In 2022, will more orphan drugs enter Medicare?

(Dust 4x/figure)

Rare diseases are a type of serious chronic diseases with low incidence and small number of patients, there is no international unified definition standard, "China Rare Disease Definition Research Report 2021" issued to propose, rare disease refers to "newborn incidence is less than 1/10,000, prevalence rate is less than 1/10,000, the number of patients is less than 140,000 diseases." "In 2018, the "First Rare Disease Catalogue" (hereinafter referred to as the Catalogue), jointly released by the National Health Commission and other five departments, included 121 rare diseases, and the treatment of these rare drugs was called orphan drugs due to scarcity.

The research and development of orphan drugs is difficult, the return on investment is low, the unit price of drugs is high, and all developed countries have adopted various sponsorship measures to encourage enterprise research and development. For a long time, China has been constrained by economic conditions and has made slow progress in the research and development of orphan drugs, as of 2019, of the 481 clinical trials for rare diseases carried out worldwide, 53% of the projects carried out in North America. 01% (255), Europe 14. 76% (71), compared with less than 10 in china.

Therefore, China's rare patient treatment drugs mainly rely on imports, but there are "can not buy" and "can not afford" the problem, although the incidence of rare diseases is low, but China's population base is large, the "catalog" included in the 121 rare diseases involving 3 million patients, how to protect the drug needs of these patients, is a natural consideration of social and economic development to a certain extent, therefore, the "CPC Central Committee and the State Council on deepening the reform of the medical security system Opinions" emphasizes that to promote the development of multi-level medical security system, Explore the mechanism of drug protection for rare diseases. This not only requires sufficient conditions for economic and social development, but also requires the consensus of the whole society, but there are always some controversies on the core issue of "orphan drugs into medical insurance".

A "fairness theory" believes that allowing more orphan drugs to enter medical insurance is fairness and justice. Another "fairness theory" believes that the incidence of rare diseases is low, the efficacy of drug treatment is unstable, and the medical value is low. At the same time, the medication time is long, the unit price of drugs is high, it is easy to cause high medical cost cases, in the case of China's aging population, the trend of low birthrate, more orphan drugs into medical insurance, will cause greater pressure on the already tight medical insurance fund.

Existing scientific research shows that 80% of rare diseases are caused by genetic genes, 50% of rare diseases begin in childhood, 30% of children die before adulthood, the diagnosis and treatment period is long, the misdiagnosis rate is high, and more than 90% of rare diseases do not have a clear treatment plan. After the synthesis of these factors, it is not difficult to find that patients who can receive orphan drug treatment are also a very small number of rare disease patients, and ensuring the drug demand of this part of the patient will theoretically not cause medical resources and medical insurance funds to be crowded out by "a very small number of people". If we explore the incentive mechanism for the research and development of orphan drugs at the same time, realize the localization of some drugs, and at the same time establish a multi-party sharing mechanism for the medical costs of rare diseases, and share them through various channels such as medical assistance and medical commercial insurance, this cost is expected to be further reduced, the payment pressure is controllable as a whole, and the drug guarantee mechanism as a whole is feasible.

Therefore, it is entirely ideal that the orphan drugs that treat rare diseases will inevitably be prioritized in the list of medical insurance drugs, so that more rare disease patients can be treated; at the same time, the inclusion of orphan drugs has not affected the level of protection of other diseases, the overall coverage of medical insurance has been expanding, and the well-being of the people has been increasing.

However, the reality is much more complicated than this, since March 1, 2021, the new version of the "National Basic Medical Insurance, Industrial and Commercial Insurance and Maternity Insurance Drug Catalog" was launched, 119 kinds of drugs were transferred in, 29 kinds of drugs were transferred out, and the final list had 2800 kinds of drugs, including 1426 kinds of Western medicines, 1374 kinds of Proprietary Chinese medicines, and the proportion of Chinese and Western medicines was basically the same. This is very different from the structure of medical insurance drugs in Western developed countries, under this structure, in the absence of major changes in the total amount of medical insurance funds, orphan drugs are included in the Western medicine part of the medical insurance drug list, which means that some Western drugs may withdraw from the list, resulting in damage to the right to life and health of another part of patients, making the ideal "not suffering from widowhood but suffering from inequality" Datong design into a move to cut flesh and make up for sores, with an unfair compensation for another kind of unfairness.

So is it possible for the Medicare Fund to make the pie bigger and meet the needs of all patients? In the long run is unrealistic, from the development trend of China's fund and demographic changes, the general disk of the medical insurance fund can not continue to expand, but in many areas, the intergenerational payment difference of the medical insurance fund has affected the enthusiasm of young people, especially the independent entrepreneurs to pay insurance, simply put, that is, the young people who pay the funds are small, and the elderly who use the funds are more, so that the young people have a sense of distrust of their future, which is a dangerous trend, when the treatment of common disease patients can not be guaranteed, The fate of patients with rare diseases can also be imagined.

In addition, some drugs with controversial efficacy, together with other drugs and preparations whose "adverse effects and contraindications are not yet clear", occupy a considerable proportion of the list, and naturally become the bulk of the payment. In the case of the controversial GV-971, medicare pays billions of dollars a year based on the capacity advertised by its R&D companies.

Orphan drug protection is not a train dilemma, and rare disease relief still has a long way to go. In the fairer, more just society we all seek, no one should be sacrificed or abandoned. But at the same time, in this society, the interests of the minority are always closely related to the interests of the majority, and on the issue of orphan drugs and even any drug in medical insurance, we should consider the improvement of cost-effectiveness ratio and overall fairness and justice, so that medical insurance can better help more people.

(The author is a university teacher)

(This article is only the author's personal opinion and does not represent the position of this newspaper)

Liu He