Bojian’s high-priced SMA drugs are on the hot search again, where is the “difficult” drug for rare diseases?

It is more desperate than no medicine to cure.
It is probably that there is no medicine to use.
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The cost of treatment is worth a house, and the sky-high orphan medicines are repeatedly on the hot search, reflecting that it is not only the “difficult” medicine for rare diseases.
Dilemma
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A few days ago, some media reported that a patient under the age of 2 was diagnosed with spinal muscular atrophy (SMA) in the Children’s Hospital of the Second Affiliated Hospital of Xi’an Jiaotong University
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However, this is not the first time that Nosina Sodium has been searched
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Biogen's Noxinagen Sodium Injection (Spinraza) is the world's first SMA precision targeted therapy drug.
The drug was approved in the United States for the first time on December 23, 2016.
In terms of pricing, Spinraza's first year price was 750,000 US dollars.
After that, the annual price is 375,000 US dollars, the 5-year cost is 2.
25 million US dollars, and the 10-year cost is 4.
5 million US dollars
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In February 2019, Noxinassen Sodium Injection was formally approved by the National Food and Drug Administration for the treatment of 5q spinal muscular atrophy (SMA), and it became China's first drug for the treatment of SMA
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In 2019, not long after the launch of Nosinagen Sodium, Biogen and the China Primary Health Care Foundation launched the "Spine Revitalization-SMA Patient Assistance Project"
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On January 4, 2021, the assistance program for patients with spinal muscular atrophy of Biogynocinacine Sodium Injection was launched
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For families with average economic conditions, this is undoubtedly a huge burden, and it may not even be able to bear it
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Since the drug requirements for medical insurance negotiations in 2019 must be approved before December 31, 2018, Bojian’s Noxinagen Sodium Injection failed to catch up with the “shuttle” of the negotiations that year
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In 2021, the adjustment of the National Medical Insurance Catalogue will be launched, and Bojian's Noxinagen Sodium Injection has also entered the preliminary review list
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This time, whether Noxinagen sodium can enter the medical insurance catalog has also attracted much attention
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According to the previous introduction of the National Medical Insurance Bureau, the national level has successively released 5 editions of the medical insurance drug catalog, and the number has increased from 1,535 to 2,709
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Recombinant human coagulation factor, riluzole and other rare disease drugs and most symptomatic treatment drugs are included
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In 2019, a new Levocarnitine oral solution for the treatment of primary carnitine deficiency in children was added, and teriflunomide tablets for multiple sclerosis and wheat for the treatment of type C Niemann-Pick disease More than 10 rare disease drugs such as glestat capsules and selepagram tablets, bosentan tablets, maxitentan tablets and lioxigua tablets for the treatment of pulmonary hypertension are included in the medical insurance list
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However, the Medical Insurance Bureau also admitted that some particularly expensive drugs for special rare diseases cannot be included in the basic medical insurance payment due to reasons such as far exceeding the fund and the affordability of patients
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From the standpoint of the medical insurance fund, the per capita funding for medical insurance for urban and rural residents in 2019 is only about 800 yuan, and basic medical insurance cannot afford some particularly expensive drugs for rare diseases
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According to statistics from the Pain Challenge Foundation, a rare disease charity organization, the 2020 medical insurance catalog includes 55 drugs for rare diseases, involving 24 types of diseases, and the annual treatment cost of these drugs is less than 700,000 yuan
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As we all know, a new drug usually takes an average of 10 years from research and development to market, and costs 1 billion US dollars.
The research and development of a new drug is a high-risk and high-yield activity
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The reason why SMA medication is so expensive is that compared with common diseases and chronic diseases, the number of patients is small, and there are not enough patients to share the cost, and the price paid by a single patient is naturally high
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Moreover, before Bojian’s Nosinagen Sodium was listed, there was no drug to treat SMA in China, and the manufacturing companies were naturally strong
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According to the procedures disclosed by the National Medical Insurance Administration, the adjustment of the National Drug List is divided into five stages: preparation, declaration, expert review, negotiation, and announcement of results
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The 2021 medical insurance catalogue will be adjusted soon, and it has now entered the expert review stage
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According to the previous introduction of the People's Daily Health Client, the drugs included in the preliminary review list after passing the application will be calculated by the pharmacoeconomics expert group and the fund calculation expert group
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After two independent calculations based on pharmacoeconomic evaluation and fund calculations, the drug negotiation price range is determined according to certain rules, and finally the medical insurance negotiation price is formed
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(Pharmaccoeconomic evaluation is calculated from the clinical value, patient benefits, adverse reactions, international prices, and the competitiveness of the drugs themselves; the fund measurement is based on the impact of these drugs on the fund if they are included in the catalog
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Another authoritative person said that only medicines with an annual treatment cost of less than 500,000 yuan in medical insurance negotiations in 2021 will be eligible for negotiation
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From this point of view, after the price cut, if the price of Noxinagen Sodium Injection is lowered again during the negotiation, it is still possible to enter the medical insurance list
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So, according to the second-chong medical insurance catalog, can the price-reduced Noxinagen Sodium Injection be included in the medical insurance catalog? If you want to enter, how much should the price drop at least?

Jin Chunlin, director of the Shanghai Hygiene and Health Development Research Center, said that when medicines are developed, they are wasteful if they are not used.
It is undoubtedly hoped by many parties to reduce the price of medicines and let more patients use them, but there are many factors that need to be considered.

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The Medical Insurance Bureau decides whether to include a drug or not, and it will also be considered after a comprehensive calculation
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It is not easy to say what kind of reduction can be achieved in the end
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However, judging from the past >
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Another industry insider said frankly that whether the sky-high rare diseases can be included in medical insurance, and at what price should be included in the medical insurance, this is not only a price issue of concern to the outside world
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First of all, to enter the medical insurance catalog, it is generally a drug with sufficient market competition and a reasonable price.
However, the nature of rare diseases determines that it can be difficult to enter a particularly reasonable and appropriate range
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On the one hand, pharmaceutical companies spend a huge amount of research and development costs, and they are not charity organizations, and they must also consider their own input-output benefits
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On the other hand, China's medical insurance catalogue mainly guarantees basic insurance.
From the perspective of medical insurance, the less expensive it can be considered
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Data show that spinal muscular atrophy (SMA) is a rare hereditary neuromuscular disease with an incidence of about 1/6000-1/10000 in newborns.
According to the age of onset and the acquisition of exercise mileage, SAM is divided into SMA-I, II, III and IV.
Without treatment, most children with SMA-I cannot survive to two years of age
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According to the "White Paper on the Survival Status of SMA Patients in China", there are currently about 30,000 SMA patients in China, and 80% of them are infants
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Based on the estimation of China's annual new born population of about 15 million, there are about 1500-2500 new cases of SMA patients in China each year
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The attribute of China's medical insurance catalogue is basic insurance.
For the entry of high-priced drugs, especially such orphan drugs, whether medical insurance can afford the entry of such drugs has always been controversial
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Behind the controversy, it also reflects that China's medical insurance system needs to be further enriched and improved
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In this regard, the National Medical Insurance Bureau has repeatedly stated that it will vigorously promote the development of commercial health insurance with a sound medical insurance system at multiple levels, give full play to the role of commercial insurance risk management and protection functions, and improve the medical protection level of insured patients at multiple levels
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It is worth noting that some places are currently exploring rare disease medical insurance strategies
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For example, Zhejiang established the insured person to establish a special fund for rare disease medication protection at the standard of 2 yuan per person per year, and the upper limit of the patient's annual self-finance is 100,000
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In addition, some commercial insurance products also include the medical expenses of rare disease patients in the scope of protection, providing patients with medical expenses protection
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As of the end of July this year, inclusive insurance has been launched in many regions across the country.
It is understood that, for example, Guangzhou "Suisuikang", Foshan "Ping An Buddha", Hangzhou "Xihu Yilianbao", Wenzhou "Yikangbao", Qingdao "Qinbao" Dao E Bao, Jiaxing, Jiaxing Critical Illness, and Zibo Qi Huibao, etc.
have included the rare-disease drug Nosina Sodium
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On the whole, patients can get medical insurance of up to more than 2 million to more than 3 million yuan each year for only about 100 yuan to 180 yuan
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On the other hand, SMA treatment drugs have also been approved one after another
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After Biogen's Nosinagen Sodium was launched, Novartis' gene therapy Zolgensma for the treatment of spinal muscular atrophy was approved by the FDA in May 2019.
At the beginning of the market, the drug once cost US$2.
1 million (approximately US$2.
1 million).
(14.
48 million yuan), known as the most expensive drug in history
.
However, unlike Spinraza, Zolgensma can complete the treatment in one go
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In March and May 2020, Zolgensma was approved by Japan and the European Union, and the drug is not currently on the market in China
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At the same time, Bojian's Spinraza also faces the challenge of Roche Risdiplam
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Spinraza and Zolgensma are both used for patients with type 1 SMA, and Risdiplam is aimed at SMA patients who have no treatment options, and is also the first oral drug that can treat all types of SMA
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Roche's Risdiplam was approved by the FDA in August 2020 for the treatment of spinal muscular atrophy in adults and children over 2 years of age
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So far, the drug has been approved for marketing in more than 40 countries and regions, including China
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Roche's Risdiplam (Chinese generic name: Lisporan oral solution powder, trade name: Ai Manxin) was approved in China in June 2021 for the treatment of spinal muscular atrophy in patients 2 months of age and older
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According to industry sources, the price of 60 mg/bottle of Lisporan oral solution is priced at 63,800 yuan, and Roche has also launched a related charity assistance program
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In the preliminary review list for the 2021 medical insurance catalogue adjustment announced a few days ago, like Bojian’s Spinraza, Roche’s is also among them.
If everything goes smoothly, two SMA treatment drugs will be negotiated at the same time
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In recent years, the country has also attached great importance to the diagnosis and treatment of rare diseases and the protection of medications
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In June 2018, five ministries and commissions including the National Health Commission jointly formulated the "First Batch of Rare Disease Catalog", and a total of 121 diseases were included
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In 2019, the Health Commission issued the "Guidelines for the Diagnosis and Treatment of Rare Diseases (2019 Edition)".
In addition, China has established a national rare disease diagnosis and treatment collaboration network covering 324 hospitals, and established a two-way referral and remote consultation system within the collaboration network.
The Supervision Bureau will give priority to the clinical examination and approval of drugs that are urgently needed.
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It can be seen that the problem of difficult diagnosis and treatment of rare diseases is slowly being resolved
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Drugs for rare diseases are also called orphan drugs.
The above-mentioned industry insiders also suggest that in terms of rare disease protection, a legal system should be established, including rare disease drug research and development, business licenses, medical insurance, and medical services.
Personnel training and other aspects, from the overall macro framework, combined with the strengths of all parties, may be able to truly and better solve the problem of difficult medication for rare diseases
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