ReviewOrphanet journal of rare diseases2017
Challenges in orphan drug development and regulatory policy in China.
Review in Orphanet journal of rare diseases, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
10 citing papers in PubMed.
- Analysis on the Marketing Trend and Approval Lag of Imported Orphan Drugs from 2010 to 2021 in China.Therapeutic innovation & regulatory science · 2023Article
- Analysis of Incentive Policies and Initiatives on Orphan Drug Development in China: Challenges, Reforms and Implications.Orphanet journal of rare diseases · 2023Article
- Is sub-national healthcare social protection sufficient for protecting rare disease patients? the case of China.Frontiers in public health · 2023Review
- Long way to go: Progress of orphan drug accessibility in China from 2017 to 2022.Frontiers in pharmacology · 2023Article
- Pharmaceutical policy and innovation for rare diseases: A narrative review.F1000Research · 2023Review
- Financing of Rare Diseases and Orphan Drugs in A Sanctioned Country: A Qualitative Study.Medical journal of the Islamic Republic of Iran · 2022Article
- Economic burden and health related quality of life of ultra-rare Gaucher disease in China.Orphanet journal of rare diseases · 2021Article
- Article
- Innovative highlights of clinical drug trial design.Translational research : the journal of laboratory and clinical medicine · 2020Review
- Trends of Clinical Trials for Drug Development in Rare Diseases.Current clinical pharmacology · 2018Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
While regulatory policy is well defined for orphan drug development in the United States and Europe, rare disease policy in China is still evolving. Many Chinese patients currently pay out of pocket for international treatments that are not yet approved in China. The lack of a clear definition and therefore regulatory approval process for rare diseases has, until now, de-incentivized pharmaceutical companies to pursue rare disease drug development in China. In turn, many grassroots movements have begun to support rare disease patients and facilitate drug discovery through research. Recently, the Chinese FDA set new regulatory guidelines for drugs being developed in China, including an expedited review process for life-saving treatments. In this review, we discuss the effects of these new policy changes on and suggest potential solutions to innovate orphan drug development in China.
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Registered trials
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.