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國家藥監局藥品審評中心(CDE)近日公示,總部位於瑞士巴塞爾的跨國製藥及生物技術公司諾華(Novartis),旗下治療脊髓性肌肉萎縮症(Spinal Muscular Atrophy, SMA)的AAV基因治療藥物「Zolgensma」(OAV101注射液),向中國遞交的臨床試驗申請已獲得臨床試驗默示許可。

Zolgensma目前定價高達212.5萬美元,折合約1348萬元人民幣,被稱是製藥史上單價最貴的藥物

據介紹,SMA也是一種運動神經元性疾病,患者控制肌肉活動的運動神經細胞無法正常工作,導致逐漸喪失各種運動功能,甚至無法呼吸和吞咽。SMA在2018年5月被列入國家衛健委等部門聯合制定的《第一批罕見病目錄》。在2019年5月24日,美國食品藥品監督管理局(FDA)批准諾華公司的第一個用於治療2歲以下SMA患者的基因療法Zolgensma,患者只需接受一次性靜脈注射給藥,就能實現長期緩解甚至治癒。目前Zolgensma已經在全球近40個國家和地區獲批准上市

報道提到,現時全球共有3款用於治療SMA的藥物,除了Zolgensma,還包括渤健的Spinraza(諾西那生鈉注射液)以及羅氏的Evrysdi,後兩款藥物都已在中國獲批。其中諾西那生鈉注射液就是在去年經歷「靈魂砍價」,從約70萬元降至3萬多元後,被納入國家醫保目錄。

但對於Zolgensma能否納入醫保,就要視乎會否再出現「靈魂砍價」。內地傳媒分析,雖然國家醫保目錄納入的罕見病藥物數量逐年增多,去年更是實現高值罕見病藥物在國家醫保目錄中零的突破,但事實上醫保不能為所有高值藥物托底也是業界共識。國家醫保談判藥品基金測算專家組組長鄭傑曾經公開表示,目前國家醫保目錄內所有藥品年治療費用均未超過30萬元,未來國家醫保局將「堅決杜絕天價藥進醫保」,「保基本」是國家醫保目錄的功能定位,決定能夠被納入其中的藥品必須具備「價格合理」這一基本條件

值得一提是,對於質疑Zolgensma的價格高昂,諾華的CEO表明,批評者並沒有真正思考醫療系統的運作,給予患者進行移植手術治療SMA,每人的花費是300萬至500萬美元,但效果仍遠不如合適的藥物。使用Zolgensma的話,由於只需要接受一次性注射,故此實際所花的費用是比較便宜。
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乜中國唔係已經成為世界第二大經濟體,富強,厲害咩?點解一班不幸患罕見疾病嘅人都得唔到治療?

"決定能夠被納入其中的藥品必須具備「價格合理」這一基本條件" ,何謂合理?


新聞來源連結:
https://www.hk01.com/即時中國/726391/罕見病藥zolgensma獲批臨床試驗-定價1300萬將再-靈魂砍價

[ 本帖最後由 hfceddie 於 2022-1-23 02:37 AM 編輯 ]



引用:
原帖由 hfceddie 於 2022-1-23 02:23 AM 發表
https://cdn.hk01.com/di/media/images/dw/20220120/560843968770740224803649.jpeg/jP9kxiqpXTA5JCg4P7_b_SlGYe696GOmDJVRIwyVUSM?v=w1280r16_9

國家藥監局藥品審評中心(CDE)近日公示,總部位於瑞士巴塞爾的跨國製藥及生物技術公司諾華(Novartis),旗下治療脊髓 ...
其實你知唔知自己講緊乜?
呢個病全世界都係得果幾隻治療方法
自己上NORD搵睇下美國過去五年有幾多人患SMA投訴得唔到足夠支援既又有幾多
張LIST有哂



引用:
原帖由 vincent.AC7 於 2022-1-23 02:42 AM 發表

其實你知唔知自己講緊乜?
呢個病全世界都係得果幾隻治療方法
自己上NORD搵睇下美國過去五年有幾多人患SMA投訴得唔到足夠支援既又有幾多
張LIST有哂
我當然知自己講緊乜。做乜吓吓要同美國比,崇洋?美國唔好嘅嘢中國要跟住做?

英國病人打,免費。 NHS 埋單。  英國比中國有錢,富強?

Essex baby given £1.79m drug has new lease of life

baby who received the world's most expensive drug has got a new lease of life, his mother said.
One-year-old Edward, from Colchester, Essex, has spinal muscular atrophy (SMA) which means he lacks a protein vital for muscle development.

He was given the new gene therapy Zolgensma, which costs £1.79m, through the NHS in August.

Megan Willis said her son has since achieved milestones she never thought would be possible.

He can now roll over, sit on his own and has taken his first assisted steps, she said.

"We are so proud of Edward," she said. "He's doing incredibly well. He is far surpassing our expectations. We are so incredibly lucky.

"All I ever wanted was for him to be able to sit and I knew then he would have an amazing life.

"We have been working on his walking for a few weeks and he moves his own legs in the gaiters, which is a really good achievement.

"It brought a tear to my eye when I first saw it. I thought 'this is what it should be like', it was an incredible moment."

The family moved to London so that Edward can have physio up to five times a week.

Ms Willis, who documents Edward's journey on Instagram, said his energy levels have really improved and he has gone from being lethargic to a normal, cheeky, playful baby.

"This drug has given Edward his life back. He has got a new lease of life.

"It's a marathon, not a sprint. It's going to take a long time for Edward to catch up but he is slowly getting there," she said.

Zolgensma is thought to be the most expensive drug in the world, though NHS England said it had negotiated an undisclosed discount on its £1.79m list price.

About 65 babies are born with SMA in England each year. It causes muscle weakness and affects movement and breathing, meaning most babies do not live past the age of two without intervention.

Edward, who was diagnosed at two months old, was receiving another drug called Spinraza, which involves regular injections in the spine for life, compared to the one-off injection of Zolgensma.

https://www.bbc.com/news/uk-england-essex-59565966

[ 本帖最後由 hfceddie 於 2022-1-23 02:57 AM 編輯 ]



呢隻藥係咪有個香港病人, 幾經哀求林鄭, 最終獲得政府付款使用嗰隻?


[隱藏]
有罕見病還比武漢肺炎更麻煩


引用:
原帖由 為香港人鼓掌 於 2022-1-23 03:18 AM 發表
呢隻藥係咪有個香港病人, 幾經哀求林鄭, 最終獲得政府付款使用嗰隻?
我唔使 check 都知唔係。



引用:
原帖由 hfceddie 於 2022-1-23 03:31 AM 發表

我唔使 check 都知唔係。
啊, 第一時間聯想到, 香港嗰個類似病人~



引用:
原帖由 hfceddie 於 2022-1-23 02:23 AM 發表
https://cdn.hk01.com/di/media/images/dw/20220120/560843968770740224803649.jpeg/jP9kxiqpXTA5JCg4P7_b_SlGYe696GOmDJVRIwyVUSM?v=w1280r16_9

國家藥監局藥品審評中心(CDE)近日公示,總部位於瑞士巴塞爾的跨國製藥及生物技術公司諾華(Novartis),旗下治療脊髓 ...
you are blocked. sucker



NHS England said it had negotiated an undisclosed discount on its


[隱藏]
引用:
原帖由 場外吶喊人 於 2022-1-23 04:16 AM 發表
NHS England said it had negotiated an undisclosed discount on its
So what?  China is unable to negotiate a discount?

On a simple pro-rata, I estimate that the no. of new born suffering from the disease will be around 1400 each year, compared to 65 of England.  China will be in a stronger position to seek a better deal.



現實就係, 所有國家政府都寧用千幾萬去醫幾十個病人, 而唔係用千幾萬黎醫一個病人, 人地唔會講, 但事實就係咁, 無論你接唔接受


希望我有生之年可以見到西方醫藥被中醫藥及順勢療法顛覆!!!


提示: 作者被禁止或刪除 內容自動屏蔽
引用:
原帖由 hfceddie 於 2022-1-23 02:23 AM 發表
https://cdn.hk01.com/di/media/images/dw/20220120/560843968770740224803649.jpeg/jP9kxiqpXTA5JCg4P7_b_SlGYe696GOmDJVRIwyVUSM?v=w1280r16_9

國家藥監局藥品審評中心(CDE)近日公示,總部位於瑞士巴塞爾的跨國製藥及生物技術公司諾華(Novartis),旗下治療脊髓 ...
我想問第一大經濟體美國,公民係咪可以免費用藥 ?



[隱藏]
是否一公升眼淚嗰種病?係就好了。


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