The "medicine basket" for rare disease patients in our country has been comprehensively upgraded. Recently, the National Healthcare Security Administration and other departments released a new version of the National Healthcare Security Drug Catalog, which includes 91 new drugs, including 13 rare disease drugs such as Maverick Capsules used to treat hypertrophic cardiomyopathy. As of now, over 90 rare disease treatment drugs have been included in the national medical insurance drug catalog. Rare diseases, due to their extremely low incidence rate, limited number of patients, and high drug research and development costs, have been "forgotten corners" in the medical field for a long time. Prevention and treatment of rare diseases require care and attention. With the efforts of multiple departments, China is accelerating the construction of a rare disease prevention and control guarantee road, building a bridge of life for patients, and continuously achieving breakthroughs in the accessibility and affordability of medication from 0 to 1. A small pill ignites the hope of patients' lives. In this year's negotiation of the national medical insurance drug catalog, the "life-saving drug" chlorpromazine for tens of thousands of children with refractory epilepsy has been included in medical insurance. The small adjustment in the price of pills by a few cents brings real hope to the families of patients. Refractory epilepsy has the characteristics of "low cure rate, high disability rate, and high mortality rate". According to relevant statistics, the mortality rate of children with refractory status epilepticus in China is over 20%. The family members of the sick child told reporters that when the child fell ill, "sometimes he had convulsions seven or eight times a day, and each seizure felt like he was hovering between life and death. For a long time, due to its dual attributes of rare disease medication and second-class psychotropic drugs, strict control, and limited market size, Chlorpromazine has been unable to be approved for market in China, and patients often choose to purchase "life-saving drugs" from abroad. In 2020, a controversial case involving overseas purchasing of chlorpromazine has attracted widespread attention. Subsequently, multiple families of children with rare epilepsy jointly issued a plea for help, calling for attention to the import and use of chlorpromazine. It is not only medicine, but also people's livelihood. In June 2022, the National Health Commission and the National Medical Products Administration jointly issued the "Work Plan for Temporary Import of Clinically Urgent Drugs" and the "Work Plan for Temporary Import of Chlorpromazine", prioritizing the use of temporary import methods to alleviate the dilemma of "no drugs available" for patients, while supporting the localization of Chlorpromazine drugs. Domestic chlorpromazine will be approved for market in 2022 and enter medical insurance in 2024. This small pill ignites the hope of patients' lives. The level of drug protection for rare diseases is steadily improving. Similarly, medication for patients with paroxysmal nocturnal hemoglobinuria (PNH) is also a focus of attention in this year's adjustment of the national medical insurance drug catalog. PNH, an extremely rare acquired hemolytic disease, is clinically characterized by intravascular hemolysis, bone marrow hematopoietic failure, and recurrent thrombosis, posing a serious threat to the patient's life. In March 2021, a PNH patient experienced a discontinuation of medication and, with a glimmer of hope, sought help from the China Rare Disease Alliance. The China Rare Disease Alliance has joined hands with Peking Union Medical College Hospital, the National Medical Products Administration, and pharmaceutical companies to embark on a compassionate journey of "compassionate drug use". After more than 80 days, the new drug introduced from Switzerland successfully landed at Peking Union Medical College Hospital, and the patient received treatment. The new version of the national medical insurance drug catalog includes this innovative drug, Ipropan Hydrochloride Capsules, which will bring hope to more patients. In recent years, drugs for the treatment of rare diseases such as spinal muscular atrophy (SMA), Gaucher's disease, and myasthenia gravis have been included in the list... Since the establishment of the National Medical Insurance Administration in 2018, the list of medical insurance drugs has been adjusted for seven consecutive years. At present, more than 90 rare disease drugs have been included in the national medical insurance drug catalog, and the level of rare disease drug protection has steadily improved. Along the way, it has been both effort and hope. The adjustment of the medical insurance drug catalog is the country's respect and care for every life, and special care for special groups Li Linkang, Executive Director of the China Rare Disease Alliance, said that every adjustment and increase in the "medicine basket" for rare disease patients is not easy. Strengthening drug research and development to make patients more "medically reliable". From "no drugs available" to "drugs available", and then to "affordable drugs"... Placing the protection of people's health in a strategic position of priority development, China actively explores new models of rare disease medication, strengthens drug research and development, and makes patients more "medically reliable". The pain and helplessness of rare disease patients always hold the hearts of doctors. Bringing more good and life-saving drugs into medical insurance is the common aspiration of doctors and patients Zhang Shuyang, Chairman of the Rare Diseases Branch of the Chinese Medical Association and President of Peking Union Medical College Hospital, said that on this challenging and hopeful path, we work hand in hand to light the beacon of life for patients. The level of civilization in a society involves both how to treat the 'majority' and how to care for the 'very few'. The power of providing better protection for rare disease patients is gathering into a warm current, infiltrating every heart in need of care. (New Society)