To summarize and analyze what the listing in market of orphan drugs in China, the United States, the European Union, and Japan in 2023, and to provide empirical reference to Chinese pharmaceutical enterprises, pharmaceutical management, and new drug research and development departments.

Collect the 2023 drug market data released by official drug regulatory agencies in four countries and international organization-China, the United States, the European Union, and Japan categorize the market of rare disease drugs and and their indications, approval dates, R&D status in China, component therapeutic areas, and special review and approval pathways were analyzed.

Findings in 2023, 28 orphan drugs were listed in the United States, mostly anti-tumor related, accounting for 32.1% (9 out of 28); 17 orphan drugs were listed in the European Union, anti-tumor related accounting for 47.0% (8 of 17); 22 listed in Japan; and 45 listed in China. In 2023, over 70% orphan drugs listed in the European Union and Japan entered the phase of clinical trials/marketing and applications/approved clinical applications in China (86.4% from Japan and 70.6% from European Union).

Currently, there is no qualification accreditation mechanism for orphan drugs in China. However, in 2023, the number of rare disease drugs listed in China was the greatest among the four countries/international organization. It reflects that China′s Catalog for Rare Diseases played a guiding role in drug research and development. Meanwhile, the reform of drug approval and review in China and the issue of rare disease catalogs took place in a short period of time, so the review and approval, as well as research and development of rare disease drugs are still in the catching up stage.