Rebirth of the God-level Xueba

Chapter 757 Rare Disease

Yang Rui is not a person who speaks freely. In fact, because he is young and a student, he deliberately pursues rigorousness in the laboratory. △↗, x.

Therefore, when Yang Rui said that he needed to get deferiprone out as soon as possible, Xu Zhengping felt bad all over, and hurriedly said: "Director Yang, let's go back and find out the situation first, I haven't seen deferiprone or anything like that. "

"What Director Xu said is true." Wang Sisheng also realized with a shock that this is a matter of resource consumption, and it is a huge resource.

Yang Rui thought for a while, and said: "If you all disagree, I will leave it to Huarui Laboratory to do it. Huarui is considered a Hong Kong company. If there is thalassemia, Hong Kong also has it."

With the last question, Yang Rui looked at Yong Xiaodong.

Yong Xiaodong pursed his lips, his thin body trembled twice, and said, "Hong Kong does have thalassemia, but the level of pharmaceutical companies in Hong Kong..."

"As long as there is a laboratory, right."

"That's right... I hope I can wait." Yong Xiaodong is a clinician, not a researcher. He doesn't know the complicated process of pharmaceuticals, but he knows the time it takes for a new drug to be launched.

Most medicines can be made in 10 years, which is considered a blessing.

For those simple compounds, when you are given the synthetic formula and key points to do it, the experimental dog can make it after a few tries, but if there is no formula and key point explanation, hehe, the masters of synthetic chemistry will die The brain cells of the brain can be strangled to death 300 bragging undergraduates at a time.

Unlike normal pharmaceutical company researchers with receding hairlines, Yang Rui's mind is full of synthetic formulas and key points of various compounds.

Drug patents in the 1980s and 1990s have basically expired after 2000 or 2010, and domestic generic drug companies have long since copied how many versions. For example, the most popular cephalosporin antibiotics nowadays, whether it is cefaclor or cefaclor, its synthesis method can be found not only in papers and various monographs, but also everywhere in the Internet. When Yang Rui was studying, he could just scan it. meet.

Of course, there are too many companies imitating them in China, so that the price of raw materials of tens of thousands of yuan per kilogram has plummeted to thousands or even hundreds of yuan after 2000...

The synthesis of deferiprone is not a secret in Yang Rui's eyes, but, after all, he is engaged in biology, and he is not very proficient in chemical synthesis. It may take a while to practice.

"Let me ask Huarui Hong Kong. Deferiprone is a kind of medicine anyway. If it is made, it should be profitable. They should think about it." Yang Rui didn't say anything dead, so as not to leak Too much information to come.

After hearing this, Yong Xiaodong believed in Yang Rui even more, and quickly said: "Yes, yes, if ironone is produced, it will definitely make money, and there are still many patients who need it. We Chinese are relatively poor, but Southeast Asian countries still need it. There are rich people, right?"

"Look, if we can make it, even if we don't earn much, it will be a lot of money in the long run." Yang Rui looked at Xu Zhengping and said, "If we do it, the future scientific research funding will be guaranteed. "

"Forget it, even if the results in our lab are profitable, they have to be handed over to the school. Otherwise, the school's appropriation will not be considered in the future." Xu Zhengping was afraid that Yang Rui would ask the ion channel laboratory to do it. Then all his time and energy have to be invested in it.

It's fine if it's basic biological work. Xu Zhengping doesn't have to live by pointing at interacting proteins. Mutual eggs or mutual ostrich eggs can also do research for a lifetime. But the pharmaceutical field is too far away from him.

Xu Zhengping's age and qualifications did not allow him to switch to other fields.

Yang Rui could guess Xu Zhengping's answer, although he was slightly disappointed,

But it is also reasonable, just nodded and said: "Since Director Xu disagrees, then so be it."

Yong Xiaodong didn't care who did it. Seeing that the two had finished talking, he said again: "Director Yang, I have some information, you can show it to Hong Kong people."

As he said that, Yong Xiaodong took Yang Rui and others to the office, searched for documents from various filing cabinets, and piled up the things he found in the center of the room.

After a while, a chair-high hill was piled up in the room.

Yang Rui picked up a few files and read them.

Most of them are clinical records made by Yong Xiaodong. In addition, there seem to be some introductions and related information about thalassemia.

And all of these, except for a few mimeographs and newspaper clippings, are mostly hand-copied.

The handwriting of all the materials is the same, but if you look carefully, you can still see slight differences. After analyzing the paper, you can recognize that these records may have been running for a long time.

"I have been doing research on thalassemia for more than ten years. I have been doing it since the appearance of deferoxamine, the first batch in China." Yong Xiaodong noticed Yang Rui's expression, forced a smile, and said: "To be honest , I have studied thalassemia for so many years, but there is no result. If there is no more hope, I will sort out the information and publish a few more papers, and then I will stop."

Yang Rui froze for a moment, but didn't speak.

"It's useful or not, that's all." Yong Xiaodong got dust all over his body, went outside the door to pat it, and then came back, saying: "When the feroxamine first came out, in the 1960s, we got a document. At that time My colleagues in the Department of Hematology were all overwhelmed, saying that thalassemia can be cured. I was appointed by my superiors and joined the thalassemia working group to be responsible for research and thoroughly understand the data. The working group also contacted the pharmaceutical factory and produced generic drugs. Later, after deferoxamine was used clinically, its efficacy was indeed outstanding. In the past, patients with the intermediate type could only live to be in their twenties and thirties. After treatment, their life expectancy was extended by ten or even twenty years. The quality of life has improved even more, and some of them can still live normally now. Patients with severe thalassemia can also be considered to have outstanding curative effects. However, from more than ten years ago to the present, our work has produced limited changes... Without new drugs, we are busy. I'm exhausted, at most I can solve some small problems, and I can't even solve more complications..."

Yang Rui nodded slightly, he could understand Yong Xiaodong's sense of powerlessness.

Like most genetic diseases, the treatment of thalassemia is actually very simple, and it is difficult for clinicians to come up with a better treatment plan.

Blood transfusion to continue life, and iron removal to alleviate complications. This plan was established in the 1960s, and it will not change much in 2016. It has changed from spleen removal to stem cell transplantation, but the basic idea remains the same.

Without new drugs, no matter how good a clinician is, they cannot solve this disease better.

If there is a perfect iron-removing agent that does not cause side effects, or does not cause serious side effects, and is not expensive, then thalassemia can actually be considered under control.

Because the causes of premature death from thalassemia and bone deformities are the excessive accumulation of iron ions in the body.

However, the world is always so imperfect.

Only in an imperfect world can people's existence have value.

Yang Rui supported Yong Xiaodong and said, "I will take a good look at your documents. When the time comes, I will copy them out and hand them over to Huarui, and then send you the original documents."

"Would it be too expensive to copy..." Yong Xiaodong is also willing to keep the original materials. For him, the records of more than ten years are not only important memories, but also important resources.

Yang Rui smiled and said, "Hong Kong people are rich."

"Don't scare people." Yong Xiaodong said very concerned, and then said: "By the way, do you know the orphan drug system in the United States?"

Yang Rui thought for a while before he came to his senses: "Are you talking about Aofen?"

Orphan is a literal translation of Orphan, and in the environment that Yang Rui is familiar with, orphan drugs have been generally called rare drugs.

Yong Xiaodong nodded repeatedly and said, "Yes, that's right, English is Orphen. Thalassemia should be a rare disease. Therefore, thalassemia drugs, such as deferiprone, should be a rare disease drug, which is in line with a bill in the United States. , called... "Orphan Drugs Act", it is said that it is a preferential policy... I will look it up again, and I will tell you when the time comes, this can be regarded as an advantage."

"You are talking about ODA approved by the United States last year, which is indeed a very big preferential policy." Yang Rui knew more about this, and said casually: "If the application for rare disease drugs is approved, there should be seven years of market exclusivity. Tax relief for development costs and application fees..."

"That's right, this is it, is it useful?"

"It's useful to be useful."

"But it's of no use?"

"I'm afraid this won't be the deciding factor." Yang Rui smiled wryly. If it is after 2000, the development of drugs for rare diseases is indeed profitable, and some drugs can even become "blockbusters" with annual sales exceeding US$1 billion. For example, ese, which treats Hunter syndrome, has only 2,000 patients in the world, but the annual treatment cost for each patient is 375,000 US dollars, which means that ese's annual sales can easily exceed 100 million. Gleevec, which treats chronic myelogenous leukemia, has a certain reputation in China. The monthly fee is 20,000 RMB, and if the charity plan is included, the annual fee is as low as 70,000 RMB.

However, Americans can't actually afford the $370,000, or $10,000 a year fixed medical expenses, they rely on the help of insurance companies, state aid and rescue systems, and non-profit patient aid organizations.

And in the 1980s, at least now in 1984, Americans couldn't get these things. Their insurance companies will not pay high-priced drugs to patients with rare diseases, and patients with rare diseases cannot afford high-priced drugs...

Therefore, at the beginning of the establishment of the "Rare Diseases Act", there were not many drugs for rare diseases released. The era that really benefited patients with rare diseases was the era after insurance companies solved the problem of high-priced drugs.

Deferiprone is even more special because it does not completely replace deferoxamine, which means that deferiprone is a rare drug with an alternative product.

Rare diseases mean fewer patients, fewer patients mean lower drug sales, and alternatives mean lower drug prices.

The sales volume is low and the price is low, so how can it be possible to make money.

What's more, Yang Rui's original intention of developing deferiprone was to reduce the burden on patients with thalassemia and solve the problem that deferoxamine was too expensive.

"Hong Kong Huarui Company doesn't necessarily have to focus on making money." Yang Rui said slowly: "I'll contact the laboratory of Huarui Company first to see if it can make norferiprone. As for the price, it should be considered in advance Be proficient in clinical trials, as well as the process of drug sales. Rare drugs do have advantages in this regard."

"Saving a life is better than building a seven-level pagoda..." Yong Xiaodong held Yang Rui's hand tightly. He has been treating thalassemia patients for 20 years, and he has seen thalassemia patients for 20 years. The shock in his heart is beyond words And metaphor.

Yang Rui shook Yong Xiaodong's hand twice, took his leave and left without giving him any promises. This is not a problem that can be solved by promises.

...

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