美国国家公共电台 NPR Drugs For Rare Diseases Have Become Uncommonly Rich Monopolies

 

STEVE INSKEEP, HOST: 

We have an investigation¹ this morning of drug companies gaming a system designed to help desperate patients. It's a story of orphan² drugs.

DAVID GREENE, HOST: 

These are life-saving drugs that pharmaceutical³ firms don't develop because they wouldn't help enough people to be profitable. People with rare diseases are left with no health.

INSKEEP: Orphan drugs were a big issue decades ago and were featured on an old TV show, "Quincy, M.E."

(SOUNDBITE OF TV SHOW, "QUINCY, M.E.")

UNIDENTIFIED ACTOR: (As character) The truth is that the only real chance of research money going to these diseases is if you, the Congress, make the orphan drug bill a reality.

GREENE: And Congress did make the Orphan Drug Act a reality in 1983. It offers incentives⁴ to companies that develop drugs for rare diseases.

INSKEEP: Now, a Kaiser Health News investigation finds drugmakers are manipulating the system to collect the incentives. Sarah Jane Tribble has the first of two stories.

SARAH JANE TRIBBLE, BYLINE⁶: A few years ago, something strange began to happen in the world of drug development. The FDA was approving a growing number of drugs that had been given orphan status. Orphan drugs, so-called because they were once abandoned by large pharmaceutical companies, are defined as those that treat a disease that affects fewer than 200,000 people. By 2015, 47 percent of new medications were orphan drugs. That raised red flags for medical researchers like Dr. Martin Makary at Johns Hopkins University medical school because many of these are household names.

MARTIN MAKARY: Some of these medications clearly serve large populations and to find out that they've been submitted for orphan drug approval for a very narrow indication when we all know in the real world they're being used broadly.

TRIBBLE: They include familiar names like arthritis⁷ drugs Enbrel, Remicade and Humira. In fact, 7 of the 10 top-selling drugs of any kind in the U.S. in 2015 were orphan drugs, and Makary says this is a big problem.

MAKARY: What we're doing is diluting⁸ the impact of the Orphan Drug Act to create incentives for companies to develop drugs for diseases that really do affect underrepresented populations in medicine.

TRIBBLE: The Orphan Drug Act gives companies three economic incentives. The biggest one is market exclusivity. So it's a government promise of no direct competition for your drug for seven years when treating that disease. The other incentives are tax credits and a couple of million in savings⁹ on fees paid to the FDA.

GAYATRI RAO: There are still tremendous unmet needs that are out there.

TRIBBLE: Dr. Gayatri Rao runs the FDA's Office for Orphan Products. She says she knows there's talk that companies are taking advantage of the system. It's her job to safeguard the law.

RAO: I want to ensure that we continue to keep our eye on that prize and we ensure that we utilize¹⁰ the Orphan Drug Act in a way that the framers really intended.

TRIBBLE: But it's not clear if that is happening. The Kaiser Health News investigation reveals more than 70 orphan drugs were approved to treat common diseases before winning an approval to treat a rare disease and, with that, the financial incentives that come with being an orphan drug. Bernard Munos is a former executive at drug giant Eli Lilly and now with FasterCures at the nonprofit Milken Institute. He reviewed our analysis and was surprised.

BERNARD MUNOS: I looked into it and the more I looked into it and the more I discovered that there is something there that is going on that doesn't seem to be in keeping with the intent of the law. It looked like somewhere some smart person perhaps had found a way to hijack¹¹ the system, but it had become, according to the data, it looked very pervasive¹².

TRIBBLE: Munos says there are drugs that meet the spirit of the law - helping¹³ small populations of patients. He questions so whether some drugs, particularly the drugs that were already on the market, should get the financial incentives.

MUNOS: We're wasting resources. We're not doing any good. We're not adding any patient to the population of those that could benefit from the drug. As far as the patients are concerned, it is a complete waste.

TRIBBLE: Top-selling drug Humira won five approvals for orphan treatments after 2002 when it was already on the market to treat millions of people who suffer from rheumatoid arthritis. AbbVie, the maker⁵ of Humira, declined to comment on why it sought multiple orphan approvals for their drug. Anne Pritchett of PhRMA says drugmakers are following the science, and she warns that the Orphan Drug Act's incentives are necessary to support the research and development of life-saving drugs.

ANNE PRITCHETT: When you look at cystic fibrosis, it was 25 years to the development of an effective therapy. I think we would be concerned about anything that would undermine the current incentives.

TRIBBLE: Treatments for cystic fibrosis have been hugely successful, and they are hugely expensive. The FDA's Dr. Rao says the law is still needed to create drugs for about 7,000 rare diseases that have no treatments. But after being briefed on what we found, she also wants to examine the issue more closely.

RAO: Where I am is I want to understand more what the truth is really.

TRIBBLE: Rao says some drugs should get more than one approved use because there are scientific benefits to studying specific rare diseases. Still, she worries about companies using the law to drive up the prices of drugs.

RAO: Our goal is to try to get it right, you know, like, figure out what the right balance is and making sure that we're appropriately incentivizing things.

TRIBBLE: The FDA is doing its own evaluation¹⁴ of the drugs that want orphan status during 2010 and 2015. If necessary, the agency could propose changes to the Orphan Drug Act's regulations. I'm Sarah Jane Tribble in Washington.

(SOUNDBITE OF KINSKI'S "BULLETIN OF THE INTERNATIONAL STRING FIGURE ASSOCIATION")

1 investigation   

n.调查，调查研究

参考例句：

• In an investigation,a new fact became known, which told against him.在调查中新发现了一件对他不利的事实。
• He drew the conclusion by building on his own investigation.他根据自己的调查研究作出结论。

2 orphan   

n.孤儿；adj.无父母的

参考例句：

• He brought up the orphan and passed onto him his knowledge of medicine.他把一个孤儿养大，并且把自己的医术传给了他。
• The orphan had been reared in a convent by some good sisters.这个孤儿在一所修道院里被几个好心的修女带大。

3 pharmaceutical   

adj.药学的，药物的；药用的，药剂师的

参考例句：

• She has donated money to establish a pharmaceutical laboratory.她捐款成立了一个药剂实验室。
• We are engaged in a legal tussle with a large pharmaceutical company.我们正同一家大制药公司闹法律纠纷。

4 incentives   

激励某人做某事的事物( incentive的名词复数 )； 刺激； 诱因； 动机

参考例句：

• tax incentives to encourage savings 鼓励储蓄的税收措施
• Furthermore, subsidies provide incentives only for investments in equipment. 更有甚者，提供津贴仅是为鼓励增添设备的投资。 来自英汉非文学 - 环境法 - 环境法

5 maker   

n.制造者，制造商

参考例句：

• He is a trouble maker，You must be distant with him.他是个捣蛋鬼，你不要跟他在一起。
• A cabinet maker must be a master craftsman.家具木工必须是技艺高超的手艺人。

6 byline   

n.署名；v.署名

参考例句：

• His byline was absent as well.他的署名也不见了。
• We wish to thank the author of this article which carries no byline.我们要感谢这篇文章的那位没有署名的作者。

7 arthritis   

n.关节炎

参考例句：

• Rheumatoid arthritis has also been linked with the virus.风湿性关节炎也与这种病毒有关。
• He spent three months in the hospital with acute rheumatic arthritis.他患急性风湿性关节炎，在医院住了三个月。

8 diluting   

稀释，冲淡( dilute的现在分词 )； 削弱，使降低效果

参考例句：

• A companion would have been a distraction, diluting the pathos of the moment. 要是有一个伴侣在旁就会分散我的注意，冲淡此时此刻的哀婉之情。
• Diluting agent has certain transparency for ink multi-color overprint. 冲淡剂具有必定的透明量，适分油不朱的众色叠印。

9 savings   

n.存款，储蓄

参考例句：

• I can't afford the vacation,for it would eat up my savings.我度不起假，那样会把我的积蓄用光的。
• By this time he had used up all his savings.到这时，他的存款已全部用完。

10 utilize   

vt.使用，利用

参考例句：

• The cook will utilize the leftover ham bone to make soup.厨师要用吃剩的猪腿骨做汤。
• You must utilize all available resources.你必须利用一切可以得到的资源。

11 hijack   

v.劫持，劫机，拦路抢劫

参考例句：

• Most countries refuse to give sanctuary to people who hijack aeroplanes.大多数国家拒绝对劫机者提供庇护。
• The hijack take place just after the plane take off.劫持是飞机刚起飞后发生的。

12 pervasive   

adj.普遍的；遍布的，(到处)弥漫的；渗透性的

参考例句：

• It is the most pervasive compound on earth.它是地球上最普遍的化合物。
• The adverse health effects of car exhaust are pervasive and difficult to measure.汽车尾气对人类健康所构成的有害影响是普遍的，并且难以估算。

13 helping   

n.食物的一份&adj.帮助人的，辅助的

参考例句：

• The poor children regularly pony up for a second helping of my hamburger. 那些可怜的孩子们总是要求我把我的汉堡包再给他们一份。
• By doing this, they may at times be helping to restore competition. 这样一来, 他在某些时候,有助于竞争的加强。

14 evaluation   

n.估价，评价；赋值

参考例句：

• I attempted an honest evaluation of my own life.我试图如实地评价我自己的一生。
• The new scheme is still under evaluation.新方案还在评估阶段。