Source: Original manuscript of People’s Daily Online
People’s Daily Online, Beijing, March 17 (Reporter Du Yanfei) “We need to standardize the standard treatment plan.” “Optimize the medical insurance catalog and treatment guarantee, and improve the medical treatment of thalassemia. Security system.” Recently, the “People’s Meeting Room” column of People’s Daily Online invited experts and scholars in the field of thalassemia prevention and treatment and representatives of thalassemia patient organizations to discuss and share on the topic of “building a full-cycle thalassemia prevention and treatment system to improve access to innovative drugs”. Help the continuous development of thalassemia prevention and control in my country, and better benefit thalassemia patients.
Improving the availability of innovative drugs to benefit more patients
Thalassemia for short is a hereditary blood disease caused by mutation or deletion of the globin peptide chain gene. Birth defect disease with serious harm and high incidence in southern regions. Although thalassemia is difficult to treat, it can be prevented. In recent years, the National Health Commission has launched thalassaemia prevention and control projects in many southern provinces with high thalassemia gene carrier rates, and promoted the implementation of thalassaemia prevention and control measures, and has achieved positive progress and good results. However, the comprehensive strengthening of thalassaemia prevention and control still requires the joint attention and support of all sectors of society.
Thalassemia is a highly hereditary and fatal disease. Wu Depei, member of the National Committee of the Chinese People’s Political Consultative Conference, chairman of the Chinese Medical Association Hematology Branch, and director of the Department of Hematology of the First Affiliated Hospital of Soochow University, believes that it is very important to establish a full-cycle thalassemia prevention and control system, and it is necessary to attach great importance to the limitations of treatment methods faced by adult patients, serious economic burden.
He suggested to further promote the formulation and practice of the standardized standard treatment of thalassemia, and accelerate the integration of treatment concepts and innovative drugs with international standards. Through multi-disciplinary cooperation, we provide comprehensive care for patients and promote the quality of life and physical and mental health of patients.
During the National Two Sessions in 2022, Wu Depei prepared a proposal around “strengthening and improving the medical security system for the treatment of thalassemia throughout the course of the disease”. The contents include: optimizing the existing medical methods to cure thalassemia; improving the availability of innovative treatment methods that can alleviate blood transfusion dependence; guiding the provincial medical insurance bureau to adjust the relevant drug catalogues, treatment guarantees, and medical assistance according to local conditions.
Wu Depei said: “It is necessary to actively promote the medical insurance evaluation and access of innovative drugs, improve the scope of medical insurance, effectively improve the availability of drugs, and reduce the possibility of patients becoming impoverished and returning to poverty due to illness. At the same time, It is necessary to actively explore the research and development of cutting-edge technology including gene therapy, in order to benefit more patients in the future.”
Innovative thalassaemia diagnosis and treatment methods improve the quality of life of patients
At present, hematopoietic stem cell transplantation It is a curative treatment for patients with β-thalassemia major. For severe or transfusion-dependent adult patients who cannot accept transplantation, the available treatment options mainly include blood transfusion and iron removal therapy, palliative therapy, and erythroid maturation therapy.
Lai Yongrong, director of the Department of Hematology at the First Affiliated Hospital of Guangxi Medical University, said that he expects forces from all walks of life to actively promote the research and development and clinical application of innovative drugs, so as to reduce the dependence of patients on blood transfusion and iron removal therapy, and improve the lives of patients quality, and reduce the economic and social burden of patients.
In recent years, treatment methods in the field of blood diseases have emerged one after another, and many innovative drugs are also accelerating the pace of entering the domestic market. Lai Yongrong introduced that after decades of hard work, the innovative treatment of thalassemia has made great progress: first, hematopoietic stem cell transplantation has continued to mature, and adult hematopoietic stem cell transplantation is also being explored; second, the development and application of innovative drugs, such as red blood cells Maturation agents also bring more options to the clinic; thirdly, gene therapy and other therapies that are undergoing clinical research will provide more possibilities for innovative treatments in the future.
Optimize the medical insurance catalog and treatment guarantee and improve the thalassemia medical insurance system
Recently, Beijing New Sunshine Charity Foundation, together with Shandong University Health Management and Policy Research Center, National Health and Health Commission Health Economics Together with the Key Laboratory of Policy Research, the “Report on the Economic Burden of Disease in Adult Patients with β-Thalassemia Major in China” was released.
Beijing New Sunshine Charity Foundation Secretary-General Liu Zhengchen introduced that the foundation has long been committed to helping patients with blood diseases and tumors. Thalassaemia can be prevented and controlled. Strengthening the prevention and control of thalassemia is of great significance to consolidate the achievements of poverty alleviation, especially to promote the health of population in areas with high incidence of thalassemia in my country.
Liu Zhengchen suggested that the national medical insurance coverage of innovative therapies should be further improved to alleviate the dependence of thalassemia patients on blood transfusion therapy. Expand the scope of pilot projects and disease types for outpatient special chronic disease settlement, so that thalassemia patients in more regions can receive treatment in outpatient clinics and conduct direct settlement across provinces. In addition, it is hoped that relevant government departments can adapt measures to local conditions and optimize the drug and medical insurance catalogue and treatment guarantees according to the characteristics of high incidence of local diseases.
Hu Shanlian, a professor of health economics at the School of Public Health of Fudan University, suggested that prevention should be put first, and prevention and control measures throughout the life cycle should be emphasized. After the list of medical insurance reimbursement benefits is formulated, it is also necessary to consider the characteristics of local high incidence, include thalassemia in “outpatient special diseases and serious diseases”, give preference to medical insurance policies, and reduce the economic burden of patients. For related innovative drugs, clinical and pharmacoeconomic evaluations should be done well, and standardized clinical diagnosis and treatment guidelines should be established.
“In order to rapidly improve the availability of innovative drugs such as red blood cell maturation agents, we need to do our best to evaluate the pharmacoeconomics of innovative drugs, evaluate health technologies, and study the impact of the national medical insurance budget.” Hu Shanlian believes that, For the research and development of innovative drugs, researchers also need to conduct extensive clinical research, accumulate experience, and include some standardized treatment methods in clinical diagnosis and treatment guidelines as soon as possible. “