Analysis of the dosage forms difference of orphan drugs in China and abroad in the First List of Rare Diseases
Objective To compare the difference between orphan drugs included in the First Rare Disease Catalogue in the U.S.FDA(Food&Drug Administration,FDA)and NMPA(Natimal Medical Products Ad-ministration).It provides a reference for the management of drug therapy for rare diseases and the research and development of new dosage forms.Methods The varieties of orphan drugs listed in the U.S.FDA and approved in the domestic NMPA for diseases included in the First List of Rare Diseases were included.Com-pared with the U.S.FDA and China's NMPA,there are differences in dosage forms with generic drug-listed varieties.The orphan drugs without relevant dosage forms in the U.S.FDA orphan drug-qualification and approval time,approval indication and usage,as well as the approval status in other countries and the clini-cal trials in China were analyzed.Results As of September 2022,among the diseases in the First List of Rare Diseases,308(43.5%,308/608)orphan drugs-listed by the U.S.FDA have been approved in China with the same generic name,of which 55(17.8%)have dosage forms different from the U.S.FDA,and 8 drugs(11 dosage forms)are approved by the U.S.FDA for the treatment of diseases in the First List of Rare Diseases,involving 6 rare diseases.Conclusion Compared with foreign countries,there is still a phenome-non that"drugs canbe obtained,but the relevant dosage forms can not meet the clinical needs"in China,and further attention should be paid to whether different dosage forms of the same generic name drugs can meet the treatment needs of rare diseases.
NETL
NSTL
万方数据
