The former "sky-high" new drug "nusinersen sodium injection" will be included in the medical insurance at a price of 33,000 yuan in 2022.
Although the price has dropped significantly from the initial price of 697,000 yuan, the negotiators are still trying their best to negotiate the price and strive for a lower price for the people.
This "special drug" for the treatment of the rare disease spinal muscular atrophy (SMA) has entered the medical insurance, which has given hope to many patients' families.
Just when Chinese patients were happy about the "successful results" of the medical insurance negotiations, the news that "Australian patients can be treated for only 184 yuan" sparked widespread discussion.
Why can the "sky-high miracle drug" of 700,000 yuan a shot be sold in Australia? Is it true that such a low price is true?
1. Introduction to the condition of the rare disease SMA
The rare disease SMA, or spinal muscular atrophy, is a neurological disorder caused by a genetic defect on chromosome 5.
The severity of SMA varies, and clinicians classify it into four types. The most common and serious type 1 SMA is in love with infants, who have not yet seen the prosperity of the world, and the haze of disease envelops their immature lives.
The statistics are even more palpitating, and among many genetic diseases, SMA has one of the highest mortality and disability rates in infancy and early childhood.
In the face of SMA, an "old adversary", the medical community has been helpless for a long time. The lack of treatment has irreversible damage.
Patients with this type, usually after six months of life, show generalized muscle weakness, particularly decreased muscle tone and decreased reflexes.
As the disease progresses, it becomes difficult for children to breathe and swallow, and more than 90% of children with type 1 do not survive their second birthday.
Types 2 and 3 have a late onset, mostly between 1 and 2 years of age and after 2 years of age, and have milder symptoms and longer survival.
Type 4 is more common in adulthood, progresses slowly, and can survive for decades. Although the incidence of SMA is very low, its harm and impact on the human body is very large.
From the current data, it can be seen that SMA has become one of the genetic diseases with the highest mortality and disability rates in infancy, infancy and early childhood.
The lack of effective "treatment" for SMA and the lack of timely intervention often lead to irreversible motor function impairment and life-threatening conditions, which will bring great "suffering" to children with SMA and their families.
But in recent years, there has been a "revolution" in the field of SMA treatment that gives hope. The advent of nusinersen sodium injection is a milestone breakthrough.
It cleverly rewrites the fragmented "script" by regulating the "scissorhands" of the SMN2 gene to obtain a functional SMN protein.
Clinical trials have shown that three-quarters of children with type 1 treated with nusinersen sodium injection survive at 13.6 months of life and do not require continuous ventilation.
Second, 700,000 sky-high drugs have been negotiated into medical insurance!
It took a full decade for the biopharmaceutical company Cyagen to bring nusinersen sodium injection to market.
Countless R&D personnel worked day and night, experienced countless failures and setbacks, and finally got it done.
Ascletis was also under tremendous financial pressure. The research and development of new biological drugs costs a huge amount of money and billions of dollars at every turn, which is a "gamble" for start-up companies.
If R&D is blocked or fails, it means "losing everything".
Even if the new drug is finally approved for marketing, the company must recover the cost and achieve profitability within the limited patent protection period, and the "patent protection period" of nusinersen sodium injection is 20 years.
As a rare disease, SMA has a very limited patient base, with an estimated number of only about 30,000 patients worldwide and about 3,000 patients in China. Even if all patients were to use the drug, it would be difficult to reduce costs through economies of scale.
Behind the high pricing of innovative drugs, in the long run, only by giving pharmaceutical companies enough profit margins and return expectations can they truly stimulate the "endogenous power" of developing new drugs.
If the "low pricing" of nusinersen sodium injection will cause Ascletis to "lose all its money", will other pharmaceutical companies still be willing to get involved in rare diseases such as SMA?
Patients with SMA will lose valuable opportunities to take medication. Whether from the micro perspective of the enterprise or the macro perspective of the society, it is necessary to fully understand the necessity of reasonable pricing of innovative drugs.
July 2022 was a major negotiation involving the "medication rights" of more than 500,000 SMA patients.
On one side of the negotiating table are representatives of multinational pharmaceutical companies holding "life-saving medicines", and on the other side are the negotiation team of the National Health Insurance Bureau, which is responsible for protecting the medical insurance fund and fighting for drug opportunities for patients.
"The sky-high price of 700,000 is definitely not good, this is far beyond the affordability of medical insurance, and the price must be greatly reduced!"
The negotiators of the Health Insurance Bureau got straight to the point, pointing out that the prices of drugs were inflated.
Considering factors such as R&D investment, patent protection, and the size of the patient population, this is their bottom line!
Representatives of foreign companies argued with reason, insisting that there was limited room for price reductions.
After some bargaining, the representative of the foreign company finally relented and said that he could consider reducing the purchase price to 53,680 yuan.
For the Health Insurance Bureau, this concession is still difficult to accept. After all, the government is not lavish in paying the bill, and such a high price is an overdraft of the future medical insurance fund!
After several price bargains, the representatives of pharmaceutical companies finally came up with a "final offer" of 33,000 yuan.
Although the price has dropped significantly from the initial price, the negotiation team of the Health Insurance Bureau still feels that the price is too high.
One conundrum was before the negotiation team:
Further forcing pharmaceutical companies to reduce prices is conducive to saving medical insurance expenses, but it will affect the enthusiasm of pharmaceutical companies and make it difficult to continue follow-up innovation.
If it is included in the medical insurance without moderation, it may benefit patients in the short term, but in the long run, how can the medical insurance fund afford it?
SMA is just one of many rare diseases, and there are many more "life-saving drugs" waiting to be included in medical insurance.
After some difficult trade-offs, the negotiation team of the Medical Insurance Bureau finally finalized the landing price of 33,000 yuan.
The negotiator said with a wry smile that the hardships of this negotiation process are unimaginable to outsiders.
The final transaction price of 33,000 yuan has reduced the drug burden of patients to a certain extent and met the actual needs of patients.
For the long-term operation of the medical insurance fund, leave room for transfer and avoid overdrawing in the future.
3. Horizontal comparison of Chinese and foreign prices, is it true that Australia has such a low price?
When nusinersen sodium injection was negotiated into the medical insurance at a price of 33,000 yuan, many netizens pointed the finger at the medical insurance bureau, and the negotiation power of the medical insurance bureau was too weak, right?
Industry insiders shook their heads again and again, this is a misreading, equating the welfare subsidy price with the purchase price, is confusing the concept!
It turns out that "41 Australian dollars" is actually a special subsidy program of the Australian government.
Under the scheme, the government will subsidise the cost of some children with SMA under the age of 18, reducing their out-of-pocket costs to $41, or $184.
This preferential price is not the actual purchase price of the drug, let alone applicable to all patients.
The Australian government's "actual purchase price" is 110,000 Australian dollars per bottle, or about 494,000 yuan, which is much higher than China's purchase price.
The same is true in the United States, where the starting price is $125,000, equivalent to about 796,000 yuan.
Although there is no nusinersen sodium on the market in Japan, Zolgensma, another drug with similar efficacy, is also priced as high as 157 million yen, with a total cost of more than 100 million yuan based on the use of 6 bottles.
The price of 33,000 yuan per bottle reached in China's medical insurance negotiation is the lowest among major countries in the world.
In developed countries, the high cost of orphan drugs cannot be fully borne by individuals or health care systems.
For example, providing exemptions, assistance, and even free medication for eligible children with SMA is a manifestation of the "government support".
As for adult SMA patients, they can only pay the full amount out of their own pockets and cannot enjoy any discount. In the U.S., Medicaid programs vary from state to state, and many patients often exceed the eligibility threshold and are not eligible for assistance.
The limitations of welfare subsidies stem from the limited nature of the medical insurance fund. Blindly emphasizing price reductions and profits is bound to crowd out medical insurance resources and is difficult to last.
In a populous country like China, it is unrealistic to implement full subsidies for all patients with rare diseases.
Under the premise of the current limited medical insurance fund, the China Health Insurance Bureau can rather say that it has achieved a win-win balance for all parties:
The negotiated price of 33,000 yuan, on the one hand, minimizes the out-of-pocket expenses of patients, especially the expenditure pressure of pediatric patients is no longer unbearable.
On the other hand, it does not excessively squeeze the space of medical insurance, leaving room for maneuver for the sustainability of the medical insurance fund.
Under the constraints of the tight reality of the medical insurance fund, how to rationally allocate limited resources and find a balance between the national "basic protection" and the key "serious illness".
With the aging of the population and the increase in the number of patients with rare diseases, how can medical insurance better protect people's livelihood and solve the pain points of people's livelihood that are expensive to see a doctor?
The negotiation highlighted the necessity of building a linkage mechanism such as medical insurance negotiation, medical insurance payment, and commercial insurance supplement.
On the one hand, in medical insurance negotiations, it is necessary to give full play to the role of strategic purchasers, and accurately evaluate the clinical value, financial affordability and other dimensions, so as to remove the low and retain the high.
The reform of medical insurance payment methods is the "golden key" to open the optimal allocation of medical resources.
Innovative models such as total budget and payment according to disease type are like a pair of skillful hands, accurately allocating every cent of medical insurance funds to maximize their effectiveness.
In this way, limited medical resources can be used where they are needed most, and every penny is well spent.
Although commercial health insurance cannot replace medical insurance, it can become a powerful supplement, and commercial insurance can meet people's multi-level and personalized health needs.
The negotiation of nusinersen sodium injection reflects the urgency of adapting to the trend of population aging and optimizing the operation mechanism of medical insurance financing.
China's elderly population over 60 years old has exceeded 260 million, and the three major trends of aging, aging and disability are superimposed, and the situation is very grim.
In the face of the stress test of an aging population, the medical insurance system must keep pace with the times and innovate.
With the continuous emergence of new drugs for rare diseases, how to find a balance between responding to the actual needs of patients, and the construction of a systematic system, and "walking a tightrope" between cost control and benefiting the people, will become a must-answer question in front of medical insurance.
With the feelings of people's livelihood and continuous improvement of the system, only then can expensive innovative drugs be reduced, used and more effective.
epilogue
When nusinersen sodium injection joins the medical insurance at a "value" of 33,000 yuan, tens of thousands of SMA patients will have the hope of a cure.
Although the price of many drugs is still very high, it is believed that in the near future, ordinary people will be able to use it.
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Reference Links:
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