseases research · 2026Article
- "We are ambassadors, we are advocates": rare disease patient advocacy groups as knowledge brokers across health and social systems-a qualitative study from Poland.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
- Reforming China's Rare Disease Security System: Risk Management Perspectives and a Dedicated Insurance Innovation.Healthcare (Basel, Switzerland) · 2025Article
- How do patients and other members of the public engage with the orphan drug development? A narrative qualitative synthesis.Orphanet journal of rare diseases · 2023Review
- 'Advocacy groups are the connectors': Experiences and contributions of rare disease patient organization leaders in advanced neurotherapeutics.Health expectations : an international journal of public participation in health care and health policy · 2022Article
- Evaluating the national system for rare diseases in China from the point of drug access: progress and challenges.Orphanet journal of rare diseases · 2022Article
- The involvement of rare disease patient organisations in therapeutic innovation across rare paediatric neurological conditions: a narrative review.Orphanet journal of rare diseases · 2022Review
- Treating rare diseases with the cinema: Can popular movies enhance public understanding of rare diseases?Orphanet journal of rare diseases · 2022Article
- Physicians' knowledge on specific rare diseases and its associated factors: a national cross-sectional study from China.Orphanet journal of rare diseases · 2022Article
- A questionnaire-based study to comprehensively assess the status quo of rare disease patients and care-givers 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
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Authors and funding
8 authors at 7 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundEach rare disease only affects a small number of population. However, a total of 7000 rare diseases may affect 10% of the population. Due to the severity and lack of rare disease awareness, rare disease represents a huge challenge for the healthcare system. In Western countries, patient organizations have been playing an integral role in raising awareness, advocating legislation, and supporting drug development. This study aims to assess the unmet needs of rare disease patient organizations in China, and identify their unmet needs, providing essential information for the government and legislators.
resultsA total of 28 individuals representing 28 patient organizations in China were interviewed. Most organizations do not have official registration, employees, written standard operation protocol, or reliable financial resources. Misdiagnosis or delayed diagnosis is common, and treatment is often lacking. Due to the lack of financial resources, no organizations have been able to sponsor academic research, unlike their counterparts in Western countries. As to challenges, 71.4% of interviewees listed lack of rare disease awareness among the general public, while 67.9% selected lack of financial resources. Further, only 7.3% of these organizations received support from the government, and 28.6% received support from the general public. As to recommendations to the government, 82.1% of interviewees selected special insurance programs for rare diseases because rare diseases have been generally excluded from the national medical insurance programs. In addition, 78.6% of interviewees recommended to stimulate rare disease research, 75% recommended to import orphan drugs, and 71.4% recommended legislation of an orphan drug act, highlighting the urgent need of therapies.
conclusionsDue to lack of support and rare disease awareness, patient organizations in China are still in the early phase. To empower these patient organizations, the interviewees' recommendations, including legislating orphan drug act and releasing official definition of rare diseases, should be considered by the government and legislators.
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