恶（2013）

Vice S04E16

Part 3 May 7, 2021, 11:30 a.m.

It's not right. I mean, every day that passes,
这不对日子一天天过去
he's losing more of himself.
他的行动能力越来越差
And that goes for every ALS patient.
每个渐冻症患者都是这样
Yeah, he's willing to take the risk,
是的他愿意冒险
which, I mean, as far as safety goes...
即使有安全问题
Yeah.
对
Yeah.
对
Yeah. If there were options here in America,
对如果在美国这里有选择的话
we'd prefer to do it here, but...
我们更愿意在这里做但是
there are no options right now.
现在别无选择
Wissam was forced to leave the country.
维萨姆被迫离开国家
His family and friends raised $30,000
他的家人和朋友凑了三万美金
to send him to Lebanon
送他去了黎巴嫩
for an experimental stem cell treatment
去进行一个和NurOWN有着类似工作原理的
that works in a similar way as NurOWN.
干细胞实验性治疗
We're in Beirut and we are on our way
我们在布鲁特去看维萨姆的路上
to see Wissam the day before
在他进行大型
his big stem-cell procedure.
干细胞手术的前一天
What are you expectations for tomorrow?
你对明天有什么期待吗
No, that's my goal too.
不那也是我的目标
You can kind of see it in my left hand.
你可以在我的左手看到萎缩现象
Also in my right hand I've atrophied a little bit.
我的右手也有一点萎缩
No, I know.
是的我知道
I know.
我明白
We followed Wissam as he prepared for treatment.
维萨姆准备治疗的时候我们一直陪着他
How are you doing, buddy? Hey, how are you?
你好吗伙计嘿你好吗
I'm good.
我很好
How are you feeling?
感觉怎么样
Oh, yeah.
哦是啊
Hopefully they find a cure soon
希望在严重发病之前
before anything happen.
他们能快点找到治疗方法
I love him so much.
我这么爱他
I don't want to lose him.
我不想失去他
I wanted to understand why patients like Wissam
我想弄明白为什么像维萨姆这样
who are severely disabled
重度残疾的病人
are forced to travel halfway across the world
却要被强制穿越半个世界
just to access treatment.
才能接受治疗
So, I met with Dr. Janet Woodcock,
所以我拜访了珍妮特伍德科克
the director of the FDA's
她是食品药品管理局
Center for Drug Evaluation and Research.
药品评估和研究中心的主任
For a disease like ALS,
像渐冻症这样的疾病
where a drug can work for one person
药物只对特定病人有效
and not necessarily the other
对其他病人不一定有用
because no two ALS patients are alike,
因为没有两个渐冻症患者是一样的
what's the harm in just releasing
如果它是安全的
a drug if it's safe?
那么将药品发行到市场上的危害到底是什么
First of all, most drugs have liabilities.
首先大多数药物都有副作用
If FDA would just release
如果食品药品管理局发行
a lot of ineffective drugs out there,
许多无效的药物到市面上
we would never find out...
我们永远都不会发现
we'd never find out which ones are harmful,
我们永远都不会发现哪些是有害的
we'd never find out which ones are beneficial.
哪些是有益的
We know this from long and bitter experience.
我们从漫长且痛苦的经历中了解到这些
But if a patient is willing to take the risk?
但是如果病人愿意冒这个险呢
I spoke to a Navy jet pilot who has the disease,
我和一个患有疾病的海军飞行员谈过话
and he's willing to sign off on it.
他支持这件事
I'm willing to sign off on it.
我也支持这件事
Again, what is the harm of releasing a drug
再问一下将我们愿意冒险一试的药物
that we're willing to take the risk for?
投放市场会有什么危害
Well, we don't want to stand between
是这样我们并不想阻碍
people trying investigational drugs.
那些希望尝试治疗的患者
Okay? So, there's a... large access programs
好吗有一个FDA开展的大型的访问计划
that FDA runs where we agree that
在该计划中我们同意
people who wish to try investigational products can.
那些有意愿的人去尝试实验药物
Sign on to that. We agree with that philosophy.
他们可以签署协议我们同意这种个人选择
The FDA program is called Expanded Access,
FDA的计划叫做扩展访问
and it allows patients to try unapproved drugs.
它允许病人尝试未经批准的药物
But in the months it often takes to complete the paperwork,
但是通常要花好几个月的时间去完成文书工作
ALS patients can lose their ability to walk,
ALS病人会失去他们的行走能力
speak, or breathe on their own.
说话能力或者自己呼吸的能力
Exhausted by the process, patients often give up.
被这个过程整得心衰力竭病人们经常会放弃
And as we learned from Rich Casey,
就像里奇凯西的经历
the CEO of a company that developed
里奇是一家公♥司♥的CEO 该公♥司♥
a highly promising drug known as NP001,
发明了一种非常有前途的药品叫做NP001
even innovative drug companies need revenue
创新药品公♥司♥都需要通过收费
to keep their research going.
来保持他们研究的持续进行
But in the US, drug companies are
但美国不允许制药公♥司♥
not allowed to charge for unapproved drugs,
对未经批准的药物收费
and that leaves American patients out in the cold.
这种政策使得美国的病人无法寻求帮助
How many people in the US are getting your drug
在美国有多少人能申请到爱心用药计划
through compassionate use and how many people
得到你们的药欧洲又有多少人
are getting it in Europe?
能得到药物
In the US, nobody.
在美国没人获得
And the reason it's available there and not here
药品在欧洲可用而在这不行的原因是
is that their laws allow us to be reimbursed
他们的法律允许我们获得
for our costs.
成本上的补偿
'Cause we obviously don't have the financial resources
因为我们显然没有财政资源
to just give it away.
来赠送药物
And in Europe, we can get reimbursed for our costs.
在欧洲我们可以就花费获得补偿
Here in the US, you can't.
在美国就不行
It's against the law to sell an... an unapproved drug.
出♥售♥未经批准的药物是违法的
How did it feel when ALS patients
当ALS病人找到你
who responded to your drug came to you
请求获得药物的时候
and asked you for the drug?
你有什么感受
It's... it's a horrible, horrible feeling.
非常糟糕的感觉
I mean, you feel like less than human.
我是说你觉得这不合人道
Because these... these people were doing well.
因为这些人很努力
And of course I wanted to give it to 'em, but there was no way.
我当然想给他们药但是不行
We... we had no money at that time.
我们那时没有钱
Um, there was questions
恩我们作为一个公♥司♥
about whether we would survive as a company.
面临很多生存问题
It's a really heart-wrenching,
和这些病人的谈话
you know, discussion with these patients.
真的很让人心痛
Um, and then of course, you know, most of them end up dying,
毫无疑问他们中的大多数最后都死了
so it's very... it's tragic, you know?
所以这非常悲痛
MIT economist Dr. Andrew Lo thinks
麻省理工的经济学家安德鲁卢认为
the real issue here is that the FDA's
这里真正的问题是FDA当下的
current approval system treats all diseases the same.
批准系统对待所有的药物都一视同仁
So, he developed a complex formula
所以他计算出一个复杂的公式
that judges risk on a disease-by-disease basis.
来判断多重病理的风险
You're saying that not all diseases
你是说不是所有的疾病
should be treated equally.
都应该被平等对待
ALS is a good example.
ALS就是一个很好的例子
Patients might be willing to take a bigger risk
病人们可能愿意冒更大的风险
of getting a drug that may not work
去尝试一种可能不管用的药物
if there is some chance that that drug might work.
只要这种药有奏效的可能性
Whereas somebody who is taking acne medication
然而正在使用治疗痤疮药物的人
may not be willing to take any kind of a chance
可能不愿意冒任何的风险
for adverse consequences.
来获得不利的结果
So, the idea is to use this mathematical framework
所以方法就是使用我们计算出的数学框架
that we developed and to be able to come up
并且能够提出
with a single number that says,
一个单一的数字来说明
"Here, this is the threshold you ought to use
这就是你用来参考的阈值
"For approving drugs for this disease,
为了使得治疗该疾病的药物获批
"Whereas for this other disease that's maybe not as serious,
尽管对于其他疾病来说可能没有这么严重
you can use a different threshold,
你可以使用一个不同的阈值
a more conservative threshold."
一个更加保守的阈值
Lowering that threshold would not only increase access
降低阈值不仅为更多患者打开通道
for patients, but also incentivize
也能够刺♥激♥
pharmaceutical companies
制药公♥司♥