美国国家公共电台 NPR Patients Increasingly Influence The Direction Of Medical Research

Patients Increasingly Influence The Direction Of Medical Research 

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Patients and their advocates are starting to get a louder voice in how medical research is carried out. They participate in the design of experiments and have a greater say in what outcomes they care about most. Today in your health, NPR's Richard Harris takes a look at how this movement is evolving.

RICHARD HARRIS, BYLINE²: When Sharon Terry discovered that her children had been born with a rare genetic⁴ disease that doctors knew little about, she had a rather startling encounter with medical researchers.

SHARON TERRY: Researchers came and took blood from us and our kids. And then a few days later, another set of researchers wanted to take blood, too, and we didn't understand why they weren't sharing. And we also didn't understand why they weren't working together.

HARRIS: Terry, a college chaplain, and her husband, a construction manager, came to realize that the researchers weren't so interested in treating their kids. Their first priority was to probe the biology underlying⁵ the rare disease.

TERRY: We look at things differently. We look at what matters to us and maybe not some, you know, biological pathway that absolutely is important but isn't going to give us the answers we need right away.

HARRIS: So Terry and her husband took matters into their own hands. This is back in the 1990s. They finagled the use of a lab bench at Harvard and set about to track down the gene³ responsible for their children's connective tissue disease.

TERRY: With no science background, we didn't understand the technicalities, but we could see patterns in things. So it took a couple of years. In those days, it did take a couple of years. And we did find the gene, and then we put together a diagnostic test based on the gene. And now we're actually looking at therapies that might involve different variations in the gene.

HARRIS: Of course, this is not a typical story of how parents end up getting involved in medical research, but it isn't unique either.

TERRY: When we did it, it was paving a new way, but there have been, actually, hundreds of people after us to do similar things. But more commonly, I think, what people do is they realize they have a question that's unanswered or they see something in their child that no one's paying attention to. And I think, in this day and age, it's kind of cool because they can do more quick crowdsourcing using Facebook or using Twitter and find each other very quickly.

HARRIS: There are now hundreds of groups focusing on everything from arthritis⁶ to xeroderma pigmentosum. And patient participation⁷ is no longer an informal matter. The concept is baked into federal laws, including one that funds the Food and Drug Administration.

(SOUNDBITE OF ARCHIVED RECORDING)

LINDSEY: Good morning, everyone.

HARRIS: Since 2013, the FDA has held more than 20 meetings in which they invite patients to come in and talk about what actually matters to them. In September, the topic was organ transplant recipients⁸. And among the people who spoke⁹ up was Lindsey Duquette, a 14-year-old with remarkable¹⁰ poise¹¹. She said she had been in and out of the hospital 172 times before she got a kidney transplant when she was 10.

(SOUNDBITE OF ARCHIVED RECORDING)

LINDSEY: Here's some items on my post-transplant wish list. How about a transplanted kidney that'll last the rest of my life, fewer toxic¹² medications? And it would be great if meds could be taken just once a day.

HARRIS: She wishes the drugs she must take didn't pose a lifetime cancer risk. And she wonders what will happen when, years from now, her transplanted kidney fails and she will need a new one.

(SOUNDBITE OF ARCHIVED RECORDING)

LINDSEY: Will I lose my job? Those are my thoughts and concerns. Thank you for listening.

(APPLAUSE)

HARRIS: The FDA, historically, hasn't considered its work strictly¹³ from the patient's point of view. It generally starts with a company pitching a new drug or device and is supposed to answer the more narrow question - is the product safe and effective? Kevin Longino, who heads the National Kidney Foundation, is optimistic that the FDA will broaden its horizons as it considers things from the patient's point of view.

KEVIN LONGINO: The fact that they're holding the events indicates that they're taking it very seriously.

HARRIS: How much leverage¹⁴ do you feel patients have in dealing¹⁵ directly with the drug companies and saying, hey, these are our problems, we need solutions?

LONGINO: I think patients have very little influence with drug companies.

HARRIS: The hope looking forward is that when drug companies bring new products to the FDA for evaluation¹⁶, the agency can ask whether the tests of these products will measure outcomes that patients care about. The FDA has already demonstrated that it's paying more attention to what patients want. Earlier this year, after hearing impassioned pleas from parents and their scientific allies, the agency temporarily approved a drug for Duchenne muscular dystrophy, even though the science in hand didn't provide proof positive that the drug was effective. Patients considered that a victory, but that makes consumer watchdog Diana Zuckerman nervous.

DIANA ZUCKERMAN: The bad news about what's happening is that so much of patient input¹⁷ is involving patients who are working with or recruited by drug companies.

HARRIS: These companies pour millions of dollars into advocacy organizations and provide information to patients, so the patient voices are sometimes also channeling commercial interests. Zuckerman, who heads the National Center for Health Research, worries that desperate patients hear more about the potential benefits than the risks. Clearly, patients should have a voice in how big a risk they are willing to take, she says, but within limits.

ZUCKERMAN: I think it's safe to say we need a sweet spot, and it hasn't been found. And the FDA has not done a good job of finding that sweet spot.

HARRIS: This is by no means a new tension. AIDS activists¹⁸ fought this decades ago with the FDA, and that friction¹⁹ will always exist. To get around this cozy²⁰ relationship between drug companies and patient groups, Zuckerman runs training sessions to teach ordinary people to serve as the voice of the patient, independent of the advocacy groups. The National Breast Cancer Coalition²¹ has been doing that for many years as well. Sharon Terry also frets²² about keeping the focus on patients. After her own experience in finding a gene for the rare disease that affects her children, she decided²³ to start an advocacy group called the Genetic Alliance.

TERRY: I think advocacy still has a place, but I'm starting to be concerned myself - and, again, I run one of these groups - about how much we're becoming the man. And we really need to break down our own silos because we start to be insular²⁴. We start to be competitive, and that is not going to serve the people who suffer.

HARRIS: She worries that advocates are losing their role as agents of change. The system has come a long way over the past 20 years, but it still has plenty of room for improvement. Richard Harris, NPR News.

1 browser   

n.浏览者

参考例句：

• View edits in a web browser.在浏览器中看编辑的效果。
• I think my browser has a list of shareware links.我想在浏览器中会有一系列的共享软件链接。

2 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。

3 gene   

n.遗传因子，基因

参考例句：

• A single gene may have many effects.单一基因可能具有很多种效应。
• The targeting of gene therapy has been paid close attention.其中基因治疗的靶向性是值得密切关注的问题之一。

4 genetic   

adj.遗传的，遗传学的

参考例句：

• It's very difficult to treat genetic diseases.遗传性疾病治疗起来很困难。
• Each daughter cell can receive a full complement of the genetic information.每个子细胞可以收到遗传信息的一个完全补偿物。

5 underlying   

adj.在下面的，含蓄的，潜在的

参考例句：

• The underlying theme of the novel is very serious.小说隐含的主题是十分严肃的。
• This word has its underlying meaning.这个单词有它潜在的含义。

6 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。

7 participation   

n.参与，参加，分享

参考例句：

• Some of the magic tricks called for audience participation.有些魔术要求有观众的参与。
• The scheme aims to encourage increased participation in sporting activities.这个方案旨在鼓励大众更多地参与体育活动。

8 recipients   

adj.接受的；受领的；容纳的；愿意接受的n.收件人；接受者；受领者；接受器

参考例句：

• The recipients of the prizes had their names printed in the paper. 获奖者的姓名登在报上。 来自《简明英汉词典》
• The recipients of prizes had their names printed in the paper. 获奖者名单登在报上。 来自《现代英汉综合大词典》

9 spoke   

n.（车轮的）辐条；轮辐；破坏某人的计划；阻挠某人的行动 v.讲，谈（speak的过去式）；说；演说；从某种观点来说

参考例句：

• They sourced the spoke nuts from our company.他们的轮辐螺帽是从我们公司获得的。
• The spokes of a wheel are the bars that connect the outer ring to the centre.辐条是轮子上连接外圈与中心的条棒。

10 remarkable   

adj.显著的，异常的，非凡的，值得注意的

参考例句：

• She has made remarkable headway in her writing skills.她在写作技巧方面有了长足进步。
• These cars are remarkable for the quietness of their engines.这些汽车因发动机没有噪音而不同凡响。

11 poise   

vt./vi. 平衡，保持平衡；n.泰然自若，自信

参考例句：

• She hesitated briefly but quickly regained her poise.她犹豫片刻，但很快恢复了镇静。
• Ballet classes are important for poise and grace.芭蕾课对培养优雅的姿仪非常重要。

12 toxic   

adj.有毒的，因中毒引起的

参考例句：

• The factory had accidentally released a quantity of toxic waste into the sea.这家工厂意外泄漏大量有毒废物到海中。
• There is a risk that toxic chemicals might be blasted into the atmosphere.爆炸后有毒化学物质可能会进入大气层。

13 strictly   

adv.严厉地，严格地；严密地

参考例句：

• His doctor is dieting him strictly.他的医生严格规定他的饮食。
• The guests were seated strictly in order of precedence.客人严格按照地位高低就座。

14 leverage   

n.力量，影响；杠杆作用，杠杆的力量

参考例句：

• We'll have to use leverage to move this huge rock.我们不得不借助杠杆之力来移动这块巨石。
• He failed in the project because he could gain no leverage. 因为他没有影响力，他的计划失败了。

15 dealing   

n.经商方法，待人态度

参考例句：

• This store has an excellent reputation for fair dealing.该商店因买卖公道而享有极高的声誉。
• His fair dealing earned our confidence.他的诚实的行为获得我们的信任。

16 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。

17 input   

n.输入(物)；投入；vt.把(数据等)输入计算机

参考例句：

• I will forever be grateful for his considerable input.我将永远感激他的大量投入。
• All this information had to be input onto the computer.所有这些信息都必须输入计算机。

18 activists   

n.（政治活动的）积极分子，活动家( activist的名词复数 )

参考例句：

• His research work was attacked by animal rights activists . 他的研究受到了动物权益维护者的抨击。
• Party activists with lower middle class pedigrees are numerous. 党的激进分子中有很多出身于中产阶级下层。 来自《简明英汉词典》

19 friction   

n.摩擦，摩擦力

参考例句：

• When Joan returned to work,the friction between them increased.琼回来工作后，他们之间的摩擦加剧了。
• Friction acts on moving bodies and brings them to a stop.摩擦力作用于运动着的物体，并使其停止。

20 cozy   

adj.亲如手足的，密切的，暖和舒服的

参考例句：

• I like blankets because they are cozy.我喜欢毛毯，因为他们是舒适的。
• We spent a cozy evening chatting by the fire.我们在炉火旁聊天度过了一个舒适的晚上。

21 coalition   

n.结合体，同盟，结合，联合

参考例句：

• The several parties formed a coalition.这几个政党组成了政治联盟。
• Coalition forces take great care to avoid civilian casualties.联盟军队竭尽全力避免造成平民伤亡。

22 frets   

基质间片； 品丝（吉他等指板上定音的）( fret的名词复数 )

参考例句：

• The river frets away the rocks along its banks. 河水侵蚀了两岸的岩石。
• She frets at even the slightest delays. 稍有延误她就不满。

23 decided   

adj.决定了的，坚决的；明显的，明确的

参考例句：

• This gave them a decided advantage over their opponents.这使他们比对手具有明显的优势。
• There is a decided difference between British and Chinese way of greeting.英国人和中国人打招呼的方式有很明显的区别。

24 insular   

adj.岛屿的，心胸狭窄的

参考例句：

• A continental climate is different from an insular one.大陆性气候不同于岛屿气候。
• Having lived in one place all his life,his views are insular.他一辈子住在一个地方，所以思想狭隘。