The last day of February every year is World Rare Disease Day.
In fact, rare diseases are not rare, although the number of people affected by each rare disease is not large, but the total number of these rare diseases added together is still a relatively large group.
According to the World Health Organization, there are currently more than 7,000 known rare diseases in the world, and more than 400 million patients have been patiented, of which 80% are caused by genetic defects.
The number of rare disease patients in the mainland is also quite large, and the current number has reached more than 20 million.
Rare diseases, what exactly is it?
In our daily lives, we always hear some very fairy tale disease names, such as frostbite, porcelain doll disease, sleeping beauty syndrome, Alice in wonderland, Progeria, werewolf disease and so on.
In fact, behind these beautiful names, it means that patients are destined to be "different" when they come to this world, and the situation of illness without dependence, medicine without medicine, and medicine without protection all make them have to face.
Why is this happening?
Due to the rapid progression of rare diseases and the high mortality rate, less than 10% of diseases can be effectively treated and improved. So far, almost none of the rare diseases have been cured.
Because there are fewer patients with rare diseases, the price of its treatment drugs is generally higher, such as Gaucher disease (a rare autosomal recessive disease), patients need to inject drugs 2-3 times a month, and the price of each drug reaches 23,000 yuan, and as the weight increases, the dose also increases, and it is necessary to take drugs for life.
Therefore, such a disease is an unbearable burden for an ordinary family.
What are these rare diseases?
We have excerpted some representative diseases, through which we can get scientific understanding of rare diseases:
Let more "orphan drugs" be able to enter medical insurance
According to the "2019 Comprehensive Social Survey Report on Rare Disease Patients in China", data shows:
About 70% of doctors do not understand the basic treatment of rare diseases, resulting in 42% of rare disease patients being misdiagnosed, and 40% of rare disease patients diagnosed for more than 1 year.
The most important thing is that at present, most of the rare disease treatment drugs in the mainland rely on imports, not only the price is unaffordable, but also whether it can be bought is also a problem.
Fortunately, with the attention of the Health Commission and the whole society, more and more "orphan drugs" have also entered the national medical insurance, reducing the economic burden of some rare families to a certain extent.
Up to now, more than 60 kinds of rare disease drugs have been approved for marketing, and more than 40 kinds of rare disease drugs have been included in the national medical insurance drug list........
Among the rare disease drugs newly included in medical insurance, 2 were originally high-value drugs, the so-called "sky-high drugs".
It is believed that with the efforts of the state and the support of society, the families of children with rare diseases are no longer alone.
However, we also call on the whole society to pay attention to rare diseases, and call on more professionals to devote themselves to relevant research and drug development, medical and social security, so that "rare" can be "seen" by more people!