How to solve the difficulty of medication for patients with rare diseases? Experts call for the establishment of a national special fund


Rare diseases, also known as orphan diseases, are generally chronic and serious diseases that often endanger life. Some of these diseases are called "ultra rare diseases" due to their extremely small number of patients. How to improve the medication guarantee mechanism for rare diseases is a topic of concern for all parties. February 29th of this year is the 17th International Day for Rare Diseases, and many experts called for exploration at the national level to establish and improve a diversified protection system for rare diseases during interviews. They hope to establish a national special fund for rare disease medication to fill the gaps in rare disease protection. In fact, in recent years, many regions have started exploring ways to build a diversified guarantee system for rare diseases, attempting to solve medication difficulties for patients with rare diseases, especially those with ultra rare diseases. For example, Jiangsu Province and Zhejiang Province have respectively established provincial-level special funds for rare diseases led by the government to provide medication support for rare disease patients who have not been included in basic medical insurance; Shanghai and Guangzhou have significantly reduced the economic burden of medication for patients by incorporating medication for extremely rare diseases into the coverage of local urban universal insurance. Taking Shanghai as an example, in April 2021, the "Shanghai Huibao" was released, which included some extremely rare diseases in the specific high-value drug protection catalog, so that the reimbursement rate for medication in this group of extremely rare patients reached 70%. Professor Sun Jie, Vice Dean of the School of Insurance at the University of International Business and Economics, praised local exploration in an online interview. She said, "Whether it is the 'Jiangsu Zhejiang model' or the 'Shanghai Guangzhou model', both are beneficial explorations to solve the problem of high value rare disease medication guarantee through innovative and multi-level guarantee models. From the current perspective, these models benefit patients, are stable and sustainable, and have a positive impact on promoting the exploration and innovation of rare disease guarantee mechanisms." However, this scholar believes that "in the long run, we will explore the establishment of a special fund for rare diseases at the national level, which will be coordinated and dedicated by the state, to fill the gaps in rare disease protection in one fell swoop, so that patients can take medication within reach." Chen Wei, Vice Chairman of the Quzhou Municipal Committee of the 93 Society and Vice President of Quzhou Traditional Chinese Medicine Hospital, expressed the same hope: by establishing the central "Charitable Medical Assistance Special Fund for Rare Diseases", Help rare disease patients solve the cost burden of high medication costs. Chen Wei believes that "special funds can be tailored to the characteristics of long treatment cycles and heavy economic burden of rare diseases, with stable funding sources and clear funding standards to support specific patients, so that patients who truly need protection can receive long-term and stable assistance." It is reported that in September 2023, the "Second Batch of Rare Disease Catalogue" was released, which includes 86 rare diseases, Increase the number of rare diseases listed in China to 207. The new version of the medical insurance catalog released in December of that year included 15 rare disease medications. The continuous favorable policies have ignited hope for the lives of many rare disease patients and families. In consecutive interviews, the reporter learned that there are still many rare disease patients waiting in the "difficulty of medication". Especially for some "extremely rare diseases" patients, the number of confirmed cases in China is currently only over a thousand. The life-saving drugs they hope for are difficult to develop, have long cycles, high investment, and higher costs, making many patients unable to afford continuous and standardized treatment. For example, Pompeii disease is an extremely rare disease. Seven years ago, China

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