Study On Mechanism For Rare Disease Medication Security In Jiangsu Province

Objective: Rare disease medication security is an integral part of social security.It is a comprehensive and basic policy that guarantees basic people’s livelihood,promotes social equity,and maintains social stability.Therefore,exploring rare disease medication security mechanism and solving the problem of rare disease medication security is of great practical significance to the continuous improvement of the multi-level medical security system with Chinese characteristics and the realization of a comprehensive well-off society.At present,the exploration of rare disease medication security in my country started relatively late,a complete security mechanism for rare diseases has not been formed.According to the goals and tasks of the national medical insurance system,based on systematically analyzing and summarizing the domestic and foreign rare disease medication and policy practice,combined with the exploration and practice of rare disease medication security in Jiangsu Province,this paper provides suggestions for the construction of the rare disease medication security mechanism in Jiangsu Province.Methods: Relevant concepts,theoretical basis,domestic and foreign rare disease medication security mechanism were systematically summarized and analyzed by literature research method;comparative study method was used to analyze the specific practices of rare disease medication security in typical countries and regions,and to compare and analyze the status quo of policy practice and exploration related to rare disease medication security in China,so as to summarize the experience that can be used for reference;the inductive and deductive method was used to analyze the deficiencies in the practice of rare disease medication security in China,summarize the common problems faced by various regions,and provide a reference basis for the establishment of long-term operation of rare disease medication security mechanism in Jiangsu Province based on the exploration.Results:(1)The exploration of drug guarantee for rare diseases in China started late,and there are still many difficulties: such as lack of clear definition of rare diseases,reliable epidemiological data,complete rare disease diagnosis and treatment system,drug screening access,pricing,coverage of benefits,effective supply of rare disease drugs,sources of security funds and payment methods.These all pose obvious challenges,which directly affect the establishment and improvement of rare disease medication security mechanism.(2)Typical countries and regions have commonalities in establishing and improving the rare disease medication security mechanism: formulate a clear definition of rare diseases;formulate laws and regulations related to rare diseases,and support the rare disease medication security mechanism through legislation;attach importance to the research and development production of rare disease drugs,and issue a series of incentive policies;formulate governmentled rare disease medical security policies;set up special institutions to manage the development and marketing of rare disease drugs.(3)China’s rare disease medication security policy practice results: national level,China has issued a series of policies,such as the establishment of a rare disease catalog,a diagnosis and treatment collaboration network,an information registration platform,accelerated review and approval,tax incentives,and inclusion of rare disease drugs in medical insurance,to gradually solve the problems of rare disease groups in diagnosis and treatment,drug accessibility and affordability.At the local level,the thinking of multi-party co-payment and risk sharing has been generally adopted in exploring the rare disease medication security.Considering the high cost of rare disease diagnosis and treatment and the high price of medicines,most regions have adopted a multi-channel co-payment method based on special funds,critical illness insurance or medical aid,rather than a single security method,to reduce high rare diseases.It has reduced the impact of high medical expenses for rare diseases on the medical insurance fund,and improves the treatment of patients.(4)China rare disease medication security policy existing problems: at the national level,there is a lack of special protection policies.The rare disease medication security is mainly based on basic medical insurance.According to the current access standards,it is difficult to include rare diseases in the medical insurance catalogue.In view of the characteristics of high price and rare audience of rare disease drugs,special evaluation standards and special financing plans should be formulated.In addition,the coverage of rare disease drugs is relatively small,and the annual treatment cost of some rare disease drugs that are not covered by medical insurance exceeds one million yuan,causing a heavy burden on patients.The government should pay more attention to such high-priced drugs and actively explore multi-channel supporting measures for high-priced orphan drugs.At the local level,on the one hand,the policy connection is not clear enough,and the main responsibility for the rare disease medication security is not clear,which leads to confusion in the way of security,scope of security,and management services.On the other hand,the sustainability of the fund is insufficient.In the absence of a clear financing mechanism,access mechanism and management mechanism,the continuous inclusion of high-value rare disease drugs will have an impact on the medical insurance fund.It is necessary to establish a special security fund and improve the supporting system,and reserve space for future adjustments and updates.(5)Problems in the practice of rare disease medication security policy in Jiangsu Province:In terms of coverage,Lack of clear,standardized and unified standards and access mechanism,lack of supplementary assistance programs for rare diseases beyond the coverage of medical insurance,and pay little attention to high-value drugs for rare diseases.In terms of fund raising,at present,Jiangsu province is mainly exploring rare disease insurance under the framework of basic medical insurance.The low pooling level will limit the expansion of insurance coverage and the improvement of treatment level,and weaken the anti-risk ability of medical insurance fund,thus affecting the sustainability of rare disease insurance.In terms of diagnosis and treatment capabilities,the distribution of medical insurance resources for rare diseases in the province is uneven,and coordination and cooperation should be strengthened to improve the regional rare disease diagnosis and treatment capabilities as a whole,and release the radiation effect of diagnosis and treatment resources of hospitals in the cooperative network.Conclusions:(1)The establishment of a scientific,reasonable and clear definition of rare diseases is the premise for the development of rare disease-related medical care,drugs,and security.When defining rare diseases in China,it should be guided by the needs and resources within the scope of policy coverage,and define rare diseases from multiple perspectives so as to better manage and maintain health services for patients with rare diseases.(2)Exploring the construction of rare disease medication security mechanism,and addressing the basic needs of patients with rare diseases as the bottom line of security is the core and key to guarantee the basic rights and interests of patients with rare diseases.Adhering to the security that patients with rare diseases "medical treatment,medical insurance,and medicine",and alleviating the risk of serious diseases for the poor,can promote the improvement of the multi-level medical security system and better reflect social fairness and justice.(3)Based on the actual situation of Jiangsu Province,specific design suggestions are put forward to establish rare disease medication security mechanism in Jiangsu Province from the aspects of clarifying the significance of security mechanism,overall requirements for mechanism construction,core elements and main contents,specific implementation management and organizational guarantee.The core elements should include: define the source of security fund,establish drug access selection,application,evaluation criteria and negotiation mechanism,establish rare disease drug use management and evaluation supervision mechanism,define security object,security scope and security level;improve the supervision and evaluation system for fund payment and the policy evaluation system for rare diseases.