A large number of rare disease drugs are included in the guarantee!

Medical Network, March 22.
On March 18, the Chengdu Municipal Medical Security Bureau officially issued 6 documents in a row, announcing that in accordance with the principle of The rare-disease drug guarantee mechanism based on “control and shared responsibility” has established a separate guarantee fund for rare-disease drug use, and announced the first batch of 7 rare-disease drugs included in the guarantee
.
Fair and appropriate treatment guarantee is an inherent requirement for improving people's health and well-being
.
In May last year, the "Hui Rong Insurance" under the guidance of the Sichuan Medical Security Bureau and the Chengdu Medical Security Bureau was officially launched, using a policy-based commercial insurance model to include 16 high-incidence tumor drugs and 4 rare disease treatment drugs into the guarantee
.
This time, Chengdu further improved the protection mechanism for drugs for rare diseases, aiming to integrate effective resources such as medical insurance, medical assistance, commercial insurance, social charity, and corporate responsibility through the establishment of a special fund to ease the financial burden on families of patients with rare diseases and increase the use of drugs for rare diseases.
Accessibility, improve the physical health of patients with rare diseases
.

Clarify the
“460,000 annual fee” threshold for medication protection.

In recent years, China has attached great importance to the protection of rare diseases and “orphan drugs”.
It has issued a number of policies at the national level and prioritized review and approval of “orphan drugs”.
In 2018, the five national ministries and commissions jointly issued the "First Batch of Rare Disease Catalogues"; at the same time, the 2018 and 2020 editions of the National Medical Insurance Catalogue have successively included rare disease-related drugs, and the 2020 edition of the National Medical Insurance Drug Catalogue has included a total of 24 rare diseases.
55 kinds of therapeutic drugs have improved the availability of medicines for patients with rare diseases in our country
.
Regarding the medical protection of rare disease drugs, many regions have carried out unique local practices based on local conditions.
Zhejiang, Shanghai, Shandong, Shenzhen, Anhui and other cities have comprehensively used financial subsidies, established special funds, and carried out major disease negotiations.
And other models, explore and accumulate experience, introduce policies to encourage active participation of multiple parties, and provide assistance to patients with rare diseases through multiple channels and diversification
.
Expert analysis pointed out that Zhejiang, Taiwan and other regions established and improved the medical assistance system for major and rare diseases when the per capita GDP reached or exceeded 100,000 yuan.
According to data from the Chengdu Development and Reform Commission, the per capita GDP exceeded the 100,000 yuan mark for the first time in 2019.
At this stage, it is the right time to perfect and construct the guarantee system for rare disease drugs
.
According to the policy issued this time, Chengdu's rare disease medication guarantee funds mainly come from Chengdu's critical illness medical mutual aid supplementary insurance funds, with separate account accounting and zero balance
.
The fund will raise rare disease protection funds based on 2% of the total funding of the critical illness medical mutual assistance supplementary insurance in the previous year.
The Municipal Medical Insurance Bureau and the Municipal Finance Bureau will adjust the financing ratio according to the operation of the guarantee funds
.
Previously, the Chengdu Municipal Medical Security Bureau, Chengdu Municipal Finance Bureau, and Chengdu Municipal Health Commission jointly issued the "Guiding Opinions on Establishing a Mechanism for the Use of Drugs for Rare Diseases", implementing an annual total cost limit access system for rare disease drugs: Annual total The cost limit does not exceed the sum of the medical insurance payment limit and the patient’s personal burden limit.
The patient’s personal burden limit should be determined between 1 to 2 times the per capita disposable income of Chengdu residents; medicines whose annual total cost of medicines are lower than the limit standard shall be determined.
It can be included in the coverage of rare disease medication
.
In accordance with this principle, the Chengdu Municipal Medical Insurance Bureau clarified in the "Notice on Issues Concerning the Protection of Drugs for Rare Diseases" that domestically approved drugs for rare diseases that have been marketed can apply to be included in the scope of the drug protection if they meet the following requirements: (1) Drug adaptation The disease is a disease listed in the National Rare Disease Catalog; (2) The drug is a rare disease innovative drug that meets the characteristics of a breakthrough treatment drug; (3) Under the conditions of a medical insurance payment of 400,000 per year and a personal burden of 60,000 per year , Patients can obtain continuous and standardized medication
.
As the most economically powerful city in Sichuan Province, Chengdu has consistently led the province in terms of residents’ income, and has a relatively high per capita disposable income.
Taking 2019 as an example, the city’s per capita disposable income of residents was 39,503 yuan.

.
With reference to economic data calculations, and comprehensively considering the income differences and burden levels between urban residents and rural residents, the “personal burden per year” limit is basically in line with the local economic situation
.

　　Sustainable multi-party financing
　　promote a multi-level security system
Medical security is a major institutional arrangement to reduce the burden of medical care, improve people's livelihood and well-being, and maintain social harmony and stability
.
In order to thoroughly implement the decision and deployment of the 19th National Congress of the Communist Party of China on the comprehensive establishment of a medical security system with Chinese characteristics, and to focus on solving the problem of unbalanced and insufficient medical security development, the "Opinions of the Central Committee of the Communist Party of China and the State Council on Deepening the Reform of the Medical Security System" specifically pointed out the need to "promote The development of a multi-level medical security system" "exploration of a guarantee mechanism for drugs for rare diseases"
.
The industry generally believes that diversified and multi-channel models need to be explored around the protection of rare disease drugs.
At the same time, the breadth, depth, diversity of scenarios, and sustainability of multi-party financing of the rare disease protection management system have received extensive attention
.
In terms of the breadth of coverage, since January this year, Chengdu’s drugs for the protection of rare diseases have been solicited and applied for.
The first batch of 7 shortlisted drugs was released during the same period when the policy was implemented, covering tetrahydrobiopterin deficiency and spinal muscular atrophy.
Drugs in the treatment of seven rare diseases, such as disease, mucopolysaccharidosis, Gaucher disease, idiopathic cardiomyopathy (ATTR-CM), pulmonary hypertension, and Pompe disease
.
These drugs not only include arabinosidase alpha for injection and imiglucerase for injection that have been included in the local rare disease drug protection system in some provinces before, but also include noxinasheng sodium injection that has not entered the protection scope of other places.
Price variety
.
Taking into account that the National Medical Insurance Bureau has included more than 40 of the more than 50 rare disease drugs on the market in China with indications into the national medical insurance drug list, the coverage of Chengdu has been further expanded
.


In terms of the depth of protection, Chengdu attaches great importance to the actual payment of various types of insurance for rare disease drugs.
The cost of drugs adopts the method of "accumulative calculation and reimbursement" within a treatment year: 200,000 yuan (including 200,000 yuan) in a treatment year The reimbursement ratio of the drug expenses within the scope is 70%, and the drug expenses in the range of 200,000 yuan to 460,000 yuan (including 460,000 yuan) shall be settled by the guarantee funds according to the facts
.
At the same time, companies are strictly required to “do not reduce the strength of the original donation of drugs that have a domestic drug donation program that is included in Chengdu’s drug protection for rare diseases
.
” In terms of multiple scenarios, Chengdu’s rare disease drug guarantee mechanism requires that those with the ability to diagnose and treat rare diseases Medical institutions are responsible for providing identification and diagnosis and treatment services for rare disease patients.
Special drug pharmacies selected by rare disease drug manufacturers (suppliers) are designated as the designated distribution organization for rare disease drugs.
Patients with rare diseases should seek medical treatment at designated rare disease medical institutions.
Purchase drugs and settle accounts at designated pharmacies for rare disease drugs
.
Multiple scenarios such as hospitalization, outpatient clinics and dual-channel pharmacies are being opened up
.
In terms of multi-party financing and sustainability, Chengdu’s rare disease drug protection mechanism proposes to integrate effective resources such as medical insurance, medical assistance, commercial insurance, social charity, and corporate responsibility.
Critical illness medical mutual aid supplementary insurance funds are shared with drug manufacturers (or suppliers); drug companies are encouraged to collaborate with other social organizations to propose innovative payment schemes to reach the total annual drug cost limit; guide and support social organizations and commercial health insurance to participate in rare diseases The family burden guarantee of patients is to provide assistance to families who still have financial difficulties after the implementation of the protection of drugs for rare diseases, so as to accurately reduce the financial burden of the families of patients with rare diseases
.
In fact, the exploration of innovative protection models for rare disease drugs must be based on China's existing medical insurance system, and provide solutions for the drug protection of rare disease patients without breaking the original medical insurance rules.
No model construction can be accomplished overnight.
Chengdu The Municipal Medical Insurance Bureau has taken a crucial step
.

　　Some rare disease drugs that have been marketed but not included in the national medical insurance list

Rare disease indications	Generic name of the drug	enterprise
Atypical hemolytic uremic disease	Eculizumab	AstraZeneca
Paroxysmal nocturnal hemoglobinuria	Eculizumab	AstraZeneca
Mucopolysaccharidosis type I	Laronie Enzyme Concentrated Solution	Sanofi
Pompe disease	Arabinosidase alpha	Sanofi
Gaucher disease	Imiglucerase	Sanofi
Fabre disease	Agalsidase beta	Sanofi
Agalsidase alpha	Takeda
Hereditary Angioedema (HAE)	Lanarizumab	Takeda
Spinal muscular atrophy	Nosinagen Sodium	Bojian
Mucopolysaccharidosis Type IVA	Elosulfatase Alpha	BioMarin (Billion Miles)
Tetrahydrobiopterin deficiency	Sapropterin	BioMarin (Billion Miles)
Hyperphenylalanineemia	Sapropterin	BioMarin (Billion Miles)
Idiopathic cardiomyopathy (ATTR-CM)	Clofloxacin soft capsules	Pfizer
hemophilia	Iimerizumab	Roche
Neurotrophic keratitis	Senegemin eye drops	Topei Pharmaceutical
Mucopolysaccharidosis type II	Idol Sulfatase β	Beihai Kangcheng
pulmonary hypertension	Treprostinil Injection	Zhaoke Pharmaceutical

　 　　Chengdu Medical Security Bureau
　　Chengdu
　　City Health Commission
　　``Guiding Opinions on Establishing a Mechanism for Protecting Drugs for Rare Diseases''
Sichuan Tianfu New District Community Governance and Social Affairs Bureau, Sichuan Tianfu New District Finance and Finance Bureau, Chengdu Eastern New District Public Service Bureau, Chengdu Eastern New District Finance Bureau, Chengdu High-tech Zone Social Security Bureau, Chengdu High-tech Zone Finance and Finance Bureau, Chengdu High-tech Zone Education, Culture, and Health Bureau, District (City) County Medical Insurance Bureau, Finance Bureau, Health Bureau, and Municipal Medical Insurance Affairs Center , Municipal Medical Insurance Information Center: In order to promote the construction of a multi-level medical security system and resolve the catastrophic expenditure risk of high drug costs on the families of insured persons, according to the "Opinions on Deepening the Reform of the Medical Security System" by the Central Committee of the Communist Party of China and the State Council (Zhongfa [2020] No.
5) In the spirit of the document, we now propose the following guidelines on the establishment of a guarantee mechanism for the use of rare diseases in our city
.

　　1.
Guiding ideology Guided
by Xi Jinping Thought on Socialism with Chinese Characteristics for a New Era, let the people share the fruits of social development, adhere to people’s health as the center, and further improve a more equitable and sustainable multi-level by building a guarantee mechanism for the use of rare diseases in our city.
The medical security system continuously enhances the people's sense of gain, happiness, and security
.

　　2.
Principles and Objectives
In accordance with the principle of “doing your best, doing what you can, personal burdens can be sustained, and sustaining”, through the integrated use of effective resources such as medical insurance, medical assistance, commercial insurance, social charity, and corporate responsibility, the establishment of a “burden controllable” "Responsibility sharing" mechanism for guaranteeing drugs for rare diseases will ease the financial burden of the families of patients with rare diseases in our city, increase the availability of drugs for rare diseases, and improve the health of patients with rare diseases
.

　　3.
Main content
(1) Guarantee objects
.
Rare disease medication protection is targeted at patients who are covered by Chengdu Mutual Medical Mutual Aid Complementary Insurance for Critical Diseases
.
The conditions for setting the guarantee object should consider factors such as the number of years of insurance and the prevention of operational risks of medical insurance funds
.
(2) The scope of protection
.
The coverage of rare disease medications is based on the disease categories in the National Rare Disease Catalog.
For those diseases that are applicable to the rare diseases catalogue, are listed in China, have definite curative effect, urgently needed treatment, and affordable, and the indications are not covered by the National Basic Medical Insurance, Innovative drugs covered by the "Work Injury Insurance and Maternity Insurance Drug Catalog" can be included in the coverage of rare disease drugs
.
(3) Fund raising
.
The main source of funds for the protection of drugs for rare diseases is the supplementary insurance funds for major illness medical mutual aid in Chengdu
.
The scale of fund-raising is in line with the level of local economic development, separate accounts are set up, and the balance is cleared
.
Supplementary supplementary insurance for critical illness medical care will raise funds for rare-disease medications in a natural year and a certain percentage of the amount of funds raised, and establish a supporting mechanism for medical insurance and the risk-sharing mechanism for pharmaceutical companies to ensure the smooth operation of funds for rare-disease medications
.
(4) Guarantee level
.
The protection of drugs for rare diseases should aim to prevent catastrophic family expenditures, and comprehensively consider the income level of the city’s residents, the ability to raise funds for medical insurance, and the number of patients protected, and reasonably determine the protection standards for drugs for rare diseases
.
(5) Confirmation of drugs
.
Rare disease drugs implement an annual total cost quota access system
.
The annual total cost limit does not exceed the sum of the medical insurance payment limit and the patient's personal burden limit.
The patient's personal burden limit should be determined between 1 to 2 times the per capita disposable income of Chengdu residents
.
Only drugs whose total annual drug cost is lower than the limit standard can be included in the scope of protection for rare diseases; for drugs whose total annual drug cost exceeds the limit, pharmaceutical companies are encouraged to cooperate with other social organizations to propose innovative payment schemes to reach the total annual drug cost limit, otherwise The drug is not included in the scope of protection for rare diseases
.
(6) Other
.
Guide and support social organizations and commercial health insurance to participate in the protection of the family burden of patients with rare diseases, and provide assistance to families still in financial difficulties after the implementation of drug protection for rare diseases, and accurately reduce the financial burden of families with rare diseases
.

　　4.
Medical insurance management
(1) Fund management
.
The management of rare disease protection funds shall be implemented with reference to the relevant systems of the current social insurance funds, and fund management mechanisms such as fundraising, use, over-expenditure sharing, and liquidation shall be established
.
Establish a rare disease protection fund supervision mechanism, improve the risk management system for reporting complaints, information disclosure, internal control, and fraud prevention to ensure the safety of funds
.
(2) Handling management
.
Establish and improve the management methods for the handling of drugs for rare diseases, and specify the scope of drug security, security standards, supervision and management and other regulations
.
Optimize and improve the information system, relying on information technology, and implement the whole-process management of treatment for those included in the protection of rare diseases
.
Strengthen the management of designated rare disease medical institutions, designated rare disease treatment physicians, and designated rare disease drug supply institutions
.
Improve the handling service standards, optimize handling rules and procedures, strengthen agreement management, refine patient management, and improve the ability and efficiency of rare disease drug protection handling management services
.

　　"Notice on Issues Concerning the Protection of Drugs for Rare Diseases"
Sichuan Tianfu New District Community Governance and Social Affairs Bureau, Sichuan Tianfu New District Finance and Finance Bureau, Chengdu Eastern New District Public Service Bureau, Chengdu Eastern New District Finance and Finance Bureau, Chengdu High-tech Zone Social Governance Security Bureau, Chengdu High-tech Zone Finance and Finance Bureau, Chengdu High-tech Zone Education Culture and Health Bureau, district (city) and county medical insurance bureaus, finance bureaus, health bureaus, municipal medical insurance affairs centers, and municipal medical insurance information centers: To promote the construction of a multi-level medical security system and resolve the disasters caused by rare diseases to the families of insured persons The risk of sexual expenditure, in accordance with the "Guiding Opinions of Chengdu Municipal Finance Bureau on Establishing a Mechanism for Protecting Drugs for Rare Diseases" (Chengyibaofa [2021] No.
1) of the Chengdu Municipal Medical Security Bureau, the city is now establishing "controllable costs and responsibility The related issues of the drug protection mechanism for rare diseases (hereinafter referred to as drug protection) of "shared responsibility" are notified as follows
.

　　1.
Objects of protection
(1) For the first time applying for the protection of rare disease medication, one of the following conditions shall be met: 1.
Patients over 5 years old (including 5 years old) shall be in the benefit period of Chengdu Mutual Medical Mutual Assistance Insurance for Major Diseases.
And the accumulative number of months of insurance payment has reached 60 months
.
2.
Patients under the age of 5 should be in the benefit period of Chengdu's critical illness medical mutual assistance supplementary insurance benefits, and either of their parents has participated in the critical illness mutual medical supplementary insurance, and the cumulative number of months of participating in the insurance payment has reached 60 months
.
(2) Those who have already enjoyed the protection benefits of drugs for rare diseases, the re-application should be in the benefit period of Chengdu's critical illness medical mutual assistance supplementary insurance benefits
.

　　2.
The scope of protection The
domestic approved drugs for rare diseases that have been marketed, and meet the following requirements, can apply for inclusion in the scope of protection for this medication: (1) The indications of the drugs belong to the diseases listed in the National Rare Disease Catalog; (2) The drugs are compliant Innovative drugs for rare diseases with characteristics of breakthrough therapeutic drugs; (3) Under the conditions of a medical insurance payment of 400,000 per year and a personal burden of 60,000 per year, patients can obtain continuous standardized medication
.
If the medicines that meet the above conditions are included in the "National Basic Medical Insurance, Work Injury Insurance, and Maternity Insurance Drug Catalog" under the second paragraph of their indications, the corresponding drug indications will not be included in or withdrawn from this medication guarantee Range
.
Newly approved drugs for rare diseases in China that meet the above conditions and are not higher than the annual drug costs of drugs with the same indication that have been included in the coverage can apply to be included in the coverage of drug coverage
.

　　3.
Guarantee funds
Rare disease medication guarantee funds (hereinafter referred to as "guarantee funds") mainly come from Chengdu's critical illness medical mutual aid supplementary insurance funds, and separate accounts are set up for accounting, and the balance is cleared
.
(1) Source of funds
.
Raise funds for rare disease protection based on 2% of the total funding of the critical illness medical mutual assistance supplementary insurance in the previous year
.
The Municipal Medical Insurance Bureau, in conjunction with the Municipal Finance Bureau, will adjust the fund-raising ratio according to the guarantee fund operation
.
(2) Over-expenditure sharing
.
When the annual security funds is expected to exceed the annual amount of money to use to protect from catastrophic medical insurance funds and mutual supplement pharmaceutical production enterprise shared (or supplier)
.
The specific sharing method shall be separately formulated by the municipal medical insurance agency
.
(3) The balance is returned
.
The current balance of the guarantee funds shall be cleared to zero
.

　　4.
Guarantee standard
Guarantee funds for the cost of medicines used by patients in compliance with the provisions of this notice shall be calculated cumulatively and reimbursed in stages within a treatment year
.
The annual drug cost limit is 460,000 yuan, of which the medical insurance payment limit is 400,000 yuan and the personal burden limit is 60,000 yuan
.
The reimbursement rate for medicines within 200,000 yuan (including 200,000 yuan) in a treatment year is 70%, and the medicine expenses within the range of 200,000 yuan to 460,000 yuan (including 460,000 yuan) shall be settled by the guarantee funds according to the facts
.

　　5.
Management services
Rare disease management services mainly include the scope of protection of rare disease drugs, certification standards, institution management, medical treatment management, settlement management, and agreement management
.
The scope of drugs is determined by enterprise declaration and expert review
.
Experts formulate the "Chengdu City Rare Disease Drug Coverage and Drug Recognition Standards" based on rare disease diagnosis and treatment guidelines, clinical drug requirements, drug instructions, drug guidelines, and expert consensus, and establish a dynamic adjustment mechanism
.
Medical institutions with the ability to diagnose and treat rare diseases are responsible for the identification and diagnosis and treatment of rare diseases
.
Special drug pharmacies selected by rare disease drug manufacturers (suppliers) are designated as the designated distribution agency for rare disease drugs
.
Patients with rare diseases should seek medical treatment at designated rare disease medical institutions, and purchase and settle drugs at designated pharmacies for rare disease drugs
.
The Municipal Medical Insurance Affairs Center shall sign an agreement with the rare disease drug manufacturer (or supplier), and the agreement shall be signed for a period of 2 years
.
Pharmaceutical manufacturers (suppliers) shall not voluntarily propose to suspend or terminate the agreement (except for the withdrawal of drugs covered by rare diseases protection from the Chinese market)
.
Based on this, the Municipal Medical Security Affairs Center formulates relevant management measures and organizes their implementation
.

　　VI.
Implementation time
This notice will be implemented from January 1, 2021, and will be valid for 5 years
.

　　7.
Other
municipal medical insurance agencies may formulate handling and management measures based on this notice
.
Prior to the implementation of this notice, the medical insurance policies related to rare diseases will continue to be effective; drugs that have a drug donation program in the country are included in the city’s rare disease drug protection, and the original donation of drugs shall not be reduced
.