ArticleOrphanet journal of rare diseases2015
A proposed definition of rare diseases for China: from the perspective of return on investment in new orphan drugs.
Article in Orphanet journal of rare diseases, 2015. 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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Who cites it
14 citing papers in PubMed.
- From presence to provenance: Building substantive infrastructure for China's rare disease ecosystem.Intractable & rare diseases research · 2026Review
- Optimizing regional rare disease management systems within China's policy framework: practical insights from the shantou experience.Orphanet journal of rare diseases · 2026Review
- Health-related quality of life and productivity burden for non-professional caregivers of adults with rare diseases: a real-world study.Orphanet journal of rare diseases · 2025Article
- Shaping national rare diseases definition in Saudi Arabia: outcome from health ecosystem multisectoral workshop.Frontiers in pharmacology · 2025Article
- Long-term distress throughout one's life: health-related quality of life, economic and caregiver burden of patients with neurofibromatosis type 1 in China.Frontiers in public health · 2024Article
- Policy analysis in the field of rare diseases in China: a combined study of content analysis and Bibliometrics analysis.Frontiers in medicine · 2023Article
- Health Policies for Rare Disease Patients: A Scoping Review.International journal of environmental research and public health · 2022Article
- The definition of rare disease in China and its prospects.Intractable & rare diseases research · 2022Article
- Demographics, in-hospital analysis, and prevalence of 33 rare diseases with effective treatment in Shanghai.Orphanet journal of rare diseases · 2021Article
- Epidemiology of benign giant cell tumor of bone in the Chinese population.Journal of bone oncology · 2018Article
- Defining rare diseases in China.Intractable & rare diseases research · 2017Article
- Challenges in orphan drug development and regulatory policy in China.Orphanet journal of rare diseases · 2017Review
- Urine-derived induced pluripotent stem cells as a modeling tool to study rare human diseases.Intractable & rare diseases research · 2016Review
- Using inpatient data to estimate the prevalence of Wegener's granulomatosis in China.Intractable & rare diseases research · 2016Article
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2 authors.
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Abstract
A prevalence threshold to define rare diseases is needed for orphan drug designation. Here, we propose a bottom-up approach to defining rare diseases for China, based on the minimum number of patients needed for the industry to make a reasonable profit on a new drug. To obtain this patient population size, we considered three factors: (1) the industry research and development cost per new drug; (2) the sales per new drug to recoup its research and development costs and generate profit; (3) the highest affordable cost for one patient's treatment in a given healthcare system. Using this model, we estimate that, with the current level of innovation in the pharmaceutical industry in China, between 300,000 and 500,000 patients could be a reference threshold to define rare diseases. Compared with other proposals, this evidence-based definition is more useful for designing rare diseases and orphan drug policies for China.
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