The Dravet syndrome (DS) community, like other rare disease communities, currently lacks the education and training necessary to advance their priorities for research to the point of design, implementation, and dissemination. This project aims to prepare the Dravet syndrome community as a whole, while establishing a best-practice paradigm. By defining and establishing this process, the project team will advance the development of patient-centered outcomes research (PCOR)-readiness in Dravet and other rare disease communities.
To assure engagement of stakeholders across the DS community, the team will embrace a multipronged strategy that fosters inclusion and infrastructure. The Dravet Syndrome Foundation (DSF) recognizes that stakeholder involvement will affect the success of this initiative and the project team plans to include a diverse group of stakeholders that will represent all aspects of the community. DSF has a strong connection to its patient/caregiver community through its support group, parent ambassadors, regional meetings, and biennial conference. The project team is well connected with professional stakeholders through medical and scientific advisory boards as well as the organization’s annual research roundtable. Because of recent clinical trials in the DS community, the project team has established strong working relationships with industry that will allow it to include their perspective in this project. In recognition that some stakeholders will have limitations that may affect their participation, the team will offer remote opportunities, such as educational webinars, to allow for maximum participation.
The initial objectives for this project are:
- Educate and involve more researchers in the PCOR process and facilitate interaction with caregivers and patients
- Educate and engage the broader caregiver community in participation in the PCOR process
- Disseminate the results of these efforts to stakeholder groups. The project team anticipates that by educating and involving a larger portion of the community, additional objectives will arise through this process. The team will do its best to incorporate additional objectives and allow them to evolve within the project as time and resources permit.
This project will produce measurable outcomes through activities that include:
- A cross-syndrome meeting, where researchers with an interest in sodium channel epilepsies will have the opportunity to brainstorm and collaborate, as well as meet patient families
- Sessions at regional workshops (2018 and 2019 Day of Dravet) that provide a local option to learn about PCOR/comparative effectiveness research (CER) and how to participate
- PCOR/CER training webinars in English and Spanish to allow caregivers to participate remotely
- Expansion of DSF’s family network to identify participants for future participation in research
- DSF parent ambassador education to allow them to better serve their local communities
- White papers to summarize the cross-syndrome meeting and the Day of Dravet workshops
- Development of a best-practice paradigm for other rare disease communities
The project team’s approach and lessons learned will be useful in other rare diseases and will allow smaller rare disease communities with fewer resources a comprehensive and systematic paradigm to help them more efficiently build their patient community to the point of PCOR/CER research readiness in a timely manner.