Trends, lag and characteristics of orphan drug approvals in the USA and China, 1983-2022

Abstract

Abstract Background Over the past approximately 40 years, Chinese drug regulations have undergone many major reforms to accelerate the approval of drugs and keep pace with the scientific innovation of drugs in the world, especially developed countries. In 2018, China’s National Health Commission, the Ministry of Science and Technology, and the Ministry of Industry and Information Technology jointly released China's "First Batch of Rare Diseases Catalogue". However, there is currently less relevant research on the overview and speed of the approval of rare disease drugs (orphan drugs) in China. Methods This mixed-methods study comprised a systematic review and cross-sectional analysis of orphan drugs approved in China and the USA from 1983 to 2022 through official drug search databases and systematically analyzed and compared orphan drugs approved in the USA and China, including the number, marketing time, chemical types, ingredient classes, and special approval pathways. Results During 1983–2022, the US Food and Drug Administration (FDA) marketed a total of 693 orphan drugs (including 1,228 dosage forms/specifications) and a total of 201 (29.00%, 201/693) orphan drugs (including 425 (34.58%, 425/1228) dosage forms/specifications) marketed by the National Medical Products Administration (NMPA) in China. The number of China's orphan drugs on the market is increasing year by year, and the approval speed has gradually accelerated. The average lag time for orphan drug marketing by the NMPA compared to the FDA has decreased from 28.81 years (1983–1987) to 1.66 years (2018–2022), showing a progressive downward trend (down by 94.24%). The time lag between FDA orphan drug marketing and designation is becoming increasingly shorter, especially after 2006. The special procedures for drug marketing have a great promoting effect on orphan drug approval. Conclusion This research provides evidence of breakthroughs in the review and approval of orphan drugs in China and demonstrates the tremendous boost to orphan drugs from China's ongoing restructuring and reform of the drug regulatory ecosystem, as well as a stimulus for future orphan drug development in China and the world.