Objective To analyze relevant policies and measures on the management of orphan drug reimbursement in foreign countries to provide a reference for future reimbursement management in China.Methods According to the perc...Objective To analyze relevant policies and measures on the management of orphan drug reimbursement in foreign countries to provide a reference for future reimbursement management in China.Methods According to the percentage of health care expenditure in GDP,the completeness of rare disease policies,and the total population,Russia,Australia,and India were selected as the reference.Based on the existing literature,the main content and characteristics of the reimbursement of rare disease drugs were analyzed.Results and Conclusion Russia manages rare diseases in the form of lists.Special rare diseases are reimbursed by federal or regional finances,and ordinary rare diseases are reimbursed by statutory medical insurance funds.Orphan drugs in Australia are included in the pharmaceutical benefits scheme(PBS)and the lifesaving drugs program(LSDP),LSDP provides fully reimbursed drugs for eligible rare disease patients.India’s proposal takes health system sustainability into consideration.China should carry out epidemiological research to legally determine the rare diseases,establish reasonable reimbursement standards,and improve the multi-level reimbursement system.展开更多
According to global annual estimates hunger/malnutrition is the major cause of death(36 of 62 million). Cardiovascular diseases and cancer(5.44 of 13.43 million) are the major causes of death in developed countries, w...According to global annual estimates hunger/malnutrition is the major cause of death(36 of 62 million). Cardiovascular diseases and cancer(5.44 of 13.43 million) are the major causes of death in developed countries, while lower respiratory tract infections, human immunodeficiency virus infection/acquired immunodeficiency syndrome, diarrhoeal disease, malaria and tuberculosis(10.88 of 27.12 million) are the major causes of death in developing countries with more than 70% of deaths occurring in children. The majority of approximately 800 million people with other rare diseases, including 100000 children born with thalassaemia annually receive no treatment. There are major ethical dilemmas in dealing with global health issues such as poverty and the treatment of orphan and rare diseases. Of approximately 50000 drugs about 10% are orphan drugs, with annual sales of the latter approaching 100 billion USD. In comparison, the annual revenue in 2009 from the top 12 pharmaceutical companies in Western countrieswas 445 billion USD and the top drug, atorvastatin, reached 100 billion USD. In the same year, the total government expenditure for health in the developing countries was 410 billion USD with only 6%-7% having been received as aid from developed countries. Drugs cost the National Health Service in the United Kingdom more than 20 billion USD or 10% of the annual health budget. Uncontrollable drug prices and marketing policies affect global health budgets, clinical practice, patient safety and survival. Fines of 5.3 billion USD were imposed on two pharmaceutical companies in the United States, the regulatory authority in France was replaced and clinicians were charged with bribery in order to overcome recent illegal practises affecting patient care. High expenditure for drug development is mainly related to marketing costs. However, only 2 million USD was spent developing the drug deferiprone(L1) for thalassaemia up to the stage of multicentre clinical trials. The criteria for drug development, price levels and use needs to be readdressed to improve drug safety and minimise costs. New global health policies based on cheaper drugs can help the treatment of many categories of orphan and rare diseases and millions of orphan patients in developing and developed countries.展开更多
文摘Objective To analyze relevant policies and measures on the management of orphan drug reimbursement in foreign countries to provide a reference for future reimbursement management in China.Methods According to the percentage of health care expenditure in GDP,the completeness of rare disease policies,and the total population,Russia,Australia,and India were selected as the reference.Based on the existing literature,the main content and characteristics of the reimbursement of rare disease drugs were analyzed.Results and Conclusion Russia manages rare diseases in the form of lists.Special rare diseases are reimbursed by federal or regional finances,and ordinary rare diseases are reimbursed by statutory medical insurance funds.Orphan drugs in Australia are included in the pharmaceutical benefits scheme(PBS)and the lifesaving drugs program(LSDP),LSDP provides fully reimbursed drugs for eligible rare disease patients.India’s proposal takes health system sustainability into consideration.China should carry out epidemiological research to legally determine the rare diseases,establish reasonable reimbursement standards,and improve the multi-level reimbursement system.
文摘According to global annual estimates hunger/malnutrition is the major cause of death(36 of 62 million). Cardiovascular diseases and cancer(5.44 of 13.43 million) are the major causes of death in developed countries, while lower respiratory tract infections, human immunodeficiency virus infection/acquired immunodeficiency syndrome, diarrhoeal disease, malaria and tuberculosis(10.88 of 27.12 million) are the major causes of death in developing countries with more than 70% of deaths occurring in children. The majority of approximately 800 million people with other rare diseases, including 100000 children born with thalassaemia annually receive no treatment. There are major ethical dilemmas in dealing with global health issues such as poverty and the treatment of orphan and rare diseases. Of approximately 50000 drugs about 10% are orphan drugs, with annual sales of the latter approaching 100 billion USD. In comparison, the annual revenue in 2009 from the top 12 pharmaceutical companies in Western countrieswas 445 billion USD and the top drug, atorvastatin, reached 100 billion USD. In the same year, the total government expenditure for health in the developing countries was 410 billion USD with only 6%-7% having been received as aid from developed countries. Drugs cost the National Health Service in the United Kingdom more than 20 billion USD or 10% of the annual health budget. Uncontrollable drug prices and marketing policies affect global health budgets, clinical practice, patient safety and survival. Fines of 5.3 billion USD were imposed on two pharmaceutical companies in the United States, the regulatory authority in France was replaced and clinicians were charged with bribery in order to overcome recent illegal practises affecting patient care. High expenditure for drug development is mainly related to marketing costs. However, only 2 million USD was spent developing the drug deferiprone(L1) for thalassaemia up to the stage of multicentre clinical trials. The criteria for drug development, price levels and use needs to be readdressed to improve drug safety and minimise costs. New global health policies based on cheaper drugs can help the treatment of many categories of orphan and rare diseases and millions of orphan patients in developing and developed countries.
