Rare Epilepsy Network

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PCORI funded the development of PCORnet^®, the National Patient-Centered Clinical Research Network, to make it easier and more efficient to conduct research. PCORnet is made up of Partner Networks that harness the power of large amounts of health data and patient partnerships.

Patient-Powered Research Networks (PPRNs) are networks operated and governed by patients, families, caregivers, and community members in close collaboration with clinical researchers, with the goal of shaping and conducting research to better serve their communities and improve their health outcomes. Several PPRNs, including the Rare Epilepsy Network (REN), participated as Partner Networks in PCORnet.

PCORI funded REN’s participation in PCORnet from 2015 to 2018. This report outlines REN’s achievements in building its research infrastructure capacity to

1. Create ways to involve patients, families, and caregivers in decision making about the network
2. Create a way to collect and share data reported by patients for research
3. Involve patients, caregivers, families, and communities in the research process, including recruiting people to participate in research studies
4. Lead or join in research studies that focus on the community’s priorities

Network at a glance

REN is a PPRN that includes people who have one of the more than 40 rare types of epilepsy and their caregivers. Epilepsy is a disease in which abnormal brain activity causes seizures. Many types of epilepsy exist; any type that affects fewer than 200,000 people is considered rare. REN connects patients, caregivers, families, advocates, and researchers to encourage research that improves the diagnosis, treatment, and quality of life of people with rare epilepsies. As of April 2019, the REN network included 1,459 people who were affected by rare epilepsies, including patients and caregivers.

How does the network operate?

REN created online surveys to gather data from adults with rare epilepsies, caregivers of children with rare epilepsies, and caregivers of adults with rare epilepsies who couldn’t complete the surveys on their own. These surveys asked about participants’ age and sex and where they lived; diagnosis and treatment; medical history; and quality of life. Patients or their caregivers could also upload medical reports.

REN set up a steering committee to make network decisions. The committee included a representative from the Epilepsy Foundation, researchers, and one representative from each of 32 rare epilepsy organizations. An executive committee helped foster communication between PCORnet and the steering committee. The executive committee also included researchers, one of whom is a caregiver for a child with a rare epilepsy, and representatives from the Epilepsy Foundation and a rare epilepsy foundation.

How did the network involve patients and other partners?

Patients, families, and caregivers were important contributors to REN. Patients and families were members of the steering committee and helped create the online surveys.

Who is in the network?

REN had enrolled 1,459 participants as of April 1, 2019.

How does the network support future research?

The Epilepsy Foundation will maintain and share REN survey data and continue to support REN, which is part of the Epilepsy Learning Healthcare System, a PCORI-funded project that provides funding and technical assistance to help communities, clinicians, and institutions improve health outcomes through quality improvement and research.

Glossary

Institutional Review Board (IRB): A group that follows federal regulations, state laws, and institutional policy to review, monitor, and approve research in order to protect the ethical rights and privacy of the subjects involved.

Patient-Powered Research Networks (PPRN): PPRNs are operated and governed by patient groups and their partners and are focused on particular conditions or populations.

PCORnet: PCORnet is a network of networks that brings together patients, clinicians, researchers, and healthcare systems to share information and participate in research.

PCORI funded the development of PCORnet^®, the National Patient-Centered Clinical Research Network, to make it easier and more efficient to conduct research. PCORnet is made up of Partner Networks that harness the power of large amounts of health data and patient partnerships.

Patient-Powered Research Networks (PPRNs) are networks operated and governed by patients, families, caregivers, and community members in close collaboration with clinical researchers, with the goal of shaping and conducting research to better serve their communities and improve their health outcomes. Several PPRNs, including the Rare Epilepsy Network (REN), participated as Partner Networks in PCORnet.

PCORI funded REN’s participation in PCORnet from 2015 to 2019. This report outlines REN’s achievements toward the PPRN goals of developing and optimizing research and engagement infrastructure capacity to

• Establish a governance structure that fully engages patients and other stakeholders for the purposes of contributing to research
• Create a data infrastructure conducive to the ongoing collection of patient-reported data
• Meaningfully engage patients, caregivers, families, and community members in the research process, including identifying and recruiting participants for clinical trials and other research studies
• Lead or partner in the conduct of comparative effectiveness studies responsive to the research priorities of their communities

Network Infrastructure

REN is a PPRN made up of people who have one of the more than 40 rare types of epilepsy and their caregivers. The network’s goal is to connect patients, caregivers, families, advocates, and researchers to advance patient-centered research that improves the diagnosis, treatment, and quality of life of people with rare epilepsies. As of April 2019, REN had baseline survey information for 1,459 people affected by rare epilepsies, including patients and caregivers.

Network Governance and Operations

REN created a patient registry as its primary data-gathering mechanism. The network developed an 11-module online survey to collect registry data from cognitively able adults with rare epilepsies, caregivers of children with rare epilepsies, and caregivers of adults with rare epilepsies who were not able to complete the survey on their own.

The Epilepsy Foundation led overall network administration and stakeholder engagement, RTI International led data management, and Columbia University led research analysis and dissemination. A steering committee served as REN’s decision-making body. This committee included a representative from the Epilepsy Foundation, two primary investigators, and one representative from each of the 32 rare epilepsy organizations who are partners in the network. The network also created an executive committee to serve as the liaison between REN and PCORnet. This committee included representatives from the Epilepsy Foundation and an organization focused on rare epilepsies as well as two researchers, one of whom is a caregiver of a child with a rare epilepsy.

Stakeholder Engagement

Patients, caregivers, and families were engaged in all aspects of REN. In particular, patients and families played active roles on the steering committee and helped develop the online survey that REN used to collect registry data.

Demographics and Research Capacity

REN had 1,459 enrolled participants in the patient registry as of April 1, 2019. The online survey used to collect registry data addressed patients’ demographics; seizure history and characteristics; diagnosis; genetic testing; treatment, including medications and surgery; developmental progress; and patient and caregiver quality of life. Participants could also provide electroencephalogram and MRI reports.

Future Opportunities

The Epilepsy Foundation will maintain and share REN baseline data and continue to support REN, which is part of the Epilepsy Learning Healthcare System, a PCORI-funded project that provides funding and technical assistance to help communities, clinicians, and institutions improve health outcomes through quality improvement and research.