February 28 this year is the first 15 International Rare Disease Day. Porcelain dolls, gradually frozen people, butterfly babies… Behind these names, all correspond to a rare disease. It is understood that there are about 20 million rare disease patients in my country, with more than 200,000 new patients every year (Xinhua News Agency, February 28).
Rare diseases are actually not uncommon in terms of numbers. However, this group of patients with a large number faces common problems – difficult to diagnose, expensive to treat, no drugs available, and so on. The cost of rare disease treatment drugs is often beyond most people’s imagination. Nosinagen sodium injection, which can be used to treat spinal muscular atrophy (SAM), has been searched on Weibo due to the sky-high price of “700,000 yuan per injection”. After it entered the medical insurance catalogue, the price of an injection at the patient’s own expense dropped to about 10,000 yuan.
There are many drugs for rare diseases like this, some of which are still difficult to popularize due to their high price. However, we must also see that rare disease drugs have a small audience, difficult research and development, and high risks. There are fewer companies willing and able to conduct research and development. It is unrealistic to blindly demand that drugs be sold at lower prices. In recent years, the National Medical Insurance Administration has included rare disease drugs in the medical insurance catalog through negotiation every year. The author believes that negotiating access to rare disease drugs or speeding up the approval time of rare disease drugs is one of the effective ways to reduce the pressure on patients and their families, but not all. We will continue to explore the establishment of a multi-layered guarantee mechanism to help patients with rare diseases from different aspects such as rare disease screening, treatment, drug and drug development, and medical insurance.
Recently, Changsha has launched the “Healthy People’s Livelihood Project – Comprehensive Prevention and Control Project of Inherited Rare Diseases”, which will carry out large-scale screening of age-appropriate groups for rare diseases in the city. The author believes that such extensive intervention measures for the development of genetic rare diseases can achieve the effects of early detection, early intervention, and early treatment, and effectively help couples of childbearing age to eliminate reproductive risks. It is also worth exploring in more places.
Focusing on rare diseases requires us to continue to explore, optimize the system, play a good combination of punches, and provide multi-level guarantees for patients. This is a major and important event that concerns the vital interests of the tens of millions of people, and it is also the due meaning of the supremacy of the people and the supremacy of life.
Editor: Deng Jinqiu