The rare disease drug market may approach 40 billion!

　　In recent years, the state has issued a series of policies to encourage the research and development and production of drugs for rare diseases
.
According to data from Minet.
com, 53 rare disease drugs have been included in the national medical insurance, including human immunoglobulin, rosuvastatin, ezetimibe and other large varieties with terminal sales of over 1 billion yuan in China's public medical institutions in 2021
.
September is approaching, and the 2022 edition of the National Talks is gradually approaching.
18 rare disease drugs are ready to be released.
The final medical insurance access situation is expected to be known by the end of the year
.
Rare diseases are "not uncommon", and the demand for drugs is surging.
Statistics show that the prevalence of rare diseases in the population is about 3.
5%-5.
9%.
Although the prevalence of 84.
5% of the diseases is less than one in a million, China The total number of rare disease patients has reached about 20 million, and the number of new patients is increasing at a rate of more than 200,000 every year
.
In view of China's huge population base, compared with other countries and regions, many rare diseases have become "not rare" in China
.
Driven by the demand for clinical drugs, the rare disease drug market continues to heat up
.
A professional organization in the industry pointed out that the Chinese rare disease drug market accounted for 0.
4% and 1% of the global rare disease market in 2016 and 2020 respectively.
43.
8 billion yuan)
.
However, domestic pharmaceutical companies have insufficient research and development motivation for rare disease drugs .
On the one hand, the coverage of rare disease drugs is not wide; relatively low
.
Aside from the economic attributes of rare disease drugs, from a technical point of view, the main dilemma in the current development of rare disease drugs is still the long-term clinical advancement problem, and the main reasons for the slow clinical advancement are as follows: 1.
Rare disease registration The improvement of the system must be accelerated.
The establishment of the system can objectively, truly and systematically collect relevant data on epidemiology, genomics, clinical manifestations, and treatment outcomes.
It can not only provide accurate epidemiological data of rare diseases in China, but also clarify the disease .
It can also explore the pathogenesis to improve the level of diagnosis and treatment, and provide a basis for the formulation of rare disease-related policies
.
2.
The clinical trial plan should be more innovative.
In the process of clinical trials, drugs for rare diseases will encounter many difficulties such as small number of patients, dispersion, and difficulty in enrolling.
Compared with other new drugs, the clinical process will definitely be relatively delayed; There are technical differences in the diagnosis of patients, resulting in undiagnosed diagnosis, lack of biomarkers for prognostic assessment, and lack of efficacy and prognostic indicators in clinical trial design
.
Incentive policies emerge one after another, and the research and development of rare disease drugs has come.
In order to encourage domestic pharmaceutical companies to increase the research and development and production of rare disease drugs, in recent years, national government departments have successively issued a series of incentive policies
.
Sources of some of the relevant policies issued by the state to encourage the R&D and production of drugs for rare diseases in the past ten years: the State Food and Drug Administration, the National Health Commission, etc.
, compiled by Minet.
In 2022, CDE has successively issued the "Technical Guidelines for Clinical Research and Development of Drugs for Rare Diseases", The "Statistical Guiding Principles for Clinical Research of Rare Disease Drugs (Trial)" has two clinical related guiding principles for rare disease drugs, which undoubtedly provides new ideas for the formulation of rare disease drug clinical trial plans, and also forms an important technical assistance
.
In the first batch of rare disease catalogs, the medical insurance coverage rate is only 21%.
The implementation of the policy fully drives research and development, but it is not ideal from the perspective of rare disease drugs approval and "insurance"
.
According to data from Minet.
com, the 2009 version of the National Medical Insurance Catalog included 22 drugs for rare diseases, covering 13 rare diseases; the 2017 version of the National Medical Insurance Catalog included 30 drugs for rare diseases, covering 15 rare diseases; the 2019 version of the National Medical Insurance Catalog included 39 rare diseases The 2020 version of the National Medical Insurance Catalog includes 45 drugs for rare diseases, covering 23 rare diseases; the 2021 version of the National Medical Insurance Catalog includes 53 drugs for rare diseases, covering 26 rare diseases
.
In June 2018, the National Health and Medical Commission announced the "National First Batch of Rare Disease List", involving 121 diseases; currently only 26 rare diseases are covered by medical insurance, and the coverage rate is only 20%
.
Sources of rare disease drugs that have been included in the medical insurance catalogue: Minet.
com China Medical Insurance Catalogue Daquan Database Among the 53 rare disease drugs included in the national medical insurance, Parkinson's disease (young-onset, early-onset), hemophilia, multiple sclerosis and idiopathic Most of the diseases such as pulmonary arterial hypertension are 5 or more
.
Considering the new drugs approved in recent years and their entry into medical insurance, the indications for rare disease drugs currently on the market still do not fully cover the indications in the first batch of rare disease catalogues, and there is still room for unmet clinical needs
.
The sales of 5 varieties have exceeded 1 billion, but rare diseases are not their main indications.
According to the data from Minet.
com, 5 of the 53 rare disease drugs will be used in urban public hospitals, county-level public hospitals, urban community centers and township health care in China in 2021.
The terminal sales of the hospital (referred to as Chinese public medical institutions) exceeded 1 billion yuan, including intravenous human immunoglobulin (pH4), rosuvastatin calcium tablets, recombinant human coagulation factor VIII for injection, ezetimibe tablets and detemir Insulin injection
.
However, rare diseases are not the main indications of the above products, such as human immunoglobulin, which is a systemic anti-infective drug, mainly used to prevent measles and infectious hepatitis, and used in combination with antibiotics to improve the susceptibility to certain serious bacterial and viral infections.
Efficacy; Rosuvastatin calcium tablets and ezetimibe tablets are classic hypolipidemic drugs; recombinant human coagulation factor VIII for injection was originally used as a hemostatic agent in emergency or elective surgery; insulin detemir injection is commonly used in clinical practice Insulin hypoglycemic agents
.
5 rare disease drugs with sales exceeding 1 billion yuan (unit: 100 million yuan) Source: Minet database At present, most of the rare disease drug market is still small.
On the one hand, it may be because the drug has just been launched and has not yet achieved large volume.
; On the other hand, it may also be because the hospital monitored by the market is not the main hospital for medication
.
However, the market potential of some varieties with rare diseases as the main indications should not be underestimated
.
Nosinagen Sodium Injection for the treatment of spinal muscular atrophy, after negotiation, reduced the price by 95% and entered medical insurance.
According to the data of Minet, the terminal sales of the drug in public hospitals in key provinces and cities in China in 2021 will be less than 50 million yuan, and the Q1 of 2022 It has exceeded 100 million yuan, and the growth rate is amazing
.
In recent years, the sales trend of Nosinagen Sodium Injection in public hospitals in key provinces and cities in China (unit: RMB 10,000) Source: The competition pattern of terminal competition in public hospitals in key provinces and cities in China Ambrisentan tablets for the treatment of idiopathic pulmonary hypertension, entered in 2019 After medical insurance, the market is rapidly increasing.
According to data from Minet, the terminal sales growth rate of this product in public hospitals in key provinces and cities in China will exceed 49% from 2020 to 2021.
In 2022, Q1 sales will reach 5.
58 million yuan, and the market potential is being released
.
In recent years, the sales trend of Ambrisentan tablets in public hospitals in key provinces and cities in China (unit: 10,000 yuan) Source: Minet.
com, the competition pattern of public hospitals in key provinces and cities Improving the availability of medication for patients is the ultimate goal and mission of the company
.
In 2022, the national talks are on the way, and 18 rare disease drugs have become "hot players" to "enable more rare disease drugs to be included in medical insurance as soon as possible", and the country will spare no effort
.
In June this year, the 2022 National Medical Insurance Catalog Adjustment Plan (hereinafter referred to as the Adjustment Plan) was officially released
.
Compared with last year, the new round of medical insurance negotiation process is basically the same as the key policies.
The difference is that the focus on rare disease treatment drugs is emphasized, and the application conditions are relaxed: the application conditions include approval before June 30 this year.
of rare disease drugs, but not required to be approved after 2017
.
According to incomplete statistics, there are currently 18 drugs for rare diseases that are expected to participate in the 2022 national talks, among which there are many therapeutic drugs for rare diseases such as mucopolysaccharidosis, hemophilia and multiple sclerosis; the dosage forms are injections, capsules In terms of sub-categories, other gastrointestinal and metabolic drugs, anti-tumor drugs and immunosuppressants occupy 4 or more seats respectively
.
Source of some rare disease drugs that have the opportunity to participate in the 2022 national talks: Minet.
com MED2.
0 Chinese drug review database has come to an end in August.
According to the work arrangement of the "Adjustment Plan", the drugs and information that have passed the preliminary review of medical insurance are expected to be carried out in the near future.
According to the announcement, the varieties in the list will be regarded as the "tickets" for this round of state talks
.
At that time, which varieties can be successfully selected, we will wait and see! Data sources: Minet database, official website of the State Food and Drug Administration, etc.
Note: Minet's "Competitive Landscape of Public Medical Institutions in China", the statistical scope is: China's urban public hospitals , county-level public hospitals, urban community centers and township health centers, no Including private hospitals, private clinics, and village clinics; the chemical drug terminal competition pattern database of public hospitals in key provinces and cities of Minet The sample hospital database of sample provinces and cities for continuous monitoring of all categories; the above sales are calculated based on the average retail price of products in the terminal
.
Rare diseases are "not uncommon", and the demand for drugs is surging .
Drugs　　1.
The registration system for rare diseases must accelerate the improvement of diseases　　.
2.
Clinical trial plans must be more innovative.
The medical insurance coverage rate is only 21%.
The sales of 5 varieties have exceeded 1 billion, but rare diseases are not their main indications .
Enterprises　　are on the way in 2022, and 18 rare disease drugs have become "hot players" for　hospital procurement.