The Rare Epilepsy Network (REN) initiative is created by and for patients with catastrophic rare epilepsies. The goal is to build a patient-centered and driven data base designed to provide patients and their families an opportunity to participate in research that will improve lives and quality of care for people with rare epilepsies. Our PPRN is led by the Epilepsy Foundation, a patient advocacy organization dedicated to the welfare of the almost 3 million people with epilepsy living in the United States.
Each of seven partnering organizations is represented by a caregiver who has a loved one with the disorder (Figure 1). They are joined by two co-investigators/epidemiologists, Drs. Hesdorffer (Columbia University) and Kroner (RTI International), and a consultant neurologist Dr. French (NYU). Columbia University will design the clinical database and manage the data and harmonization. RTI will host the database and serve as the genetics and biospecimen repository. Dr. French, director of the Epilepsy Clinical Trials Consortium, will assign epilepsy seizure type and syndrome.
The key considerations for our patient network governance include policy creation, standards development, outreach, and member engagement. The inclusion of the leaders of the partnering organizations as members of the Steering Committee ensures that caregivers and individual organization members are involved directly or indirectly in all aspects of decision making on data elements, policy formulation, research questions, data sharing, dissemination and social participation, responsiveness of patients, safeguards of privacy, and sustainability.
The REN will use Consilience Software’s Maven database. All data will reside on secure network servers in two RTI data centers. RTI’s networks are fully compliant with FISMA data security standards and are protected by industry-standard firewalls that require data to be encrypted in transit thereby protecting submitted data.
There are currently 857 patients in the network and we expect it to increase to 1500 in 18 months. Each organization has identified specific recruitment strategies including web-based and face-to-face recruitment. Where possible, we will use NIH common data elements (CDE) for epilepsy and other data elements for caregiver-reported patient outcomes. At baseline, the epilepsy CDEs will be used for demographics, seizure type, and adaptive behavior. Additionally, we will collect other data such as assessment of intellectual function and comorbidities. Follow-up information will be collected every six months.
To sustain the REN, we will apply for future funding through public and private sources. The Epilepsy Foundatoin is prepared to help sustain the REN database through their own fund-raising efforts and to make the database available to all rare epilepsy organizations in order to foster research. We believe that the REN database represents a strong mechanism to support our cause.