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    Home > Medical News > Medical World News > Solving the "expensive medication" of rare disease groups Experts call for strengthening top-level design and speeding up independent research and development

    Solving the "expensive medication" of rare disease groups Experts call for strengthening top-level design and speeding up independent research and development

    • Last Update: 2021-08-02
    • Source: Internet
    • Author: User
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    Medical Network, July 19, reported that the difficulty and high cost of medication for patients with rare diseases are common problems faced by countries all over the world
    .
    A multidisciplinary expert seminar on rare diseases focusing on hyperammonemia was held here on the 17th
    .
    Many experts such as Professor Wang Lin, Secretary-General of the Bureau of the China Rare Disease Joint Conference, said that attention to rare diseases should be based on improving the national basic medical security system, safeguarding the interests of patients with rare diseases, and maintaining the concept of social justice for patients with rare diseases, so as to achieve "basic Medical insurance is basic"; at the same time, it is based on the concept of government-led and strengthened national top-level system design; based on the concept of multi-level and multi-disciplinary joint participation and precise policy implementation
    .
    The conference was co-sponsored by the National Joint Conference of Chairpersons of the National Rare Disease Academic Group, the Shanghai Rare Disease Prevention Foundation and the Rare Disease Specialist Branch of the Shanghai Medical Association
    .
    How to solve the predicament of high-value medicinal drug protection for rare diseases, Professor Li Dingguo, chairman of the Shanghai Rare Disease Prevention and Control Foundation, believes that under the premise of encouraging local exploration, we must still adhere to the government as the leading factor and strengthen the top-level design of the system
    .
    Driven by the basic policy of basic medical insurance, all parties should be mobilized to jointly provide medical assistance to patients with rare diseases, so as to realize that patients with rare diseases "have medical care and medical insurance"
    .
    It is understood that, with the huge wave of innovative drug research and development set off around the world, major pharmaceutical companies are optimistic about the uncultivated orphan drug hotspot and are competing to join the fierce competition in the development of drugs for rare diseases
    .
    Professor Li Dingguo pointed out that in the process of seeking development, enterprises must adhere to both economic and social benefits
    .
    Enterprises should reasonably reduce the cost of medication for patients, improve the availability of drugs among patients with rare diseases, and increase independent research and development of drugs for rare diseases
    .
    Professor Wang Lin said that in recent years, the issues of social equity and efficiency behind rare diseases, the development of medical science and the overall planning of pharmaceutical industry innovation, etc.
    , have been increasingly raised to the national level by some countries and regions, and they have begun to formulate national strategies and plans.
    , Bring rare diseases into the scope of national public governance
    .
    It is understood that in China’s 121 rare diseases catalogue, there are still more than 20 rare disease patients who have to take drugs "over-indication"; from the national rare disease system level, there are still many rare disease drugs that have not yet been included in the country.
    In the scope of medical insurance payment, the demand for high-value rare disease drugs with high annual treatment costs has not been met
    .
    At the seminar, many experts believed that: the types and diseases of rare diseases are dynamically changing.
    With the gradual in-depth research on diseases and the development of medical technology, some rare diseases may become common diseases, and there will be new ones every year.
    Types of diseases are included in the category of rare diseases
    .
    During the interview, many experts agreed to establish a government-led multi-party co-payment mechanism for rare disease drugs.
    Companies can cooperate with the government to participate in critical illness insurance and medical assistance; companies can cooperate with the market to participate in commercial health insurance; companies and society Work together and participate in charitable donations
    .
    During the interview, the reporter learned that hyperammonemia is mainly caused by human urea cycle disorders
    .
    Professor Yang Yanling, a pediatrician at Peking University First Hospital , said that increased blood ammonia can cause progressive organ damage, especially brain damage
    .
    Those who do not receive timely treatment for persistent hyperammonia will survive in time and will also cause intellectual disability
    .
    Professor Yang Yanling said that rare diseases caused by urea cycle disorders involve 9 types of diseases, all of which are included in the first batch of rare diseases in the country
    .
    She pointed out that raising the awareness of rare diseases among the Chinese people, increasing the popularization of rare diseases, and adhering to the principles of "early detection, early diagnosis, and early treatment" and "preventable and cureable" prevention and control principles for rare diseases are conducive to promoting China Rare disease prevention and control and guarantee the sustainable development of the cause
    .
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