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since my country announced the first list of rare diseases in 2018, the problems faced by more than 20 million rare disease patients in terms of access to medical care, medicine and insurance have begun to be solved. however, there is still a lack of institutional and systematic guarantees. the industry calls for the passage of relevant legislation to promote the durability and stability of policies in the field of rare diseases.

the code rare disease center released the "research report on china's rare disease legislation" (hereinafter referred to as the "report") at the 13th china rare disease summit forum 2024 held recently. the report puts forward nine legislative initiatives, including the imperative of systematic legislation on rare diseases in china, the clarification of the legal definition of rare diseases and rare disease drugs as soon as possible, and the clarification of incentive policies for rare disease research and development and industrial development.

lou yu, professor at the school of civil, commercial and economic law of china university of political science and law and director of the institute of social law, believes that the most important thing about rare disease legislation is to achieve two functions. one is to clarify the legal definition of rare disease groups, and the other is to solve the problems of drug accessibility such as drug research and development, marketing, and inclusion in the medical insurance catalog.

how to move from catalog management to legislative protection

rare diseases are a general term for different diseases with low incidence and involving multiple systems. there are more than 7,000 rare diseases in the world, 80% of which are genetic diseases, which are difficult to diagnose and treat, have serious harm, and are a heavy burden. countries around the world have different definitions of rare diseases. for example, the world health organization's standard is that the number of patients accounts for 0.65‰~1‰ of the total population, the united states is less than 200,000 people, and the european union is less than 1/2000, chronic, progressive and life-threatening.

sun kun, president of xinhua hospital affiliated to shanghai jiao tong university school of medicine, said at the forum that there are three challenges in the diagnosis and treatment of rare diseases in my country. the first is difficult diagnosis, with 42% of patients experiencing misdiagnosis and 65% of doctors not understanding rare diseases. the second is difficult treatment, with a lack of drugs for rare diseases and poor drug accessibility. the number of orphan drugs in my country is only 3.6% of that in the united states. the third is poor homogeneity, with diverse clinical manifestations of the disease and atypical symptoms, and large disparities in diagnostic and treatment capabilities between different regions, hospitals, and doctors.

rare diseases in my country are managed in the form of catalogs. the country has released two batches of rare disease catalogs, the first batch of 121 and the second batch of 86, with a total of 207 rare diseases included. at present, there is no official definition of rare diseases in my country, no epidemiological research or database on rare diseases, and genetic screening for rare diseases has not yet been widely promoted.

huang rufang, founder and director of the code rare disease center, said that there are two major flaws in defining rare diseases through a catalog. the first is the public issue. diseases in the catalog can receive good policies and attention, while diseases outside the catalog cannot. the second is the scientific issue. there are more than 7,000 rare diseases in the world. just because the catalog defines these 200 diseases, the others are not rare diseases.

the report suggests that over the past decade, there have been rich discussions and experience accumulated at the grassroots level in my country regarding the definition of rare diseases, but there has never been a consensus at the national level or legal effect. in addition, the current rare disease catalog update mechanism can no longer meet the needs of industry development. it is recommended to confirm the legal definition of rare diseases and rare disease drugs through legislation as soon as possible to provide a legal basis for the protection of the rights and interests of rare disease patients and the development of the industry.

lou yu told china business news that the central legislation on rare diseases should define rare diseases and clarify the content related to "orphan drugs". from the perspective of legal functions, the legislation on rare diseases should be a management law and a promotion law. its main function is to define the division of power and cooperation between various functional departments of the state. in addition, it should guide and encourage social entities to actively participate in this field through promotion measures such as state subsidies and preferential policies.

clarify the "orphan drug" r&d and industry incentive policies

china business news learned that local governments have already explored legislation for rare diseases. jiangsu province's "medical security regulations" issued in 2023 include a drug security mechanism for rare diseases; shanghai, in terms of promoting the pharmaceutical and medical device industry, took advantage of the legislative advantages of pudong new area and included the "shanghai provisions on promoting the development of rare disease medicine in pudong new area" in the research project of the shanghai municipal people's congress' five-year legislative plan, and continued to conduct research and demonstration.

the r&d cost of drugs for rare diseases is high, the market capacity is small, and the return on investment is low. companies are reluctant to make them a research and development priority or even give up on research and development. therefore, drugs for rare diseases are also called "orphan drugs."

the report points out that there are more than 20 million patients with rare diseases in my country. various innovative r&d, production and service activities around this group have formed a considerable industrial effect in many regions around the world. however, my country has not yet formed a clear development path in this field. it is recommended that in the legislation of rare diseases, incentives for rare disease r&d and industrial development should be clarified, and relevant legal documents should be issued and implemented as soon as possible in some core policies, such as patent compensation, data protection law, market exclusivity period, and r&d tax incentives.

lou yu said that when promoting legislation on rare diseases, a supporting system is needed to make pharmaceutical companies profitable, especially when legislating, the market exclusivity period must be clearly defined, which can greatly increase the profits of pharmaceutical companies. at the same time, there will also be policy biases in payment methods.

in recent years, my country has introduced some policies to support the development of drugs for rare diseases, such as the "14th five-year plan for the development of the pharmaceutical industry", the "implementation plan for supporting the development of innovative drugs in the whole chain", and the "guiding principles for the study of the natural history of diseases in the development of drugs for rare diseases", which provide important technical guidance and support for the development of drugs for rare diseases. however, overall, there is a lack of full-chain support for the industry, and companies are also worried about whether the orphan drug policy will maintain its support and whether it will stagnate after a few batches are issued.

the report states that incentive policies are the key to strengthening industrial development, and legislation is necessary to integrate existing policies to form a synergy and provide comprehensive and lasting protection for patients with rare diseases. in terms of specific laws, the report recommends that governments at all levels use cash rewards or tax reductions to stimulate pharmaceutical and medical device companies to invest in the production of rare disease products, extend the protection period and market exclusivity period for rare disease drugs and medical devices, and accelerate the listing of rare disease drugs and medical devices in various ways.

payment for rare disease drugs is also a focus of this legislative survey. huang rufang said that the most prominent contradiction in my country's rare disease industry is the payment issue for innovative drugs and high-value rare disease drugs.

the cost of treating some rare diseases is very high, more than 10 times the cost of ordinary diseases. the cost of treating a single patient for some rare disease drugs exceeds 500,000 yuan or even 2 million yuan, and the patient needs to take the medicine for life. at present, my country has not yet promulgated special policies and regulations for rare disease patients at the national level, nor has it established a specific medical insurance system.

at present, the medical insurance department is exploring the establishment of a multiple-level protection system for rare diseases. more and more rare disease treatment drugs have been included in the national medical insurance drug list through national negotiated price reductions. among them, 7 new drugs were added in 2021, 7 new drugs were added in 2022, and 15 new drugs were added in 2023. to date, more than 80 rare disease treatment drugs have been included in the national medical insurance drug list. the national medical insurance administration also encourages localities to explore rare disease drug protection models.

the report also recommends setting up a special medical insurance fund to cover rare diseases, integrating various forces (such as the ministry of finance, ministry of civil affairs, national health commission, medical insurance bureau, etc.) to participate in the financing and operation of the special fund, and allowing and encouraging the exploration of special funds for rare diseases or other innovative payment models at the local level.