The most expensive in history, a needle of 13.48 million can reach a suite in Beijing! The prototype development of "I am not a medicine god"

Source of this article: Time Weekly Author: Du Sumin

"One injection of 13.48 million yuan" and "the most expensive drug in history", because of its own high-price drug label, on January 20, the news that sovarevir obtained clinical license in China quickly attracted public attention.

Sovarevir is a gene therapy based on adeno-associated viral vectors for the treatment of children under 2 years of age with mutations in the bialelectrons of the SMA and survival motor neuron 1 (SMN1) genes. After listing, sovalevine will become the first gene therapy in mainland China to be used to treat SMA, which can be cured in principle with one dose. Novartis is the prototype of the pharmaceutical company in the sky-high drug controversy in "I Am Not a Medicine God".

In May 2019, soverevir was approved for listing in the United States at US$2.125 million, equivalent to nearly RMB13.48 million, making it the most expensive drug to date. However, due to the high price, the drug has caused controversy after its advent, and some netizens angrily asked "Do poor people have no right to live?" "The price is so expensive, what is the significance of inventing this medicine for the common people?"

Subsequently, Novartis quickly adjusted the payment plan according to market feedback, paying in five years according to the actual efficacy, paying 400,000 US dollars (equivalent to about 2.54 million yuan) per year, which is equivalent to the price of Northinassan sodium injection when it was just listed abroad. Novartis said that the payment is based on the outcome of treatment, and if there is no effect, the fee can be reduced.

Sovarevir has been listed in nearly 40 countries and territories. According to the data of Yaorongyun, in 2020, sovalevil achieved global sales of $920 million; in the first three quarters of 2021, a total of $1.009 billion was sold.

SMA is a difficult condition in which muscle strength decreases due to a loss of specific gene function. According to the highest level of motor function and the age of onset, it is mainly divided into I, II and III types. There are also adult-onset type IV, and type 0, which occurs prenatal and dies within a few weeks.

Among them, type I patients are also called infant type, because children with SMA usually need to bring a ventilator and can talk about life, so such children are also called "robot babies" and "elephant trunk babies". Type I patients account for about 45% of all SMA cases, generally develop illness within 6 months of life, and most live to no more than 2 years. The proportion of type II and type III is 30% to 40% and 20% respectively, the onset is slightly later, although it will not die very early, but it will also gradually weaken, suffer from pneumonia, scoliosis and other diseases, and be accompanied by wheelchairs for life.

According to genetic probabilities, there is about 1 in every 40-50 people with SMA carriers, and if the two carriers are combined, there is a one-in-four chance of giving birth to a child with SMA. According to the White Paper on the Survival Status of SMA Patients in China, there are about 20,000-30,000 SMA patients in mainland China.

At present, there are only two related drugs approved for the treatment of SMA in China, namely Bojian's Northinacana injection and Roche Pharmaceutical's risprolane oral solution. In December 2021, the news that the original price of 700,000 yuan of Nosinasana injection was reduced to 33,000 yuan to enter the medical insurance directory has given many SMA patients hope.

However, Northinacana Injection does not cure SMA patients at once, and patients need to inject 6 injections in a year and then 1 injection every 4 months, year after year. Therefore, despite the high price of sovarevir, it is still expected by many families of children with SMA.

In this year's medical insurance negotiations, Aguilense injection (trade name: Yikaida) worth 1.2 million yuan passed the preliminary formal examination, but did not enter the medical insurance directory negotiation process. In this regard, at the press conference held by the National Medical Insurance Bureau on December 3, 2021, Zheng Jie, head of the 2021 Medical Insurance Negotiation Fund Calculation Team, said that a 1.2 million injection of anti-cancer miracle drugs may have a good efficacy, but because the price far exceeds the affordability of the fund and the burden level of the people, it does not have economics, so it has not been able to pass the review.

"The fund's calculation is not to pursue the lowest price of drugs, but to give a reasonable institutional standard." Zheng Jie said that according to the current drug payment range, the maximum annual expenditure on drugs in the national catalogue is not more than 300,000 yuan, and the basic principle of guarantee can make the limited fund play the maximum efficiency. "In the future, we will also prevent sky-high prices from entering medical insurance." Zheng Jie stressed.