Since the enactment of China's first “Drug Administration Law of the People's Republic of China” in 1984, China has entered a phase of legal and standardized drug management. The drug regulatory authorities have continuously strengthened the construction of the legal and regulatory framework for drug supervision and have optimized the drug review and approval system. The accessibility of drugs for rare diseases is a significant challenge worldwide. To meet the medication needs of patients with rare diseases, China has introduced a series of incentive policies to fully support the development of drugs for rare diseases and accelerate their review and approval. This paper systematically reviews the historical evolution, development process, and reform achievements of China's rare disease drug review and approval system, and looks ahead to the future, aiming to further optimize the system to better meet the medication needs of patients.