Tag Archives: medicine

A listening cure: PatientsLikeMe gives patients voice in clinical trial design


Until now, patients have had very little to do with the design of clinical trials — even though they’re key to their success. Online patient networking and research platform PatientsLikeMe, is changing all that. Last week, PLM launched a new service that lets pharmaceutical companies collaborate with patients to create clinical trials that serve their needs — amplifying the chances of success for both researchers and patients. We asked PLM’s Paul Wicks to tell us more about why this is a game-changer for medical research.

Why is this latest development so important?

The most important path to creating new medical breakthroughs is the randomized clinical trial (RCT). Hundreds, sometimes thousands, of patients volunteer to be randomized for either a new treatment, placebo or existing treatment in these large and expensive studies. This isn’t an easy process. It can be bewildering for patients to wade through incomprehensible medical jargon in order to understand what they’re signing up for. Then they have to undergo frequent testing — which includes traveling frequently to the hospital, and undergoing blood tests, brain scans, biopsies, and other procedures, some of which can be time-consuming, painful, or both. They also have to adhere to a strict set of rules while they take part.

All the while, patients don’t know if they’re on a treatment that might help or harm them, or whether they are in fact being systematically fooled by a placebo. Patients have misgivings about how they perceive the experience will be, too: 22% of cancer patients in a survey reported they thought they would be “treated like a guinea pig” in a clinical trial.

So not surprisingly, only 5% of patients take part in clinical trials. While the trials cost millions of dollars, 1 in 5 enroll no patients whatsoever, and half miss their targeted number of participants.

How has PatientsLikeMe solved this problem?

We’ve developed the first rapid, robust, representative system that lets patients have a voice in the design of study protocols. RCTs have been designed by doctors and scientists ever since the mid-20th century — when they first started being used widely – with input from regulators (like the FDA) and the business interests of pharmaceutical company sponsors, but with none from patients themselves. We share patient data from PatientsLikeMe with people designing RCTs to identify exactly the type of patients that might be eligible for a study, and get their input and feedback on every aspect of trial design.

We address everything from the language used to describe the study (e.g., “What does this word mean? Patients would probably say X instead of Y”) to the outcome measures used (e.g., “This outcome measure doesn’t really sound like something that’s important to me. Why aren’t you measuring pain?”), and aspects of the study design that are a “deal-breaker” for patients (“If I have to travel more than 20 miles to come to the study center, I’d refuse to take part”).

How will this help those who design clinical trials, and, more importantly, patients?

By listening to patients in advance, study designers can be proactive rather than reactive. They’ll more likely be able to minimize the burden on patients, and they’re far more likely to recruit enough patients to take part in their study. They’ll also glimpse the types of problems their study is likely to encounter, leaving them better prepared to shift gears or make changes if necessary.

This all seems very sensible. Why has no one ever done it before? 

A lot of people in industry are afraid of listening to patients, worried that there might be legal or regulatory barriers to speaking with them. We’ve also lacked ways to quickly gather systematic quantified and qualitative feedback from a large number of patients. That’s something patient-powered research networks like PatientsLikeMe have only recently enabled.

There have been some smaller-scale attempts to get patient input, but these have tended to be within academia, and still aren’t widely used.

What’s your ultimate goal? 

We’re trying to make it so fast, simple, and downright useful to gather this type of data that we’ll move from the status quo (almost nobody uses quantitative patient research in the design of trials) to it being the norm in the next five years.

It’s all a part of our mission to put the patient voice in the decision-making heart of healthcare — and as you can tell, I think it’s pretty exciting! This year, there will be hundreds of clinical trials that fail to meet their targets because of off-putting study protocols that place too much burden or confusion on patients. By bringing a patient focus into the system we could streamline research, increase recruitment rates, and maybe even get faster cures. Just by listening.

To find out more about Paul Wicks and PatientsLikeMe, visit the TED Blog >>>

A snapshot of recent survey results, based on 1,600 patients' views. Image: PatientsLikeMe

A snapshot of recent survey results, based on 1,600 patients’ views. Image: PatientsLikeMe






The Rare Genomics Institute celebrates World Rare Disease Day 2014

Last Friday was World Rare Disease Day – an event launched in 2008 to galvanize public awareness and research momentum for rare diseases. In the United States, a disease is considered rare if it affects fewer than 200,000 people. Yet there are more than 7,000 known rare diseases. This ratio means that there’s little funding for rare-disease research, so even getting a diagnosis can be a years-long odyssey, never mind treatment. Rare disease patients — the majority of them children — too often fall through the cracks.

“World Rare Disease Day unites all the thousands of rare diseases to speak with one voice and raise attention to this often overlooked sector of health research,” says Jimmy Lin, a TED Fellow and the founder of the Rare Genomics Institute, which serves the needs of patients suffering from rare diseases.

To mark World Rare Disease Day, the RGI has released a free ebook — Diagnosing Rare Diseases: Giving Families Hope Through DNA Testing, Crowdfunding, and Access to Experts — for families of those living with rare diseases. Written by Ana Sanfilippo and Jimmy Lin, the 150-page resource features interviews with the first-ever patients to be diagnosed using genomic sequencing, inspiring stories of those whose lives have been saved, advice for parents, as well as conversations with respected rare-disease physicians and genomics experts.

“We hear so many amazing stories of heroic parents fighting for their rare disease children and the amazing scientific results from this work that we think it will inspire many families in the trenches,” says Lin. “Plus, we get a lot of questions about the science and thought an information guide may be helpful.”

RGI helps individual patients with by giving them access to state-of-the-art genomics sequencing technology (see video, above). It offers a crowdsourced platform to raise funding for research, pairs patients with doctors and researchers, and helps families tap into the support of other affected families. To learn more about the Rare Genomics Institute, read the TED Blog’s conversation with Lin.

Illuminating an illness without end: Fellows Friday with Jennifer Brea


Three years ago, Jennifer Brea, then a PhD student in political science, was struck down by what appeared to be a severe flu. It turned out to be the beginning of a long illness — including neurological dysfunction and extreme exhaustion — that she has yet to recover from. Discovering that the medical community did not recognize her illness and worse, dismissed it as hysteria, Brea did her own research and discovered that there is a name for what she was experiencing: myalgic encephalomyelitis (ME), a devastating, misunderstood and ignored disease affecting millions.

To call attention to the plight of those suffering from ME, Brea is making a film, titled Canary in a Coal Mine, to offer firsthand insight into what it feels like to live with this debilitating disease. Its Kickstarter campaign, launched just days ago, has already almost met its target, a clear indication that the time for this film has come. Here, Brea tells the TED Blog her story.

When you did you become ill?

I first became ill in 2010. I had 104.7-degree fever that lasted for 10 days. A year later, in 2011, I was at a restaurant with some friends. The check came, and I couldn’t sign my name. I would look at the paper, and I couldn’t move my hand. I found I could no longer draw curves or circles. Over the course of the next day, I went in and out of periods where I would be totally lucid, I could understand everything anyone was saying to me, but became completely unable to think in any language. It was a little like being a dog. I could understand speech, and I could conceive of things in general impressions, and pictures, but there was no monologue in my head.

That sounds terrifying.

I think in some ways it was actually a blessing, because I could not be afraid of what was happening to me. Something I’ve learned is that in order to experience fear, you have to be able to project the future, and for that you need language. You need the future tense. You might even need the past tense. I was living in the present, and that was all that there was.

My neurologist told me I was suffering from conversion disorder: my illness was caused by stress or a psychological trauma that I might not even recall. Although my physical symptoms were real, there was no organic basis of my illness. It was all very Freud.

This doesn’t speak very well for doctors.

Since starting the film, I’ve become much more sympathetic to doctors, because it’s really not their fault. It’s a product of the training they have received. They’re taught to cure the world’s known diseases, and my disease is not taught in medical school. So if they can’t put me in a box, they can’t treat me.

That said, I was shocked. It was the first time I had ever been not taken at my word, had ever been condescended to like that. I started to wonder, well, if I — with my personality and education and privilege — am being treated that way by all of these doctors, what the hell is happening to people all around the country? What would happen if I were walking into a public clinic, or if I’m in a rural area? And what if I’d been someone who hadn’t had the education to feel like I can challenge people who are in positions of authority? This is one of the reasons I wanted to make this film.

Brea and her husband Omar. Still image from Canary in a Coal Mine.Brea and her husband Omar. Still image from Canary in a Coal Mine.

What were you doing while all this was happening?

Throughout this experience, I was doing tons of research. I was getting my PhD in political science, but I was also doing a master’s in statistics, and so had some training to know how to read and understand research. My husband and I were just constantly reading. We’d go to the doctors with stacks of journal articles, and they would say, “Where did you find that, on the internet?” I’d say, “Yes I did. Here’s an article from Nature, here’s an article from Science, here’s an article from the New England Journal of Medicine.” I had one doctor actually make a guttural sound when he tossed the articles on the floor. I mean, I was really naïve — I didn’t realize that most doctors don’t read medical journals. It’s just not the way that system works. Even one researcher — who was a fantastic doctor — was not reading anything on my illness that was being done by people in other fields. She was an infectious disease doctor, but she wasn’t reading what’s happening in immunology, or people who were looking at gastrointestinal stuff.

What’s the difference between chronic fatigue syndrome and ME?

Chronic fatigue syndrome was a name created by a CDC committee in 1988 in response to a series of outbreaks, most notably the Incline Village, Nevada, outbreak of 1984. It was really unfortunate, not just because the name sucks, but because one, there was already an internationally recognized name for the disease — myalgic encephalomyelitis — and two, the definition they came up with said nothing about some of the most severe neurological and autonomic dysfunction many of us experience. So some of our most devastating symptoms are said to be impossible because they are not in the definition.

The horrid name and the exclusion of those symptoms means that many people diagnosed with CFS probably don’t have ME. A lot of people want to lump chronic fatigue syndrome, fibromyalgia and so on into one group. I think we need to study people who are patently self-similar in terms of their histories and symptoms and stop saying “It’s all very murky” when the murkiness is man-made.

That said, I do, at the end of the day, think that strict diagnostic criteria are useful for clinical trials but harmful for treatment. Our bodies don’t obey medicine’s boundaries, and I don’t think many diseases do either. For example, I’ve learned through my own research that what is probably happening to me right now, physiologically, has a lot in common with certain features of multiple sclerosis, HIV, diabetes and certain genetic mitochondrial diseases. Now, who would ever think those literatures should be speaking to each other? But they should be.

To read the full interview, visit the TED Blog >>>