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新藥備受爭議,但這家公司卻仍不放棄

新藥備受爭議,但這家公司卻仍不放棄

Sussie Gharib 2019-12-17
公司的首席執行官稱,在服用了渤健藥物18個月后,阿爾茨海默病的患者出現了“明顯的”好轉跡象。

有關渤健(Biogen)和其引發爭議的治療阿爾茨海默病的藥物,將有更多好戲上演。在加州圣迭戈面對一大群研究阿爾茨海默病的科學家時,渤健展示了其名為aducanumab藥物的新數據,稱這種藥物可以緩解阿爾茨海默病患者的癥狀。在詳細的闡述中,公司稱臨床試驗顯示,使用該藥物最高劑量的患者,在認知能力的退化方面要比服用無效對照劑的患者更加緩慢。

今年10月,渤健宣布重新啟動該藥物研究,并將尋求監管機構許可,震驚了整個生物醫藥界,此后也引來了諸多的懷疑。因為幾個月前,渤健曾經宣布放棄推出該藥物,并終止對該藥物的研究,因為試驗結果令人失望。

如今,這家位于馬薩諸塞州劍橋市的生物科技公司稱,這一藥物有效,他們已經重拾信心,并已經向美國食品與藥品管理局申請許可。申請結果預計將在2020年公布。如果監管機構同意,那么該藥物將成為能夠實際幫助日益衰弱的阿爾茨海默病患者的首種藥物。

隨著故事的起伏,渤健的股價也隨著每條新消息漲漲跌跌,而華爾街也是莫衷一是。

當我在幾周前與渤健的首席執行官米格爾·維納特索斯對話時,他顯得積極而充滿信心。他說,他覺得令人鼓舞的是,阿爾茨海默病的患者在服用了渤健藥物18個月后,出現了“明顯的”好轉跡象。

“我們要誠實、謙虛,并遵守監管流程。”他說,“在慶祝之前,我們得先等一等。但確實有希望。”

那么,如果維納特索斯成功地將這款突破性的治療阿爾茨海默病的新藥推向市場,它的售價將是多少呢?一開始,他推脫說:“談價格還太早。”

考慮再三后,他提議說,這種藥物投放市場后,將采用一種更加有創意的支付方式,用以覆蓋數百萬因為阿爾茨海默病而喪失記憶的患者。據美國疾控中心的數據,在美國患有阿爾茨海默病的患者人數,到2060年時將翻兩番,達到1400萬人。

“這需要多方努力以及政府參與,”他說,“我們公司和管理層,將對創新的商業方式也保持開放態度,讓藥物治療覆蓋更多的人群。我將竭盡所能破除那種舊式的繳費才治療的模式,這種模式在治療大范圍人群時已經顯示出局限性。”(財富中文網)

譯者:宣峰

There was more drama this week about Biogen and its controversial Alzheimer’s drug. Speaking before a large crowd of Alzheimer scientists gathered in San Diego, California, Biogen showed new data that its drug, called aducanumab, could offer relief to people struggling with Alzheimer’s. In a detailed presentation, the company said that in clinical trials, patients receiving the highest dose of the drug had slower declines in their cognitive abilities than patients who received a placebo.

There’s been plenty of skepticism about those claims ever since Biogen shocked the biotech world in October when it announced it was resurrecting its research and would also seek regulatory approval for the drug. Just a few months earlier, Biogen said it had given up on aducanumab and terminated its research because of disappointing results.

Now the Cambridge, Massachusetts-based biotechnology company says it has renewed confidence that the drug works and has returned to the Food and Drug Administration for approval. A decision is expected in 2020. If regulators give the okay, the drug could be the first treatment that actually helps people with the debilitating Alzheimer’s disease.

As the saga continues, Biogen shares have been soaring and tumbling on every new piece of information, and opinions on Wall Street have been mixed.

But when I spoke with Biogen CEO Michel Vounatsos a few weeks ago, he was positive and hopeful. What he finds encouraging, he says, is that Alzheimer’s patients who have received Biogen’s drug treatment showed “significant” improvement after 18 months.

“We have to be modest, humbled, and follow the regulatory process now,” he tells me. “We have to wait before we celebrate. But there is hope.”

So, if Vounatsos succeeds in delivering a breakthrough drug to treat Alzheimer’s, what will it cost? At first, he pushes back saying, “it’s too early to speak about cost.”

After some thought, he suggests that it will take a more innovative payment model to cover the millions of people suffering from Alzheimer’s memory loss. According to the CDC, the number of people living with Alzheimer’s disease is expected to nearly triple to 14 million people in the United States by 2060.

“This will take multi-party and engagement with authorities,” he says. “And the company and its leadership will be open to innovative contracting in order to treat the larger population. I will do everything I can in order to disrupt from the old models of fee-for-service that have certainly demonstrated their limitation in terms of treating a broader population. ”

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