dmims 019 One piece of good news
019 One piece of good news
More than a year ago, Yu Wenhao went to Professor Zheng for consultation. He said he saw some patients in the group who couldn't wait for a fully matched bone marrow transplant and had a half-matched transplant. He asked if Qianqian's situation could also be treated as a half-matched transplant using his bone marrow.
"Of course it can be done. We've done several cases already, and the surgery itself is not a problem."
Professor Zheng crossed his fingers over his chest, pressing them together, looked at Yu Wenhao, and continued:
"But in cases like Qianqian's, my advice is not to do it, but to wait. Compared to a fully matched transplant, a half-matched transplant has a higher risk of rejection and a lower five-year cure rate. In addition, although there is no data to support the relapse rate, based on my experience, it is definitely higher."
"Half-matched transplantation is a last resort for patients with acute illnesses, those whose physical condition can no longer withstand the disease, or those who are not suitable for targeted therapy and have to undergo transplantation immediately. We are friends, and I can be frank with you. To put it bluntly, it's like trying anything as a last resort, taking a gamble."
"If Qianqian had reached that stage, I would definitely advise you to have a haploidentical transplant immediately. But Qianqian hasn't reached that stage yet. She still has time to wait, to wait for a fully matched transplant, or to wait for advancements in medicine and healthcare. By the way, Xiaoyu, I have some good news for you."
Upon hearing Professor Zheng's good news, Yu Wenhao's eyes lit up, and he quickly leaned over to ask, "What good news?"
"The new drug Nerlikon, co-developed by Ascentage Pharma and Innovent Biologics, is generically known as Orabatinib tablets. It is an oral third-generation BCR-ABL inhibitor and the first third-generation BCR-ABL-targeted drug for the treatment of drug-resistant CML approved in my country. It has outstanding effects on BCR-ABL and various BCR-ABL mutants, including the T315I mutation, and can be said to have reached the world's leading level."
"What do you mean? Professor Zheng, please explain in a way that I can understand," Yu Wenhao said, sounding a little anxious.
"Okay, to put it simply, it's very effective for patients with chronic myeloid leukemia (CML) and can cure them without a bone marrow transplant," Professor Zheng said. "I have several cases where patients have been able to work and live normally after taking this medication."
"A cure without a transplant? Is that really possible? That's amazing!" Yu Wenhao exclaimed.
"This drug is remarkable. In March 2021, it was included in the list of breakthrough therapies by the CDE. In July 2019, it received FDA approval for clinical trials and directly entered Phase Ib clinical research. For four consecutive years since then, it has been selected for oral presentation at the American Society of Hematology Annual Meeting and was nominated for Best Study at the 2019 ASH Annual Meeting."
"In May 2020, they successively obtained orphan drug designation and fast track designation from the U.S. FDA, and also obtained orphan drug designation from the European FDA..."
"Orphan drugs, that means they're especially effective for children, right?" Yu Wenhao interrupted Professor Zheng hastily.
Professor Zheng couldn't help but laugh. He said:
"Don't take it literally. Orphan drugs are not drugs for orphans, but rather rare diseases, which are used to prevent and treat rare diseases. Because rare diseases affect a small population, have low market demand, and are costly to develop, pharmaceutical companies rarely invest in their research and development. Therefore, these drugs are figuratively called 'orphan drugs.'"
Yu Wenhao chuckled, then immediately realized what he meant and asked, "Is it very expensive?"
"Yes, orphan drugs are not cheap. The market is small and the research and development investment is large, so they are definitely not cheap. There are orphan drugs abroad that cost millions of dollars per dose. But for some people, they also need to take them, like Steve Jobs. If a drug is effective for him and can save his life, he would take it even if it cost tens of millions or hundreds of millions of dollars per dose, right?"
"There's a saying that sounds cruel, but it's true: the more advanced medicine becomes, how long a person lives is not determined by God or the King of Hell, but often by your wallet. The thicker your wallet, the longer you can live compared to others."
"I don't care, I don't care about Steve Jobs, or God or Yama, I just want to know, how much does this medicine cost?" Yu asked.
"Thirty-seven thousand a box," Professor Zheng said.
"So expensive?"
Yu Wen was taken aback. He knew that even when Novartis' imatinib was first imported from Switzerland, it cost only a little over 20,000 yuan per box and was not covered by medical insurance. This forced many patients with chronic myeloid leukemia to buy generic drugs from India, which led to the story of "Dying to Survive".
Now that imatinib is covered by medical insurance, the price has dropped to around 4,000 yuan, so Qianqian only needs to pay a little over 1,000 yuan per month out of pocket.
Professor Zheng separated his intertwined fingers, lightly tapped the table with his right hand, and greeted Yu Wenhao:
"This is a world-class drug. The company sets the price based on the global pricing system. According to the price of this drug, a chronic myeloid leukemia patient would need about 450,000 RMB per year. But the company takes into account the actual affordability of domestic patients and has a third-party patient assistance program. Several of my patients have applied for it through us, and the annual cost of the drug is about 185,000 RMB."
"Moreover, the National Healthcare Security Administration has included them in the drug price negotiation. As long as the drug is included in the medical insurance, it will not be a problem for most patients."
"Use it, Professor Zheng. Even if it's 185,000, you can use it for Qianqian too. Don't wait for it to be included in the medical insurance list; just pay for it out of pocket now," Yu Wenhao said.
"I know you'd sell everything you own to treat Qianqian, but it's not that I'm not going to use it on her, there's just a special situation."
"What special circumstances?" Yu asked.
"There is reliable data to support the use of this drug in adult patients, 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 started research in this area, but they cannot provide specific data at the current stage of clinical progress."
Yu Wenhao understood, and he sighed, "So you've basically just painted a pie in the sky for me."
"No, why is it a pancake? The medicine is there, and the real efficacy is there too. There is no clinical research data on patients under the age of 18. It only means that the side effects on adolescents have not been fully understood. Once there is research data in this regard, we doctors will know how to guide the use of the medication."
Professor Zheng looked at Yu Wenhao and said to him:
"Why are you in such a hurry? There are over four million leukemia patients nationwide, half of whom are teenagers. Do you think pharmaceutical companies will let this market go? They will definitely speed up their progress to fill this gap, so don't worry. Qianqian can wait for the pharmaceutical companies' progress while also waiting for a fully matched tissue sample. I'm not telling you this as a doctor, but as a friend, and I assure you that Qianqian will definitely be able to get it."
Yu Wenhao nodded, pondered for a moment, and then asked Professor Zheng:
"Is it possible, Professor Zheng, for example, that we could start giving Qianqian this medicine now, under your guidance?"
"I also want to, and I also want Qianqian to be able to use it right away."
Professor Zheng and Yu Wenhao said:
"But I dare not. This is for your sake and for Qianqian's sake. Before the side effects of this drug on teenagers are fully understood, I cannot provide medication guidance. Otherwise, the consequences will be uncontrollable and irreversible. I cannot take this risk, and Qianqian's current condition is not worth taking this risk. Let's wait."