Pay attention to rarity and light up the light of life. On February 29, 2024, the 17th International Day of Rare Diseases was celebrated. Not long ago, the latest version of the national medical insurance drug catalog adjusted and added 15 rare disease drugs outside the catalog, covering 16 rare disease types. Some rare diseases that have not been effectively solved for a long time, such as Gaucher's disease and myasthenia gravis, are also included in it. So far, over 80 rare disease treatment drugs have been included in the list of national medical insurance drugs. This is expected to bring more blessings to over 20 million rare disease patients in China. The World Health Organization defines rare diseases as diseases or lesions that account for between 0.065% and 0.1% of the total population. Difficulty in diagnosis, medication, and high drug prices are the "three hurdles" that rare disease patients face. For the global challenge of rare disease diagnosis and treatment, the National Health Commission and other departments have joined hands with various sectors of society to care for rare disease patients, continuously exploring "Chinese solutions" for rare disease prevention and treatment, especially in terms of accessibility and affordability of rare disease medication, and striving to make "medical orphans" not lonely. Starting from January 1st this year, patients with atypical hemolytic uremic syndrome have ushered in a turning point in their fate: a life-saving drug called "Icuzumab" that once cost over 20000 yuan to treat the disease has been reimbursed by medical insurance, with each drug priced at about 1000 yuan. Many patients and families have regained hope. In addition to making some rare disease patients "able to afford medication" and solving problems such as difficulty in medication and lack of medication, relevant departments and various sectors of society have also been taking action. Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired hemolytic disease known as a "super rare disease", which causes patients to suffer from complications such as repeated hemolysis, hemoglobinuria, and renal dysfunction. In March 2021, a PNH patient experienced drug discontinuation and, with a glimmer of hope, sought help from the China Rare Disease Alliance. There is no precedent to follow, no detailed rules to follow. The China Rare Disease Alliance, Beijing Union Medical College Hospital, the National Medical Products Administration, and pharmaceutical companies are working together in a team to achieve sympathetic medication. More than 80 days later, the new drug introduced from Switzerland successfully landed at Peking Union Medical College Hospital, and the patient received new life. Not only sympathizing with medication, but also seamlessly connecting relevant departments and medical institutions to prioritize the review and approval of rare disease treatment drugs, opening up a green channel for some rare diseases that are in urgent need of drugs that have been cut off. More and more "orphan drugs" such as Mitotan, Laronidase, and Chlorobenzoic Acid are being introduced to solve the urgent needs of rare disease patients. With the support of relevant departments, some enterprises have established rare disease patient care centers to provide one-stop professional pharmaceutical services for rare disease patients, including drug supply guarantee, health management and medication consultation, charitable drug donations, medical insurance settlement, and other full process pharmaceutical services. "Making medication available for rare disease patients has shifted from consensus to intensive action," said Li Linkang, Executive Director of the China Rare Disease Alliance. In order to strengthen the research and development of rare disease drugs, a series of documents such as the "Guidelines for the Development of the Pharmaceutical Industry" and the "14th Five Year Plan for the Development of the Pharmaceutical Industry" have been intensively issued, guiding enterprises to strengthen the research and development of special drugs for treating rare diseases; Medical and research institutions such as Beijing Children's Hospital have made corresponding arrangements for the research and evaluation of rare disease drugs... taking small steps without stopping! Medication