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A rare disease is a disease with a low incidence
.
At present, more than 7,000 rare diseases have been identified, 80% of which are genetic diseases, such as albinism and hemophilia
.
In recent years, the number of rare patients and the market size have been increasing year by year
.
According to incomplete statistics, as of 2021, there are expected to be more than 300 million rare disease patients worldwide
.
Among them, China already has more than 20 million rare disease patients
.
According to data from Frost & Sullivan, as the demand for rare disease treatment continues to grow, the market size of the global rare disease drug market is expected to increase from US$135.
1 billion in 2020 to US$383.
3 billion in 2030, with a compound annual growth rate of 11%
.
At the same time, China's rare disease drug market may also grow rapidly, from US$1.
3 billion in 2020 to US$25.
9 billion in 2030, equivalent to more than 160 billion yuan, with a compound annual growth rate of 34.
5%
.
However, it should be noted that, contrary to the rapidly growing market size, for a long time, due to the small number of rare patients in China, the small dosage of drugs and weak profits, etc.
, the enthusiasm of pharmaceutical companies for production has not been high, and a large number of patients can only rely on imports.
The medicine lives
on.
However, in recent years, under the background of intensifying competition among pharmaceutical companies and seeking differentiated competitive advantages, the field of rare disease drugs is attracting a lot of capital, and a large number of pharmaceutical companies are also constantly developing and expanding the market
.
Of course, in addition to the fact that the market prospects are attracting more and more companies to deploy, in fact, favorable policies are the main driving force for the development of the rare disease industry
.
According to the "2020-2024 Comprehensive Market Research and Investment Analysis Report of China's Rare Disease Drug Industry" released by the Xinsijie Industry Research Center, in order to encourage the research and development and marketing of rare disease drugs, China has issued a number of policies to support it
.
For example, since 2018, the National Medical Security Administration has begun to dynamically adjust the list of medical insurance drugs once a year, and many drugs for rare diseases have been included in it.
.
At present, in addition to local companies, with favorable policies, overseas products have also begun to enter the domestic market.
The industry expects that a large number of rare disease drugs with different targets will be launched in the domestic market in the next five years
.
In fact, since the beginning of this year, domestic companies have gradually begun to make new progress in the field of rare diseases
.
For example, on January 4, Shufang Pharmaceutical and Santhera Pharmaceuticals announced that they had reached an exclusive licensing agreement for the new rare disease drug Vamorolone
.
According to the agreement, Shufang Pharmaceutical has obtained the exclusive rights to develop and commercialize Vamorolone in Greater China for Duchenne muscular dystrophy and other rare disease indications
.
The former will pay an upfront payment in the tens of millions of dollars and milestone payments related to U.
S.
registration filings, totaling $20 million
.
In addition, double-digit percentage royalties will be paid
.
Due to the huge unmet demand, the industry believes that in the future, Shufang Pharmaceutical will usher in more development opportunities after Vamorolone is approved
.
However, it should be noted that the discovery, treatment and commercialization of rare diseases is a complex systematic project, and a multi-party cooperation system needs to be established, including from policy support to legal protection, from drug production to capital supply, from talent training to standard setting,
etc.
Therefore, for pharmaceutical companies, when developing rare disease drugs in the future, continuous efforts are needed to explore and improve
.
.
At present, more than 7,000 rare diseases have been identified, 80% of which are genetic diseases, such as albinism and hemophilia
.
In recent years, the number of rare patients and the market size have been increasing year by year
.
According to incomplete statistics, as of 2021, there are expected to be more than 300 million rare disease patients worldwide
.
Among them, China already has more than 20 million rare disease patients
.
According to data from Frost & Sullivan, as the demand for rare disease treatment continues to grow, the market size of the global rare disease drug market is expected to increase from US$135.
1 billion in 2020 to US$383.
3 billion in 2030, with a compound annual growth rate of 11%
.
At the same time, China's rare disease drug market may also grow rapidly, from US$1.
3 billion in 2020 to US$25.
9 billion in 2030, equivalent to more than 160 billion yuan, with a compound annual growth rate of 34.
5%
.
However, it should be noted that, contrary to the rapidly growing market size, for a long time, due to the small number of rare patients in China, the small dosage of drugs and weak profits, etc.
, the enthusiasm of pharmaceutical companies for production has not been high, and a large number of patients can only rely on imports.
The medicine lives
on.
However, in recent years, under the background of intensifying competition among pharmaceutical companies and seeking differentiated competitive advantages, the field of rare disease drugs is attracting a lot of capital, and a large number of pharmaceutical companies are also constantly developing and expanding the market
.
Of course, in addition to the fact that the market prospects are attracting more and more companies to deploy, in fact, favorable policies are the main driving force for the development of the rare disease industry
.
According to the "2020-2024 Comprehensive Market Research and Investment Analysis Report of China's Rare Disease Drug Industry" released by the Xinsijie Industry Research Center, in order to encourage the research and development and marketing of rare disease drugs, China has issued a number of policies to support it
.
For example, since 2018, the National Medical Security Administration has begun to dynamically adjust the list of medical insurance drugs once a year, and many drugs for rare diseases have been included in it.
.
At present, in addition to local companies, with favorable policies, overseas products have also begun to enter the domestic market.
The industry expects that a large number of rare disease drugs with different targets will be launched in the domestic market in the next five years
.
In fact, since the beginning of this year, domestic companies have gradually begun to make new progress in the field of rare diseases
.
For example, on January 4, Shufang Pharmaceutical and Santhera Pharmaceuticals announced that they had reached an exclusive licensing agreement for the new rare disease drug Vamorolone
.
According to the agreement, Shufang Pharmaceutical has obtained the exclusive rights to develop and commercialize Vamorolone in Greater China for Duchenne muscular dystrophy and other rare disease indications
.
The former will pay an upfront payment in the tens of millions of dollars and milestone payments related to U.
S.
registration filings, totaling $20 million
.
In addition, double-digit percentage royalties will be paid
.
Due to the huge unmet demand, the industry believes that in the future, Shufang Pharmaceutical will usher in more development opportunities after Vamorolone is approved
.
However, it should be noted that the discovery, treatment and commercialization of rare diseases is a complex systematic project, and a multi-party cooperation system needs to be established, including from policy support to legal protection, from drug production to capital supply, from talent training to standard setting,
etc.
Therefore, for pharmaceutical companies, when developing rare disease drugs in the future, continuous efforts are needed to explore and improve
.
