13 million per injection, the most expensive drug in history, has really entered China!
This year’s medical insurance negotiation has reduced the 700,000-injection Noxinagen Sodium Injection to 33,000-in-a-shot, bringing hope to countless spinal muscular atrophy (SMA).
It is worth noting that Nosinagen Sodium Injection is not the only drug for the treatment of spinal muscular atrophy. Zolgensma is also a specific drug for the treatment of spinal muscular atrophy, and it is the first drug in the world. Gene therapy for the treatment of spinal muscular atrophy does not require life-long injections like Nosinagen Sodium Injection. It can be cured or relieved with just one injection.
Behind the special effects is the sky-high price. Zolgensma is priced at 2.125 million US dollars (equivalent to about 13.58 million yuan) for one injection. Zolgensma’s clinical trial application in China was accepted in October last year, and the clinical trial was officially approved this year. .
Mid-March, 21st Century Business Herald, A randomized, double-blind, controlled study in age ≥2 years to <18 years old, newly diagnosed, late-onset 2 who can sit alone but cannot walk The evaluation of the efficacy and safety of intrathecal (IT) injection of OAV101 (Zolgensma) in patients with SMA type SMA has begun, marking the real first step in the history of the most expensive drug in China.
One, one needle is 13 million, and if you die, you may not live for a year.
According to statistics, SMA The incidence in newborns is about 1/10,000. For the general population, about 1 in 40 to 50 people is a carrier of the SMA pathogenic gene. The number of SMA neonates in my country is about There are 1,200 new cases, and there are about 30,000 existing patients.
People with SMA will gradually develop symptoms of limb weakness, which will become more severe and difficult to reverse. Without treatment, the patient’s muscle strength slowly deteriorates, and eventually, problems such as swallowing or breathing can lead to infection or death. Heartbreakingly, more than half of babies with SMA live less than a year.
Let’s see how Zolgensma transforms decay into magic, and how does it work?
The results of a recent phase 3 clinical trial abroad showed that children with 3 copies of the SMN2 gene who received treatment before symptoms appeared, have been able to resume the amount of exercise at their age, and can She stood and walked normally without ventilation or feeding tube support, and there were no serious, treatment-related adverse events.
Second, one injection can cure or relieve, how amazing is the sky-high life-saving medicine?
At present, there are three kinds of drugs for the treatment of SMA in the world:
1. Nosinagen Sodium Injection
Approved for marketing in 2016, it is the world’s first SMA precision targeted drug, with an original price of $750,000 in the first year and then $375,000 per year. However, Nosinagen Sodium Injection will enter my country’s medical insurance list in 2021. The price will be reduced from the original 700,000 injections to 33,000 yuan per injection, and patients will eventually need to pay about 10,000 yuan out of pocket. Yuan a needle. The patient needs 4 injections in the first 63 days, and 1 injection every 4 months thereafter, lifetime medication is required.
2. Risdiplam oral solution
This is the first drug in the world that can be taken orally to treat SMA. According to the frequency of oral administration once a day, needs Lifetime medication , patients need to pay about 340,000 US dollars per year, Risdiplam oral solution was approved for marketing in my country in June 2021, and can be used to treat all types of SMA patients aged 2 months and above.
3. Zolgensma
The price of Zolgensma abroad is US$2.125 million, which is about 13.58 million yuan in RMB.. However, patients only need to inject Zolgensma once, because Zolgensma belongs to gene therapy, a single injection can make the body produce a protein that SMA patients lack, and solve the genetic root of SMA. In short, it is A single injection can provide long-term relief or even cure SMA.
However, it is still unknown whether Zolgensma can be successfully launched in my country, because There are still many uncertain factors in the process of recruiting local participants to the end of the Phase 3 clinical trial >, even if it is successfully listed, it is difficult for medical insurance to bear the sky-high price.
Third, why is life-saving medicine always expensive?
Faced with sky-high prices for life-saving drugs, many people will wonder why these drugs are so expensive?
Song Hualin, an expert in pharmaceutical law and associate dean of Nankai University Law School mentioned that due to the small number of rare disease patients, the dosage of drugs is very small, In addition, the research and development cost of drugs is very high, so the prices of drugs for the treatment of rare diseases are generally relatively expensive.
So, how high is the research and development cost of a new drug? According to data from foreign research institutions, the research and development cost of a new drug is about 1 billion to 2 billion US dollars, and the research and development time is generally 10 to 15 years. Going through multiple stages, such as the clinical trial stage, takes 5 to 7 years. Once the research and development fails, it is almost a waste.
4. Is there any risk in participating in free clinical trials?
Faced with more than 13 million new drugs, many people may want to seize the opportunity of clinical trials, because the drugs in clinical trials are all free. What are the risks?
1. It may not be possible to use new drugs
Participating in free clinical trials does not necessarily mean that new drugs can be used, and there is a high probability of being classified. To the control group, the control group was on a standard treatment regimen.
2. New drugs may not be 100% effective for themselves
New drugs in clinical trials are not necessarily 100% effective, and may not reach patients. expectations.
3. Unpredictable adverse reactions may occur
The drugs in clinical trials may cause unpredictable adverse reactions in patients. Tolerated, may need to face withdrawal from clinical trials.
If Zolgensma can successfully enter China, there will be one more choice for SMA patients, but Zolgensma also has limitations and high price, which is not an easy choice for patients’ families. It is also hoped that the country can help more rare disease patients to afford medicines as soon as possible, and increase the research and development of rare disease medicines.
References:
[1] “13 million yuan a shot of “the most expensive drug in history” was approved for clinical use in China, and three world-renowned SMA drugs gathered in the Chinese market”. 21st Century Business Herald. 2022-01-19
[2] “21 Health News Daily｜Bidding for the first centralized procurement of novel coronavirus antigen detection reagents in China; Novartis’ SMA drug starts clinical trial in China” 21st Century Business Report. 2022-03-17
[3] Why is the new drug for rare diseases 13.48 million yuan per injection so expensive in China? 》.Poster News.2022-01-21