4-year-old Liang Liang was bleeding, blood trickled out, my mother took out a roll of paper towels to block it, soaked it, plugged a roll, and soon it was wet again, and the bleeding still didn't stop.
On the night of Chinese New Year's Eve 2010, Liang Liang fell, his head on the radiator, and swollen a bag. At first, the family did not care, but the bag did not disperse for a few days, but became more and more popular. After 2 months, the large bag broke open a small opening and began to bleed.
In the panic, the family hugged Liang Liang to Jinzhou Children's Hospital. The doctor said it was a hemangioma and had to hurry up for surgery.
The mother did not believe it, thinking that it was caused by the bump, and the family transferred Liang Liang to the city hospital. The doctor of the city hospital looked at it, shook his head, and let it be sent to the provincial city quickly. After arriving in Shenyang, Liang Liang's blood continued to flow, and finally the doctor of Shenyang Children's Hospital was diagnosed: Liang Liang suffered from hemophilia.
Drugs for the treatment of hemophilia are commonly known as "eight factors", and once the injection is given, the blood stops at eight o'clock the next morning. Mom said that the doctor thought it was a miracle that Liang Liang survived, and that he shed at least as much blood as two mineral water bottles one night.
01 Survive on drugs
"What does blood taste like?" Salty, astringent, very fishy, burning throat, very disgusting... What does it feel like to be painful? Painful, very painful, painful to eat all the morphine, durandine, can not stop the pain. In the year of Liang Liang's illness, a patient wrote this text.
Pain and bleeding are common experiences for hemophilic patients. Because the body lacks coagulation factors, if there is no external supplement to stop the bleeding, blood may flow out of the epidermis, a certain injured joint, and even internal organs through broken blood vessels, causing more pain than childbirth. Data show that more than 75% of hemophilia patients have a physical disability.
There are more than 140,000 people in China suffering from hemophilia, the patients are usually male, the current treatment methods, they can only supplement the coagulation factor to be the same as normal people, otherwise it is easy to bleed "glass people".
Bright must avoid any bumps. When she was in elementary school, her mother applied for a school companionship and became a special table with Liang Liang, and for six years, she took care of her son and cleaned the toilets of the whole school in her spare time.
Relying on the replacement treatment of eight factors, Liang Liang survived, and he began to help other patients. Liang Liang is now 16 years old, he loves to draw, since the age of 7, he has painted 70 or 80 paintings for the blood friend's home every year, and all the proceeds of the public welfare auction are donated to small patients.
Bright paintings
Exogenous coagulation factors are mainly extracted from human plasma, which is not only low in production, but also expensive. China Hemophilia Home is committed to helping hemophiliacs fight for drugs. In May 2000, Hemophile Patient Guan Tao founded the Hemophile Home with 9,000 yuan raised, and since then has rescued countless distressed and critically ill hemophilic patients, including Liang Liang.
More than twenty years have passed, and the development of Chinese hemophilia drugs, Guan Tao has always been in the eyes. Now in addition to the domestic blood-borne human coagulation factor VIII. and the imported recombinant human coagulation factor VIII., in recent years, there are innovative drugs that hope to cure hemophilia, such as gene therapy and bispecific antibody drug emesizumab, which can enable patients to produce clotting factors on their own and give hemophilic patients the hope of restoring normal life.
However, for the vast majority of patients with hemophilia, coagulation factors are still the most commonly used life-saving drugs. Guan Tao introduced to the Health Bureau that the efficacy of foreign innovative coagulation factor drugs is much better than that of domestic products, and the half-life has become longer, which can be administered once a week or even once a month.
However, such cutting-edge drugs must be used by patients, and there are two levels to be approved for marketing and payment. Liang Liang knew that there were more advanced good medicines, but could not buy them in China.
02 They need more good medicine
"Have you ever seen red tears?"
Chen Haochang, vice president and general manager of Jetbelling's greater China commercial operations, often asks friends this. It is one of the world's leading manufacturers of hemophilia drugs and has access to many hemophilic patients.
Patients with severe symptoms will have eye bleeding, and when they cry, red tears will fall down, which gives Chen Haochang a great shock. He understands how desperate hemophilia patients can be without very effective medicines.
Chinese hemophilia drugs have bid farewell to the stage of shortage. The Guangdong Inter-Provincial Alliance, which has just begun, involves a variety of eight-factor varieties, and the Health Bureau noted that only one foreign company product is Green Cross, and the market demand accounts for less than 8%.
What patients with hemophilia need is a more effective coagulation factor. At a hemophilia patient exchange meeting one year, Chen Haochang asked Guan Tao, president of the Blood Friends Home, at lunch: "As a pharmaceutical company, what is the greatest support we can give them?" ”
"Medicine", Guan Tao directly threw out these two words: "Do you understand what I mean?" Bring the most advanced and cutting-edge and effective treatment programs from abroad to China. ”
Jetbelling is a global pioneer in the field of biotherapy, bringing products to China in the early 1980s and becoming the main supplier of human blood albumin in China.
In terms of hemophilia drugs, Jetbelling has a number of innovative drugs, and has a highly innovative and diverse product portfolio in the field of hemophilia type B and a portfolio of early and late product candidates with continuous innovation in the field of hemophilia treatment.
In June 2020, Jetbelling spent $450 million to introduce the global development and commercialization rights of EtranaDez, the gene therapy for hemophilia B, expanding Jetbelling's ability to continue to develop in the field of hemophilia B gene therapy.
But getting good medicines into the hands of Chinese patients is not an easy task. According to the requirements, China's approval of drugs to carry out clinical research and declaration work, the number of hemophilia patients is small, clinical trial organization is very difficult, and the cost is high, many patients who urgently need imported drugs can only hope for drugs.
After the publication of the First Catalogue of Rare Diseases in 2018, the situation changed radically. The Food and Drug Administration has accelerated the approval of rare disease drugs, including hemophilia, and compressed the approval time for rare disease drugs from 130 working days to less than 70 working days. In addition, the State Food and Drug Administration has also released a list of 3 batches of urgently needed new drugs in clinical practice, and accelerated the approval and listing of urgent new drugs in clinical needs.
At the same time, in Places such as Hainan and the Guangdong-Hong Kong-Macao Greater Bay Area, the state has formulated relevant approval and access procedures for drugs that are urgently needed clinically. Jetbelling has the opportunity to introduce drugs to China faster.
In addition to coagulation factor drugs for bleeding, preventive treatment and humanistic care are also needed by patients with hemophilia. Guan Tao said: "Some places with relatively good economic conditions have begun to carry out preventive treatment, and the quality of life of patients has been improved, and it can be pain-free. In the past, treatment of patients with hemophilia was only to sustain life.
Professor Yang Linhua, director of the Institute of Hematology of Shanxi University, is full of hope: "I believe that the prevention and treatment of hemophilia will be better and better, and there is still a lot of space for us to develop." We also want more new drugs and more tests for patients. ”
What Guan Tao is doing now is to stop letting the next generation of small patients repeat the pain. This requires the joint efforts of doctors, pharmaceutical companies and society.
Liang Liang was in his first year of high school, and he had a dream to be admitted to the China Academy of Art and help more blood mate patients.
(In order to protect the privacy of the respondents, Liang Liang in the article is a pseudonym)
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