19 million needles! The sky-high price of 'life-saving medicine' caused controversy, 'every small group should not be abandoned'

Yuanyuan, a 7-year-old girl from Shaanxi, suffers from beta-thalassemia major. People who are concentrated on this rare disease will need blood transfusion throughout their lives to survive. Today, long-term relief or even cure can be achieved almost with a single injection. Such a "life-saving drug", Zynteglo gene therapy, was finally approved for marketing, with 19.08 million injections, which is the most expensive drug in history.

Before the gene therapy hit the market, there were almost only two treatment options for the rare disease. One is regular permanent blood transfusion to maintain life, and the other is hematopoietic stem cell transplantation to achieve a complete cure, but this program is expensive and it is extremely difficult to find a suitable matching source. The rare disease group has been forgotten by society for a long time because of its niche.

"No small group should be abandoned".

Now the U.S. FDA has announced the approval of Zynteglo gene therapy, which is specially used to treat the rare disease β-thalassemia. According to experimental data released by the FDA, after receiving Zynteglo therapy, 89% of patients can no longer need blood transfusion, as long as they are cured almost once Can be cured, "one needle changes the patient's life".

But at the same time, its price of 2.8 million US dollars (about 19.08 million yuan) also hit a record high in the pharmaceutical industry and was rated as "the most expensive drug in history", causing social controversy. "This price is equivalent to two suites in Beijing", "What's the point of developing such a drug", "This is no different from death". For a while, there was controversy.

In the face of social controversy, the chief commercial officer of the manufacturer, Bluebird Bio, publicly stated that "the pricing of the therapy is actually reasonable."

Bluebird Bio also gave a corresponding pricing basis. The current cost of Zynteglo gene therapy is $2.8 million, but drug pricing regulators have also revealed that the cost-effectiveness of this treatment is actually $3 million per dose. And according to the current standard of care in the United States, the lifetime medical cost of this rare disease actually exceeds $6.4 million. From these perspectives, Bluebird Bio's pricing seems reasonable.

At the same time, in order to reduce the burden on patients, Bluebird Bio has implemented a one-time upfront payment model in the United States. The most important of which is the realization of 80% risk sharing. That is to say, if the patient fails to maintain blood transfusion-free for two years after receiving this treatment, then the patient or commercial institutions such as insurance will be able to get 80% of the cost reimbursement. But obviously the premise is that you must first have the ability to come up with 19.08 million.

The sky-high price is still an insurmountable "last mile", and the "gap between the rich and the poor" in life has become a reality that has to be acknowledged. According to a study published in the Journal of Gerontology, the average life expectancy gap between rich and poor men in the United States is about 31 years. Coincidentally, the Norwegian Institute of Public Health also found that the average life expectancy difference between the rich and the poor in Switzerland is about 14 years.

In addition to the living environment, habit differences, and medical treatment caused by wealth level, "rich people who chase time" are not satisfied with this, and further focus on high-end biotechnology that can delay aging, trying to open a larger gap in lifespan . Take the "Seleray" anti-aging technology as an example. In recent years, its astronomical price of 1.5 million has been called "the lifespan game of the rich" from Japan to China. The richest man Li Ka-shing has also publicly stated that he has taken such longevity substances.

It is reported that the core substance of this kind of longevity technology was developed by Harvard in 2013. It helps the human body to delay aging by empowering cells. Subsequently, universities such as the University of Tokyo, Belle-Houston School of Medicine, and Tsinghua University and Peking University in China have also successively confirmed in clinical experiments that through the reversal of the internal cellular aging process, the external aging indicators such as human skin, muscle strength, and bones appear. positive improvement.

In recent years, with its introduction on platforms such as JD.com, it has further opened up the domestic high-end consumer market. Although the price of a single bottle of "Selori" is still as high as 5 digits, it seems that it is still difficult to prevent high-income groups from entering the market. Up to now, the data of Double Eleven shows that "Selerui" technology has reached a new high and topped the sales list. Some of them reported that they were "willing to pay for young people". However, it is worth noting that with the breakthrough of enzymatic technology by Chinese researchers, its cost is decreasing year by year, and it is expected to further benefit more groups in the future.

There is a saying in the movie "I'm not the God of Medicine", there is only one disease in this world, and that is the disease of poverty. Although high prices are still the "last mile" that has not yet been crossed, whether in the medical field or in the field of longevity technology, we can also see that rare disease groups that were once forgotten by society have been paid attention to by the medical community and have been troubled by The aging problem of all human beings is also constantly being broken through.

As the technology matures in the future, its cost will inevitably be further reduced. I hope that people in the world are free from diseases, so why not dust the medicine at home. There may be more technological breakthroughs in the future, bringing hope to patients.