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Chapter 114 The Rescued Rare Disease Patient

Kunming, Sanqing Headquarters.

Wei Kang looked at the report in his hand with a look of surprise on his face.

The report listed the results of the Orphan Drug Generic Project of the generic drug department led by Gu Xian in the past few months.

A long list of completed and pending drug listings.

Wei Kang looked at the completed list of generic drugs for rare diseases, and the corners of his mouth twitched.

Nine generics!

Gu Xian, are you living in the laboratory 24 hours a day, using all your might?

He looked at Gu Xian who was sitting across from him, nodded slowly, and praised: "Very good, I did not misread you. Let's arrange the listing as soon as possible."

"However, you should also find time to rest for a few days, so as not to overwhelm your body." Looking at Gu Xian's two big dark circles, he couldn't help persuading.

Gu Xian nodded, took the signed report, and left excitedly.

Soon, Sanqing issued an announcement on its official website announcing the listing of the following nine generic drugs for rare diseases.

Nitisinone (capsule): Congenital Tyrosinemia

Gratiere (injection): patients with relapsing multiple sclerosis

Fabuzan (algalsidase beta for injection): Fabry disease

pyridostigmine bromide (tablet): myasthenia gravis

Treprostinil (injection): pulmonary hypertension

Bosentan (tablet): pulmonary hypertension

Icibant (injection): hereditary angioedema

Deerasirox (dispersible tablet): iron overload in thalassemia

Sodium Dimercaptosulfonate (Injection): Hepatitis

Except for patients who frequently refresh the official website of Sanqing, not many people know this information.

However, when those patients with rare diseases saw the listing information of their specific medicines, their eyes lit up and their eyes filled with tears.

*****

Ning City, People's Hospital.

32-year-old Li Lei is an ordinary Fabry patient. He came to the hospital again today for a daily follow-up visit, planning to prescribe some painkillers and other medicines.

He began to suffer from the disease since he was a child. Every time his fingertips and legs hurt as if they were broken. Later, he started to have a headache when he was in high school. During his stay at school, he beat himself with a wooden stick every day to relieve the pain. The pain was unbearable So I asked for leave and went to the dormitory, crying alone.

The pain didn’t get any better after I went to university, and the pain continued. The whole body was in rotation, and the pain changed in two weeks. Sometimes the pain was severe and sometimes the pain was mild. There was no regularity. The pain continued until I graduated from university and started working.

During this period of time, he went to all the hospitals in Haishi, but he couldn't find anything wrong.

Later, he began to feel dizzy and nauseated. One day, when he got up in the morning, his right eye suddenly became dark, and he could not see anything. He never recovered.

At that time, he was only 23 years old and had just graduated from college.

After graduation, he was supposed to usher in a bright future, but he was confronted with round after round of blows.

It was not until the age of 31 that he underwent a genetic test that he was diagnosed with Fabry's disease.

This disease is a rare hereditary disease, inherited from his mother. He was married at that time, but because of taking medicine for a long time, he had no children. After discovering this disease, it was even more impossible to have children, so he made peace with his wife not long after. separate.

The average life expectancy of a patient with Fabry's disease is 40 years old, but severe organ failure will occur at the age of 30, and he will die soon. His mother died of a sudden stroke. At that time, he did not know that it was due to Fabry's disease. Only now do I understand the truth.

"Li Lei," the follow-up doctor was already familiar with him. After calling out, he hesitated and said, "Your illness can't be delayed any longer. There is a special medicine that is currently on the market in China, which should be effective for your condition."

"I know, it's Fabuzan, a subsidiary of Sanofi, right? I was overjoyed when this drug was first launched, and I felt so lucky that there would be a cure after the diagnosis." Li Lei shook his head and sighed: " It's a pity that although there are medicines that I can use, I can't afford them."

There was a wry smile on his face, and he couldn't help complaining: "Fabzan's imported specific medicine is really expensive. The official 5mg bottle costs 9,000 yuan. Calculated according to the adult weight standard, the annual treatment cost is 2 million yuan. Medication for life. How the hell can I afford it."

He looked at the doctor, his eyes were moist: "Doctor, I want to live too, but there is only one disease in this world, and that is the disease of poverty. I can't cure this disease, so I don't deserve to live at all. Let me just drag it on until now." It’s the end of my life, and it’s a relief for me.”

The doctor shook his head and said with a smile: "Li Lei, it's not what you think. Fabuzan is indeed very expensive, but what I just said is a domestic generic drug, which is much cheaper and has just been on the market. It should be very helpful for your condition. Great relief."

When Li Lei heard it, a hint of surprise appeared in his despair-filled eyes, and he said in a trembling voice, "How much...how much?"

"Fabzan's domestically produced generic drug costs 300 yuan for a 5ml bottle, which is 30 times cheaper. Calculated, your annual treatment cost is only 60,000 yuan."

Li Lei trembled all over, with an expression of disbelief: "It's so cheap? Is it real or not?"

The doctor sighed: "Of course it is true. Why is it so cheap? Because it is a generic drug of Sanqing. I heard that it has launched a generic drug plan for rare diseases. The generic drug of Fabuzan is the first batch. Fortunately, there is Sanqing. Your life is finally saved."

"It is said that we are still talking about getting medical insurance. If you can get in later, it is estimated that you can save half of it."

"Ah..." It is said that men do not shed tears lightly, but when Li Lei heard this, two lines of hot tears rolled down his face in an instant, and he suddenly burst out with a strong desire to survive: "Doctor, does medicine have this medicine now? You can open it for me quickly."

The doctor smiled and nodded: "I just arrived yesterday. There are not many patients with this disease in the country. At present, there are only more than 300 people diagnosed in China. After I heard the news, I contacted Sanqing specially to order this batch of goods for you. Today Let me test it out for you, let’s see what happens next.”

Li Lei wiped away the tears on his face with a tissue on the table, and kept nodding his head: "Okay, I'll follow your orders, doctor, thank you."

*****

Beijing, Harmony Hospital.

Fang Chao hurried into the outpatient room with the child's urine test report, followed by his wife holding the one-year-old child.

"Doctor, what's the matter with the child? Why is the urine black?" Fang Chao looked at the doctor anxiously.

After reading the report, the doctor was silent for a long time before saying, "It may be Garrod's syndrome, a rare autosomal recessive metabolic defect disease. The typical symptom is dark urine, commonly known as alkuria. This The disease is related to the metabolic disorder of tyrosine and phenylalanine, and the homogentisic acid in urine is toxic to the human body and can cause damage to bones, liver, kidneys and other organs.”

"The incidence of this disease is one in a million, and there are very few patients in China. At present, there is only one specific drug, which is nitisinone, which is used to treat congenital tyrosinemia type Ⅰ."

The doctor suddenly smiled and said: "Your child is very lucky. Originally, Nitisinong was only available in foreign countries, and it was not on the market in China. I couldn't prescribe it for you, but Sanqing's first batch of orphan drug generic plans happened to have This medicine."

"This is the first drug for the treatment of type I tyrosinemia in China. It has just been launched recently. I will apply to the hospital immediately. The drug should be prescribed in a few days."

Fang Chao was both surprised and delighted: "Great, then I will come back for a follow-up visit on the weekend. How long will I have to take this medicine? Do I have to keep taking it?"

"Your child is only one year old, right? Generally speaking, after taking the medicine for 6 months, the blood succinylacetone level will drop to normal, and the blood tyrosine level will drop, and the condition will improve, but the dose will still need to be reduced later. take.

"Also, in addition to this medicine, you must strictly control the intake of tyrosine and low phenylalanine in your diet. I will make a list for you. You should pay attention to these foods."

"Yeah." Fang Chao took out his mobile phone and kept recording, with an extremely serious expression on his face.

*****

Haishi, Huashan Hospital.

The 60-year-old Mr. Zhou, supported by his son, walked out of the hospital with a look of disappointment on his face, with his head down, and he couldn't help sighing.

His family lived in Su City, but he came to the hospital in Hai City just to find a medicine.

Mr. Zhou has been suffering from myasthenia gravis for more than a year, and has been taking a drug tablet called "pyridostigmine bromide".

This drug is not expensive, about 30 yuan per bottle, and it is the only drug for myasthenia gravis.

Recently, however, the drug has suddenly disappeared from the market.

Mr. Zhou searched all the major hospitals and pharmacies in Su City, and even came to Huashan Hospital in Hai City to inquire, but he couldn't buy this medicine.

Afterwards, he continued to search for more than a month, but he did not find any trace of this cheap life-saving medicine.

He has been off the drug for half a month, and he began to show signs of serious illness. His eyelids are drooping, he can’t see people at all, his legs and feet are weak, and he can’t walk. If he continues without the drug, it will become difficult to breathe slowly. Just wait Death came.

He is not the only one who is looking for this medicine, many patients in patient groups all over the country are looking for this medicine.

They were all confused, how could such an important life-saving medicine suddenly disappear?

Mr. Zhou once asked the doctor, and the doctor said that since three months ago, the hospital could not get the medicine.

Myasthenia gravis is an autoimmune disease. The main symptom is that some or all skeletal muscles are prone to fatigue, unable to open the eyelids, unable to swallow food, and it becomes difficult to breathe slowly. Using this medicine can relieve the symptoms Significantly relieved or relieved.

Mr. Zhou's family was in a panic. Later, his son found out on the website of the Food and Drug Administration that there were three manufacturers of pyridostigmine. However, currently only one pharmaceutical company in Haishi is actually producing it.

His son contacted the person in charge of the sales of the pharmaceutical factory. According to the other party, the company also plans to stop production, because this kind of medicine has no profit, and there is still no profit after adding the price. Even the production cost is not enough, so they want to lower the price. The medicine was thrown away and I didn't want to do it.

The other party also said that due to factors such as rising raw material and labor costs over the past decade, the production of cheap medicines has become unprofitable. Therefore, many pharmaceutical companies have given up the production and supply of cheap drugs. In fact, there are many pharmaceutical companies across the country that have approval for the production of this drug, but all of them have stopped production one by one due to cost issues.

After Mr. Zhou found out, he was completely desperate. He felt that he had to wait for death.

The whole world is gray to him, and now he rests on the bed every day, and doesn't want to do anything except read the speeches of everyone in the patient group.

Suddenly, Mr. Zhou's eyes froze, and he saw a message from the group leader, and then the entire patient group erupted.

"I found out that pyridostigmine bromide produced by Sanqing Pharmaceutical is already on the market. I just bought this drug at a pharmacy in Kunming."

Then I took a few photos, all of which were pictures of medicines, and the words "Sanqing" on the packaging box were impressive.

Before he had time to read the news from other patients, Mr. Zhou sat up instantly and shouted to his son: "Master, hurry up! There is medicine, there is medicine!"

Mr. Zhou's son hurried into the bedroom, took his father's mobile phone, looked at the photos, and jumped up in surprise.

"Dad, don't worry, I'll go to the pharmacy to buy medicine right away."

Like an arrow leaving the string, he turned around and rushed out of the door, disappearing.

Mr. Zhou sat on the bed restlessly, waiting anxiously for fear that something might go wrong.

Half an hour later, the son came back with a bag of medicine with a happy face.

As soon as he entered the door, he yelled: "Dad, I bought it, I bought it. It's still the same price. The pharmacy owner said that the supply is sufficient, and there is no fear that it will run out of stock in a short time."

Mr. Zhou's hanging heart finally let go, and he asked in a trembling voice: "Are you sure? Should we stock up on some medicine? What if the production is stopped again?"

The son smiled and waved his hands and said, "That's impossible. It's made by Sanqing, and they don't need money. If you're really worried, I'll call the manufacturer and make sure."

Mr. Zhou nodded and watched his son call Sanqing's customer service number.

However, it has always been a blind tone, and it is impossible to communicate with each other.

The son smiled bitterly and said, "I'll check the Internet, and you always check the group to see if there's any new news."

Mr. Zhou took the medicine tremblingly, opened the WeChat patient group, and only then did he know what happened.

It turns out that Sanqing has launched a rare disease generic drug plan, and pyridostigmine is in it. According to the group leader, Sanqing will continue to produce this drug, even if it loses money, so everyone can rest assured.

Mr. Zhou was finally relieved, with a long-lost smile on his face.

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