The last day of February each year is the International Day of Rare Diseases. This article was first published on 22 July 2020 and reissued today on International Rare Disease Day to focus on the difficulties encountered by people with rare diseases.
Each of us has the potential to be a rare disease patient or a carrier of a rare disease gene.
On average, 7 to 10 groups of genes are defective. Of the more than 7,000 rare diseases known worldwide, 80% are caused by genetic defects. As long as life multiplies, there is a possibility of rare diseases.
Of the more than 7,000 rare diseases that have been identified, only about 10% have symptomatic medications. For patients, it is fortunate that drugs are available. However, in China, due to the small number of patients and the unclear prospect of commercial returns, rare disease drug research and development has rarely been discussed by enterprises. Rare disease drugs that have been marketed are difficult for patients to reach because most of them are highly priced. This creates puzzles outside of medicine.
In view of the high cost, from the local to the national, different levels of guarantee mechanisms have been introduced. Local governments have taken the lead in exploring rare disease protection mechanisms with a view to reducing the burden on local patients. At the same time, the problem arises: out-of-pocket expenses after reimbursement are still unbearable for most patients – the "last mile" of Medicare is still bumpy.
Not only that, under the background of low overall planning level, local exploration objectively exacerbates national injustice, and "the same disease and different lives" has become a cruel reality that many patients need to face.
This article is written by Liang Zhen
Wang Yuanjun of Shaoxing, Zhejiang Province, had already set a "death date" for his son Wang Yu - 3 years.
In January 2019, two-year-old Wang Yu was diagnosed with "Pompeii disease". This is an alias for the rare disease glycogen accumulation disease type II. Before the onset of the disease, the patient is no different from a healthy person. After the onset of the disease, early-onset patients often die of heart failure and respiratory failure around the age of 1 year, and late-onset patients often die of respiratory failure, more often after the age of 1 year, and even later than after the age of 60 years.
Pompeii disease is curable, but expensive. At present, the only effective drug, cytarabase α (trade name: Meerzan), needs to be calculated according to the patient's weight and taken for life. The cost of medicine will increase from hundreds of thousands of yuan a year to several million yuan.
If used in sufficient quantities, Wang Yu can grow up healthy and no different from ordinary people; if the dose is reduced or the drug is stopped, it will be disabled and killed with the course of the disease. According to Wang Yuanjun's plan, he and his wife each lived in a dormitory, and then sold the only property in the family, which could get more than 1 million yuan. The money was only enough for my son to take as much medication as possible for 3 years.
At that time, there was no reimbursement policy for the relevant drugs in the local area. "At the time, it looked like a bottomless pit." Wang Yuanjun said. He felt responsible for another daughter and planned for the future of the whole family, so he set a 3-year deadline for his son.
Because of the high cost of medicine, parents of children can generally only choose to reduce the amount of use or find a foreign purchasing agent. Because it is a genetic disease, many domestic Pompeii disease families have multiple children with disease, and they can only take turns to take medication or give the only opportunity to use drugs to the worst situation. The vast majority of adult patients simply choose to abandon the drug.
But by the end of the year, Wang Yuanjun and his son were "saved."
On December 31, 2019, Zhejiang Province issued a notice on the establishment of a rare disease drug guarantee mechanism in the province, clarifying that the annual drug cost of individual patients will be capped at 100,000 yuan, and will organize negotiations on rare disease drugs.
On June 24, 2020, Zhejiang Province announced the results of the negotiation of special drugs for rare diseases, and cytarabidase α was included in the scope of payment for rare disease drugs in Zhejiang Province.
A health insurance policy expert, who did not want to be named, commented on the current situation of medical protection for rare diseases in China:
"The general trend is good, but there are more problems and more challenges. At present, the drug supervision has gone the fastest, and in terms of priority review and approval, an institutional arrangement has been made for rare disease drugs. But in the medical insurance sector, in the past 10 years, some rare diseases (drugs) have successively entered the medical insurance, but they have not yet formed a systematic operation mode."
Dilemma: There is medicine, but I can't afford it
In the past three years, the "drug-free problem" of rare diseases in China has been alleviated. Patients have strongly called for attention to the problem of "foreign drugs, domestic drugs", which is also very prominent among rare disease patients. However, with the blessing of various policies, rare disease drugs have been listed abroad, and the addition of China has been significantly accelerated. New drug research and development enterprises focusing on the field of rare diseases have also begun to appear in China.
At the end of drug approval, priority review, accelerated approval and other new drug approval system reform policies are clearly inclined to rare diseases. At the end of disease diagnosis and treatment, the National Health Commission (hereinafter referred to as the "National Health Commission") and other five departments jointly published the "First Rare Disease Catalogue", which includes 121 rare diseases. This is the first time that a disease catalogue has been defined at the national level as a rare disease.
By the end of 2018, 74 of the 121 rare diseases on the list had been approved for marketing abroad, involving a total of 162 therapeutic drugs. Of the 162 treatments, 83 are available in China, involving 53 rare diseases.
Previously, the approval of relevant drugs in China took a long time.
Arbosidase α, which treats Pompeii disease, was available in nearly 60 countries around the world before it was approved for marketing in China in 2017, 10 years after its first approval in the United States. The agasase β (trade name: Fabzan), which treats Fabry's disease, has been on the MARKET in the EU for 18 years before it was approved for entry into China in 2019.
The national high-level has also continuously given clear support for rare disease protection. At the executive meeting of the State Council in February 2019, the use of drugs for rare diseases, as a major disease prevention and treatment work related to the well-being of the people, enjoyed the same import tax reduction benefits as anti-cancer drugs to ensure the use of drugs for more than 20 million rare disease patients in China.
But when there is a cure, the question arises – whether the patient can afford it. Because of the small number of patients and limited market space, rare disease drugs tend to be priced higher.
Image source: Figureworm creative
In China, the public medical insurance system occupies the main position, and whether patients can afford high-priced drugs is directly related to whether related drugs are included in the medical insurance reimbursement system. In two national drug price negotiations for high-priced drugs in 2017 and 2018, four rare disease drugs eventually made it into the national health insurance list.
According to the "China Rare Disease Medical Insurance City Report 2020" jointly completed by the Rare Disease Development Center and international consulting company IQVIA, as of the 2019 national medical insurance catalogue update, 38 rare disease drugs have been included in the national medical insurance catalogue, involving a total of 21 rare diseases. Of these, 9 drugs are classified as Class A, i.e. 100% reimbursed, and patients do not have to pay out of pocket.
However, in the current medical insurance policy for rare disease drugs, the most breakthrough is local medical insurance.
In practice, various forms of disease protection with huge economic burdens such as rare diseases have been explored, including major illness insurance, medical assistance, and commercial insurance. Among them, it is relatively common to include rare diseases in major illness insurance, but the total amount of annual reimbursement will be limited, ranging from tens of thousands of yuan to hundreds of thousands of yuan.
The total amount limit means that after a year of medical insurance reimbursement for serious illnesses, if the price of the corresponding rare disease drugs is high, patients will need to pay a considerable amount of money out of pocket each year. "For example, the reimbursement limit is 300,000 yuan a year, but if a drug costs 1.3 million patients, where will he go to solve the remaining 1 million?" It should be a logic to set a limit on the out-of-pocket ratio, tell the patient how much he can bear at most, and then find a way to solve the others." Huang Rufang, director of the Corde Center for Rare Diseases, said.
This problem is particularly acute in the patient population that needs "ultra-high value rare disease drugs". Lysosomal deficiencies such as Pompeii disease, Gaucher disease and Fabry disease, which are included in the rare disease protection mechanism of Zhejiang Province, belong to this category. The annual drug cost is as high as millions of yuan.
"Because of the policies that come out of some places, patients may have hundreds of thousands of yuan to be reimbursed for a year, and they can't reach it." At least on our side, I feel like we can reach out and get it." Wang Yuanjun said.
Not all rare disease drugs are "sky-high prices".
"Not all rare diseases are so expensive. For patients with scleroderma, more than 30 yuan a month in Concordia prescribe a hospital preparation is well controlled. Another example is multiple sclerosis, which is about 200,000 yuan a year, which is already high, and after being included in the medical insurance reimbursement, the annual out-of-pocket payment is only about 20,000 yuan." Ma Tao, deputy secretary general of the Pain Challenge Foundation, said.
Taking the medical assistance project of the Pain Challenge Foundation as an example, the average annual drug cost of rare diseases it assists accounts for the majority of 300,000 to 400,000 yuan.
However, the cost of hundreds of thousands of yuan a year is still embarrassing for the average Chinese family. According to the 2019 Statistical Bulletin of China's National Economic and Social Development, the median per capita disposable income of urban residents is less than 40,000 yuan, and the median per capita disposable income of rural residents is less than 15,000 yuan.
The exploration of the protection of "ultra-high value rare disease drugs" is also exploring the way for the protection of the entire rare disease drug protection.
Zhejiang test, up to 100,000 out-of-pocket payment per year
"Multi-party co-payment" is considered to be an effective way to solve the problem of drug protection for rare diseases. The academic community and the industry have been calling for many years, but they have not landed. Many parties include governments, pharmaceutical companies, commercial insurance, patients, charitable organizations and other stakeholders. In recent years, multi-party co-payment has been summarized as "1+N".
In terms of functional positioning, resource mobilization ability and other aspects, the government is considered to be the best choice for the "1" role." Government leadership includes two meanings: Health care should dominate, and the government should take the initiative to do something." The medicare expert said.
At the end of December 2019, the policy of drug guarantee mechanism for rare diseases in Zhejiang Province was officially introduced, which was the first in the country. Provincial overall planning, special funds, and individual out-of-pocket ceilings are its three major characteristics. "Zhejiang Province is designed from the overall system, which is more comprehensive or one-step in place." Huang Rufang commented.
Because of the implementation of the municipal medical insurance policy (that is, local self-financing), even if it is implemented in the same province, even neighboring cities may be different. Zhejiang implements "provincial overall planning", that is, the same standard is implemented throughout the province in five aspects: the scope of protection, financing standards, treatment levels, diagnosis and treatment norms and medication management.
The "special fund" means that the Zhejiang Rare Disease Drug Protection Fund is specially established according to the amount of 2 yuan per person per person per year for basic medical insurance participants. The fund sets up a sub-account under the special financial account of the provincial medical insurance fund, and the two implement separate account management, and each account is independently accounted for. As of the end of 2019, the number of people insured by basic medical insurance in Zhejiang Province was 54.61 million, and based on this calculation, the total amount of the Rare Disease Drug Protection Fund in Zhejiang Province was 109.22 million yuan.
Xie Junming, vice chairman and alternate chairman of the Rare Disease Branch of the Zhejiang Medical Association, said that from the perspective of insurance, in the current domestic medical insurance, the basic medical insurance guarantees the basic medical treatment of the insured, and the major illness insurance guarantees the treatment of major diseases, and everyone may also suffer from rare diseases, so the public medical insurance fund should also guarantee the payment of rare diseases.
"The inclusion of rare disease protection in the health insurance fund is conducive to overall arrangements in the system. The establishment of special funds is a professional thing for professional people." Xie Junming has participated in the policy design and consultation of rare diseases in Zhejiang Province for many times.
The biggest highlight of the rare disease protection mechanism in Zhejiang Province is that the maximum out-of-pocket expense of individuals is 100,000 yuan per year. According to the design, the patient's drug expenses in a settlement year are reimbursed in segments: 0-300,000 yuan, reimbursement of 80%; 300,000-700,000 yuan, reimbursement of 90%; more than 700,000 yuan of expenses, full reimbursement.
The above-mentioned health insurance expert explains: "This is related to our health insurance concept of special emphasis on financial management in the past. Basically, the break-even, even if the task is completed, the value of this piece of consideration is less. (Zhejiang) This is very meaningful, it has changed from the pursuit of financial balance as the main value pursuit to the value pursuit of patients' medical needs."
The policy design of the out-of-pocket cap also allows social forces such as non-profit organizations to see hope.
For out-of-pocket expenses of up to 100,000 yuan, if patients still can't afford it, the Zhejiang Rare Disease Drug Guarantee Mechanism proposes two ways: medical assistance and charitable assistance.
On June 4, 2020, the Pain Challenge Foundation and other social organizations jointly launched the "Rare Disease Medical Assistance Project - Zhejiang Special Aid Fund Kick-off Meeting" online live broadcast. Through this project, patients in Zhejiang Province whose diseases are on the "121 Catalogue" and who are treated in designated institutions can apply for financial assistance with a maximum limit of 50,000 yuan.
"We want to send a message to the outside world: as long as the government says it moves, the people will follow." The private sector has kept up and helped to revitalize local resources, and insurance resources have come in again, and there will be a model model." Ma Tao, deputy secretary-general of the Pain Challenge Foundation, told "occasional cures".
According to Ma Tao, the foundation is already in contact with insurance companies and pharmaceutical companies to discuss solutions to solve the out-of-pocket costs of patients.
Prior to the Zhejiang Project, the Pain Challenge Foundation launched medical assistance for rare diseases as early as 2018. In two years, it finally assisted 533 patients and spent 7.03 million yuan. However, in January 2020, this nationwide, non-targeted assistance program was suspended.
"We found that the supply costs 1 million patients 50,000 yuan a year, which is a drop in the bucket for him." Ma Tao said. Therefore, the Pain Challenge Foundation decided to adjust the direction of assistance and target the medical assistance project for rare disease patients whose corresponding drugs or means have been included in medical insurance. "Most of the cost has been reimbursed by medicare, we push it, and the patient can continue to take the drug if he tries harder."
There is another objective reason for making this adjustment, "In the past two years, Internet fundraising has not been so easy."
Addressing the out-of-pocket costs of patients with rare diseases is key to restoring normal social functioning in families hit by the disease.
Many people with rare diseases can restore most of their social functions if they can use their medications normally, including working and living like healthy people. On the other hand, after taking the drug, regardless of whether the patient can return to normal social functioning, at least a stable state can be maintained, so that the labor force of other family members can be liberated, and the whole family can enter a normal operation and recyclable state.
Zhejiang Province has introduced a drug guarantee mechanism specifically for rare diseases, which has actually been carefully considered and prepared. According to Xie Junming, according to many factors such as time to market, disease severity, treatment effect, patient social appeal, etc., the rare diseases included in the guarantee mechanism were finally delineated. More importantly, Zhejiang conducted research and mapping on these rare diseases in the province and grasped the scale of patients in the province.
Mr Tse said: "In the middle of this process, the government is very cautious. From July 1, the government is also observing what problems will be encountered. In general, our wishes and designs are considered and should be able to hold the bottom. We hope to be able to evaluate and adjust the policy in a shorter period of time to evaluate the performance of the guarantee. The entire fund must be safe and controllable in order to have continuity. For follow-up arrangements (for other rare diseases), it is necessary to advance in an orderly manner."
Local differences are exacerbated, and the same disease is not the same life
Zhejiang is at the forefront of the country, and behind it is mostly regions with huge differences. The randomness and non-institutionalization of the national rare disease medical insurance policy are prominent.
According to the "China Rare Disease Medical Security City Report 2020", among the local models, the most practiced is to include rare disease drugs in the local medical insurance directory through local supplementary authority, but only involving individual rare disease drugs.
There are also some places that have negotiated the inclusion of some rare disease drugs in local major illness insurance. The reimbursement ceiling line ranges from tens of thousands of yuan to hundreds of thousands of yuan. A small number of cities include some rare diseases in the scope of local medical assistance or cooperate with commercial insurance companies to launch policy-based commercial insurance. However, the latter has strict limits on the scope of protection.
In addition, there is another way - "sporadic additions".
"Sporadic supplements", that is, the inclusion of drugs for certain rare diseases in the local medical insurance directory. The reasons for inclusion are mostly media reports that have attracted attention or that local people's congress deputies and CPPCC members have appealed. There is also the ability of patients to mobilize resources in the local area or are willing to work hard to visit and lobby the local authorities for many years.
At present, the domestic rare disease protection policy is highly fragmented. Even patients with rare diseases with reimbursement policies have different situations depending on the region.
Zhao Yumei's daughter cried badly when she got the first shot, and she cried herself. It was the end of April 2019, zhao Yumei's daughter took the first medication. Her daughter, 23, waited 10 years from the time she was diagnosed with Pompeii disease to the time she was medicated. Their family lives in a county seat under the jurisdiction of Xinzhou City, Shanxi.
In April 2019, Shanxi Province issued a document to include Pompeii disease in the local major illness insurance coverage, and reimbursed the cost of medication at a rate of 50%. The remaining expenses are reimbursed by 60% of the special relief funds. In addition, eligible patients can apply for medical assistance after the completion of the two reimbursements, at a rate of 70%.
Zhao Yumei's daughter is 1.5 meters tall, weighs only 32 kilograms, and the cost of using cytarabase α is as high as more than 1 million a year, and after two reimbursements, Zhao Yumei's family still needs to pay nearly 250,000 yuan at their own expense.
"It took almost a year and a half from 2019 to now, and the out-of-pocket expenses totaled a little more than 300,000. Now it's almost exhausting. Borrow everything that should be borrowed." Zhao Yumei said.
Every time the daughter's medicine money, Zhao Yumei's family is almost all cash raised. "After using it once, if you have raised enough money, you may go again, maybe three weeks or 20 days, anyway, just push it back." This condition is fairly common in the population of pompeii patients.
Zhao Yumei's husband's monthly income of more than 4,000 yuan is the only source of income for the whole family. The family's annual income is only more than 50,000 yuan. After Zhao Yumei was laid off, she had no formal job. In recent years, because her daughter has been inseparable from the ventilator, she can only barely walk with the help of the outside world, plus she has cirrhosis of the liver, and Zhao Yumei is at home full-time.
From 1999, when Zhao Yumei found something wrong to her daughter's diagnosis in 2009, Zhao Yumei had been bitterly uncomfortable.
The daughter who knew the result asked, "Mom, is there any medicine for this disease?" She didn't lie: "Yes, but we can't afford it."
After 10 years, my daughter finally took the medicine. But Zhao Yumei was once again in a dilemma.
Although there are relevant medical insurance reimbursements, the annual maximum reimbursement of Shanxi Major Illness Insurance is 400,000 yuan. From January this year, the limit of Zhao Yumei's family is about to arrive. Zhao Yumei has also applied for medical assistance from the Pain Challenge Foundation, but the gap of 50,000 yuan a year is still huge compared with out-of-pocket expenses.
According to external standards, compared with Zhao Yumei, Wang Yuanjun's family in Shaoxing, Zhejiang Province, has much superior conditions. Both husband and wife are urban workers, and their annual income is about 200,000 yuan.
But Wang Yuanjun told "occasional cures": "There is no family on our side who can support adequate medication, even if it is a child." My family's conditions are relatively better, but this disease can make ordinary well-off families suddenly become poor families."
Since the birth of the child in 2017, Wang Yuanjun's family has spent nearly 700,000 yuan on the treatment of the child.
Huang Rufang believes that the differences in rare disease protection policies in various places are mainly caused by three reasons: local financial strength, local medical insurance experience in the past exploration and practice, and the relevant awareness of local governments.
The above-mentioned domestic medical insurance expert further explained that this is related to the low level of domestic medical insurance co-ordination. Behind the low level of overall planning, it reflects the basic contradictions of the domestic medical insurance system.
"Because of the limitations of national conditions, the development of various regions is not balanced. The imbalance has led to an imbalance in the allocation of medical and health resources, and has also led to an imbalance in the allocation of medical insurance resources. This is a basic contradiction, that is, the contradiction between the people's growing demand for medical security and the unbalanced and insufficient allocation of medical security funds."
Drug price negotiation, health insurance "last mile"
Solving the problem of affordable drugs for rare diseases and ensuring the use of drugs for rare diseases has gradually reached a consensus at the national level. But under the consensus, the game of testing the determination and wisdom of all parties has also emerged.
First, the price game.
According to the "occasional cure", the rare disease drug negotiations in Zhejiang Province actually invited more than one. But in the end, the two sides did not negotiate, the direct reason is that the company does not want to reduce prices.
Judging from past drug price negotiations, it is difficult for local-based negotiations to achieve the same effect as national drug price negotiations. In cases where the number of rare diseases may be below double digits, local bargaining power is weak. On the other hand, the gap between the innovation ability of domestic drugs and the mature markets in Europe and the United States is obvious, and the rare disease drugs of multinational pharmaceutical companies have almost no market competition in China.
In general, multinational pharmaceutical companies will consider two aspects in pricing: the cost of existing treatments and the pricing of neighboring countries. Whether to reduce prices is considered: the impact on the global price system and whether the expected benefits can be obtained after price reductions.
A former Sanofi strategic planning department told "occasional cures" that the pricing considerations of multinational pharmaceutical companies in China are now in many cases from the perspective of medical insurance. "You can't get into health insurance, basically this drug can't sell much (sales)." Therefore, when listing pricing, it will leave room for future negotiations on price reductions. Companies are willing to cut prices, but it is another question, and it is unlikely to fall to a very low level."
The issue of expected benefits is more realistic.
On the one hand, China's rare disease market is currently only expected to be large, but it is far from being developed. "In theory, there are more patients, but the diagnosis is minimal." The market source said. According to him, for example, the rare disease Gaucher disease is expected to have 5,000 to 8,000 people in China, but less than 500 people have been diagnosed; Pompeii disease is expected to be about 10,000 people in China, but only more than 100 people are now diagnosed.
"This means that the initial investment, such as diagnosis, doctor education, market education, etc., will be very large." You can't make much money upfront, and it may take 5 years and 10 years to pay off. This is also a consideration for businesses. From an investment point of view, you may prefer to do something that pays off quickly, such as tumors."
On the other hand, after the company cuts prices, if patients still can't afford to use it, their revenue loss will be even greater. "For example, even if the average annual cost of medicine for Gaucher's disease drops from one million to 500,000, how many people can afford it?" If according to the current reimbursement ratio in some places, patients also have to pay about 30% out of pocket, that is, 150,000. Not everyone can afford it. So it's important to solve the 'last mile' problem of Medicare payments."
For companies planning to expand the introduction of rare disease drugs to the Chinese market, the momentum brought about by the reform of the drug review and approval side may be weakened by the uncertainty on the payment side.
A person from the market access department of a rare disease foreign pharmaceutical company said: "Every product enters a country, the initial investment in all aspects is very large, if there is no sales at the terminal in the end, it is equivalent to this investment is a loss." As a headquarters, we may consider whether to continue to invest in this market."
The other game is even more invisible: fairness.
After Zhejiang Province announced the final negotiation results, the rare disease patients who had drugs but were not included in the scope of protection sent a joint letter directly to the Zhejiang Provincial Medical Insurance Bureau. They questioned why the drug they used was also newly on the market, but it did not appear on the list like another rare disease drug. For some time afterwards, the two patient communities were at odds.
Such contradictions, whether explicit or implicit, occur in many places where rare disease protection policies have been introduced.
The medicare expert said of "Occasional Cure": "The benefit of drugs for rare diseases involves three relationships: accessibility, fairness and sustainability. Accessibility is fundamental, sustainability is key, and equity is at the core. Fairness is actually a big challenge right now."
There has been action at the national level. In July 2019, the National Medical Insurance Bureau publicly solicited comments on the management system of the list of medical security benefits. The so-called treatment list system, in short, is to clarify what the domestic medical insurance system can do and what it cannot do, and strictly comply with it, reducing local discretion.
The document said that the purpose of this move is to fairly and appropriately protect the people's basic medical security rights and interests, and requires local governments not to establish other medical security systems beyond the framework of the basic system.
In the treatment list system, two points are considered to be of great importance to the protection of rare diseases.
One is decision-making authority. The basic policies of domestic medical security shall be formulated, adjusted and issued by the State Medical Insurance Bureau in conjunction with relevant departments, and local governments can only formulate specific financing and treatment policies within the scope of national regulations; one is that the payment items of medical insurance funds in various localities should be strictly implemented in accordance with the national medical insurance directory, and in principle, they must not formulate their own catalogues or use alternative methods to increase the drugs in the catalogue.
The "Zhejiang model" that has been concerned is actually to separate rare disease protection from major illness insurance. Major illness insurance is on the list of benefits, but rare disease medication coverage is not. At present, a considerable part of the exploration of various places is supplemented by the local medical insurance directory. Once the treatment list system is implemented, it means that local governments do not have the right to make these policy explorations.
"Why the state should use the right to collect rights and go in the direction of the list of benefits is precisely to ensure the fairness of all places." Now the unfairness is already manifested." said the above experts.
Source: Station Cool Helo
The implementation of the local overall planning model of the domestic medical insurance system, how to coordinate the relationship between the state and the locality, especially how to coordinate the directory access and reimbursement standards, tests the wisdom of all parties. "It's advantageous in the long run[ but it shouldn't be one-size-fits-all when local differences are taken into account." Once the knife is cut, it will "press the gourd and get up the scoop.""
In the view of the above-mentioned person from the market access department of foreign pharmaceutical companies, "the treatment list system shows that the policy is being collected, but it seems that there is no specific mention of rare diseases." Although the National Medical Insurance Bureau did not express its opinion on Zhejiang's approach, in other words, at least did not say that it was opposed."
According to the "occasional cure", there have been many discussions in the academic community about the rare disease protection policy at the national level. Before Zhejiang released its provincial policy, discussions on rare disease protection policies at the national level had already been carried out, and reached the level of the National Medical Insurance Bureau. At that time, the version proposed to establish an independent medical security system for rare diseases in China, which is characterized by universal financing, universal coverage, multi-party co-payment, and multi-level security.
Huang Rufang pointed out that many local models are not models for long-term development in the future, and need to be nationally oriented and guided at the national level.
"The pattern of exploration in each place has its own subjective and objective reasons, and it cannot be directly compared. However, some models are objectively flawed or have problems with sustainability. Not a rare disease problem should be borne by the government all the pressure and risk, and it must be paid for by multiple parties. The state can put the framework in place so that the localities have full initiative to participate in it, while giving guidance on a macro level."
The above-mentioned domestic medical insurance expert said to "occasional cure": "The institutional design of national rare disease protection is no longer a problem at the theoretical level, the problem lies in the practical level, that is, how to do it and who will do it."
Before the local or national government began to think and solve, the fates of the two families of Wang Yuanjun and Zhao Yumei have been divided in the "last kilometer" of the road to rare disease medical insurance.
Wang Yuanjun is now full of hope. "We are glad that the policy came out relatively early, and my family has less debt now, which can only be said to be fortunate."
"If you don't (continue) to use (medicine), the child's life will be turned back again, and the money was spent in vain before, and the child is still not good." 」 (Continue) use, or no money. Now the child is more tired and feels that it is difficult to ride the tiger." Zhao Yumei said.
In the text, Wang Yuanjun, Wang Yu, and Zhao Yumei are pseudonyms
Planner: Earth Cat, Yu Lu
Executive Producer: Gyouza
Title image source: Figureworm Creative