Needed: Standardized outcome measures for patient-generated data

By: Jonah Comstock | Feb 25, 2013        

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patientslikemeIn order to conduct meaningful clinical trials, it’s important to have solid metrics for tracking disease trajectories and patient experiences. Developing these patient outcome measures is the goal of PatientsLikeMe’s new open-participation research platform, funded by a $1.9 million grant from the Robert Wood Johnson Foundation’s Pioneer Portfolio.

PatientsLikeMe President and co-founder Ben Heywood said that having good patient outcome measures, and seeing them as widely used as possible, is a huge boon for disease research.

“We’re not measuring pain in 45 different ways unless we need to,” he said. “One of the goals we have is a unified medical framework to think about that disease more broadly.”

In other words, the more studies use the same scale, the easier it is to compare their outcomes. At the same time, Heywood believes some diseases don’t have a good existing measurement system for patient-generated data. He said PatientsLikeMe discovered this early on in developing its online patient community, in which patients share data with each other about their diseases. He cited an example from their multiple sclerosis community.

“When we started our MS community, we looked at the way the disease is measured broadly. The general consensus among the research and scientific community was that those measures were inadequate, outdated, and not very good,” he said. “So we built a rating scale, that we then deployed on the PatientsLikeMe platform. Over time we had thousands and thousands of patients measuring their symptoms. We did a lot of work, making sure it was validated internally, and then we validated it clinically outside the system. And it became a much better scale.”

Now, with the new open access system, any researcher who wants to develop a scale can have access to the PatientsLikeMe disease community in order to test it out, as well as to a number of development tools built into the platform. In return, PatientsLikeMe asks researchers to share the scales they develop, making them available for users of the site and people conducting offline clinical trials. If someone uses the scale and adapts it or improves upon it, they’re asked to report those changes back to the PatientsLikeMe database as well.

Heywood hopes the platform will trigger a shift in how patient outcome measures are developed and used. He said currently even when good scales are developed, the system doesn’t always work well.

“One of the big problems with outcome measures today is once they get validated, they never get improved upon. The reality is our understanding of disease, and patients needs, evolve over time, and our cultural understanding of questions evolves over time,” he said. “This system is going to greatly lower the barrier in improving those steps.”

In general, PatientsLikeMe has a philosophy of open access to research. Paul Wicks, the director of research and development at PatientsLikeMe, who announced the new platform in his TED talk today, wrote a blog post on the subject last year. The company has published 25 peer-reviewed studies, many of which they’ve paid the publishers to open up to the public.

“It’s about allowing the people who were engaged in the research, ultimately the patients, to have access to it. We really think data should be open and available, so people can validate and understand the raw data and anyone can learn from the work of everyone else,” said Heywood. “Morally, if patients are engaged in doing this research, they should have access to the results. It’s similar in an open platform, having everyone see it and have access to it and understand it will evolve the science much more rapidly.”

At present, the grant allows PatientsLikeMe to offer the platform free of charge. However, PatientsLikeMe, a for-profit company, might charge for access in the future.