The domestic rare disease industry has huge development potential, and the market may exceed 60 billion yuan in 2030

According to the definition of the World Health Organization (WHO), a rare disease is a disease in which the number of patients accounts for 0.
65‰-1‰ of the total population
.
At present, there are more than 7,000 rare diseases confirmed in the world, and about 80% are caused by genetic defects
.
However, it should be noted that only about 5% of these 7,000 rare diseases have effective treatments, and most rare diseases still lack effective treatments
.
For example, of the 121 diseases included in the first batch of rare disease catalogs in China, only 60% are "curable"
.
In this regard, the industry believes that there is a huge unmet demand in the field of rare disease treatment
.
It is understood that in recent years, people with rare diseases have actually received more and more attention in the industry due to problems such as difficult diagnosis, high drug prices, and even no available drugs
.
At home, in order to accelerate the development of orphan drug benefit more patients, countries are more and more emphasis on the treatment of rare diseases work, has been targeted issued a series of documents, including the catalog of rare diseases, rare disease diagnosis and treatment guidelines and so on
.
It is worth mentioning that, under the strong promotion of the state, various regions have also issued documents since this year, and began to include more rare disease drugs into medical insurance reimbursement
.
For example, at the beginning of July this year, Jiangsu Medical Insurance included the medications for five rare diseases into the medical insurance reimbursements.
These five rare diseases include Gaucher disease, Pompe disease (also known as glycogen accumulation disease type II), and French medicine.
Bray's disease, spinal muscular atrophy (SMA), mucopolysaccharidosis type IV rare disease medication
.
In addition, on June 30, the Shaanxi Provincial Medical Insurance Bureau also issued the "Announcement on the Results of Negotiations on Specially Effective Drugs for Rare Diseases", and decided to use velaglucerase α for injection (Editor’s note: treatment of Gaucher’s disease type I) and injection Seven drugs including agalsidase β (for the treatment of Fabry disease) are included in the payment scope of Shaanxi Provincial Critical Illness Insurance (large amount of medical assistance)
.
Of course, in addition to the release of rare disease catalogues, rare disease diagnosis and treatment guidelines and other documentary measures to speed up the approval of rare disease drugs into the domestic market to benefit patients; the state is also encouraging pharmaceutical companies to actively invest in the research and development of rare disease drugs
.
It is understood that since 2015, more and more local pharmaceutical companies have begun to deploy the rare disease market
.
It mainly includes companies that focus on rare diseases such as Beihai Kangcheng, Kangdini Pharmaceutical, Deyi Sunshine, Shufang Pharmaceutical, and Langyu Group; and Boya Gene, Ruibo Biological, and Shumi Technology, which take rare diseases as the entry point and main focus Technology platform companies; and pharmaceutical companies such as Rongchang Biological, BeiGene, and Platinum Pharmaceuticals
.
At present, from the perspective of industry development trends, thanks to the efforts of many pharmaceutical companies, China's rare disease industry has begun to shift from being dominated by foreign companies in the past to local pharmaceutical companies and foreign companies, and from generic drugs to "fast-following" and first-in-class drugs; From the "special drug model" that focuses on drug treatment, to an ecological model that is oriented to the needs of patients
.
The industry predicts that according to the current industry development trend, as domestic pharmaceutical companies continue to make efforts in the rare disease drug market in the future, the market potential will continue to grow substantially
.
In fact, the domestic rare disease market has reached about 1 billion yuan in 2016, and it will exceed 3 billion yuan in 2020, accounting for about 1% of the global share
.
According to the current development trend, by 2030, China's rare disease market is expected to reach 60-90 billion yuan, and it will account for more than 5% of the global market by then
.
In general, there is still a big gap in the domestic rare disease drug market
.
However, with the country’s introduction of a series of orphan drug incentive policies, as well as the advancement and upgrading of drug policy reforms and medical insurance payments, local pharmaceutical companies have quietly emerged.
In the future, the launch of domestic drugs for rare diseases will have more possibilities, and it will also be expected to give Patients with rare diseases bring more good news
.