The purpose of our project partnership, Developing Comparative Effectiveness Research in Dravet Syndrome, is to further engage community stakeholders in a collaborative partnership to develop patient-centered outcomes research that reflects the care gaps and unique health concerns of patients with Dravet syndrome. Dravet syndrome is more than a severe epilepsy syndrome, affecting many aspects of a patient’s life. It can be difficult to know what to study beyond seizures. Patients face multiple health issues, including difficulty with sleep, gait, digestion, developmental delays, behavioral problems, and risk of sudden death, which together lead to a decreased quality of life.
The focus of our one-year project partnership is to further refine the potential comparative effectiveness research (CER) questions developed in Tier I down to a few that are strong and viable options with relevance to our community. We will accomplish this through additional outreach to caregivers and community stakeholders, including focus sessions and surveys. We will also determine the tools necessary to measure effectiveness in the top candidate categories. In addition, we will develop a communications plan and a stakeholder engagement plan to allow dissemination of the CER progress.
*All proposed projects, including requested budgets and project periods, are approved subject to a programmatic and budget review by PCORI staff and the negotiation of a formal award contract.