According to the China Rare Disease Drug Accessibility Report (2019), which was just released, 42% of patients with rare diseases still do not receive any treatment. Members suggested that in order to solve the problem of rare disease groups, it is necessary to gradually establish a medical security strategy for rare diseases that is suitable for Chinas national conditions and should be tailored to local conditions.
In recent years, the State Council and relevant government departments have paid more and more attention to rare diseases. Especially in May 2018, the State Health and Health Commission jointly released the first batch of rare diseases catalogues in China, which caused great social repercussions.
At the ongoing National Two Sessions, Ding Jie, a member of the CPPCC National Committee and professor of pediatrics at the First Hospital of Peking University, and the Chinese Womens Federation, jointly submitted the Recommendations on Developing Medical Security Strategies for Rare Diseases that are in line with Chinas actual situation, pointed out that in order to solve the problems of rare disease groups, it is necessary to classify them scientifically, and gradually establish measures according to local conditions and bottlenecks. The strategy of medical insurance for rare diseases is suitable for Chinas national conditions.
Ding Jie, member of the CPPCC National Committee and professor of pediatrics, First Hospital of Peking University. Photographer/Beijing News reporter Wang Kara
[Problem] It is difficult for a single system to solve the problem of medical security for patients with rare diseases.
On February 11, 2019, when Li Keqiang, Premier of the State Council, chaired the executive meeting of the State Council, it was clearly pointed out that more than 20 million patients with rare diseases should be guaranteed medication. In February 2019, the State Health Commission announced the establishment of a national collaborative network of rare diseases diagnosis and treatment to strengthen the management of rare diseases in China and improve the level of diagnosis and treatment of rare diseases. Relevant work on rare diseases in China is gradually being refined and implemented.
However, on the day of the International Rare Diseases Day just past, the China Rare Diseases Drug Accessibility Report (2019), published by the Rare Diseases Development Center in conjunction with IQVIA Ikunwei China, showed that 42% of the patients with rare diseases still did not receive any treatment. Even in the patients receiving treatment, the vast majority of them fail to take treatment drugs in time and in sufficient quantities; more than half of the patients are disabled due to illness.
Ding Jie pointed out that although rare diseases are rare, they are not rare in China. Because of the huge population base in China, although the incidence of single disease is very low, the total number of patients with rare diseases is large and the demand for medical treatment is huge. It is difficult to solve the problem of medical security for patients with rare diseases by relying on a single system. On the other hand, there are thousands of rare diseases with complex clinical manifestations and different means of diagnosis and treatment. Even the 121 diseases in the first batch of rare diseases catalogue issued by five ministries and commissions at present involve different kinds of therapeutic drugs, costs and urgent problems to be solved. Therefore, it is very important to formulate a medical security strategy for rare diseases that is suitable for our countrys reality.
[Strategies] Gradually determine the range of rare diseases by stages and batches
The proposal suggests that, first of all, the scope of protection should be clearly defined, and the current level of economic and social development, the balance of medical insurance fund, the incidence of rare diseases and the diagnosis and treatment of rare diseases should be synthesized. The scope of rare diseases and the amount of medical payment needed can be gradually determined by stages and batches after the organization of expert demonstration. According to the status of basic medical insurance, the duration of insurance, household registration and other information, the members of the rare disease collaboration network are graded to determine the protection objects that each province needs to be covered by the rare disease medical insurance system.
Patients with rare diseases included in the scope of protection are classified according to the medicines needed for treatment of diseases and their expenses, and their compliance medical expenses are separately shared by basic medical insurance, major diseases insurance and medical assistance step by step. Compliant medical expenses include the medical expenses necessary to maintain diagnosis and treatment within the scope of basic medical insurance policy, and the expenses for special drugs for rare diseases. Ding Jie pointed out that the path of realization should be clearly guaranteed. For patients who meet the requirements of rare disease protection objects, they should go to the National Rare Disease Diagnosis and Treatment Collaborative Network Hospital (designated hospital) announced by the State Health and Health Commission to diagnose, issue the Diagnosis Certificate and reexamine the work.
In terms of cost guarantee, patients should first reimburse the medical expenses for rare diseases in proportion to the basic medical insurance, major diseases insurance and medical assistance policies of each province. The remaining medical expenses for rare diseases should be financially allocated and solved through special relief channels. Special relief funds shall be subsidized by the financial department through civil relief channels. At the same time, the proposal proposes the establishment of a multi-participatory and multi-financing fund for rare diseases to overcome the plight of patients with rare diseases and their families abandoning treatment or returning to poverty due to illness.
[Implementation] Multi-participation and multi-financing
In order to ensure the implementation of the above measures, the proposal suggests that the National Medical Security Bureau should take the lead in planning and establish a multi-participated and multi-funded Fund Management Committee for rare diseases and its expert committee and medical ethics committee in conjunction with the Ministry of Civil Affairs, the Ministry of Finance, the Health Planning Commission, the Drug Administration and the Development and Reform Commission. The specific executing agencies are set up in the Health Care Commission or the Medical Insurance Bureau. The provincial governments refer to the implementation.
The Health and Health Commission guides the formulation of rare disease catalogues (which can be formulated in stages and batches dynamically), the definition of designated medical institutions, the classification and classification of therapeutic drugs, and the guidance of diagnosis, treatment and information management. Medical security departments, in conjunction with the Health and Health Commission, determine the rare diseases and the scope of special medication, and incorporate them into the payment scope of major diseases insurance, so as to reimburse the medical expenses of patients with basic medical insurance and major diseases insurance. It also puts forward the calculation of the required funds. On the basis of the calculation of funds proposed by the Medical Insurance Bureau and the Health Care Commission, the financial department allocates special funds according to the governments financial resources, and supervises the operation and management of the funds. Civil affairs departments are responsible for the qualification examination, archives management and reimbursement of rescue funds for the rare patients.
In order to ensure the supply of rare disease drugs, the proposal also proposed that the management model of rare disease special drugs should be established, and the negotiation access model similar to that of special drugs for disease insurance should be adopted, but orphan drugs should not be competitive negotiations. Medical insurance agencies are responsible for negotiating and signing drug management service agreements with drug manufacturers (suppliers); special drugs for rare diseases are not included in the proportion of drugs in hospitals and the average cost assessment of medical insurance. ?
[Call] Establish a management model for special drugs for rare diseases
In order to improve the accessibility of rare disease drugs, Ding Jie proposed that we should establish a management model of rare disease special drugs, adopt a negotiation access model similar to that of special drugs for disease insurance, and negotiate by the state. After all, there are many rare patients in China, and the market is not small. Through national negotiations, drug prices can be reduced appropriately. Dante stressed that orphan drugs do not enter into competitive negotiations.
In addition, in order to ensure that drug prices are not affected by intermediate links and rise, Ding Jie suggested that medical insurance agencies should be responsible for negotiating and signing drug management service agreements with drug manufacturers (suppliers) to turn on the green light for rare disease drugs in order to ensure drug accessibility. At the same time, special drugs for rare diseases are not included in the assessment of the proportion of drugs in hospitals and the average cost of medical insurance. Because quite a number of rare orphan medicines are very expensive, if the proportion of medicines and the average cost of medical insurance are included, hospitals will not have the motivation to introduce these medicines. Even if they are included in medical insurance, patients will not necessarily be able to buy medicines in hospitals.