Recently, a "seeking medicine" message released from the media has attracted many attentions. According to the article, the infant in Hunan who has just turned 1 year old this year is suffering from a rare disease called spinal muscular atrophy (SMA) and is in urgent need of special medicines, but he has to pay “700,000 yuan” for medical expenses, and the medicine is in Australia. The price is "$41".
However, according to public reports, the price of one dose of the drug "Nosinagen Sodium Injection" that fits the disease is US$125,000. The drug requires five to six injections in the first year and will cost between US$625,000 and US$750,000.
In addition, a reporter from China Business News learned that Nosinagen Sodium Injection was launched in China on April 28, 2019. Earlier in May 2018, "Spinal Muscular Atrophy" was also included in the "First List of Rare Diseases" jointly formulated by the National Health Commission and other departments. It is understood that the 121 rare diseases that have entered the list are expected to be given priority consideration at the levels of drug approval and listing, inclusion in medical insurance payment, and participation in assistance projects.
How to improve the accessibility of expensive rare disease drugs? What kind of payment model is more acceptable?
Need 700,000 per stitch?
"Spinal muscular atrophy" is a rare genetic neuromuscular disease with an incidence of about 1/6000~1/10000 in newborns.
In addition to the Noxenagenic Sodium Injection produced by Biogen, another drug for the treatment of SMA is the gene therapy Zolgensma developed by Novartis' AveXis company, but the latter is currently only available in the European Union, the United States, and Japan. The SMA drug developed by Roche also submitted an application for listing in China on April 23 this year, and entered priority review in June, and is expected to be approved within the year.
Wang Yi, Director of the Management Office of Fudan University Hospital, said that since 2004, SMA antisense oligonucleotide (ASO) therapeutic targets have been identified, which can be used to selectively bind target RNA and regulate gene expression.
On the basis of this research, the intrathecal injection of Noxinassen Sodium Injection (administered through the nervous system by lumbar puncture) started a phase I clinical study in 2011. In October 2019, China's first case of intrathecal injection of Noxinassen Sodium Injection was completed in the Children's Hospital of Fudan University. The patient was an 8-year-old child.
So how to buy and how to price the "Noxinagen Sodium Injection"? The reporter saw on the official website of "Shangyao Kangdele" that the drug's sales page showed "temporarily out of stock". The official website of "Hong Kong Jimin Pharmaceutical" has a purchase page for the drug, but you still need to "consult WeChat customer service" to know the price. .
The WeChat customer service told reporters, "The price of 12mg of Nosina Sodium is HK$820,000 each, and the model is the German version."
Biogen has publicly stated that the price of a dose of Noxinarine Sodium Injection is $125,000. The drug requires five to six injections in the first year and will cost between US$625,000 and US$750,000. After that, three doses must be injected each year at a cost of approximately US$375,000. Patients may need to inject Noxinagen sodium for life.
The domestic price is far less than the above-mentioned pricing. "In China, there is the'PAP Patient Assistance Program' for the treatment of SMA patients. Under this assistance program, patients need to inject 4 loading doses of Noxinassen Sodium Injection within 2 months. Form; after that, it will be injected once every 4 months, using the form of buying 1 needle and giving 1 needle.” Huang Rufang, founder and director of the Corder Center for Rare Diseases (CORD), said in an interview with reporters, “ Currently, 64 patients have been treated under this project."
That is to say, under this project, patients need to pay about 700,000 yuan for the cost of Nosinagen Sodium Injection in the first 2 months, but the average injection is about 175,000 yuan; after that, the average injection is once in 4 months. Calculated on the basis of 6 injections in 2 years, a total of 2.1 million is needed, and the average annual cost is 1.05 million.
What is the price of Novartis' Zolgensma? Novartis’s official website shows that “one-time treatment can be used to replace chronic treatments that need to last a lifetime.” The reporter also learned that Novartis Zolgensma costs about 425,000 US dollars a year, which will last for 5 years, which is equivalent to nearly 1,500 yuan in total. Ten thousand yuan.
Huang Rufang further told reporters: "The'MAP Patient Assistance Program' developed by Novartis overseas has 100 places, but the conditions are more demanding. For example, patients are required to issue a test report at one of the few testing institutions in the world, and the patients must be children under 2 years old. Wait."
How to improve access to medicines?
In Australia, Nosinassen Sodium Injection was included in the local Pharmaceutical Benefits Program (PBS) on June 1, 2018 for the treatment of spinal muscular atrophy type 1, type 2 and type 3a patients under 18 years of age.
According to PBS regulations, patients need to pay a certain amount for the subsidized drugs in the plan. From January 1, 2020, patients (holding an Australian health insurance card) will only need to pay up to $41 for most PBS drugs, and if they have a concession card, they will only need to pay $6.60. The remaining costs are paid by the Australian government. The amount that patients need to pay will be adjusted according to the Consumer Price Index (CPI) on January 1 of each year.
It is reported that PBS started in 1984. According to official data, as of May 2004, the cost of the drug benefit program has increased by nearly 13% annually in the past 10 years. Among them, the factors that increase costs include newly developed expensive drugs, over-prescription, aging population, and increased patient awareness and expectations.
"In China, what we have been discussing is the '1+N' multi-party co-payment model." Huang Rufang said, "For example, a treatment fee of 1 million yuan will be borne by the government for 60% to 70%, and the rest is through the enterprise Price reduction, social assistance, commercial insurance, personal commitment, etc. are used to solve the problem."
At the same time, Huang Rufang introduced that there are seven modes of local protection for rare diseases, "including special funds, major disease negotiations, financial contributions, policy-based commercial insurance, medical assistance, sporadic supplements of medical insurance, and independent declaration."
Huang Rufang believes: "The drugs for rare diseases must be included in the medical insurance as soon as possible. The individual rights of patients are equal to those of other citizens, and the national medical insurance should bear the patient's treatment costs as much as possible."
The reporter learned that in order to increase the availability of rare disease drugs to patients, local governments often negotiate with pharmaceutical companies to reduce drug prices.
For example, after Sanofi’s "Silzan", which treats Gaucher's disease, made a price cut of 1/3 last year, on June 2 this year, the Zhejiang Medical Insurance Bureau announced the negotiation results of two special drugs for rare diseases on the official website. "Sanofi's'Meerzan' (treatment of Pompeii) and'Fabuzin' (treatment of Fabre) were both shortlisted for negotiation."
In terms of cost protection, the Zhejiang Provincial Medical Insurance Bureau stipulates that patients with rare diseases shall pay their own expenses when visiting a designated treatment hospital, and other expenses shall be directly settled by the medical insurance agency and the designated treatment hospital. Drugs for rare diseases are not included in the scope of assessment of total medical insurance budget management and the proportion of drugs in designated treatment hospitals.
my country's current medical insurance system is similar to that of the United Kingdom. The NHS (National Medical Service) system in the United Kingdom makes the welfare of individual patients highly publicized and de-commodified. Individual disease treatment and health problems are relatively independent of income and not affected by their purchasing power.
And every medication or treatment of the NHS must be evaluated by NICE (National Institute of Health and Clinical Optimization). The evaluation takes the results of the "cost-benefit" analysis of drugs as the main criteria for measuring whether they are included in medical insurance reimbursement, puts clinical efficacy and economy in the first list of medical insurance selections, and maximizes the benefits of patients under a limited budget. (Intern reporter Wang Yuezhou also contributed to this article.)
Author: Zou Jie Zhen Yue Ho Shu Guo Jinhui