February 28 is the 15th International Rare Disease Day with the theme “Sharing the Color of Your Life”. Porcelain Doll, Butterfly Baby, Moon Child… But behind these seemingly cute names is a huge pain.
Rare disease, also known as “orphan disease”. According to the definition of the World Health Organization, rare diseases refer to diseases or lesions with the number of patients between 0.65‰ and 1‰ of the total population. At present, there are more than 7,000 rare diseases in the world, including ALS, “glass man” (hemophilia), “puppet man” (multiple sclerosis), floppy child (spinal muscular atrophy), etc. Rare diseases are not only a medical problem, but also a social problem. There are about 20 million rare disease patients in my country, with more than 200,000 new patients every year. They have experienced pain beyond the comprehension of ordinary people and bear a very heavy financial burden.
Now, from the performance point of view, the atmosphere of rare disease clinics has become relaxed and optimistic, which seems to be inconsistent with the severity and difficulty of diagnosis and treatment of such diseases. But in fact, this contrast just shows that a series of policies and measures for rare diseases have achieved good results, not only let some rare disease patients get out of the predicament, but also let other rare disease patients see hope.
This is due to the fact that in recent years, the state has launched a series of combined punches for the treatment of rare diseases. For example, in response to the phenomenon of “disease without a doctor”, relevant departments have selected a group of hospitals across the country to form a rare disease diagnosis and treatment cooperation network, playing the role of weak and strong, so that even “rare” diseases can be seen in time. Another example is to increase investment in research and development and foreign introductions, so as to solve the problem of “no medicine can be cured” to the greatest extent. In addition, in response to the phenomenon of “medicine without insurance”, my country has made great moves frequently in recent years, and the effect is remarkable. In addition to the release of the “Catalog of Rare Diseases”, allowing some rare disease drugs and APIs to receive value-added tax discounts, etc., more drugs for rare diseases have been negotiated into the medical insurance catalog.
Of course, it should also be noted that the current achievements in the treatment of rare diseases are worthy of recognition, but the treatment of rare diseases is facing a very complex situation and many difficulties need to be overcome, which also needs to be addressed. There are many types of rare diseases, and only a few are included in the scope of protection. Although some “high-priced drugs” have plummeted, many rare disease drugs are difficult to reach due to their high prices. What’s more, many rare diseases still lack effective treatment drugs. These problems need to be passed Keep trying to resolve it.
“No matter how small the group should be, it should not be abandoned”, expanding from targeting some rare diseases to all rare diseases, from drug price negotiation to drug research and development and the whole process guarantee, as well as the inclusion of rare diseases in medical insurance. On this basis, we will establish mechanisms such as special funds, commercial insurance, critical illness protection, and government support, so that love can be seen everywhere, and the light of hope can shine on all rare disease patients. We are still on our way. The innovation and maturity of the model are more important than the immediate effect. Because only by forming a long-term mechanism and a scientific model, can the level of protection and treatment capacity for rare diseases be greatly improved.
Caring for patients with rare diseases depends not only on love, but also on the system. Parents cannot take care of their children for a lifetime, but a good system can do it. The life of each rare disease patient has a different color, and it may bloom with splendid light. Let us join hands to protect patients with rare diseases and create a more colorful and beautiful world!
Source: Jingchu Network
Author: Wang Hongyang (Xiangzhou District, Xiangyang)