诺华47.5万美元的癌症基因疗法到底贵不贵？

上周三，瑞士医药巨擘诺华制药开创了历史，成为第一家获美国食品药品监督管理局批准使用嵌合抗原受体T细胞免疫疗法治疗癌症的公司。这种突破性新技术可以强化病人自己的免疫细胞。它从病人体内提取免疫细胞，在实验室进行再造，然后重新植入病人体内，用于杀死肆虐的血癌细胞。诺华将这种治疗方法命名为Kymriah，医疗和生命科学界将其誉为医学的重大进步以及部分儿童和青年白血病人的福音（该基因疗法获准用于治疗某种白血病）。FDA也将批准该疗法称为“历史性举措”。

纪念斯隆·凯特琳癌症中心儿童肿瘤专家凯文·J·科尔曼在接受《财富》杂志采访时说：“这真的是一次变革。这些癌症患者的缓解率很高。它给病儿和家长带来了希望。如果其他治疗手段没有效，我们可以告诉他们我们的兵工厂里有了这样的新武器，它可以让你的细胞学会对抗癌症。”

但这种疗法的价格非常昂贵，诺华也因此受到了几个病人团体的抨击。诺华表示，Kymriah的价格为每个疗程47.5万美元（312.55万元人民币，这实际上略低于许多分析师此前的预期）。有人认为这样的价格并不合理。

近几年，药品价格丑闻一直是美国的主要乱象之一，Valeant和Mylan等制药公司都曾因为大幅提高老产品价格而备受指责。但有些人认为，考虑到Kymriah疗效极好（许多病人在接受治疗三个月后发现此前难以对付的癌症已经消失的无影无踪）而且打算在美国按新药定价，诺华首席执行官江慕忠不应与世界上的黑心制药大亨同流合污。

生物科技投资者布拉德·隆卡经营着一只专门投资癌症免疫疗法的基金。他对《财富》杂志表示：“问题在于这种事很难说清楚。无论什么时候，看到很高的绝对价格都会让人觉得非常难以接受。47.5万美元是一大笔钱，毫无疑问。但在我看来，设定这样的价格并不是出于贪婪。”

隆卡认为，和那些滥用权力，随意在美国为自家产品标价的公司不同，诺华实际上完成了生物医药行业视为“心脏”的科学创新。他还指出，诺华和美国联邦医疗保险和医疗补助服务中心将采用基于疗效的定价模式，也就是说，只有治疗一个月后见效，诺华才能拿到Kymriah的报销款。隆卡说：“江慕忠今天的领导力值得肯定。我觉得要是换一家公司，情况可能会大相径庭。我甚至不知道就这种适应症而言诺华能否盈利。”

隆卡相信，随着公司继续创新并掌握这种疗法的复杂制造过程，CAR-T领域的价格实际上将会下跌。他还说，如果更多制药公司和保险商共同采取措施，从而将药品对病人的实际疗效作为定价基础，美国就有可能不仅在科学方面看到新的发展空间，还会“在癌症治疗计费方式上进入新的时代”。

如欲进一步了解对诺华此项疗法和按疗效收费模式的有价值解读，请参见著名医疗肿瘤专家大卫·阿格斯和健康经济学家达娜·高德曼的新报告。（财富中文网）

译者：Charlie

审稿：夏林

On Wednesday, Swiss pharmaceutical giant Novartis made history as the first company to win Food and Drug Administration (FDA) approval for a groundbreaking new type of cancer treatment known as CAR-T. This technology harnesses the power of patients' very immune cells—which are extracted from them, reengineered in a lab, and then pumped back into the body—to kill aggressive blood cancers. The treatment, named Kymriah, was hailed by doctors and the life sciences community as a major advance in medicine and a boon to children and young adults with a certain form of leukemia (the group for whom the gene therapy is approved). The FDA itself called the approval a "historic action."

"It's really transformative," as Dr. Kevin J. Curran, a pediatric oncologist at the Memorial Sloan Kettering Cancer Center, told Fortune in an interview. "It's shown a massive response rate in people with these cancers. It's given hope to patients and parents. If other treatments fail, we can tell them, we have this new weapon in our arsenal that teaches your cells to fight cancer."

But Novartis immediately faced backlash from several patient groups over the therapy's massive list price. The company announced that Kymriah would ring in at $475,000 for a treatment course (and that's actually a bit less expensive than what many analysts had expected). To some, that price tag is unjustifiable.

Drug price scandals have become a prominent flashpoint in America over the last several years, with companies like Valeant and Mylan under the gun for gigantic list price hikes on old products. However, there are those who argue that Novartis CEO Joe Jimenez shouldn't be lumped in with the Martin Shkrelis of the world considering Kymriah's formidable benefit (many patients see no signs of their hard-to-treat cancers at all three months after treatment) and the first-of-its-kind pricing structure the company is pursuing in the U.S.

"The thing that’s hard to articulate about this is, whenever you see a high absolute price, it seems wildly outrageous. $475,000 is a lot of money, no question," said Brad Loncar, a biotech investor who runs a fund dedicated to cancer immunotherapies, in an interview with Fortune. "But the price in this case, in my opinion, was not set by greed."

Loncar argues that, unlike companies that abuse their prerogative to dictate whatever list prices they want in the U.S., Novartis has actually pulled off the kind of scientific innovation that the biopharma industry claims as its beating heart. He also points to the "outcomes-based" pricing model that the company will be instituting alongside the federal Centers for Medicare and Medicaid Services (CMS)—Novartis will only receive reimbursements for Kymriah if patients respond to it after the first month of treatment. "Joe Jimenez deserves a lot of credit for leadership today. I think this would have looked very different if it were another company," said Loncar. "I’m not sure if for this indication Novartis can even make it profitable."

Loncar believes that prices will actually come down in the CAR-T field as companies continue to innovate and navigate the manufacturing complexities of the treatments. And if more drug makers and insurance companies join in on a movement to price medicines based on how effective they actually are for patients, Loncar says, the U.S. could see not just a new frontier in science, but a "new era in how we pay for cancer therapies."

For more valuable insight into the Novartis drug and the pay-for-performance model, make sure to check out this new piece from renowned medical oncologist David Agus and health economist Dana Goldman.