CHINAPHARMACEUTICAL policyORPHAN drugsDRUG accessibilityDRUG analysisPOLICY analysisRare diseases have various types, low incidence rates, complex conditions, and are often difficult to diagnose. Due to China's large population, there is a significant number of rare disease patients, but there is a ...
This review of the US Orphan Drug Act (ODA) 1983 outlines how the ODA is intended to stimulate orphan drug research and development of drugs for rare disea
DOI 10.1186/s13023-016-0460, Challenges in orphan drug development and regulatory policy in China. Cheng and Xie Orphanet Journal of Rare Diseases (2017) 12:13. DOI 10.1186/s13023-017-0568-6. Recently, China's FDA has offered to grant conditional approvals for Orphan meds already approved ...
40.Xu K, Coté TR. Database identifies FDA-approved drugs with potential to be repurposed for treatment of orphan diseases. Brief Bioinform 2011;12:341–5.10.1093/bib/bbr006Search in Google ScholarPubMed 41.US Department of Health and Human Services. Rare diseases: common issues in drug dev...
Analysis of Incentive Policies and Initiatives on Orphan Drug Development in China: Challenges, Reforms and Implications Article Open access 27 July 2023 The importance of the policy framework on orphan drug accessibility Article 06 April 2023 Regulatory Considerations Toward Orphan Drug Designation ...
Alpha-1 antitrypsin deficiency (AATD, OMIM #613490) is a rare metabolic disorder affecting lungs and liver. The purpose of this study is to assess the impact of the US orphan drug act on AATD by providing a quantitative clinical-regulatory insight into t
drug use and a resurgence of epidemics in populations that were the first to be heavily impacted, like urban men who have sex with men (MSM) in North America, Europe and Australia. It is estimated that 33 to 45 million people worldwide are infected with HIV, including between 2–3 ...
FDA's Office of Orphan Products Development designates orphan status to drugs and biologics intended for the safe and effective treatment, diagnosis, or prevention of rare diseases/disorders that affect fewer than 200,000 people in the United States. If FDA approves the drug, Merrimack Pharmaceutical...
orphan drug Topics Collection Topic: Newborn Screening I - Real World Applications and Technologies Topic: Newborn Screening II - Policy, Ethics and Patient Perspectives Latest Issue All Issues Volume 3, Issue 4 (in progress) Volume 3, Issue 3 ...
Evaluating the national system for rare diseases in China from the point of drug access: progress and challenges Article Open access 10 September 2022 Background Ten years have passed since Latvia became a member of the European Union (EU) in 2004. Since then the EU laws and regulations, in...