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Discussion on the positioning of rare disease catalogue and the improvement of drug innovation and accessibility

 Last Update: 2023-01-05
 Source: Internet
 Author: User

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On November 11, 2022, AstraZeneca announced that eculizumab was officially launched in China and the first order was sold in Hangzhou, which was conditionally marketed in NMPA four years ago through the "Clinical Urgent Overseas New Drug Review and Approval Work Procedures", and is currently the only drug
approved in China for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) in adults and children.

An imported new drug approved for marketing did not officially start sales until 4 years later, which reflects the problems encountered in market access and payment for rare disease drugs approved through the accelerated channel
.
For the field of rare diseases, there is still great room
for systematic policy optimization and adjustment.

Since the release of the First Rare Disease Catalogue in 2018, the field of rare diseases has been widely valued in China, the investment and financing amount and R&D pipeline of pharmaceutical companies have increased year by year, more and more companies focusing on rare diseases have been established, and the listing of CANbridge on the Hong Kong Stock Exchange has become a milestone event in the development of innovative drugs for rare diseases in China
.
However, as the global tide of capital gradually fades, more and more problems
are gradually exposed in the field of rare diseases with immature domestic policies.

First, the problems faced by the development of China's rare disease industry

Problems faced by the development of China's rare disease industry

1.
The diagnosis and treatment needs of patients with rare diseases need to be solved urgently

China has included rare diseases with strong demand for diagnosis and treatment into the catalog management, and the definition, etiology and epidemiology, clinical examination, auxiliary examination, diagnosis, differential diagnosis and treatment
of these 121 diseases have been elaborated in detail.
A national diagnosis and treatment cooperation network was established to give full play to the leading role of high-quality medical resources, and on this basis, medical staff training was carried out nationwide to improve the clinical diagnosis and treatment level
of clinicians for these 121 rare diseases.

However, the number of rare diseases in the directory is limited, according to the official website of the American Organization for Rare Diseases (NORD), there are currently more than 7,000 rare diseases known in the world, corresponding to more than 300 million patients; According to the "China Rare Disease Definition Report 2021" released in 2021, the number of rare diseases in China exceeds 1,400, corresponding to more than 20 million patients, and the coverage of rare diseases is far less than the actual demand
.

The coverage of rare disease catalogues is much smaller than the actual need

Figure The number of rare diseases and the corresponding number of patients under different standards

Source: Sullivan, "White Paper on China's Industry Trends in the Next 50 Years", "China Rare Disease Definition Report 2021"

For more than 1,000 rare diseases outside the catalog, due to the lack of unified integration into the diagnosis and treatment collaboration network and direct reporting system, it is difficult for clinicians to improve the awareness rate of these diseases, and it is difficult to improve the diagnostic methods, and the corresponding epidemiological data is difficult to improve, which in turn affects the inclusion of such diseases in the rare disease list, forming an "endless cycle"
.

2.
Domestic innovative drugs for rare diseases are still in the "zero to one" development stage

With the deepening of the reform of the drug review and approval system and the opening of the capital exit channel, China's innovation environment has been improved, and innovation achievements have continued to emerge in recent years, and the number of domestic innovative drugs on the market in the past five years has exceeded 70, but the number of innovative drugs in the field of rare diseases is zero, compared with the overall industry of pharmaceutical innovation in China, the development of rare disease innovation field is seriously lagging behind
.

On the other hand, the new rare disease drugs listed in recent years are all imported drugs, reflecting the prominent import dependence of rare disease patients in China, and the rights and interests of rare disease patients in China cannot be protected by domestic drugs, especially in the current increasingly tense international relations, such issues need to be paid special attention
.

Table 1 30 new drugs for rare diseases launched after the publication of the 2018 catalog

Source: 2022 China Rare Disease Industry Trend Observation Report

3.
The accessibility of innovative drugs for high-value rare diseases needs to be improved

As we all know, the research and development of innovative drugs has high investment, high risk and long-term cycle attributes, in addition to facing more commercialization risks
in the field of rare diseases.
Due to the special properties of rare diseases, they face problems such as a small number of patients and difficulty in diagnosis, resulting in greater market risks
after the launch of innovative drugs for rare diseases.

The high price of rare diseases is inevitable due to "double" high risk, and China's medical insurance emphasizes "ensuring the basics", and pursues more squeeze price "moisture" to ensure the availability of drugs for all people
.
According to incomplete statistics, there are still 14 diseases of all treatment drugs not included in medical insurance, including 9 high-value drugs with an annual treatment cost of more than 500,000 yuan, the number of patients registered for such drugs is relatively small, and it is difficult to develop the market, but at the same time, it has also caused the situation
of "no drugs available" for patients.

Table 2 Statistics of drugs with an annual treatment cost of more than 500,000 yuan outside the medical insurance list

Note: The number of people with Gaucher disease type I is based on its account for 90% of all Gaucher disease Scale estimates
.
Drug prices refer to the "China Rare Disease Medical Insurance City Report 2020" and pharmaceutical company data, and consider the charitable drug donation plan
of the enterprise.
The annual treatment cost of irosulfatase α is calculated as an average adult weight of 15 kg, a child with an average body weight of 7.
5 kg, laronidase with an average body weight of 40 kg, edulfate β at 25 kg for adults and 7.
5 kg for children, imigresidase with an average body weight of 40 kg, and a arabinase α with an average body weight of 25 kg
.
Sttuximab is calculated
at body weight 60 kg.

Source: 2022 China Rare Disease Industry Trend Observation Report

Second, some thoughts on rare disease drug policy

Some thoughts on rare disease drug policy

The report of the 20th National Congress proposed that "promoting the construction of a healthy China" requires "deepening the reform of the medical and health system and promoting the coordinated development and governance of medical insurance, medical treatment and medicine"
.
The development dilemma of the rare disease industry is not caused by isolated policy problems, and systematic policy optimization and adjustment
are required to promote the development of the rare disease industry and meet the clinical needs of rare disease patients.

1.
The rare disease directory should not become a directory that "defines rare diseases"

China is the only country that manages rare diseases under the catalogue system, but the existence of rare diseases themselves is not transferred by the formulation of the catalogue
.
At present, a variety of policies and preferential treatment have been formulated for rare diseases in the catalog, and a collaborative network and direct reporting system for rare disease diagnosis and treatment have been established from the diagnosis and treatment of diseases, and the registration of patients with rare diseases in the catalog has been promoted.
From the aspect of approval and review, establish a priority review channel for innovative drugs for rare diseases; In terms of drug payment, rare disease drugs in the catalogue enjoy multi-level medical security systems such as urban universal insurance of multiple local governments and assistance systems
of various charities.

For rare diseases outside the list, they should enjoy the same policy preferences, and the rare disease list should not become a means to define rare diseases, and rare diseases should be defined from the perspective of incidence and number of patients in a scientific way, and at the same time, some rare diseases that are urgently needed clinically can be selected to be included in the directory to rationally allocate resources
.

2.
The inclusion of the rare disease catalogue should be "innovation-oriented"

The inclusion criteria of the rare disease catalogue also need to be optimized and upgraded, and two of the four major criteria currently included are "there is a clear diagnostic method" and "there is a treatment or intervention method, affordable or there is no effective treatment or intervention method, but it has been included in the national scientific research project"
.
This reflects the limitations of the current rare disease inclusion standards on the definition of rare diseases, emphasizing "disease treatment" and light on "innovative research and development", which will form an "invisible wall" from the perspective of new drug research and development, restricting the exploration
of new areas of rare diseases.

3.
The improvement of drug accessibility for patients with rare diseases requires the coordinated optimization of the "three drugs" policy

In the face of the current problems such as import dependence of rare disease drugs, payment dilemma, and obstruction of access to medical institutions, it is necessary to form a "three-drug linkage" from the perspectives of medicine, medical insurance and medical treatment to promote the development of
innovative rare disease industries.

From the perspective of medicine, it is necessary to further relax the limit on the number of clinical trials on the current basis and open up more acceleration channels, and appropriately extend the data protection period and market exclusivity period of innovative drugs for rare diseases, so as to stimulate the enthusiasm of pharmaceutical enterprises for research and development in this field;

From the perspective of medical insurance, patients with rare diseases should enjoy the same medical insurance reimbursement rights as patients with other diseases, and it is recommended that medical insurance establish a separate payment system for rare diseases, and change the "drug price" to determine the "payment standard" for rare disease drugs, and leave the rest to market competition, so that more rare disease drugs can be included in medical insurance without excessive pressure on the medical insurance fund, and this also leaves room for the development of commercial insurance;

From a medical point of view, for accelerated approval of innovative drugs for rare diseases, their use
in medical institutions cannot be limited due to insufficient clinical evidence.
It is recommended to abolish the system such as the Pharmaceutical Council, select some hospitals with strong diagnosis and treatment capabilities, and use real-world research as a means to open access to innovative drugs for rare diseases, improve the accessibility of drugs for patients, and form strong clinical evidence to further determine and adjust the scope
of use.

Caring for patients with rare diseases is the responsibility of the whole society, and whether vulnerable groups can be fully protected is also a sign of the maturity of modern society, and every small group should not be abandoned
.

Policy Research Center, China Association for the Promotion of Pharmaceutical Innovation

Author: Yangyang Zhang, Assistant Researcher zhangyy@phirda.
com

Review proofreader: Zhang Zhijuan, Liu Fapeng

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