Rebirth of the God-level Xueba

Chapter 757: Rare disease

Yang Rui is not a whisperer. In fact, because he is young and a student, he deliberately pursues rigor in the laboratory. No matter how exaggerated what he says, there are traces to follow, and he strives to achieve it. △↗, x.

Therefore, when Yang Rui said that he wanted to do desiccant as soon as possible, Xu Zhengping was not well, and he hurriedly said: "Director Yang, let's go back and find out the situation first. I have never seen desiccant or anything. "

"What Director Xu said is." Wang Sisheng also reacted in a shocking response. This is a matter of consuming resources, and it is a huge resource.

Yang Rui thought for a while, and said, "If you don't agree, I'll leave it to Huarui Lab. Huarui is a Hong Kong company. If there is thalassemia, Hong Kong also has it."

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

Yong Xiaodong pursed his mouth, his thin figure trembling twice, and said: "Hong Kong does have thalassemia, but the level of Hong Kong pharmaceutical companies..."

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

"Yes... 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 released.

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

For those simple compounds, when you give you synthetic formulas and key points to do, the experimental dog can make them several times, but if there are no formulas and key points, hehe, the majors in synthetic chemistry are dead. The brain cells of his group can strangle 300 bragging undergraduates at a time.

Unlike the researcher whose hairline is receding from a normal pharmaceutical company, Yang Rui is full of synthetic formulas and key points of various compounds.

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

Of course, there are too many domestic companies that imitate them, so that the price of raw materials of tens of thousands of yuan per kilogram after 2000 has drastically dropped to thousands or even hundreds of yuan...

The synthesis of deferiprone is not a secret in Yang Rui's eyes, but, after all, he is engaged in biology and is not very proficient in chemical synthesis. It is estimated that it will take some time to practice.

"I'll ask Hong Kong Huarui. Anyway, iron ketone is a kind of medicine. If it is made, it should be profitable. They should consider it." Yang Rui did not say anything to death, so as not to leak it. Too much information to come.

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

"Look, if you can do it, even if you don't make a lot of money, it will be a lot of money after long-term calculation." Yang Rui looked at Xu Zhengping and said: "If we do, the future scientific research funding will be guaranteed. "

"Forget it, even if the results in our laboratory yield gains, they will have to be turned over to the school. Otherwise, the school's funding will not be thought of in the future." Xu Zhengping was afraid that Yang Rui would want the ion channel laboratory to do it. Then all his time and energy have to be invested.

If it is the basic work of biology, then Xu Zhengping does not necessarily have to live with interacting proteins. Mutual eggs or mutual ostrich eggs can also be used for a lifetime of research. But the pharmaceutical field is too far away from him.

Xu Zhengping's age and qualifications do not allow him to move to other fields.

Yang Rui was able to guess Xu Zhengping's answer. Although he was a little disappointed, he was reasonable. He just nodded and said, "Since Director Xu disagrees, so be it."

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

Having said that, Yong Xiaodong took Yang Rui and the others to the office, started looking through the documents in various file cabinets, and piled up the found things in the center of the room.

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

Yang Rui picked up a few documents and looked at it.

Most of them are clinical records made by Yong Xiaodong, in addition to this, there seems to be some introduction and related information about thalassemia.

And all of these, except for a few mimeographs and newspaper clippings, most of them were copied by hand.

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

"I have done research on thalassemia for more than ten years. I have done it since the appearance of desferoxamine. It is the first batch in China." Yong Xiaodong noticed Yang Rui's expression and reluctantly smiled and said, "To be honest. , I have studied thalassemia for so many years, and there is no result. If there is no hope, I am going to sort out the information, publish a few more papers, and stop."

Yang Rui was stunned for a moment, and said nothing.

"Useful or useless, that's it." Yong Xiaodong got all the dust out, patted outside the door, and came back, saying: "When the iron amine just came out, in the 1960s, we got a copy of it. The colleagues in the Department of Hematology are all overwhelmed, saying that thalassaemia can be saved. I was assigned by my superiors to join the thalassaemia working group to be responsible for research and thorough data. The working group also contacted the pharmaceutical factory and produced generic drugs that year. Later, after deferoxamine was used clinically, it was indeed effective. In the past, patients of the intermediate type could only live to their 20s or 30s. After treatment, their lifespan was extended by ten or even twenty years. The quality of life has improved even more. Some people can live normally now. Patients with severe thalassaemia can be regarded as having outstanding curative effects. However, from a dozen years ago to the present, our work has produced limited changes... Without new drugs, we are even busy. It’s exhausting, at most it can solve some minor problems, even if you want to get rid of more complications..."

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

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

Blood transfusion continues life and iron remover slows down complications. This plan was established in the 1960s and will not have changed much in 2016. The spleen resection has changed to stem cell transplantation, but the basic idea remains the same.

Without new drugs, no matter how good a clinician is, there is no better solution to this disease.

If there is a perfect iron remover that does not cause side effects, or does not cause serious side effects, and the price is not expensive, then thalassemia can actually be controlled.

Because of the premature death of thalassemia, the cause of bone deformities is the excessive accumulation of iron ions in the body.

However, the world is always so imperfect.

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

Yang Rui supported Yong Xiaodong and said, "I will take a good look at your information. Then, I will copy it out and give it to Huarui, and then send you the original information."

"If it is copied, will it be too costly..." Yong Xiaodong is also willing to leave the original data. For him, the records for more than ten years are not only an important memory, but also an important resource.

Yang Rui smiled and said, "Hong Kong people have money."

"Don't scare people." Yong Xiaodong said very carefully, 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 woke up: "Do you mean Orpheum?"

Orphan is the literal translation of orphan, and in the environment that Yang Rui is familiar with, orphan medicine has been commonly referred to as a rare medicine.

Yong Xiaodong nodded and said: "Yes, yes, English is Orphen. Thalassemia should be a rare disease. Therefore, thalassemia drugs, such as deferiprone, should be rare disease drugs, in line with a US law. It's called... The Rare Disease Drugs Act, which is said to be a preferential policy... I will check it again and I will tell you at that time, this can be considered an advantage."

"You are talking about the oda approved by the United States last year. It is indeed a great preferential policy." Yang Rui knows more about this and casually said: "If the application for rare disease drugs is approved, there should be 7 years of market exclusivity. Tax relief for development costs and application fees..."

"Yes, this is it, is it useful?"

"Useful is naturally somewhat useful."

"But it's useless?"

"I'm afraid this won't be the deciding factor." Yang Rui smiled bitterly. 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 1 billion US dollars. For example, in the treatment of Hunter's syndrome ese, there are only two thousand patients in the world, but the annual treatment cost per patient is 375 thousand US dollars, which means that the annual sales of ese easily exceed 100 million. Gleevec, which treats chronic myelogenous leukemia, is well-known in China. The monthly cost is 20,000 RMB. With a charity plan, the minimum annual cost is 70,000 RMB.

However, Americans can't actually come up with $370,000 ~www.NovelMTL.com~ or $10,000 a year in fixed medical expenses. They rely on the help of insurance companies, the National Aid and Rescue System, and non-profit patient assistance agencies.

In the 1980s, or at least 84 years now, Americans did not get these things. Their insurance company will not pay high-priced drugs to patients with rare diseases, and patients with rare diseases cannot afford to pay high-priced drugs...

Therefore, when the "Rare Disease Act" was established, not many drugs for rare diseases were released. The era when rare disease patients really benefited is the era after insurance companies settled on high-priced drugs.

Deferiprone is even more special because it cannot completely replace deferoxamine, which means that deferiprone is a rare medicine with alternative products.

Rare diseases mean fewer patients, fewer patients mean lower sales of medicines, and alternative products mean lower prices of medicines.

With low sales and low prices, how can it make money?

What's more, Yang Rui's original intention to develop deferiprone was to reduce the burden of thalassaemia patients and solve the problem of excessively expensive deferipamine.

"Hong Kong Huarui Company does not necessarily have to make money to do it." Yang Rui said slowly: "I first contact Huarui Company's laboratory to see if I can make deferiprone. As for the price issue, it should be done in advance. Be proficient in clinical trials and the process of drug sales. Rare drugs do have advantages in this regard."

"Saving a life is better than building a seventh-level Buddha..." Yong Xiaodong held Yang Rui's hand tightly. He had treated thalassemia for 20 years, and he had also seen thalassaemia patients for 20 years. The shock in his heart is not to say. Yu.

Yang Rui shook Yong Xiaodong's hand twice and left without giving him any promise. This was originally not a problem that could be solved by promise.

...

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