This is not the first time that Yang Linhua, a deputy to the National People's Congress and chief physician of the Department of Hematology of the Second Hospital of Shanxi Medical University, has spoken out for hemophile patients at the Two Sessions of the National People's Congress.
In 1996, she began diagnosing and treating a variety of blood disorders, including hemophilia. Hemophilia is a hereditary bleeding disorder, due to the lack of important clotting factors in the human blood clotting process, the body's blood clotting disorders, mainly manifested as spontaneous bleeding that occurs repeatedly since childhood or bleeding after trauma.
In China, there are nearly 40,000 registered haemophilia patients, and it is estimated that in fact, this group may reach 140,000.
As a rare disease, hemophilia patients have faced many problems in the past many years, such as insufficient timely diagnosis, insufficient standardized treatment, lack of drugs and heavy economic burden. These problems have been gradually seen and solved in the past decade or so.
How many steps are needed to achieve "early diagnosis and treatment"
In the consensus on the diagnosis and treatment of hemophilia, there has always been an emphasis on "timely diagnosis and early and appropriate treatment". Because the consequences of not getting timely intervention are very painful – disabling or even dying of bleeding from organs. There is currently no radical cure, and patients require lifelong treatment with supplementation with clotting factors.
But in reality, only a subset of patients with hemophilia can be diagnosed and treated in a timely manner.
"The first is that patients, families and grassroots doctors don't recognize the disease."
Clinically, Yang Linhua always found that in such patients, joint swelling - the clinical manifestations of hemophilia include bleeding from joints, muscles and deep tissues, and can also have bleeding from the gastrointestinal tract, urinary tract, central nervous system and bleeding after tooth extraction.
"Some mild and medium-sized patients have bleeding and swelling that is not very severe, just rest a little." Some doctors in primary medical institutions, if they are not specialized in blood diseases, may not be aware of this, and may also diagnose arthritis or other diseases."
At the same time, some patients with hemophilia are screened out before surgery. Yang Linhua told us that before the 1980s, China's preoperative requirements were not strict, and there was a great risk in the surgery of hemophilia patients without a well-prepared and thorough surgical plan.
"Therefore, the older generation of hematologists have repeatedly called for preoperative examination of coagulation so that mild to medium-sized hemophilia patients can be identified in the process."
Yang Linhua attended the 10th China Rare Disease Summit Forum.
Image source: Courtesy of respondents
In addition, the dilemma faced by primary medical institutions is not only here, but also limited to hardware, and some tests that need to be completed in the laboratory cannot be realized.
"This is why we have been promoting the establishment of hemophilia diagnosis and treatment centers at all levels across the country, which need to achieve homogeneous and standardized diagnosis and treatment, and patients cannot be introduced to big cities such as Beijing or Shanghai for examination," Yang Linhua said.
At present, the mainland has established designated diagnosis and treatment units for hemophilia in 31 provinces and cities, and various localities are also actively exploring and improving the hemophilia diagnosis and treatment network at all levels in provinces, cities and counties.
From myrrh to medicine
Hemophilia is mainly divided into two categories, of which type A hemophilia is the lack of clotting factor VIII., the number of patients accounts for about 80% to 85%, and the lack of clotting factor IX.
Both groups of patients require lifelong treatment with coagulation factors, including hematogenous coagulation factors and recombinant coagulation factors.
For many years, the former has been the main drug used by patients with continental hemophilia. In the past three decades, the use of blood-borne coagulation factors has run through a series of reforms in the domestic blood products industry.
"In the past, we were not yet mature in the detection of viral diseases, and some of them could not be detected during the window period. In the past, there was no recombinant coagulation factor, no imported ones, all of which came from hematogenous coagulation factors, so some hemophilic patients were infected with viral hepatitis, and a small number of other diseases," Yang Linhua said.
Entering the twenty-first century, the state vigorously rectified the blood products industry, some local plasma stations closed, raw plasma supply is insufficient, coupled with the small number of hemophilia patients, insufficient research and development power of enterprises and other issues, since 2006, the mainland has occurred a number of hemophilia patients drug shortage, 2007 and 2013 have outbreaks of national coagulation factor shortage, causing a sensation.
In 2021, as a deputy to the National People's Congress, Yang Linhua called for an amendment to the Blood Donation Law, including increasing the age of blood donors from the current 55 years old to 60 years old; shortening the interval between two blood donations, "Now the Blood Donation Law stipulates that the two blood donations must be more than half a year apart, but because the average lifespan of red blood cells is 120 days, some blood that can be used is invisibly rejected." If you shorten the interval between two blood donations to 4 months, you will increase the number of blood donors."
Yang Linhua attended the NPC session.
Yang Linhua hopes to expand the source of blood donation, "Any person can get sick, whether it is internal medicine, surgery, or trauma, in fact, it involves the use of blood." 」
In addition to blood-borne coagulation factors, there must also be mentioned genetic recombinant coagulation factors. It does not require the use of plasma raw materials, avoids the contamination of plasma proteins and viruses, and is safer.
Therefore, in order not to repeat the infection tragedy of that year, doctors try to encourage patients to use recombinant coagulation factors.
In 2007, taking into account the actual situation of the shortage of coagulation factors in China, the State Food and Drug Administration at that time quickly approved the import of genetic recombinant coagulation factor VIII.
"Overall, the problem of drug supply in our hospital has improved a lot compared to the past, but there will also be some emergencies. For example, a hospital has 300 hemoglottal coagulation factors in a month, but a patient may use 200 because of heavy bleeding."
Yang Linhua told us that the medication of patients with hemophilia varies greatly from individual to individual, and the drug is administered according to the patient's kilogram weight. "If it is only preventive treatment, the basic amount can be guaranteed, but once the bleeding, such as gastrointestinal bleeding or cerebral hemorrhage, or the need for surgery, the amount used is very large."
What if I don't have medicine?
Yang Linhua replied that it is possible to urgently transfer goods from pharmacies or pharmaceutical companies, and there are also mutual assistance between patients, such as brothers who are all sick, and borrow from each other, and cannot find other patients to solve temporary urgent needs.
Medication "Last Mile"
In recent years, some hemophilia patients are still stuck in the "last mile" node from the drug.
Source: State Drug Administration
Because patients with hemophilia require lifelong treatment, patients need to pay different fees because of different individual needs. Public information has calculated that the annual treatment cost of hemophilia patients is about 288,000-312,000 yuan, which is still a large expense for many patients.
From 2004 to 2017, the government introduced various policies to include products for the treatment of hemophilia in medical insurance, especially preventive treatment of children.
But some patients will still get stuck.
In February 2020, due to the closed management of the new crown epidemic in Wuhan, some hemophilia patients could not go to the hospital to buy drugs, and were told that they could only go to the pharmacy to buy drugs - according to the relevant policies of the Wuhan Medical Insurance Clinic, this means that their reimbursement in the hospital can be settled immediately, but they can only pay in advance when purchasing drugs in pharmacies. According to the dosage of previous patients, some people may need to pay about 6,000 yuan per month, and patients with poor economic conditions face the risk of drug discontinuation.
Some patients called to inquire and were told that they needed to report to the district medical insurance bureau before purchasing drugs, and after the epidemic ended, patients needed to take the invoice to the medical insurance bureau for reimbursement.
In addition, some places have exposed the shortage of recombinant human coagulation factor VIII in 2021. According to the response, because the drug is a prescription drug, it needs to be used by the doctor to the patient, and the hospital has a procurement demand, and the manufacturer can supply it. Some hospitals have not purchased this expensive drug considering the proportion of drugs, and patients can only go to other places to find drugs.
With medicine, it is only the last kilometer away to use medicine.
At present, in the hospital where Yang Linhua is located, medical insurance can solve 70% to 75% of the medical expenses of patients, but even the remaining 25% to 30% is also a burden for some patients. "Because the patient is a lifelong medication, sometimes bleeding, it takes a lot at a time, and the personal payment is too high to pay."
"We all say that Medicare is widely covered, not special. But rare patients are also human and should also enjoy medical insurance. Therefore, we have been calling for medical insurance to not follow the path of common diseases and introduce a separate medical insurance system for rare diseases."
"At the same time, we can't let medicare solve the full burden of patients, but we need to establish a multi-level medical security system."
At this year's two sessions, Yang Linhua mainly suggested exploring the establishment of a provincial rare disease drug guarantee mechanism.
Among them, it mobilizes enterprises or various economic entities to make targeted donations and establishes a provincial rare disease charity special medical assistance fund to solve the problem of payment for high-value drugs for rare diseases outside the medical insurance drug list.
At the same time, she stressed that it is necessary for the government to guide and strengthen supervision, and departments at all levels to jointly formulate disease lists and drug lists, and at the same time establish a dynamic adjustment mechanism for the catalog.
"Clarify the lead department, formulate the work system and operation process, clarify where the money comes from, where it goes, whether it enters the payment account of the patient's medical treatment, and other operation methods and details."
Author: Su Weichu
Producer: Li Chen
Source: Courtesy of respondents
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