In recent years, China has made significant progress in the field of rare disease management, with a continuously improving policy system and gradually enhancing medication security.
The policy system is constantly improving
In 2024, China will introduce multiple policies related to rare diseases. The Government Work Report proposes to strengthen rare disease research, diagnosis and treatment services, and medication security. The "Key Tasks for Deepening the Reform of the Medical and Health System in 2024" issued by the General Office of the State Council will accelerate the review and approval of rare disease treatment drugs as a key task. The "Guidelines for the Diagnosis and Treatment of Rare Diseases (2024 Edition)" have been officially released, providing important references for clinical diagnosis and treatment.
The National Medical Products Administration will continue to increase policy support for the research and development, production, and other aspects of rare disease drugs, issue relevant guidance principles and plans, and promote the research and marketing of rare disease drugs.
The medication guarantee is gradually improving
The National Healthcare Security Administration has established a dynamic adjustment mechanism for the medical insurance drug catalog, shortening the time for new drugs to be included in the medical insurance drug catalog. In 2024, 13 rare disease drugs will be added to the medical insurance drug catalog, covering a variety of diseases. As of the end of 2024, 126 rare disease drugs have been included in medical insurance, involving 68 rare diseases.
Local governments are also exploring mechanisms for ensuring medication for rare diseases. As announced by Anhui Province, the cost of some rare disease drugs in the national rare disease catalog will be paid separately by the major illness insurance fund, and policy preferences will be given to extremely poor individuals, low-income recipients, etc.
Progress has been made in drug research and development
With the introduction of encouragement policies, China has made good progress in the research and development of rare disease drugs. In 2024, there will be 47 rare disease drugs in the market application stage in China, involving 21 rare diseases. Domestic pharmaceutical companies are increasing their investment in the research and development of drugs for rare diseases, while multinational pharmaceutical companies are also continuing to pay attention to the field of rare diseases.
Public welfare organizations assist in ensuring medication security
China's public welfare and charity forces have made a series of efforts to ensure the use of drugs for rare diseases. The Pain Challenge Foundation has launched a rare disease medical assistance project. As of the end of January 2025, a total of 4677 people have been assisted, and over 50.3 million yuan has been disbursed, involving 114 rare disease types.
Although China has made significant progress in rare disease management, it still faces some challenges. In the future, with the improvement of policy and regulatory systems, the advancement of innovative therapies, and the deepening exploration of multi-level payment guarantee systems, the field of rare diseases is expected to achieve breakthroughs in policy coordination, technological innovation, and patient empowerment, further improving the accessibility and affordability of rare disease drugs and meeting the multi-level needs of patients.
