Patients’ Online Data

August 25, 2009
Research Trove: Patients’ Online Data
By SARAH ARNQUIST

CAMBRIDGE, Mass. — After Amy Farber learned she had the rare and fatal disease called LAM in 2005, she became determined to increase and speed up research into her illness with the hope of finding a cure in her lifetime.

Dr. Farber, now 39, was a law student with a doctorate in anthropology who was about to start a family. She quit law school and founded the LAM Treatment Alliance to raise money and connect a network of scientists around the world to research this mysterious disease, which destroys young women’s lungs.

To her dismay, she says, she encountered a cumbersome research system fraught with obstacles to collaboration and progress — one that failed to focus on patient needs.

“We can do better,” she remembers thinking.

She took her frustrations to Dr. George Demetri, a member of her organization’s advisory board. A professor and cancer researcher at Harvard Medical School, Dr. Demetri had long wanted to use the Internet to connect patients around the globe and mine their collective wisdom for new insights into the rare cancers he studies.

That led her to Frank Moss, director of the Massachusetts Institute of Technology Media Laboratory, and a new collaboration between her group and the Media Lab: LAMsight, a Web site that allows patients to report information about their health, then turns those reports into databases that can be mined for observations about the disease.

Since the Internet’s earliest days, patients have used the Web to share experiences and learn about diseases and treatments. But now advocates like Dr. Farber say that online communities have the potential to transform medical research — especially into rare diseases like hers that lack the number of patients needed for large-scale studies and rarely attract research financing from the drug industry. Also, she said, it empowers patients to contribute, ask questions and help lead the way to discoveries.

“Patients have been a tremendously underutilized resource,” she said.

Mr. Moss, who came to the laboratory in 2005 from the drug industry, agrees. Patients’ everyday experiences in living with an illness are an enormous source of untapped data, he said; aggregated, those data could generate new hypotheses and avenues for research. “We’re really turning patients into scientists and changing the balance of power between clinicians and scientists and patients,” he said.

Scientists and entrepreneurs are increasingly exploring ways to tap that potential, and not just for rare diseases.

Several private companies are now collecting patient data and genetic information online to use in recruiting patients for clinical trials, conducting research internally or to sell to drug and biotechnology companies.

Supporters of this model — sometimes called crowd-sourcing or open-source research — call it democratization of research and say they are pioneering new models that put patients in control of their data and build bridges between researchers, patients and their doctors. They say these methods are far cheaper and faster than traditional research, which has high start-up costs and relies heavily on clinicians.

Still, some experts are skeptical. Questions abound about how these sites will guarantee patient privacy; whether patients fully understand what it means to share their medical information online; whether private businesses should have to follow the same strict patient protection rules that govern most researchers; and the quality problems of user-generated data.

The Web offers great potential to reach large numbers of patients quickly, but self-reported data creates considerable research dilemmas, said Dr. James Potash, an associate professor of psychiatry at Johns Hopkins School of Medicine.

Dr. Potash cited two studies that examined the quality of information reported online by depressed patients. Patients reported their diagnosis online; doctors then interviewed them to confirm it. In one study, only two-thirds of the online responses were validated; in the other study, it was three-fourths.

Those numbers are not good enough for high-quality research, Dr. Potash said. Without the ability to ensure a correct diagnosis and accurate patient information, usually gained in face-to-face interviews, researchers can end up with a “garbage in, garbage out” problem, he said.

“Faster is only better if the work is done well,” he said. “You don’t want to speed the train up and have it fly off the tracks.”

Dr. Demetri, of Harvard, acknowledges the challenges of using data self-reported online. “We all are sensitive to the fact that we are making the rules up as we go along,” he said. “I worry about going back to observations of low quality and low power, and I want to be careful that we avoid misleading observations.”

No one expects that observational research using online patient data will replace experimental controlled trials, said Ian Eslick, the M.I.T. doctoral student developing the LAMsight project.

The data generated by the project will be used mainly for exploratory analysis and hypothesis generation, Mr. Eslick said, although he added that the online approach could eventually yield new models for conducting experimental research.

“There’s an idea that data collected from a clinic is good and data collected from patients is bad,” he said. “Different data is effective at different purposes, and different data can lead to different kinds of error.”

In June, the Belgian pharmaceutical company UCB announced a partnership to build an online epilepsy community with PatientsLikeMe, among the first private companies to develop a platform for data sharing by patients. PatientsLikeMe, based in Cambridge, has as members tens of thousands of patients who contribute detailed information about their diseases, drugs, doses and side effects.

Mark McDade, UCB’s chief operating officer, said the regulatory approval process should be changed to incorporate not just safety and efficacy but also measurements on how drugs affect patients’ lives — data that is now slow and expensive to collect.

Genetic companies have also taken up patient-driven research. The Silicon Valley company 23andMe, for example, started a program this summer called “Research Revolution.” People can buy a stripped-down version of 23andMe’s genetic service, which gives people DNA information on ancestry and risk for certain diseases, for $99 and then contribute their genetic data toward research into the disease of their choice.

The company plans to store the genetic profiles of thousands of people to use for research internally and in partnerships with other companies. “We call it research 2.0,” said Linda Avey, a founder of 23andMe. “It’s the Wikipedia approach versus Encyclopaedia Britannica approach.”

Such databases could be a valuable resource for researchers needing to recruit huge numbers of patients quickly, said Dr. Robert Cooke-Deegan, director of the Center for Genome Ethics, Law & Policy at Duke University’s Institute from Genome Sciences and Policy.

But private companies like 23andMe and PatientsLikeMe are not bound by the same patient protection rules that govern traditional medical researchers who receive federal financing. Company leaders say they have detailed patient privacy statements and ethics policies.

As these companies evolve, however, Dr. Cooke-Deegan said he expected them to have to deal with more issues of privacy and informed consent, since maintaining patient trust is crucial to their success.

Ben Heywood, co-founder and president of PatientsLikeMe, said his company’s business model was built on trust.

“We are only successful if our patients are engaged and using the site,” Mr. Heywood said. “If we break their trust, we lose our community and we have nothing.”

Dr. Cooke-Deegan said the model was so new that its implications had yet to be thought through. “I’m very suspicious of a company that has tons of private data getting too cozy with the drug or biotech industry,” he said. “But I don’t want to say it’s not going to work, because I can see all kinds of value that could come out of this.”

Dr. Farber hopes her Web site will become the world’s largest database of active LAM patients. More than 100 registered users on five continents are using the site, which has no advertising, she said.

LAM, short for lymphangioleiomyomatosis, kills by slowly destroying the lungs. Breathing problems have not yet impeded Ms. Farber’s push for new research, but she says each healthy day with her husband and young daughter is a blessing.