Postmarketing safety of orphan drugs: a longitudinal analysis of the US Food and Drug Administration database between 1999 and 2018

Orphanet Journal of Rare Diseases

Vincent K. C. Yan¹  Eliza Y. T. Tam¹  Min Fan¹  Lauren K. W. Lau¹  Eric Y. F. Wan²  X. Li³  Adrienne Y. L. Chan⁴  Ian C. K. Wong⁵  Xinning Tong⁶  Patrick Ip⁷  Terry Y. Lum⁸ 

[1] Centre for Safe Medication Practice and Research, Department of Pharmacology and Pharmacy, Li Ka Shing Faculty of Medicine, The University of Hong Kong, L2-59, 2/F, Laboratory Block, Faculty of Medicine Building, 21 Sassoon Road, Pokfulam, Hong Kong;Centre for Safe Medication Practice and Research, Department of Pharmacology and Pharmacy, Li Ka Shing Faculty of Medicine, The University of Hong Kong, L2-59, 2/F, Laboratory Block, Faculty of Medicine Building, 21 Sassoon Road, Pokfulam, Hong Kong;Department of Family Medicine and Primary Care, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Pokfulam, Hong Kong;Centre for Safe Medication Practice and Research, Department of Pharmacology and Pharmacy, Li Ka Shing Faculty of Medicine, The University of Hong Kong, L2-59, 2/F, Laboratory Block, Faculty of Medicine Building, 21 Sassoon Road, Pokfulam, Hong Kong;Laboratory of Data Discovery for Health (D24H), Hong Kong Science Park, Sha Tin, Hong Kong;Department of Medicine, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Pokfulam, Hong Kong;Centre for Safe Medication Practice and Research, Department of Pharmacology and Pharmacy, Li Ka Shing Faculty of Medicine, The University of Hong Kong, L2-59, 2/F, Laboratory Block, Faculty of Medicine Building, 21 Sassoon Road, Pokfulam, Hong Kong;Laboratory of Data Discovery for Health (D24H), Hong Kong Science Park, Sha Tin, Hong Kong;Groningen Research Institute of Pharmacy, Unit of PharmacoTherapy, -Epidemiology and -Economics, University of Groningen, Groningen, The Netherlands;Centre for Safe Medication Practice and Research, Department of Pharmacology and Pharmacy, Li Ka Shing Faculty of Medicine, The University of Hong Kong, L2-59, 2/F, Laboratory Block, Faculty of Medicine Building, 21 Sassoon Road, Pokfulam, Hong Kong;Laboratory of Data Discovery for Health (D24H), Hong Kong Science Park, Sha Tin, Hong Kong;Research Department of Practice and Policy, UCL School of Pharmacy, London, UK;Department of Medicine, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Pokfulam, Hong Kong;Department of Paediatrics and Adolescent Medicine, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Pokfulam, Hong Kong;Department of Social Work and Social Administration, Faculty of Social Sciences, The University of Hong Kong, Pokfulam, Hong Kong;

关键词: Orphan drug designation;    Adverse events;    Postmarketing surveillance;    Medication safety;    Risk–benefit trade-off;   

DOI  :  10.1186/s13023-021-02166-9

来源: Springer

【 授权许可】

CC BY