Abstract
The pathogenesis of diseases in the field of rare diseases is complex. Many rare diseases have yet to be conquered worldwide, and there are still no effective treatments for most rare diseases, resulting in limited accessibility to medications. Over the past few years, China has been committed to ensuring the availability of drugs for rare diseases, both at the national level and in all sectors of society. Through National Healthcare Insurance Negotiations (hereinafter referred to as "national negotiations"), the National Healthcare Security Administration has included several drugs for rare diseases in medical insurance coverage, addressing some of the issues with medications for rare diseases. National Negotiations have reduced the prices of drugs for rare diseases to a certain extent, but they remain expensive for many patients. By the end of 2023, out of the 165 drugs for rare diseases that had been launched, 53 were still not covered by medical insurance, leaving patients with a significant financial burden. Addressing payment issues remains a current challenge, and various regions in China are actively exploring innovative methods of paying for rare disease care to protect the socioeconomic rights and interests of patients with rare diseases.