The rare disease drug market may approach 40 billion! 5 blockbuster varieties of more than 1 billion were unveiled, and 18 products were pointed to the 2022 national talks

In recent years, the state has issued a series of policies to encourage the research and development and production

Rare diseases are "not rare", and the demand for medication is surging

Statistics show that the prevalence of rare diseases in the population is about 3.

Driven by the demand for clinical drugs, the market for rare disease drugs continues to heat up

However, the lack of motivation for the research and development of rare disease drugs by domestic pharmaceutical companies is reflected in the fact that the coverage of rare disease drugs is not wide, on the other hand, the difficulty of rare disease drug research and development is far higher than that of other drugs, and the probability of successful listing of drugs is relatively low

1.

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Incentive policies are emerging in an endless stream, and the spring breeze of rare disease drug research and development has arrived

In order to encourage domestic pharmaceutical companies to increase the research and development and production of rare disease drugs, in recent years, the national government departments have successively issued a series of incentive policies

In the past decade, the state has issued some relevant policies to encourage the research and development and production of rare disease drugs

Source: State Food and Drug Administration, National Health Commission, etc.

In 2022, CDE has successively issued two clinical guidelines for rare disease drugs, namely, the Technical Guidelines for Clinical Research and Development of Rare Disease Drugs and the Statistical Guidelines for Clinical Research of Rare Disease Drugs (Trial), which undoubtedly provides new ideas for the formulation of clinical trial plans for rare disease drugs and also forms an important boost

The first rare disease directory, medical insurance coverage is only 21%

The policy implementation has driven research and development in an all-round way, but it is not ideal

According to data from the Intranet, the 2009 edition of the National Medical Insurance Catalogue contains 22 rare disease drugs, covering 13 rare diseases; The 2017 edition of the National Medical Insurance Catalogue contains 30 rare disease drugs, covering 15 rare diseases; The 2019 edition of the National Medical Insurance Catalogue contains 39 rare disease drugs, covering 19 rare diseases; The 2020 edition of the National Medical Insurance Catalogue contains 45 rare disease drugs, covering 23 rare diseases; The 2021 edition of the National Medical Insurance Catalogue contains 53 rare disease drugs, covering 26 rare diseases

In June 2018, the National Health Commission published the First National Catalogue of Rare Diseases, involving 121 diseases; At present, there are only 26 rare diseases covered by medical insurance, and the coverage rate is only 20

Rare disease drugs that have been included in the Medical Insurance List

Source: Minneinet China Medical Insurance Directory Encyclopedia Database

Among the 53 rare disease drugs included in the national medical insurance, parkinson's disease (youth type, early-onset type), hemophilia, multiple sclerosis and idiopathic pulmonary hypertension are the majority, all of which are 5 or more

Combined with the new drugs approved in recent years and their entry into medical insurance, the indications corresponding to the rare disease drugs currently listed have not fully covered the indications of the first batch of rare disease catalogs, and there is still room

Sales of the five varieties exceeded 1 billion, but rare diseases are not their main indications

According to intranet data, 5 of the 53 rare disease drugs will have terminal sales of more than 1 billion yuan in China's urban public hospitals, county-level public hospitals, urban community centers and township health centers (referred to as China's public medical institutions) in 2021, including intravenous injection of human immunoglobulin (pH4), rosuvastatin calcium tablets, recombinant human coagulation factor VIII for injection, ezez-maibu tablets and detemir injection
.

However, rare diseases are not the main indications for the above products, such as human immunoglobulins are systemic anti-infective drugs, mainly used for the prevention of measles and infectious hepatitis, as well as in combination with antibiotics to improve the efficacy of certain serious bacterial and viral infections; Rosuvastatin calcium tablets and ezetimid tablets are classic hypolipidemic drugs; Recombinant human coagulation factor VIII for injection was initially used as a hemostatic agent in emergency or elective surgery; Insulin deuterine injection is a commonly used insulin hypoglycemic drug in clinical practice
.

5 rare disease drugs with sales exceeding 1 billion yuan (unit: 100 million yuan)

Source: Intranet Database

At present, most of the rare disease drug market size is still small, on the one hand, it may be because the drug has just been listed and has not yet achieved volume; On the other hand, it may also be because the hospital monitored by the market is not the main hospital
for medication.
However, there are still some varieties with rare diseases as the main indications for the market potential that should not be underestimated
.

Treat spinal muscular atrophy of the Nocinacon sodium injection, through negotiations after the price reduction of 95% into medical insurance, intranet data show that the drug in 2021 in China's key provinces and cities public hospital terminal sales of less than 50 million yuan, 2022 Q1 has exceeded the 100 million yuan mark, the growth rate is very amazing
.

In recent years, the sales trend of nonsinasson sodium injection terminal in public hospitals in key provinces and cities in China (unit: 10,000 yuan)

Source: Intranet key provinces and cities public hospital terminal competition pattern

Anlisheng tan tablets for the treatment of idiopathic pulmonary hypertension, in 2019 into the medical insurance after the market rapid release, intranet data show that the product in 2020-2021 in China's key provinces and cities public hospital terminal sales growth rate of more than 49%, 2022 Q1 sales reached 5.
58 million yuan, the market potential is released
.

In recent years, the sales trend of terminal Anlisheng tan tablets in public hospitals in key provinces and cities in China (unit: 10,000 yuan)

Source: Intranet key provinces and cities public hospital terminal competition pattern

In general, the price reduction of rare disease drugs into medical insurance is only the process, and accelerating product release and improving the availability of drugs for patients is the ultimate purpose and mission
of the enterprise.

2022 national talks on the road, 18 rare disease drugs into the "big hot players"

"Let more rare disease drugs be included in medical insurance as soon as possible", the state is also sparing no effort
.

In June this year, the 2022 National Medical Insurance Catalogue Adjustment Plan (hereinafter referred to as the Adjustment Plan) was officially released
.
Compared with last year, the new round of health insurance negotiation process is basically the same as the key policies, which is different from the emphasis on rare disease treatment drugs, while relaxing the declaration conditions: the declaration conditions include rare disease drugs approved before June 30 this year, but do not require approval
after 2017.

According to incomplete statistics, there are currently 18 rare disease drugs that are expected to participate in the 2022 national talks, of which there are more treatment drugs for rare diseases such as mucopolysaccharide storage, hemophilia and multiple sclerosis; The dosage form is mainly injectable and capsule; In terms of sub-categories, other gastrointestinal and metabolic drugs, antitumor drugs and immunosuppressants occupy 4 or more seats
respectively.

Some of the rare disease drugs that have the opportunity to participate in the 2022 national talks

Source: MED2.
0 China Drug Review Database

August has entered the end, according to the work arrangement of the "Adjustment Plan", the drugs and information that have passed the preliminary examination of medical insurance are expected to be announced in the near future, and the varieties in the list will be regarded as the "admission ticket"
for this round of national talks.
At that time, which varieties can be successfully selected, we wait and see!