Building a protective wall for patients with rare diseases in 6 aspects

On February 11 this year, the executive meeting of the State Council called for preferential policies on the import tariff of the first batch of 21 drugs with rare diseases and 4 APIs to ensure the use of drugs by more than 20 million patients with rare diseases Recently, with the introduction of the catalogue of rare diseases and the increase of relevant reports, the problem of medication security for rare diseases has aroused widespread concern In order to further explore the ways to improve the access of rare disease patients to medication and improve the medical security system for rare diseases, China Medical Insurance magazine held the youth drug administration Forum (20th issue) in Beijing on March 2, inviting experts in the industry to discuss the theme of "drug security for rare disease patients" There are several suggestions: the multi party collaboration to promote drug use in rare diseases is huge because of the large population base in China Although the incidence rate of single disease is very low, the number of patients with rare diseases is large, and the demand for medical treatment is huge Therefore, on the basis of basic medical insurance and serious illness insurance, we should give full play to multi-channel solutions such as social charity, medical assistance, commercial insurance and the establishment of special government subsidy funds Internationally, most countries have set up special safeguard policies for rare diseases, and seek special support outside the medical insurance system to relieve economic pressure, such as adding tobacco tax to the safeguard for rare diseases It should be noted that multiple stakeholders participate in the protection of drug use for rare diseases, and the relationship between them is complementary rather than incompatible Therefore, it is necessary to establish a communication and coordination mechanism between each other to avoid impact on the original drug use guarantee channels (for example, the state must maintain the established drug donation platform and channels of charity projects as much as possible when issuing the guarantee policies), and finally form a balance between different benefit subjects, so as to maximize the guarantee benefits Under the condition of limited resources, drug use security should focus on different rare diseases, and there is a large gap in per capita medical costs Some rare diseases require only a few hundred yuan to be treated, and some, such as gerschneider's disease, Pompeii's disease, Fabry's disease, mucopolysaccharide storage disease, etc the total annual drug cost is more than 6 billion yuan, and the average annual drug cost is about 2 million yuan People's life is priceless, but when making macro policy decisions, we need to use limited resources to obtain the maximum security benefits The opportunity cost and ethical choice of rare disease drugs must be considered Therefore, experts suggest that priority should be given to the following patients with rare diseases: first, patients who need long-term medication and may die or be disabled if the drug is stopped; second, patients who can be completely cured; third, children's patients Although great progress has been made in the application of health technology assessment, the current assessment tools are still insufficient in the practice of specific policy application Especially for some high-priced rare disease drugs, how to link the assessment process and results with the health insurance policy? How to promote scientific decision? There is still a lot of space to study We should also pay more attention to the participation of patients in the assessment, and learn more about how rare diseases affect their lives? What are the current possible treatments? Which therapeutic effects are most important to patients? What are the possible risks of the new treatment? Wait In order to improve the performance of medical insurance fund and promote the quality of drugs for rare diseases, the concept of performance-based medical insurance payment can be introduced into the strategic purchase of drugs According to the pharmacoeconomic evaluation, drugs with clear innovation value, evidence-based medicine and clinical efficacy are preferred to be included For new drugs with unclear effects, the reimbursement policy can be readjusted when the evidence is sufficient If the patient does not have the expected reaction after taking the medicine, the pharmaceutical enterprise may consider returning a certain proportion of the expenses to the national medical insurance Wait To improve the basic database of rare diseases, we should establish and improve the basic database of rare diseases as soon as possible Data can be obtained from three ways: first, collect relevant data through the existing clinical data collection platform, such as the national rare disease information registration platform; second, collect relevant clinical application specific data in the field of medical security; third, supplement with the relevant data of pharmaceutical enterprises Finally, through the cooperation of big data application and departments, we can accurately identify the patients suffering from catastrophic expenditure and provide them with accurate assistance At present, doctors who can treat rare diseases are more rare than rare diseases It is not realistic to carry out technical training and popularization of science for diagnosis and treatment of rare diseases nationwide At present, the most feasible way is to set up several provincial centers for the diagnosis and treatment of rare diseases, and try to gather high-quality doctors to treat patients with rare diseases In addition, the dynamic adjustment mechanism of drug catalog should be introduced as soon as possible to ensure that drugs for rare diseases can be negotiated in a timely manner, and more funds can be provided for the treatment of rare diseases through "cage up" and "bird replacement".