Need to Reality

One of the key moments of the April FDA meeting on drug development for ME/CFS was when Bernard Munos said that ME/CFS patients will have to collect and pool their data to attract the interest of big pharma. Many advocates were dismayed by that comment. How can we collect our data? We have no resources. We’re too sick!

Enter PCORI. The Patient Centered Outcomes Research Institute was created by the Affordable Care Act, and is a non-profit organization funded by the federal government. Their budget is huge: $320 million for FY 2013 alone. And they fund research, lots of it. In May 2013, PCORI issued a funding announcement for patient-powered research networks and it could not have come at a better time. Up to $1 million per project is available to help fund data infrastructure to collect and pool electronic data from patients in order to facilitate clinical outcomes research, which is exactly what Munos told us was needed.

Two different ME/CFS projects were proposed, one led by the CFIDS Association and one led by the Open Medicine Institute. In full disclosure, I was one of many advocates consulted by the CFIDS Association in preparing their Letter of Intent, but I have no formal affiliation with the project. Both projects were supported by multiple patient organizations. Neither the CFIDS Association nor OMI were willing to tell me on the record if they were invited to submit a full proposal.

Both organizations already have the beginnings of patient centered research networks. The CFIDS Association has a nascent research network through the SolveCFS BioBank. For the PCORI proposal, the CFIDS Association went big and partnered with Patients Like Me, one of the largest patient-powered research networks in the country. Patients Like Me has almost 10,000 patients in the ME/CFS community on its site, and has the big data expertise to create systems to capture both clinical data/electronic health records and patient reported outcomes. OMI has been building its own online patient data system. They recently received a grant to build a “new personalized medicine infrastructure.”

PCORI has set aggressive goals for the projects it funds under this opportunity. During the 18 month period of grant, funded projects must achieve the following: Membership must reach 0.5% of the US population with the condition; patient-reported data must be collected from at least 80% of the participating cohort, patients must be fully involved in network governance, and data must be standardized and suitable for sharing with other infrastructure members.

These patient-powered research networks are not just big databases sitting in server rooms. Patients themselves must be in control of the data and governance of the network. This would be a first in the ME/CFS community: none of the existing biobanks and databases are governed by patients. Traditionally, the non-profits and researchers have determined the features of these databases and how they will be used. But PCORI is requiring direct governance by the network participants themselves. This will be a unique challenge for either proposal. Getting back to the advocates’ reaction to Munos, we’re too sick! So few of us have the capacity to serve on steering committees and boards of directors, and those that have capacity are already involved (often in multiple contexts). A proposal from the Association or OMI will have to think beyond traditional forms of governance in order to collect the input and opinions of the network membership.

Another challenge will be privacy and confidentiality. Patients Like Me pools anonymized data and sells it to partners, and OMI is a for-profit health practice. Participants in either network should pay attention to the consent agreements and understand exactly what will be done with their data. PCORI networks will collect more than just demographic data. Other collected data will include patient-reported outcomes instruments and electronic health records. Managing and protecting the privacy of participants will be one of the challenges for network governance.

So what’s next? Full proposals for PCORI funding are due September 27, 2013 and funding would begin in January 2014. Let’s hope that at least one of the ME/CFS proposals is successful. One million dollars would go a long way towards building what Munos said we needed: a patient-powered network of data that can be shared with researchers and form the basis of treatment trials and biomarker identification. If there is any disease cohort that needs this kind of jumpstart funding, it is the ME/CFS community.

Update August 13, 2013: The CFIDS Association announced on its Facebook page that it has been invited to submit a full application. Dr. Suzanne Vernon said in an email to me, “The CFIDS Association partnered with 10 organizations and were invited to submit a full application to compete for $12 million that will fund 18 patient-powered research networks.  Our full application is due the end of September and organizations are notified of awards in December.  I am hopeful we will have a very competitive application!”