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
Characterized by their low prevalence, rare diseases are often chronically debilitating or life threatening. Despite their low prevalence, the aggregate number of individuals suffering from a rare disease is estimated...Characterized by their low prevalence, rare diseases are often chronically debilitating or life threatening. Despite their low prevalence, the aggregate number of individuals suffering from a rare disease is estimated to be nearly 400 million worldwide.Over the past decades, efforts from researchers, clinicians, and pharmaceutical industries have been focused on both the diagnosis and therapy of rare diseases. However, because of the lack of data and medical records for individual rare diseases and the high cost of orphan drug development, only limited progress has been achieved. In recent years, the rapid development of next-generation sequencing(NGS)-based technologies, as well as the popularity of precision medicine has facilitated a better understanding of rare diseases and their molecular etiology. As a result, molecular subclassification can be identified within each disease more clearly, significantly improving diagnostic accuracy. However, providing appropriate care for patients with rare diseases is still an enormous challenge. In this review, we provide a brief introduction to the challenges of rare disease research and make suggestions on where and how our efforts should be focused.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.
基金supported by the National High Technology Research and Development Program of China (2015AA020108, 2015AA020104)the National Science Foundation of China (31671377)Shanghai 111 Project (B14019)
文摘Characterized by their low prevalence, rare diseases are often chronically debilitating or life threatening. Despite their low prevalence, the aggregate number of individuals suffering from a rare disease is estimated to be nearly 400 million worldwide.Over the past decades, efforts from researchers, clinicians, and pharmaceutical industries have been focused on both the diagnosis and therapy of rare diseases. However, because of the lack of data and medical records for individual rare diseases and the high cost of orphan drug development, only limited progress has been achieved. In recent years, the rapid development of next-generation sequencing(NGS)-based technologies, as well as the popularity of precision medicine has facilitated a better understanding of rare diseases and their molecular etiology. As a result, molecular subclassification can be identified within each disease more clearly, significantly improving diagnostic accuracy. However, providing appropriate care for patients with rare diseases is still an enormous challenge. In this review, we provide a brief introduction to the challenges of rare disease research and make suggestions on where and how our efforts should be focused.
