Yang Rui is not a man of letters, in fact, because he is young and a student, he deliberately pursues rigor in the laboratory, and no matter how exaggerated the words he says, there are traces to follow, and he tries his best to achieve them. Pen "Fun" Pavilion www.biquge.info

Therefore, when Yang Rui said that he wanted to do ferrite as soon as possible, Xu Zhengping was not good, and said busily: "Director Yang, let's go back first to understand the situation, I haven't seen it before." ”

"What Director Xu said is true. Wang Sisheng also reacted with a stirring spirit, this is a resource-consuming thing, and it is a huge resource.

Yang Rui thought for a while and said: "If you don't agree, I'll leave it to Sinovel Laboratories to do, Sinovel is a Hong Kong company, and if there is thalassemia, Hong Kong also has it." ”

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

Yong Xiaodong pursed his lips, his somewhat emaciated figure trembled twice, and said: "Hong Kong does have thalassemia, but the level of pharmaceutical companies in Hong Kong is ......."

"As long as there's a lab, right? ”

"Yes...... Hopefully wait. Yong Xiaodong is a clinician, not a researcher, and he doesn't know the complex process of pharmaceuticals, but he knows the time it takes to launch a new drug.

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

Those simple compounds, when you are given a synthetic formula and key points to do, the experimental dog can make it after several attempts, but if there is no formula and key point explanation, hehe, the dead brain cells of the masters of synthetic chemistry can strangle 300 bragging undergraduates at a time.

Unlike a normal researcher with a receding hairline in a pharmaceutical company, Yang Rui's mind is full of synthetic formulas and key points of various compounds.

The drug patents of the 80s and 90s basically expired after 2000 or 2010, and domestic generic drug companies have long copied how many versions. For example, the most popular cephalosporin antibiotics today, whether it is cefaclor or cefixia, its synthesis method can not only be found in papers and various monographs, but also everywhere on the Internet.

Of course, there are too many companies copying them in China, so that the price of tens of thousands of yuan per kilogram of raw materials has plummeted to 1,000 yuan or even hundreds of yuan after 2000......

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

"Let me ask Hong Kong Huarui. Deferiprone is a drug no matter how it is said, and if it is made, it should be profitable, and they should think about it. Yang Rui didn't say it to death, so as not to leak too much information.

When Yong Xiaodong heard this, he believed Yang Rui even more, and hurriedly said: "Yes, yes, if you produce ferrite, you will definitely be able to make money, there are still many patients who need it, we Chinese are relatively poor, but there are still rich people in Southeast Asian countries, right." ”

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

"Forget it, even if the results in our laboratory have income, they must be handed over to the school, otherwise, the school's appropriation, don't think about it in the future. Xu Zhengping was afraid that Yang Rui would do it in the ion channel laboratory, so his time and energy would have to be invested in it.

If it's the basic work of biology, forget it, Xu Zhengping doesn't have to point to interacting proteins to live, and mutual eggs or mutual ostrich eggs can also do 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 could guess Xu Zhengping's answer, although he was a little disappointed, but it was also reasonable, so he just nodded and said, "Since Director Xu doesn't agree, then so be it." ”

Yong Xiaodong didn't care who did it, and when he saw that the two of them had finished speaking, he said again: "Yang ...... Director, I have some information that you can show to the people of Hong Kong. ”

As he spoke, Yong Xiaodong took Yang Rui and the others to the office, began to rummage through the materials from various filing cabinets, and piled the things he found in the center of the room.

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

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

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

And all of them, with the exception of a few mimeographs and newspaper clippings, were mostly copied by hand.

The handwriting of all the materials is the same, but if you look closely, you can still see the slight differences, and if you analyze the paper, you can recognize that these records may have been going through for a long time.

"I have been doing thalassemia research for more than ten years, and I started to do it since the emergence of deferoxamine, which is the first batch in China. Yong Xiaodong noticed Yang Rui's expression, smiled reluctantly, and said: "To be honest, I have studied thalassemia for so many years, and there is no result. ”

Yang Rui was stunned for a moment and didn't speak.

"It's useful or not, that's all. Yong Xiaodong got a body of dust, patted it outside the door, came back, and said: "When feramine first came out, in the 60s, we got a document, and at that time, the colleagues in the Department of Hematology were boiling, and they all said that thalassemia was saved. I was assigned by the superiors, specially joined the thalassemia working group, responsible for the amount of research and thorough information, the working group also contacted the pharmaceutical factory, made a generic drug, later, deferoxamine in the clinical use, it is indeed outstanding efficacy, before the intermediate type of patients, are only able to live to twenty or thirty years old, after treatment, have also extended the life of ten or even twenty years, the quality of life has improved greater, some can now live normally, severe thalassemia patients are also regarded as outstanding efficacy, but from more than ten years ago to the present, our work, the changes are limited…… Without new drugs, even if we are busy and tired, at most we can solve some minor problems, and we can't even solve more complications if we want to......"

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

Like most genetic diseases, thalassemia is very simple to treat, and clinicians struggle to come up with a better treatment.

Blood transfusion to continue life, iron removal to alleviate complications, this program was established in the 60s, and it will not change much in 16 years, and the middle is nothing more than from removing the spleen to stem cell transplantation, but the basic idea is still the same.

Without new drugs, no matter how good a clinician is, he or she will not be able to solve this disease better.

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

Because the cause of early death of thalassemia and the cause of skeletal deformity are excessive accumulation of iron ions in the body.

However, the world is always so imperfect.

Only an imperfect world can make people's existence worthwhile.

Yang Rui held Yong Xiaodong and said, "I will take a good look at your information, and when the time comes, I will make a copy of it and hand it over to Huarui, and then send the original information back to you." ”

"If you make copies, won't it be too expensive......" Yong Xiaodong is also willing to leave the original materials, and the records of more than ten years in a row are not only important memories for him, but also an important resource.

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

"Sensei scared people. Yong Xiaodong said very concernedly, and 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 Offen?"

Offen is the literal translation of orphan, and in Yang Rui's familiar environment, orphan drugs have been widely called rare drugs.

Yong Xiaodong nodded again and again and said, "Yes, yes, yes, English is read Aoffen." Thalassemia should be a rare disease, therefore, thalassemia drugs, such as deferiprone should be a rare disease drug, in line with a bill in the United States, called the ...... "Rare Disease Drug Act", which is said to be a preferential policy...... I'll check it out again and tell you when the time comes, this can be considered a good point. ”

"You're talking about the ODA approved by the United States last year, which is indeed a big preferential policy. Yang Rui knew more about this, and said casually: "If the application for rare disease drugs is approved, there should be 7 years of market exclusivity, and tax deductions for development costs and application fees......

"That's right, that's it, does it work?"

"Useful is naturally somewhat useful. ”

"But it's useless?"

"I'm afraid that's not going to be the deciding factor. Yang Rui smiled bitterly. After 2000, the development of drugs for rare diseases would indeed be profitable, and some drugs could even become "blockbusters" with annual sales of more than $1 billion. For example, Elaprase, which treats Hunter's syndrome, has only 2,000 patients worldwide, but the annual cost of treatment per patient is $37. $50,000 means that Elaprase's annual sales easily exceed 100 million. Gleevec, which treats chronic myeloid leukemia, has a certain reputation in China, with a monthly cost of 20,000 yuan, and a minimum annual cost of 70,000 yuan if you add a charity program.

However, Americans can't afford to spend $370,000, or $10,000 a year on regular medical expenses, and they rely on insurance companies, the National Aid and Rescue System, and nonprofit patient assistance agencies.

And in the 80s, at least now in '84, Americans don't get these things either. Their insurance companies don't pay for high-priced drugs for rare disease patients, and rare disease patients can't afford to pay for high-priced drugs......

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

Deferiprone is special because it does not completely replace deferoxamine, which means that deferiprone is a rare drug for which there are alternatives.

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

If the sales volume is low and the price is low, how can it be profitable.

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

"Hong Kong Huarui Company does not have to be aimed at making money. Yang Rui said slowly: "I will first contact the laboratory of Sinovel to see if I can make deferiprone products, as for the price, it should be a skilled clinical trial in advance, as well as the process of drug sales." Rare drugs do have advantages in this regard. ”

"Saving a life is better than creating a seventh-level floating slaughter...... Yong Xiaodong held Yang Rui's hand tightly, he has been treating thalassemia for 20 years, and he has seen thalassemia patients for 20 years, and the impact in his heart is self-evident.

Yang Rui shook Yong Xiaodong's hand twice, said goodbye and left, and did not give him any promises, which was not a problem that could be solved by relying on promises.

……