Pharmaceutical companies are digging into the blue ocean market for rare diseases, and patient treatment will be broken

 Rare disease drugs are also called orphan drugs.
Many pharmaceutical companies are reluctant to spend their energy on research and development due to difficulties in research and development, small audiences, high prices, and difficulties in promotion
.
According to the industry, the number of rare patients is small, the dosage of drugs is small, and the profit is weak, which leads to the low enthusiasm of pharmaceutical companies for production.
Profiting pharmaceutical companies brings risks and risks expanding losses
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In recent years, all parties have been making active efforts to solve the difficult problem of rare disease treatment
.
For example, in 2016, the National Health and Medical Commission established the Rare Disease Diagnosis and Protection Expert Committee to make positive contributions to promoting the management of rare diseases in China
.
In May 2018, the National Health and Medical Commission and several departments issued China's "First Batch of Rare Disease List", which included 121 diseases, including albinism, to strengthen the state's management of rare diseases
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In October 2018, more than 50 medical institutions, universities, scientific research institutions and enterprises established the China Rare Disease Alliance, actively promoting the development of rare diseases in China
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In February 2019, the "Notice on Establishing a National Rare Disease Diagnosis and Treatment Collaboration Network" was released, establishing a rare disease diagnosis and treatment collaboration network covering 324 hospitals across the country
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In 2019, the newly revised "Drug Administration Law of the People's Republic of China" stipulates that the state encourages the research and creation of new drugs, and opens green channels for new drugs for rare diseases and children's drugs that are urgently needed in the clinic, with priority review and approval
.
In October and November 2021, the "Technical Guidelines for Clinical Research and Development of Rare Disease Drugs" and "Statistical Guidelines for Clinical Research of Rare Disease Drugs (Draft for Comment)" were released successively.
Suggestions and references have greatly improved the efficiency of clinical research and development related to rare diseases
.
With the efforts of all parties, more and more pharmaceutical companies have begun to pay attention to the R&D and promotion of drugs for rare diseases
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For example, in 2019, the State Drug Administration approved the listing of 8 rare disease drugs; on February 18, 2020, WuXi PharmaTech invested US$30 million in Beihai Kangcheng to create an ecosystem for rare diseases; in July 2020, it focused on Langhua Medicine, a Chinese pharmaceutical company in the field of rare diseases, received US$80 million in Series A financing; in February 2021, Neophos received 400 million yuan in Series B financing, and a total of 500 million yuan in financing.
After the financing is completed, the company will accelerate the promotion of AAV The research and development of new ophthalmic gene therapy drugs and the clinical research of the core product NR082; in December 2021, Beihai Kangcheng officially landed on the Hong Kong stock market, becoming a "share" in the field of rare disease pharmaceuticals
.
According to the industry, due to the differentiated competitive advantages and the fact that the research and development of rare disease drugs has practically solved the clinical needs of patients, the field of rare disease drugs has become a blue ocean, which has attracted a lot of capital
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The optimistic view of capital also makes enterprises have the motivation to continuously develop and develop the market
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It is understood that in order to encourage the research and development and marketing of drugs for rare diseases, China has continuously issued policies to support it in recent years, to attract overseas products to the domestic market, and to increase the emphasis of local pharmaceutical companies on the research of drugs for rare diseases.
The domestic market may be different in the next five years.
Rare disease drugs are being launched one after another
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In addition, drugs for rare disease patients have always been the focus of attention during the adjustment of the National Medical Insurance Drug List.
Since the normalized adjustment of the National Medical Insurance Drug List in 2019, drug varieties for rare diseases have entered the medical insurance list through negotiation every year.
45 species have been reached
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In 2021, a total of 7 rare disease drugs have been successfully negotiated, and the list of high-value rare disease drugs is now listed
.
On December 3, 2021, the new version of the National Medical Insurance Drug Catalog was announced, and the precision targeted therapy drug for the treatment of the rare disease spinal muscular atrophy, Nosinagen Sodium Injection, was listed.
The price of medicine has dropped from nearly 700,000 yuan per injection to 33,000 yuan per injection
.