Before answering this question, I need to explain what a rare disease is.
Rare diseases, also known as "orphan diseases", refer to diseases with extremely low incidence. Most of them are congenital diseases, chronic diseases, and are often life-threatening. Spinal muscular atrophy (SMA), which affects 1/6,000 to 1/10,000 newborns, is clearly a rare disease. The imported drug in question, Nosinaxin Sodium, is a precision targeted therapy drug for the treatment of SMA.
Figure 1. Noxinaxin Sodium Injection
From the beginning of research and development to the launch of a new drug, billions of dollars of capital investment are required, which can be said to be sky-high. At the same time, new drug patents are only protected for twenty years. Excluding the time it takes to obtain a patent and go on the market, there is little time left for pharmaceutical companies to earn back their costs. For these rare disease drugs with few patients, raising drug prices can only be the only way for pharmaceutical companies to recover costs.
The domestic price of 700,000 yuan per dose of Nosinaxin Sodium Injection in the United States is 125,000 knives (approximately 870,000 yuan), and the so-called price of 200 yuan per dose abroad (Australia) is also Just because the drug has been included in Australia's pharmaceutical benefit plan, each drug is purchased by the government at A$110,000 (approximately RMB 550,000), so patients only have to pay out-of-pocket A$41 (approximately RMB 200).
Having said this, it is not difficult for us to understand that the imported medicine in question, which costs more than 200 yuan and sells for 700,000 in China, is just a gimmick by some media to attract attention and earn traffic.
Having said that, it is still an indisputable fact that the price of rare disease drugs is high. So the next question is, what should patients do for diseases that are so expensive that treatment costs require ordinary people to work hard for a lifetime to pay off.
First of all, these rare disease drugs cannot become medical insurance drugs in a short period of time and can only be purchased at one's own expense. However, patients can raise money from the public through some fundraising platforms.
Figure 2. Volunteers of Shuidichou
Secondly, at the social level, we hope that relevant departments and social celebrities will take the lead in popularizing rare diseases such as SMA to the public and establishing related public welfare Associations, etc., help a large number of patients through donations for rare diseases from caring people from all walks of life. For example, the Ice Bucket Challenge launched by a former Boston College baseball player in 2014 raised US$114 million in charity in the United States alone in August 2014.
Figure 3. The ALS Ice Bucket Challenge that took the world by storm in 2014
Finally, the national level. At present, the "First Batch of Rare Disease Catalog" has been released, and ensuring the medication of patients with rare diseases has been included in the key tasks of deepening medical reform in the "13th Five-Year Plan". It is believed that through the country’s unremitting efforts, rare disease drugs will be included in medical insurance soon.
At the same time, I also believe that with the advancement of science and technology and the development of society, the cost of developing a drug will gradually decrease, and one day the shortage of rare disease drugs will no longer be a problem.