Preliminary study on the global funding pattern of rare diseases / 国际药学研究杂志

ABSTRACT

Rare diseases are mostly chronic and serious diseases, which can cause multi-organ dysfunction, and generally lack effective drugs for treatment. Basic research on rare diseases and drug development of orphan drugs have received widespread attention worldwide. In 2018, ”China's First List of Rare Diseases”was officially released, and a to-tal of 121 diseases were included, achieving a historic breakthrough in the support of rare diseases and greatly promoting the development of researches on rare diseases in our country. However, China has not yet defined rare diseases, and there is no perfect research funding system for basic research on rare diseases. To this end, this paper compares the fund-ing structure of basic research projects for rare diseases in China and the world. In addition, drawing on the experience of global rare disease funding, we put forward the policy recommendations for the financing of rare diseases in China, in or-der to provide reference for the Chinese government to introduce research projects for the development of rare diseases, and to optimize the funding system.