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    Home > Active Ingredient News > Drugs Articles > The dilemma of high-value rare disease medication | the first scene

    The dilemma of high-value rare disease medication | the first scene

    • Last Update: 2022-10-14
    • Source: Internet
    • Author: User
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    High-value rare disease drugs are a pain in the hearts of rare disease patients and their families, and their accessibility is an urgent problem
    to be solved.
    At present, there are more than 80 kinds of drugs listed in China and clearly indicated for rare disease indications, of which 58 drugs have been included in the national medical insurance list, covering 28 rare diseases, and some higher-priced rare disease drugs have not yet been included
    .

    High-value rare disease drugs are a pain in the hearts of rare disease patients and their families, and their accessibility is an urgent problem
    to be solved.
    At present, there are more than 80 kinds of drugs listed in China and clearly indicated for rare disease indications, of which 58 drugs have been included in the national medical insurance list, covering 28 rare diseases, and some higher-priced rare disease drugs have not yet been included
    .

    Obviously, health insurance is not the only hope
    .
    At the 11th China Rare Disease Summit Forum, participants from different fields such as governments, pharmaceutical companies, and patient organizations discussed high-value rare disease drugs, and put forward suggestions
    such as setting up a volume-price linkage agreement and a efficacy-linked agreement, improving the rare disease database, setting up a special fund, and cooperating with Huimin Insurance.

    Obviously, health insurance is not the only hope
    .
    At the 11th China Rare Disease Summit Forum, participants from different fields such as governments, pharmaceutical companies, and patient organizations discussed high-value rare disease drugs, and put forward suggestions
    such as setting up a volume-price linkage agreement and a efficacy-linked agreement, improving the rare disease database, setting up a special fund, and cooperating with Huimin Insurance.

    How to get closer to the country?

    How to get closer to the country?

    The "soul bargaining" in last year's health insurance negotiations is still fresh in everyone's memory, and the phrase "every small group should not be abandoned" has inspired and touched many people
    .

    In fact, in recent years, the country has also paid more and more attention to rare diseases, almost every year the medical insurance catalogue update has rare disease drugs transferred, last year was included in two high-value drugs, namely nosinacon sodium injection for the treatment of spinal muscle atrophy (SMA) and Agartaxase α injection solution
    for the treatment of Fa Bray disease.
    Among them, the annual treatment costs of these two high-value drugs do not exceed the payment "ceiling" of the medical insurance fund of 300,000 yuan
    .

    It can be said that the medical insurance party is still very cautious about drugs that cost more than 300,000 yuan / year
    .
    How can we get closer to the country?

    In this regard, Jin Chunlin, director of the Shanghai Municipal Health and Health Development Research Center, believes that the biggest concern of the medical insurance side about the protection of rare diseases stems from two uncertainties
    .
    "First, the number of patients is uncertain, if the number of patients grows explosively after the inclusion of high-value drugs, which will bring a huge impact on the medical insurance fund? The second is uncertain efficacy, the approval speed of many rare disease drugs is very fast, and due to the low incidence, the number of subjects in the clinical stage is also very small, and it is uncertain whether the efficacy is good or not
    .

    Jin Chunlin proposed some solutions to the above uncertainties
    .
    For example, "when medical insurance negotiates with enterprises, a volume-price linkage agreement and a efficacy-linked agreement
    can be set.
    " "The volume-price linkage agreement stipulates the upper limit of the number of people insured by the medical insurance party according to the number of potential patients calculated epidemiological calculation in advance, and the drug costs arising from the excess number of people shall be shared by the enterprise; The efficacy linkage agreement can stipulate that after the completion of the drug cycle, the cured or patients with a large degree of improvement will be paid by medical insurance, and those who have no efficacy or poor efficacy will be borne
    by the enterprise.

    Huang Rufang, Director of Cord Rare Disease Center (first from left), moderated the discussion on the deep meaning behind the "soul bargaining" of the national talks, and the panelists were: Ms.
    Yu Lei, Head of Rare Diseases and Rare Blood Diseases Business of Sanofi (China) (second from left), Ms.
    Zhang Min, Vice President of Takeda China and Head of Access Excellence Department (middle), Mr.
    Xu Yifan, Vice President of Fabre Patients Association and Director of Fabre International Collaboration Network (second from right), and Professor Jin Chunlin, Director of Shanghai Health and Health Development Research Center (first from right).

    Huang Rufang, Director of Cord Rare Disease Center (first from left), moderated the discussion on the deep meaning behind the "soul bargaining" of the national talks, and the panelists were: Ms.
    Yu Lei, Head of Rare Diseases and Rare Blood Diseases Business of Sanofi (China) (second from left), Ms.
    Zhang Min, Vice President of Takeda China and Head of Access Excellence Department (middle), Mr.
    Xu Yifan, Vice President of Fabre Patients Association and Director of Fabre International Collaboration Network (second from right), and Professor Jin Chunlin, Director of Shanghai Health and Health Development Research Center (first from right).

    Professor Wu Jing, Vice Dean of the School of Pharmaceutical Science and Technology of Tianjin University, participated in the discussion
    online.

    Professor Wu Jing, Vice Dean of the School of Pharmaceutical Science and Technology of Tianjin University, participated in the discussion
    online.

    Professor Wu Jing, deputy dean of the School of Pharmaceutical Science and Technology of Tianjin University, said that whether it is a form review before the negotiation or a negotiated reserve price calculation, experts usually take a high look at the drugs for rare diseases, and when considering the economy, they will give special thresholds, but it is still difficult to break through the "red line", and it may still be necessary to hope for a multi-level guarantee system and innovative payment
    .

    In the construction of a multi-level security system, the highest voice in the industry is to let basic medical insurance introduce policies first, and other channels will relay, that is, the "1 + N" guarantee model, whether the model can be implemented is still being discussed
    .
    The biggest problem with innovative payment at present is the lack of a sound database, whether it is to establish a volume-price-linked agreement or to establish a curative effect-based agreement, which requires standard, perfect and standardized data
    .
    This database should include real-time follow-up of medications, so that the health insurance party can observe the number of patients and the improvement of various indicators of patients at any time
    .

    However, whether it is the improvement of the multi-level security system or the construction of the database of rare disease patients, it cannot be achieved
    overnight.
    To solve the urgent need, we also need to make full use of the "sharp weapons"
    that we currently have in our hands.
    Commercial health insurance in the form of Huimin Insurance, special funds for rare disease protection explored and set up in some provinces, and donations and assistance from civil affairs departments and charitable organizations can all contribute to the protection of rare diseases
    .

    What efforts can local health insurance make?

    What efforts can local health insurance make?

    Zhejiang Province began to implement a special fund for rare diseases in 2020, and each co-ordination district will hand over to the Zhejiang Provincial Rare Disease Drug Guarantee Fund from the Serious Illness Insurance Fund of the co-ordination area according to the number of basic medical insurance participants at the end of last year according to the number of basic medical insurance participants at the end of last year
    .
    Based on the number of 54.
    61 million basic medical insurance participants in Zhejiang Province, the annual available budget of the special fund for rare disease drug protection is 109 million yuan, and it has now included two rare disease specific drugs - emidase for the treatment of Geshe disease and salproterin for the treatment of phenylketonuria
    .

    Jiangsu Province also set up a special fund for rare disease assistance in February this year, which included 6 drugs involving five rare diseases, namely Gosher disease, Pompeii's disease, Fabre disease, spinal muscular atrophy and mucopolysaccharide storage disease type IVA, into the scope of the first batch of
    rare disease drug protection.
    For those who meet the policy conditions and are treated in designated medical institutions, the designated drug costs incurred within one settlement year shall be paid
    proportionally by the Rare Disease Drug Guarantee Fund.

    The special funds for rare diseases in these two provinces have balances in operation, and there is no situation in which the number of patients has surged and brought operational risks to the
    funds.

    Another important "weapon" is to benefit the people
    .
    As one of the most widely popular commercial health insurance insurance in all parts of the country, Huimin Insurance has several characteristics - it has the guidance and endorsement of local governments, the conditions for insurance are relaxed, and the price is "inclusive"
    .
    At present, there are about 200 Huimin insurance products in the country, of which about 50 to 60 Huimin insurance products have included
    rare diseases.

    Jiangsu Province stipulates that all varieties in the provincial rare disease special guarantee fund can enjoy the treatment of gradual reimbursement of Huimin Bao, that is, to connect the Huimin Insurance and the rare disease special guarantee fund system
    .
    At the same time, some cities will also include some rare diseases that are not included in the national catalogue and the provincial special security fund into the local scope of
    people's insurance.

    At present, the relevant state departments have communicated with the Banking and Insurance Regulatory Commission to let commercial insurance pay more attention to rare diseases in the future as much as possible, and the policy of benefiting the people will be adjusted
    in the future.

    What can businesses do now?

    What can businesses do now?

    The promotion of rare disease protection by national and local governments is obvious to all, but it is difficult to speak alone, and the improvement of the rare disease protection system is inseparable from the efforts and goodwill
    of pharmaceutical companies.

    Deng Lei, Head of Corporate Public Policy at Sanofi

    Deng Lei, Head of Corporate Public Policy at Sanofi

    "Judging from the current situation, the main problems facing rare diseases are difficult to guarantee, difficult to land and difficult to
    treat.
    In this regard, enterprises can take targeted actions
    .
    Deng Lei, head of corporate public policy at Sanofi, said
    .

    Take targeted action

    Deng Lei believes that enterprises can take action from three aspects: in terms of protection, we must find ways to effectively reduce patients' out-of-pocket payment, so that patients can really use drugs, compared with price reduction, donating drugs is also a very effective program
    .

    In terms of landing, although the country has issued a lot of favorable policies, including dual-channel, admission policies, etc.
    , but the real landing of drugs, but also need to improve the clinical awareness and diagnosis and treatment level of rare diseases, through screening projects and clinical training, so that more patients are "screened out, cured", so that doctors have the opportunity and ability to diagnose and treat rare diseases, the use of rare disease drugs, in order to effectively promote rare disease drugs to benefit patients
    .

    In terms of treatment, there are two dimensions: First, for rare diseases that are not treated by drugs now, drugs should be introduced as soon as possible; Second, for rare diseases that already have drugs, they cannot be satisfied with the status quo, but also continue to explore better solutions in the clinic, so that the quality of life of patients can be higher and higher
    .

    "Due to the vast Chinese market and large regional differences, enterprises need to formulate refined strategies, work closely with relevant parties, adapt to local conditions, and use existing policies to quickly promote the implementation
    of rare disease protection.
    " Deng Lei said
    .

    Deng Lei (first from left) poses with Cai Haiqing, former director of the Treatment Department of Jiangxi Provincial Medical Security Bureau (second from left), Hu Dayang, president of Jiangsu Medical Insurance Research Association (third from left), and Zhu Kun, deputy director of the Social Development Research Center of the Chinese Academy of Fiscal Sciences (first from right), at the forum
    .

    Deng Lei (first from left) poses with Cai Haiqing, former director of the Treatment Department of Jiangxi Provincial Medical Security Bureau (second from left), Hu Dayang, president of Jiangsu Medical Insurance Research Association (third from left), and Zhu Kun, deputy director of the Social Development Research Center of the Chinese Academy of Fiscal Sciences (first from right), at the forum
    .
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