Rare disease patients protection: theoretical discussion and institutional construction of orphan drug designation in China.
China's orphan drug reforms could expand access for millions of rare disease patients who currently lack treatment options and regulatory pathways.
This policy paper in Orphanet Journal of Rare Diseases examines the theoretical basis and institutional structure of orphan drug designation in China. It proposes regulatory reforms to better protect the approximately 20 million rare disease patients in China who face high unmet medical need.
What the study was
- Study design
- Policy analysis / review
- Population
- Rare disease patients in China; policy context
- Category
- Public Health
- Maturity
- Exploratory
- Journal
- Orphanet Journal of Rare Diseases
Why it surfaced
Policy analysis relevant to rare disease access in a high-burden setting (China); important for rare disease pipeline at population scale.
A plain-language summary of published research — not medical advice. Talk to a clinician about your own care.