This article comes from the WeChat Official Account:National Business Daily (ID: nbdnews)Editors: He Xiaotao, Wang Jiaqi, Yi Qijiang; Proofreader: Sun Zhicheng. Original Title: "A Single Injection Costs 13 Million Yuan: The 'Most Expensive Drug in History' to Launch in China! Manufacturer Once Called It 'Not Expensive'". Header Image Source: Visual China
$2.125 million per injection(Approximately RMB 13.48 million)"The most expensive drug in history" will be available in China.
Recently, the Center for Drug Evaluation of the National Medical Products Administration of China（CDE）Public Notice, Novartis（Novartis）Treatment of Spinal Muscular Atrophy（Spinal Muscular Atrophy, SMA）Novartis' AAV gene therapy drug Zolgensma(OAV101 Injection)Clinical Trial Application Submitted in ChinaHas obtained clinical trial implied permissionPreviously, on October 21, 2021, the clinical trial application submitted by the drug was accepted.
Currently, Zolgensma has been approved in nearly 40 countries and regions worldwide. Among them, Zolgensma was included in Japan's health insurance system in 2020, with patients only required to pay 30% of the cost; it was later incorporated into the UK’s National Health Service in March 2021.
1. Only one intravenous injection is required to achieve long-term relief or even a cure.
Speaking of the Ice Bucket Challenge, many people can recall "ALS patients."(Amyotrophic Lateral Sclerosis, also known as ALS)This statement.
But when it comes to its "brother disease" – SMA, many people are quite confused. In fact, SMA is also a type of motor neuron disease. After contracting SMA, the motor nerve cells that control muscle activity cannot function properly, and patients gradually lose various motor functions, even the ability to breathe and swallow.
In newborns, the incidence rate of SMA is between 1 in 6,000 to 1 in 10,000. In the general population, approximately 1 in every 40 to 50 individuals is a carrier of the SMA disease-causing gene.
In May 2018, SMA was included in the "First List of Rare Diseases" jointly formulated by the National Health Commission and other departments.
Based on the patient's age at onset and the maximum motor function they can achieve, SMA is mainly divided into four types:
Type I is the most common and severe, and patients usually face greater physical challenges. Based on various factors, their life cycle will vary significantly.
Patients with type II, III, and VI often have a normal or near-normal life cycle, but their physical functions will continue to change over time.
Similar to patients with ALS, SMA patients also progressively lose corresponding motor functions. However, while ALS typically manifests in adulthood, SMA usually begins in infancy or early childhood. Moreover, the earlier SMA symptoms appear, the more severe the condition tends to be—exactly the opposite of ALS.
On May 24, 2019, the US FDA approved Novartis' first gene therapy for treating SMA patients under 2 years old — Zolgensma. Patients only need to receive a single intravenous injection, which enables long-term expression of the SMN protein in cells, providing long-term relief or even a cure.
2. Sky-high drug prices once sparked huge controversy
Zolgensma is currently priced at $2.125 million, being dubbed the most expensive drug in pharmaceutical history by unit price.
Currently, Zolgensma has been approved in nearly 40 countries and regions worldwide. In the U.S. market, Novartis collaborates with relevant insurance companies, allowing patients' families to pay the cost over five years, averaging $425,000 per year. In May 2020, Zolgensma was included in Japan's health insurance system, with patients required to pay only 30% of the cost; in March 2021, Zolgensma was incorporated into the UK National Health Service.
Due to the pricing of up to $2.125 million, some have questioned whether Novartis is holding children's lives for ransom.
Bloomberg once noted in a report that Zolgensma has triggered two completely different reactions in the United States:Critics argue that this is a case of pricing gone out of control; on the other hand, supporters say that Novartis has found a cure for this rare disease, which can save desperate families.
In response to societal and public skepticism, the CEO of Novartis once stated: "Those critics haven't really thought about how our healthcare system works. We perform transplant surgeries on patients, costing each patient between $3 million to $5 million, but the outcomes are far less effective than these drugs." The CEO added that if this therapy is only administered once, it would be cheaper than current treatments.
According to Novartis' financial report, the sales of Zolgensma reached 920 million US dollars in 2020, increasing by 151% year-on-year. The performance of Zolgensma in the third quarter of 2021 was 375 million US dollars.(Including 126 million U.S. dollars in the U.S. market); Total of the first three quarters: US$1.009 billion(Including $351 million in the U.S. market)。
3. The Case of Sky-High Priced Drugs Entering Insurance at "Floor Prices" is Not Easily Replicable
Currently, three SMA therapies have been approved globally, including Zolgensma, Biogen's Spinraza, and Roche's Evrysdi, with the latter two already approved in China.
Spinraza(Nusinersen Injection)Approved by the National Medical Products Administration (NMPA) in February 2019 for the treatment of 5q spinal muscular atrophy.(Accounting for approximately 95% of all SMA cases), which is also the first drug approved in China for the treatment of SMA.
Previously, Spinraza was priced at nearly 700,000 yuan per dose in China, requiring repeated injections every year and being a completely self-funded medication.
Nusinersen Sodium Injection Image Source: Photo provided by the respondent
In the 2021 National Medical Insurance Drug Catalog Negotiations,Seven rare disease drugs included in the medical insurance directory, including the highly anticipated Spinraza for the treatment of spinal muscular atrophy.。
January 1, 2022, marked the implementation of the 2021 edition of the National Medical Insurance Drug Catalog in China.The price of a single dose of Spinraza drops from 700,000 yuan to more than 30,000 yuanAccording to the data from the Mei'er SMA Care Center, as of January 1 this year alone, more than 20 SMA patients in 11 provinces across China have already been treated with Spinraza.
Although the significant price reduction of Spinraza has given many patients with rare diseases hope, an industry insider admitted that the price cut of Spinraza is "(Price)Unexpectedly,(Joining Insurance)"Within Expectations."
"Spinraza is not a new drug, it has basically turned a profit in the global market. There are many children with SMA in China, representing a large drug market. The extent of Biogen's price reduction in the national reimbursement negotiations essentially reflects whether or not they want to capture the Chinese market," the aforementioned industry insider analyzed. The inclusion of rare disease drugs in the medical insurance catalog represents an individual breakthrough achieved through corporate price reductions within the established framework for medical insurance negotiations, and Biogen has strategically cast its vote in favor of China.
In recent years, the number of orphan drugs included in the national medical insurance catalog has been increasing annually. According to statistics from the Beijing Illness Challenge Foundation, after the 2021 National Medical Insurance Catalog negotiations, 58 drugs for 28 rare diseases have been included in the national medical insurance catalog. Based on the statistics of 87 rare disease indication drugs registered in China from the "First Batch of Rare Disease Catalog," the coverage of the medical insurance catalog has exceeded 60%. The 2021 medical insurance catalog negotiations achieved a "zero breakthrough" for high-value orphan drugs in the national medical insurance catalog.
But on the other hand, it is also an industry consensus that medical insurance cannot cover all high-cost drugs. Zheng Jie, head of the National Medical Insurance Negotiation Drug Fund Evaluation Expert Group, once publicly stated, "Currently, the annual treatment cost of all drugs in the national medical insurance directory does not exceed 300,000 yuan," and claimed that in the future, the National Medical Insurance Bureau will "resolutely prevent astronomically priced drugs from entering the medical insurance." The national medical insurance directory, which bears the function of "ensuring basic needs," determines that drugs included in it must meet the basic condition of being "reasonably priced."
This article comes from the WeChat Official Account:National Business Daily (ID: nbdnews), Editors: He Xiaotao, Wang Jiaqi, Yi Qijiang