Join the 14th World Congress on Rare Diseases and Orphan Drugs, June 2-3, 2025, in Amsterdam. Explore breakthroughs in research, treatment, and innovation to transform rare disease care globally.
POLICY sciencesRARE diseasesHEALTH care rationingMore than 8% of the global population is affected by a rare disease and >50% affected are children (30% of whom will die before 5 years). There are 6,000-8,000 classified rare diseases - 80% are genetically based - ...
As China continues to expand its policy support for development, registration review and commercializing of orphan drugs, China might become a more desirable market for orphan drugs. [Note] [1] The 2023 China Rare Disease Observation Report issued by Frost & Sullivan on April 27, 2023. [2] I...
however, they shifted to the rare disease specialty institution system, and its number increased to 17. Rare disease specialty institutions focus on providing intensive and specialized care for rare diseases, conducting rare disease treatment and registration statistics, and disease research. This is an...
The Rare Diseases Epidemiology book is a comprehensive text focused on the rare disease field where both general epidemiological methods and specific methods for low prevalence diseases are discussed. This is the first book to address rare diseases as a public health problem where the epidemiological ...
2022 Speakers Wendy Erler VP Global Head Patient Experie... Alexion Rare Disease Debbie Drane Senior Vice President Global C... CSL Behring Alan J Balch CEO Patient Advocate Foundation and National Patient Advocate Foundation Femida Gwadry‐Sridhar ...
Rare disease drugs (orphan drugs) approved by NMPA before June 30, 2022. *The six conditions above only apply to drugs currently outside the NRDL. For NRDL-listed drugs which have major indication changes or seek contract renewal to stay in NR...
“undiagnosed disease” has emerged as a concept and category which helps properly guide the patient's management within the health system and reduce the time to obtain a correct diagnosis. This new series fromOrphanet Journal of Rare Diseaseswill delve into the questions which need to be ...
With these limitations, a clear understanding of the variation in epidemiology and outcomes for rare disease patients is hampered. This challenges the public health of rare diseases patients through a lack of information necessary to prioritize research, policy decisions, therapeutic development, and ...
Finally, due to the lack of data from the pre-implementation period, this study was unable to assess the impacts of the negotiation policy on the utilization and affordability of the rare disease drugs. Conclusion The health insurance reimbursement policy in a tier-one city has been operating ...