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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, BYLINE: When Sharon Terry discovered that her children had been born with a rare genetic 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 underlying 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 gene 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 arthritis to xeroderma pigmentosum. And patient participation 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 recipients. And among the people who spoke up was Lindsey Duquette, a 14-year-old with remarkable poise. 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 toxic 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 strictly 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 leverage do you feel patients have in dealing 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 evaluation, 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 input 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 activists fought this decades ago with the FDA, and that friction will always exist. To get around this cozy 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 Coalition has been doing that for many years as well. Sharon Terry also frets about keeping the focus on patients. After her own experience in finding a gene for the rare disease that affects her children, she decided 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 insular. 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.