Patient-Centered Outcomes Research (PCOR) is research that provides patients and their clinicians with evidence-based information in order to help them make more informed health care decisions and allowing them to express what is most important to them. This research is designed to answer questions about the potential benefits and harms of different care options, given a patient’s personal characteristics, conditions, and preferences.
Where We Started
In 2016, we launched a caregiver survey to identify characteristics, measure comorbidities, and prioritize caregiver concerns regarding Dravet syndrome. Over 250 caregivers responded, and we published the results in Epilepsy & Behavior. What we discovered, in addition to clear patient data trends, was that caregivers’ top concerns, aside from seizure control, were: speech/communication, sibling impacts, cognition/developmental delay/regression, and behavioral issues. Each of these concerns lends itself well to patient-centered outcomes research, so we organized a group of stakeholders including caregivers, clinicians, basic scientists, and industry representatives to determine how to design a study that would address these concerns.
Read the paper here.
Our Experience with PCOR
In 2016, DSF was awarded a Pipeline to Proposal (P2P) award from the Patient-Centered Outcomes Research Institute (PCORI) to develop comparative effectiveness research in Dravet syndrome. The purpose of the P2P Awards program is to help people form new collaborations with the goal of developing proposals for research with sound scientific rigor and robust patient engagement.
In Tier I of our P2P project, a stakeholder group (including family members, clinicians, and scientists) was established. The stakeholder group met on a monthly basis to dicuss and understand the primary concerns of the community and to encourage development of collaborative research to identify comparative effectiveness research (CER) goals for people living with Dravet syndrome.
In Tier II of our P2P project, the focus was to further refine the potential CER questions developed in Tier I down to a few strong and viable options with relevance to the Dravet syndrome community. This was accomplished through additional outreach to caregivers and community stakeholders, including focus sessions and surveys. In addition, we developed a communications plan and a stakeholder engagement plan to allow dissemination of the CER progress.
After completing Tier II of our P2P project, we applied for and were awarded a Eugene Washington PCORI Engagement Award. These awards support projects that encourage active integration of patients, caregivers, clinicians, and other healthcare stakeholders as integral members of the patient-centered outcomes research/clinical effectiveness research (PCOR/CER) enterprise. Our project was on expanding patient engagement in the Dravet syndrome community and developing a best practice paradigm