Prospects for drug protection for rare diseases under the new situation

In recent years, China has paid more and more attention to the protection of drugs for rare diseases
.

In 2020, the "Opinions on Deepening the Reform of the Medical Security System" issued by the Central Committee of the Communist Party of China and the State Council clearly proposed to "explore the drug security mechanism for rare diseases"

1.
The new situation of drug protection for rare diseases

2021 is the first year of the "14th Five-Year Plan", and it is also after China has built a moderately prosperous society in an all-round way and achieved the first centenary goal, taking advantage of the momentum to start a new journey of building a socialist modernized country in an all-round way and striving towards the second centenary goal.
The first five years of the march
.

Important policies such as the "Medical Security Law (Draft for Comment)" and the "14th Five-Year Plan for Universal Medical Security" have been promulgated

01.
Formulate basic policies for medical security at the national level

On January 19, 2021, the National Medical Insurance Administration and the Ministry of Finance issued the "Opinions on Establishing a Medical Insurance Benefit List System" (Medical Insurance [2021] No.
5)¹
.

The opinion requires that localities shall not set up other medical security systems beyond the scope of the basic system framework by themselves, and all localities shall strictly follow the national basic medical insurance drug catalogue

Because it is related to the basic policy of medical security, the state also requires local governments to clean up and standardize the policies and measures that do not conform to the list before the list is issued.
The time limit is in principle three years
.

How to adjust the current special policy of paying expenses outside the scope of the policy through basic medical insurance, critical illness medical insurance and special funds derived from the above funds? In particular, can the model that has included high-value drugs for rare diseases (referring to expensive drugs, patients with high annual treatment costs, some even more than one million yuan, and often require lifelong drugs) into the coverage of local medical insurance can continue? From the current point of view, the adjustment path does not seem clear

02.
Guide social forces to participate in assistance and security

In November 2021, the General Office of the State Council issued the "Opinions of the General Office of the State Council on Improving the Medical Insurance and Relief System for Serious Diseases" (Guo Ban Fa [2021] No.
42)
.

The opinion stipulates that the payment of medical assistance funds should comply with the national regulations on the scope of basic medical insurance payment, that is, it must follow the medical insurance treatment list system and cannot break through the basic medical insurance catalogue

However, the Opinion clearly proposes to actively guide social forces such as charities to participate in assistance and security
.

Especially with regard to the protection of drugs for rare diseases, which is different from previous national-level policies, this opinion has developed ideas and directions, that is, integrating medical security, social assistance, charity assistance and other resources to implement comprehensive protection

In fact, this is in line with the "Decision of the Central Committee of the Communist Party of China on Several Major Issues Concerning Adhering to and Improving the Socialist System with Chinese Characteristics and Promoting the Modernization of the National Governance System and Governance Capability" adopted by the Fourth Plenary Session of the Nineteenth Central Committee of the Communist Party of China
.

The decision proposes to "pay attention to the role of the third distribution and develop social welfare undertakings such as charity"

03.
More rare disease drugs are included in medical insurance at reduced prices

On December 3, 2021, the National Medical Insurance Administration issued the "Notice on the National Basic Medical Insurance, Work Injury Insurance and Maternity Insurance Drug Catalog (2021)"
.

The basic medical insurance list has newly included 7 rare disease drugs, the number is second only to the fields of tumor and anti-infection

Among the newly included rare disease drugs last year, 2 were originally high-value drugs
.
This time, the substantial price reduction through negotiation was included in the national basic medical insurance catalogue, which also achieved a breakthrough of zero national medical insurance for high-value drugs for rare diseases
.
Analysis of the reasons, in addition to the large scale of patients in China, these two drugs are facing the pressure of competing products, so this enables the national medical insurance negotiation to obtain the price of imported high-value drugs that is far lower than the foreign market, or even the lowest price in the world.

.
Therefore, many people are not so optimistic that the national medical insurance will continue to accelerate the inclusion of high-value drugs for rare diseases in the future
.

04.
Policy-based commercial insurance actively includes drugs for rare diseases

In recent years, many places in China have actively explored government-guided urban customized commercial supplementary medical insurance
.
The industry even calls 2020 the "first year" of Huimin Insurance
.
The "14th Five-Year Plan for National Medical Insurance" issued last year clearly stated that: clarify the boundaries of basic medical insurance liability, support commercial insurance institutions to develop commercial health insurance products linked to basic medical insurance, and better cover basic medical insurance coverage.
fees paid
.
And it is necessary to gradually incorporate the application of new medical technologies, new drugs, and new devices into the coverage of commercial health insurance
.
The "Opinions of the General Office of the State Council on Improving the Medical Insurance and Relief System for Serious Diseases" also clearly states: support the development of commercial health insurance and meet security needs other than basic medical security
.
According to reports, as of the end of September last year, at least 120 cities at the prefecture-level and above had launched a pilot program of “Benefiting the People’s Insurance”, with a total of more than 70 million people participating in the insurance
.

These policy-based commercial insurances have begun to play a role in the protection of rare disease drugs
.
For example, the “Huhui Insurance” issued by Shanghai in April last year can pay 50% of the single-product drug expenses in the out-of-pocket medical expenses for specific hospitalizations outside the basic medical insurance catalogue for those with pre-existing conditions, and 70% for those without pre-existing conditions, up to a maximum of 300,000 yuan.
; and 5 high-value drugs for 3 rare diseases are included in the specific high-value drug catalog, 30% of the pre-existing disease can be paid, 70% of the non-pre-existing disease can be compensated, and the maximum compensation can be 1 million yuan
.

2.
Prospects of drug protection for rare diseases under the new situation

Although all parties have paid more and more attention to the protection of drugs for rare diseases in recent years, there are still many patients who need drugs that are not guaranteed support
.
Therefore, many patients cannot afford the cost of specific drugs and can only treat symptomatically or not
.
These patients can only race against time and hope that the special drugs will enter the relevant insurance coverage as soon as possible
.
Based on the above analysis of the situation, in the future, drug protection for rare diseases may need to be accelerated in the following aspects:

01.
Access to more rare disease drugs in the National Basic Medical Insurance List

Basic medical insurance should reflect fairness and inclusiveness for people with different diseases, and patients with rare diseases also have equal rights to use drugs.
Guaranteeing drugs for rare diseases is a reflection of the state and society respecting and protecting the right to health of such small groups
.
Therefore, it is expected that the state will include more drugs for rare diseases that save lives and help in times of crisis into the basic medical insurance catalog
.
Taking into account the financing and treatment level of basic medical insurance, in order to avoid excessive treatment in terms of absolute amount, medical insurance payment quotas can be set for higher-priced drugs, rather than one-size-fits-all or sticking to the reimbursement ratio
.
And you can refer to some international practices to formulate more flexible payment policies, such as setting tiered prices and implementing financial sharing mechanisms
.
This will not only enable the basic medical insurance to fully implement the "basic insurance", but also conform to the basic principle of doing your best and doing what you can at the current stage
.

Regarding the price of drugs for rare diseases, with the increase of competition and other factors, some pharmaceutical companies may enter the national medical insurance through price reduction
.
However, the investment in new drug research and development is high and the risk is high, and pharmaceutical companies also need reasonable returns to have the enthusiasm for new drug research and development
.
In particular, patients with rare diseases rely on imported drugs for a long time.
Under the current policy environment, domestic pharmaceutical companies are very lack of enthusiasm for imitation and research and development
.
If the policy mechanism fails to encourage innovation, in the end the most affected patients are the patients who have no cure
.
Therefore, medical insurance should further balance the relationship between value and price.
"The strategic purchase pursued by medical insurance is not to fight for the 'cheapest' nor to be intimidated by 'high-priced drugs'
.
Instead, medical insurance uses 'vacant cages for birds' to sustainably Realize the greatest common divisor of 'people first, health first'
.
" The price of medicines should be reasonably guided, not just relying on the negotiation and price reduction of the medical insurance sector, but also requires science and technology, industry and information technology, drug supervision, taxation, health and other departments.
Support the R&D, production, distribution, and use of pharmaceutical companies to ensure reasonable commercial returns for pharmaceutical companies
.
Especially for domestic pharmaceutical companies, it is necessary to stimulate their enthusiasm for research and development, which is related to the national economy and people's livelihood and national security
.

02.
Guide social forces to play an important role in the guarantee of drugs for rare diseases

The financing level of basic medical insurance in China is still low.
In the face of the accelerated emergence of various innovative high-value drugs, it is indeed impossible to rely too much on basic medical insurance to pay for it, and it is impossible to solve all rare disease drug problems through basic medical insurance
.
Therefore, it is necessary to give full play to the important role of social forces in the guarantee of drugs for rare diseases under the guidance of the policy of the third distribution and common prosperity
.

At the 2021 China Rare Disease Conference held recently, it was announced that the Chinese Red Cross Foundation and the China Rare Disease Alliance jointly initiated the establishment of the Rare Disease Co-Aid Fund, which will be dedicated to subsidizing rare disease patients with a heavy economic burden due to the disease
.
Some places with a relatively high level of economic and social development and strong social forces (such as Shanghai, etc.
) should have the courage to explore, develop new types of charities guided by the government, fully mobilize social forces, and establish a multi-level and integrated drug protection mechanism for rare diseases.
Establish a charity relief fund for rare diseases, which mainly guarantees drugs for rare diseases outside the national basic medical insurance catalogue, explores a new model for the protection of drugs for rare diseases under the new situation, and provides reference experience for the country and other regions
.

For regions that have introduced protection measures related to drugs for rare diseases (mainly involving drugs that are not included in the national basic medical insurance list), in order to avoid the decline in patient treatment and social conflicts, the state should appropriately extend the policy transition period, allow localities to continue to retain them, and encourage localities According to local conditions, local governments should not give up or slow down the exploration because of the treatment list system.
They should actively explore new mechanisms and methods for the protection of drugs for rare diseases after requesting and reporting according to the procedures
.
In addition, through the practice in many places in recent years, the relevant laws and regulations of policy-based commercial insurance also need to be further improved, and the relevant support policies also need to be further strengthened, so that long-term healthy development can be achieved, and the coverage of rare disease drugs can be further expanded
.

None of the above is separate.
It is necessary to promote the topic and coordinate and integrate, especially in the process of exploration by all parties
.
The comprehensive protection of drugs for rare diseases is expensive and difficult to integrate.
At present, the integration of existing protection models is not high
.
It is hoped that under the new situation, the government, the market, and the society will increase exploration and play an important role in the protection of rare disease drugs
.
It is expected to speed up the construction of a comprehensive insurance model for orphan drugs that has a high degree of integration, a high level of treatment, and can better balance corporate motivation, patient burden and government pressure
.

Author: Kang Qi Shanghai Health and Health Development Research Center

Author: Kang Qi Shanghai Health and Health Development Research Center

[1] The publication date of this document is August 10, 2021
.

[1] The publication date of this document is August 10, 2021
.

Main references:

Main References: Main References:

Illness Challenge Foundation.
The medical assistance system needs to "see" rare disease groups.
2021.

Illness Challenge Foundation.
The medical assistance system needs to "see" rare disease groups.
2021.

Learning Times.
What is a "third assignment"? .
2020.

Learning Times.
What is a "third assignment"? .
2020.

Illness Challenge Foundation.
The 2021 edition of the National Medical Insurance Directory, 7 new types of rare disease drugs + 9 types of renewal information at a glance! 2021.

Illness Challenge Foundation.
The 2021 edition of the National Medical Insurance Directory, 7 new types of rare disease drugs + 9 types of renewal information at a glance! 2021.

Health Times.
Huimin Insurance covers more than 100 cities, with more than 70 million participants.
2021.

Health Times.
Huimin Insurance covers more than 100 cities, with more than 70 million participants.
2021.

Cai Jiandao.
Bi Jingquan: New drug research and development, high risk should have high return.
2021.

Cai Jiandao.
Bi Jingquan: New drug research and development, high risk should have high return.
2021.

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