Policy support, capital favor, the domestic rare disease market can be expected in the future

In May 2018, the National Health Commission formulated the "First List of Rare Diseases", aimed at strengthening the management of rare diseases in China, improving the level of diagnosis and treatment of rare diseases, and safeguarding the health rights and interests of patients with rare diseases
.

A total of 121 rare diseases are included in the "First Batch of Rare Disease Catalog", which is the first time that China has officially defined a rare disease

Rare disease (Rare Disease), the official definition refers to a very low prevalence, rare disease, generally chronic, serious disease, often life-threatening
.

The World Health Organization defines rare diseases as diseases or lesions in which the number of patients is between 0.

With the popularity of "International Rare Disease Day" on February 28 every year, more and more people begin to pay attention to this field
.

Behind the "seeing" of rare diseases by more and more people is the surging research and development boom in this field from the world to China

01 Drugs for rare diseases-a gold mine yet to be excavated

01 Drugs for rare diseases-a gold mine yet to be excavated

A few years ago, the field of pharmaceutical research and development was highly praised and discussed by capital, and all focused on the field of tumors and chronic diseases
.

The best-selling drugs in the world have been iterating over the past two to three decades.

This is in line with the law of industry development.
In the era when chronic diseases have not been overcome, R&D resources will inevitably be tilted toward patient-intensive areas
.

But when common diseases continue to be overcome, the research and development track in this field will become particularly crowded

Moreover, the return of the rare disease market is not necessarily worse than that of tumors
.

Because most tumors are still incurable and the medication cycle is short; rare diseases are often life-long diseases and require long-term medication.

Many rare diseases are inherited diseases caused by genetic defects, and they often occur in children and adolescents
.

Once treated correctly, most of them can study, work and live normally like healthy people, creating long-term value for the country and society

02 Favorable policies drove domestic investment boom

02 Favorable policies drove domestic investment boom

From a global perspective, there have been many large mergers and acquisitions in the field of rare diseases in recent years
.

In 2018, Takeda Pharmaceuticals acquired the rare disease giant Shire at a price of US$65 billion, which not only set the record for the global pharmaceutical industry's acquisition of that year, but also made Takeda one of the top 10 pharmaceutical companies in the world

However, returning to the Chinese market, for a long time, there were not many investment projects in the field of rare diseases or local biopharmaceutical companies
.
There is a huge mismatch between supply and demand in the rare disease market in China
.

Multinational pharmaceutical giants rarely consider bringing rare disease drugs to the Chinese market
.
There are two main concerns: First, the Chinese patients and the government are unable to pay, and the products cannot be sold after they come in; second, China does not pay attention to the protection of intellectual property rights and is soon copied after entering the market
.

Since the formulation of the "First List of Rare Diseases" in 2018, the policy barriers are gradually being breached
.
In terms of rare disease diagnosis and treatment, in February 2019, the National Health and Construction Commission issued the "Notice on Establishing a National Rare Disease Diagnosis and Treatment Collaboration Network".
The first batch of 324 hospitals were selected to form a rare disease diagnosis and treatment collaboration network, led by Peking Union Medical College Hospital
.
In October of the same year, the Health Commission issued a notice on the registration of diagnosis and treatment information for rare disease cases, and organized the development of a rare disease diagnosis and treatment service information system in China
.
In March 2020, the "Opinions on Deepening the Reform of the Medical Security System" issued by the State Council included the protection of drugs for rare diseases
.

Especially after China joined ICH, various standards have been in line with international standards
.
In the past years of medical insurance negotiations, rare disease drugs have been included in the medical insurance list, and local governments are actively exploring different rare disease protection models, bringing new windows for related drugs to enter the field of rare diseases
.

As of May 2020, based on the "First Batch of Rare Disease Catalogue", 38 rare disease drugs have been included in the National Medical Insurance Catalogue, involving 21 rare diseases
.
In the 2020 national medical insurance negotiations, six rare disease drugs were included, involving rare diseases such as systemic sclerosis, multiple sclerosis, idiopathic pulmonary hypertension, and Huntington's disease
.

The expansion of the medical insurance catalog is undoubtedly a huge driving force for the entry of rare disease drugs into the Chinese market
.
Under this positive signal, high-value drugs, tumor drugs, and rare disease drugs are rapidly releasing market potential and increasing attractiveness
.

03 The rare disease market has huge potential, and the prospects for the Chinese market are promising

03 The rare disease market has huge potential, and the prospects for the Chinese market are promising

In 2017, the global rare disease drug market totaled 70.
3 billion U.
S.
dollars, with an annual growth rate of 11%, far higher than the 5.
3% growth rate of prescription drugs
.
It is estimated that by 2024, sales of rare disease drugs will reach 262 billion U.
S.
dollars, accounting for one-fifth of the global prescription drugs.
Faced with such a rapid growth in the rare disease drug market, multinational foreign-funded pharmaceutical companies have stepped up their layout early on.
stepping up the pace to move the field of rare diseases, including multinational pharmaceutical giants Roche, Sanofi, Novartis, Pfizer, GlaxoSmithKline, Ireland Hill companies
.

The global rare disease drug market pattern from 2016 to 2024 (Source: Frost & Sullivan Report)

Due to the large population base in China, the absolute value of rare diseases is not small.
There are about 20 million rare disease patients in China, which is the largest rare disease group in the world
.

In May of this year, a report on the rare disease industry released by the Boston Consulting Group (BCG) showed that there are 2,844 global oncology drug research and development that have entered the clinic, while there are only 776 rare disease research and development pipelines.
Faced with a huge variety of rare disease diseases, There are still few related drug R&D pipelines, but the market demand is huge.
Compared with the R&D pipelines of oncology drugs, the R&D competition for rare diseases is relatively small
.

With the favorable policies and the gradual release of market demand, by benchmarking other therapeutic areas, China's rare disease market is expected to reach 5-7% of the global market by 2030, that is, 60-90 billion yuan, with a compound annual growth rate of 12%
.

At present, China's rare disease cause is still in its infancy, but its development potential is obvious to all
.
Now, many R&D-oriented innovative pharmaceutical companies have begun planning and layout work in the field of rare diseases, such as the start-up Langyu Group and Beihai Kangcheng (Beijing) Pharmaceutical Technology.
The future development is worth looking forward to
.

04 Actively improve the market environment and solve the difficult diagnosis and treatment of rare diseases

04 Actively improve the market environment and solve the difficult diagnosis and treatment of rare diseases

Since 2019, the country has established a National Rare Disease Diagnosis and Treatment Collaboration Network, building a rare disease diagnosis and treatment network covering 1 national-level leading hospital, 32 provincial-level leading hospitals and 291 member hospitals
.
Establish an unobstructed collaboration mechanism, give full play to the leading role of high-quality medical resources, and promote the improvement of clinical diagnosis and treatment capabilities for rare diseases
.

At the same time, the registration of diagnosis and treatment information for rare disease cases was initiated nationwide
.
By registering basic information of patients and their families, clinical history, biological samples, multi-omics results, imaging and pathology image data and other information, it truly reflects the incidence of rare diseases in China
.

With the rapid development of the Internet, the penetration and synergy of information have been enhanced.
The use of the Internet and big data to establish and improve China’s rare disease database will fill up the lack of basic data on rare diseases in China, and help diagnose and treat diseases in China.
, Drug research and development, and policy formulation provide an important basis
.

Rare diseases, as a new area where China's policy support has been strong in recent years, have begun to enter the field of vision of major international pharmaceutical companies, and overseas companies have successively entered the Chinese market with new products
.
For domestic companies that are committed to rare disease drugs, they need to be well positioned, choose a path suitable for their own development, and make longer-term and more stable economic investment in the research and development of rare disease drugs, in order to truly bring more benefits to rare disease patients.
Many effective drugs
.

In the future, in the context of China’s increasingly sound industry policy for drugs for rare diseases, a complete payment system will greatly activate the domestic market for drugs for rare diseases, and more domestic pharmaceutical companies will invest in the research and development of drugs for rare diseases and change patients with rare diseases.
There are few or no drugs to choose from the dilemma
.

refer to:

refer to:

1.
https://doi.
org/10.
1038/s41375-019-0561-2;

1.
https://doi.
org/10.
1038/s41375-019-0561-2;

2.
Wang D, Tai PWL, Gao G.
Nat Rev Drug Discov.
2019 May;18(5):358-378;

2.
Wang D, Tai PWL, Gao G.
Nat Rev Drug Discov.
2019 May;18(5):358-378;

3.
Huajin Securities
.

3.
Huajin Securities
.