The traditional role of patients in research has been as study subjects rather than as self-advocates and partners, which limits patient priorities as compared to researcher perspectives and introduces a disconnection in effective treatment development. To address this problem, the Children’s Tumor Foundation (CTF) proposes to organize a Neurofibromatosis-specific Patient-Centered Outcomes Research (NF-PCOR) forum in Atlanta, Georgia on May 4-6, 2018. The event will help equip patients, families, and stakeholders with the expertise needed to participate in research. It will include educational sessions on patient-centered outcomes research, comparative effectiveness research, the drug development process, and hands-on training workshops and real-time examples by Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) for participating in research.
The NF-PCOR forum will have three aims:
- Patient training and education
- Patient participation in research
- Shared knowledge through partnership
Through these aims, the project team hopes to teach patients about the value of being involved in research and expand their knowledge base; encourage active participation in REiNS’ and CTF’s research programs, with the long-term objective of engagement with industry, regulatory affairs, and other stakeholders; and provide stakeholders a better understanding of the impact of NF on patients and their families. Thus, effective partnerships can develop through a shared commitment to PCOR.
The conference planning committee will meet bimonthly to discuss topics, invite speakers, draft attendee surveys, and design sessions. Sessions will include presentations with question and answers, group breakout discussions and time for networking, co-learning, and community building. There will be a dedicated workshop on the protection of human subjects and basic research concepts and ask-the-expert sessions. The team will record sessions and make them available on its website. The project team will also record a promotional video with patients and partners about getting involved in research.
- Awareness about PCOR/CER will spread through attendees, conference videos, and a summary report.
- A network of trained patient partners will be established to integrate clinicians and all stakeholders, fostering continuous interactive communication across all groups, and accelerating the research recruiting process.
- Conference presentations will be recorded and produced as online videos for the benefit of disease communities and accessible as self-paced learning webinars. This will include a special video about the REiNS initiative as a tangible way of getting involved in the research process, and will serve as a best practice tool for other disease groups to adapt.
Patient and Stakeholder Engagement Plan: The primary stakeholders are NF patients and their families, caregivers, clinicians, speakers sharing their expertise, and CTF. CTF will facilitate conversations across all stakeholders at the event. An organizing committee of two patients, two clinicians, and two CTF staff members will communicate regularly to plan the agenda, set the learning objectives, and discuss tasks such as website content, marketing, and event promotion. Over 200 participants, researchers, clinicians, and regulators will meet face to face, in group chats, and open discussions. Patients will have the opportunity to have their voices heard in REiNS workshops.
Collaborators include REINS, patients, and other speakers.