Use 11 million yuan at a time! "World's Most Expensive Drug" Incorporated into Health Insurance in Japan

On May 13, Japan's Ministry of Health, Labour and Welfare decided to list "Zolgensma" as the subject of public health insurance at a price of 1670.7 million yen (about 11.02 million yuan), Kyodo News reportedThis is good news for patients and their families waiting for treatment"the world's most expensive drug" was included in Japan's health insurancereported that Japan's Ministry of Health, Labour and Welfare decided on the 13th, the difficult disease spinal muscular dystrophy gene therapy drug "Zolgensma" as the applicable target of public health insurance, the drug price of 1670.7 million yen (about 11.02 million yuan)The drug is intended for patients under the age of 2Although treatment is done with only one use, this is the highest domestic price for a health insurance-used drugThe fastest 20 days applythe previous highest drug price in Japan was "kymriah", a drug that was introduced in May last year and was expected to effectively treat some leukemia and malignant lymphoma, at 33.49 million yenspinal muscular dystrophy (SMA) is one of the most common fatal neuromuscular diseases and the leading genetic cause of infant death, caused by a single gene motor neuron survival gene SMN1 defectOne in every 10,000 newborns is affected, and 90 per cent of these sick babies do not live to be 2 years oldAlthough the sMA etiology is clear, but there has been no medical means available for this, no medicine can be doneBut it is also because the disease-causing gene is clear, in recent years, the development of gene therapy technology, for this kind of disease has brought hopeNovartis has said that all the children who have been treated in the trial are still alive, and many are able to sit, roll, crawl, play, and some walkZolgensma is introduced into normal genes through intravenous injections in an effort to restore motor functionNovartis expects 25 people a year to use it, with sales of 4.2 billion yenaccording toKyodo, the rate of out-of-pocket payment for public insurance medical expenses for patients under the age of 2 is 20% Because of the subsidy system in Japan's central and local governments, there is virtually no need to pay out of pocket the drug has caused great controversy
gene therapy treats diseases caused by genetic defects by introducing effective genes into the human body Zolgensma treats spinal muscular dystrophy, a disease caused by a patient's lack of key genes to control muscles the use of Zolgensma therapy marks an important breakthrough in the history of SMA treatment, which requires only one intravenous infusion to achieve treatment results compared to the first SMA drug, the Soma, which required injections every four months, in The Sodium Injection public data show that between 450 and 500 babies in the United States suffer from SMA each year However, Novartis's price of up to $2.1 million has raised questions about the pharmaceutical company's use of the child's life as a threat Bloomberg reported on May 30 last year that Zolgensma had provoked two very different reactions in the United States: critics said it was an out-of-control practice, and on the other side, supporters said Novartis had found a cure for the rare disease that could save desperate families questions about society and public opinion, Novartis CEO said: "Those critics don't really think about how our health care system works We give patients transplants that cost $3 million to $5 million per patient, but they're far less effective than these drugs The CEO said that if the treatment was only done once, it would be cheaper than the current treatment The difficulty in drug pricing is how to reach a pricing consensus The current common pricing method is "cost-benefit" pricing, which estimates the benefits of a patient's healthy life for each year, and then pricing the drug by calculating the total benefits of the drug but it is difficult to value a patient's healthy life Novartis says Zolgensma's "cost-effectiveness" is between $4 million and $5 million But an analysis by the Nonprofit Institute for Clinical and Economic Review found that Zolgensma's "cost-effectiveness" should be no more than $1.5 million The difference in valuations is due to Novartis's higher valuation of a healthy life for patients each year , says pricing is a real problem because drugs that no one can afford are useless Another problem with pricing is insurance, which can be unaffordable for insurers as more gene therapies enter the market in the future.