Abstract: Objective To analyze the general situation of incentive policies on rare disease drugs research and development in the United States, so as to provide reference for the establishment of policies on rare disease drugs research and development in China. Methods By comparing the differences between China and the United States in terms of rare diseases legislation, official definition of rare diseases, and incentive measures on rare disease drugs research and development, the inadequacies of incentive policies on rare disease drugs research and development in China were pointed out, and specific suggestions were putted forward. Results The United States was the first in the world to promote development of rare disease drugs through legislation, and has a clear official definition of rare diseases. In the United States, incentive policies and measures, such as funding research and development projects and speeding up the examination and approval process, have greatly increased the number of rare diseases drug and improved medication status of rare diseases patients. Although China has carried on the exploration to policies on rare disease drugs research and development, but there is no official definition of rare disease. There is lack of incentives to develop rare disease drugs, accessibility of rare disease drugs needs to be further improved. Conclusion China should learn from foreign advanced experience, establish management incentive policies on rare disease drugs research and development, so as to protect legal rights and interests of rare diseases patients.

Key words: rare disease drugs, the United States, drugs research and development, incentive policy