"No pain, no disability", domestic recombinant human coagulation factor Ⅷ brings new hope to Chinese hemophilia patients!

*Only for medical professionals to read and refer to Shenzhou Jiayin, accessible to everyone! Hemophilia A is a coagulation dysfunction caused by the lack of coagulation factor VIII protein, and faces a very high risk of bleeding throughout life and the risk of joint deformity and disability caused by long-term inflammation
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At present, the main treatment for hemophilia A is alternative infusion of coagulation factor Ⅷ to achieve the purpose of hemostasis or prevention of bleeding.
However, due to the generally low level of diagnosis and treatment in the past and the shortage of therapeutic drugs, many patients with hemophilia cannot receive adequate timely Treatment
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In China, patients with hemophilia still face the status quo of low diagnosis rate, low treatment rate, and high disability rate
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How to improve the survival status of hemophilia patients in my country? How to "break" this dilemma? The medical community sincerely invites Professor Yang Renchi from the Hospital of Hematology of the Chinese Academy of Medical Sciences to share the challenges faced by the diagnosis and treatment of hemophilia in China and how to deal with them
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The unbearable burden of life-the real plight of hemophilia patients ""From birth to the end of life, the cost of treatment for hemophilia patients in this lifetime is very expensive," Professor Yang Renchi said: "Although the national medical insurance has A product for the treatment of hemophilia is included in the scope of reimbursement.
However, from the introduction of national policies to the actual implementation of various provinces, affected by different medical insurance budgets and different economic development conditions, the payment ratio of hemophilia patients is still relatively high.
High level
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In the past, economic constraints have made hemophilia patients "unable to use drugs," and the lack of access to drugs has made it worse.
On-demand treatment for hemophilia patients cannot be guaranteed
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Thanks to drug research and development and national policies, the current treatment model for hemophilia in China is gradually shifting from on-demand treatment to preventive treatment.
After 2017, the National Medical Insurance Catalogue has included preventive treatment for children with hemophilia in the scope of reimbursement, which means Patients with hemophilia under the age of 18 should start regular and continuous replacement therapy when they experience joint bleeding for the first time, and can be reimbursed by the national medical insurance
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Professor Yang Renchi pointed out that although the National Medical Insurance Catalogue has included the preventive treatment of children with hemophilia in the scope of reimbursement, there are still many restrictions on adults.
Due to the inability to receive adequate and effective treatment in time, adult hemophilia patients may even experience life-threatening Symptoms of bleeding
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"The level of diagnosis and treatment of hemophilia patients in China has been significantly improved, but the effective implementation of medical insurance policies, reducing patient payment ratios, and improving drug accessibility and patient compliance are still key issues that need to be addressed in the management of hemophilia.
Question
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"Professor Yang Renchi said
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A difficult city-how to create a win-win situation for patients with curative effects and economy? "The efficacy, safety, accessibility, and economic factors of drugs are the points that clinicians and patients consider when making clinical decisions together
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Professor Yang Renchi said: "For the challenges that clinicians face in the diagnosis, treatment and management of hemophilia, from From my personal point of view, it is still the issue of patients paying for medical expenses
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How to reduce the economic burden of patients as much as possible while prescribing in accordance with diagnosis and treatment standards is a major challenge for clinicians
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The second is how to reduce and deal with the production of coagulation factor inhibitors in hemophilia patients-about 30% of hemophilia A patients will produce inhibitors, and at the same time as the number of treatments and the increase in the amount of coagulation factor concentrate infusion, patients will produce The possibility of clotting factor inhibitors is also increasing
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Once coagulation factor inhibitors are produced, the curative effect of normal treatment is getting lower and lower, the bleeding is more and more difficult to control, and the commonly used doses of coagulation factor concentrates cannot quickly control the bleeding.
Problems will also "enroll", and it is also for clinicians.
Great challenge
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"" The cloud sees the day-the first domestically produced recombinant human coagulation factor Ⅷ to help patients with "pain-free, non-disabled" patients with hemophilia A.
The treatment of patients with hemophilia A is mainly an alternative infusion of coagulation factor Ⅷ, according to the "Chinese Guidelines for the Treatment of Hemophilia (2020 Edition) ) "Recommendation [1], the first choice should be recombinant human coagulation factor Ⅷ or virus-inactivated blood-derived coagulation factor VIII
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The use of blood products has the risk of viral infection, and once infection occurs, the treatment costs and social burdens of virus-positive hemophilia patients will far exceed those of ordinary hemophilia patients
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From the perspective of production capacity, the production and supply of recombinant human coagulation factor Ⅷ is also more secure than blood products
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Therefore, recombinant human coagulation factor VIII has advantages over blood-derived coagulation factor VIII
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However, for many years, recombinant human coagulation factor Ⅷ for Chinese hemophilia patients has only imported varieties to choose from, and they are facing practical problems such as price and supply
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On July 23, 2021, Recombinant Human Coagulation Factor Ⅷ (An Jiayin) for injection by Shenzhou Cell Engineering Co.
, Ltd.
was officially approved for marketing by the National Medical Products Administration (NMPA), breaking the situation in one fell swoop and filling the domestic recombinant human coagulation factor Ⅷ The blank [2]
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Figure 1 Screenshot of the official website.
As the main researcher of Anjiayin's clinical registration research, Professor Yang Renchi introduced: Anjiayin is China's first recombinant human coagulation factor Ⅷ product with independent intellectual property rights, and it also means another national enterprise in China Innovation and breakthrough
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From the research data, compared with imported products, domestic recombinant human coagulation factor Ⅷ has no difference in terms of effectiveness and safety
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And because Anjia has leading production technology and capacity, it will make up for the shortage of factor VIII supply in China and even the world, and greatly improve the accessibility of factor VIII
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Professor Yang Renchi pointed out that after imported medicines are marketed in China, due to patent protection and other reasons, prices are very high for patients
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Therefore, if domestic drugs are safe and effective enough, clinicians also hope that domestic drugs can replace imported products, so that more Chinese hemophilia patients can afford and use drugs, improve the quality of life of patients, and reduce disability due to illness.
Incidence rate, to help more patients return to normal life
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"In addition, due to the cost advantage, the listing of Anjiayin can also reduce the burden of national medical insurance.
As the total amount of drugs that the country needs to pay is reduced, the burden of payment for hemophilia patients will also be reduced, which will ultimately be implemented in the individual benefits of patients
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The reduction of treatment costs and the timely update of professional diagnosis and treatment knowledge are of great benefits to both doctors and patients and the society
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"Professor Yang Renchi said
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"I believe that with the launch of the first domestically-made recombinant human coagulation factor Ⅷ, more domestic hemophilia patients will be able to truly receive preventive treatment, changing the situation of on-demand treatment or low-dose preventive treatment, so as to achieve Chinese hemophilia The patient's treatment goal of "no pain, no disability"! Expert profile Professor Yang Renchi, chief physician and doctoral supervisor, Director of Thrombosis and Hemostasis Center, Chinese Academy of Medical Sciences Hematology Hospital (Institute of Hematology) Director of the Asia-Pacific Hemophilia Working Group Steering Committee Member Vice Chairman of the Hemostasis and Thrombosis Branch of the Chinese Medical Education Association Executive Director of the Rare Diseases Branch of the Chinese Research Hospital Association Standing Committee of the Thrombosis and Hemostasis Professional Committee of the Chinese Research Hospital Association Member of the Committee of Guarantee Experts, Chairman of the Hemophilia Group of the China Alliance of Rare Diseases, Deputy Leader of the Vascular Biology Group of the Experimental Hematology Professional Committee of the Chinese Pathophysiology Society Leader of the Chinese Rare Blood Disease Working Group and Leader of the National Hemophilia Collaborative Group References: [1] Yang Renchi.
Chinese Guidelines for the Treatment of Hemophilia (2020 Edition) [J].
Chinese Journal of Hematology, 2020, Volume 41, Issue 4, Pages 265-271, MEDLINE ISTIC PKU CSCD CA, 2021: Chinese Academy of Medical Sciences Medicine Major collaborative innovation projects with health technology innovation projects[2]https://mp.
weixin.
qq.
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