More than a year ago, Yu Wenhao went to see Professor Zheng and consulted him. He said that he saw some patients in the group who could not wait for a fully matched match and had a haploidentical transplant. In Qianqian's case, it was possible
Can't you also do a semi-identical transplant using my bone marrow?
"Of course it can be done. We have done several cases. There is no problem with the operation itself."
Professor Zheng crossed his fingers on his chest, supporting each other. He looked at Yu and said hello, and continued:
"But in a situation like Qianqian's, my suggestion is not to do it, or to wait. Compared with a fully identical transplant, a haploidentical transplant has a greater risk of rejection, and the five-year cure rate is definitely lower. In addition,
In terms of recurrence rate, although there is no data to support it, from my experience, it is definitely higher.
"Haploidentical transplantation is also a helpless choice for those patients with acute onset, or whose physical condition cannot support them, or who are not eligible for targeted drugs and have to undergo transplantation immediately. We are friends, I
I can tell you straight, to put it bluntly, I treat a dead horse as a living horse doctor, let it go and take a gamble.
"If Qianqian has reached this level, I would definitely advise you to perform a haploidentical transplant immediately. But Qianqian has not reached this level yet. She still has time to wait for a fully matched match, or for medical and medical experts.
Development, yes, less than, let me tell you a good news."
As soon as Professor Zheng said the good news, Yu Wenhao's eyes lit up and he quickly walked over and asked, "What's the good news?"
"The new drug Nelic, jointly developed by Ascentage Pharma and Innovent Biologics, is commonly known as orebatinib tablets. It is an oral third-generation BCR-ABL inhibitor and the first approved third-generation BCR-ABL target in my country. Drugs for the treatment of drug-resistant CML have outstanding effects on BCR-ABL and a variety of BCR-ABL mutants including the T315I mutation, which can be said to have reached the world's leading level."
"What do you mean? Professor Zheng, please tell me something I can understand." Yu said hello a little anxiously.
"Well, to put it simply, it is very effective for patients with chronic myeloid arthritis. It can also cure patients without bone marrow transplantation." Professor Zheng said, "I have several cases on my hands. After taking this drug, they can now
Work and live normally."
"Can it be cured without transplantation? Is there really such a thing? That's great!" Yu Wenhao shouted.
"This drug is not simple. In March 2021, it was included as a breakthrough therapy drug by CDE. In July 2019, it obtained clinical trial approval from the US FDA and directly entered Phase Ib clinical research. The clinical trial has been in progress for four consecutive years and has been selected by the American Society of Hematology.
Oral presentation at the annual meeting, and was nominated for the Best Research at the 2019 ASH Annual Meeting.
"In May 2020, they successively obtained orphan drug qualifications and fast-track review qualifications granted by the US FDA, and also obtained European orphan drug qualifications..."
"Orphan drugs mean they are particularly effective for children, right?" Yu Wenhao interrupted Professor Zheng anxiously.
Professor Zheng couldn't help laughing and said:
"Don't take it literally. Orphan drugs are not drugs for orphans, but rare drugs, drugs used to prevent and treat rare diseases. Because there are few people suffering from rare diseases, little market demand, and high research and development costs, general pharmaceutical companies are very
There is little investment in research and development, so these drugs are vividly called 'orphan drugs'."
Yu Wenhao smiled. He immediately thought of it and asked, "Isn't that expensive?"
"Yes, orphan drugs are not cheap. The market is small and R&D investment is huge. They will definitely not be cheap. There is also a multi-million-dollar orphan drug abroad. But for some people, they still have to take it. For example, like Steve Jobs,
If any medicine is effective for him and can save his life, let alone millions, even tens of millions, 100 million US dollars per dose, he will take it, right?
"There is a saying that sounds cruel, but the truth is correct. The more medical science develops, how long a person lives is not decided by God or the King of Hell. In many cases, it is decided by your wallet. People with thicker wallets have better health than those with thicker wallets.
If you just say it, you will get more lives than others."
"I don't care, I don't care about Jobs, God or Hell, I just want to ask, how much does this medicine cost?" Yu Wenhao asked.
"Thirty-seven thousand one box." Professor Zheng said.
"So expensive?"
Yu Wenhao was surprised. He knew that even imatinib from Novartis of Switzerland cost more than 20,000 per box when it was first imported. It was not covered by medical insurance, which forced many chronic myeloid patients to buy generic drugs from India.
Only when it comes to pharmaceuticals can we have the story "I'm Not the God of Medicine".
Now that imatinib is included in the medical insurance, the price has dropped to more than 4,000, and Qianqian only needs more than 1,000 out-of-pocket every month.
Professor Zheng spread his fingers that were supporting each other, patted his right hand on the table, and said hello to Yu:
"This is a world-class drug. Enterprises must consider the global pricing system when setting prices. According to the price of this drug, a chronic myeloid patient needs about 450,000 yuan a year. But other companies, considering the actual payment ability of domestic patients,
, there is a third-party patient assistance program, and several of my patients applied for it through us. The annual drug cost is about 185,000.
"Moreover, the National Medical Insurance Administration has included them in drug price negotiations. As long as the drug is included in medical insurance, it will not be a problem for most patients."
"Yes, Professor Zheng, it's only 185,000 yuan. You can also use it for Qianqian. You will pay for it out of your own pocket now before it is included in the medical insurance list." Yu Wenhao said.
"I know that for the sake of Qian Qian, you will give her the treatment even if you are trying to sell it. This is not because I don't want to give it to her, but because there is a special situation."
"What special situation?" Yu Wenhao asked.
“This drug is currently used by adult patients and has reliable data support, but there is no clinical research data on patients under the age of 18.” Professor Zheng said, “I contacted them and they told me that they have already started to conduct research in this area.
Research, but based on the current clinical process, specific data cannot be provided."
When Yu Wenhao understood, he sighed: "Then you are just drawing a cake for me."
"No, why is it a cake? The medicine is there, and the real efficacy is there. There is no clinical research data on patients under the age of 18. It just says that its side effects on teenagers have not been fully understood. Once there is research data in this area,
, our doctors will know how to guide medication."
Professor Zheng looked at Yu Wenhao and said to him:
"Why are you anxious? There are more than 4 million leukemia patients in the country, half of them are teenagers. Do you think pharmaceutical companies will let this market go? They will definitely speed up the progress and make up for this market. Don't worry. Qian Qian can definitely
While I'm waiting for the progress of other pharmaceutical companies, and for the matching match, I'm not telling you as a doctor, but as a friend, Qianqian will definitely be able to wait."
Yu Wenhao nodded, he pondered for a while, and asked Professor Zheng:
"Is it possible, Professor Zheng, for example, that under your guidance, we could start giving Qian Qian this medicine now?"
"I want it too, and I also want Qianqian to be able to use it right away."
Professor Zheng and Yu Wenhao said:
"But I don't dare. I am responsible for you and Qianqian. Before the side effects of this drug on teenagers are fully understood, I cannot give medication guidance. Otherwise, the consequences will not be controllable.
It’s also irreversible. I can’t take this risk, and Qianqian’s current situation is not worth taking this risk, so I’d better wait.”
Thank you for the reward from Xinghai Bookworm 1977 at around three o'clock in the middle of the night. Thank you for the reward from Xinghai Bookworm 1977! Thank you Shachen Mood and Brother Sanpi for your monthly pass! Thank you for all the recommendation votes and reading! I wish everyone good health!
