Real World Data Research Readiness: Engaging the Prader-Willi Syndrome Community
Background: Real-world data (RWD) is an emerging area of data acquisition and analysis, with the potential to transform clinical research and patient care. Wearable devices, smartphone technology, electronic health records, insurance databases, patient registries, and social media provide an opportunity for patients to partner in developing RWD patient-centered outcomes research (PCOR), including comparative effectiveness research (CER). However, patient partnering in the development of RWD research and tools is lacking. Prader-Willi syndrome (PWS) is an ideal model to pilot RWD strategies for rare diseases. PWS is a complex neurodevelopmental disorder encompassing broad clinical, behavioral, and mental health issues. It presents opportunities for RWD development that are transferable to other rare diseases. These include hypotonia, developmental delays, intellectual disability, excessive appetite, reduced metabolism, obesity, growth hormone deficiency, sleep disorders, scoliosis, autism, anxiety, temper outbursts, self-injurious behavior, and psychosis. However, the PWS community has limited understanding of RWD and its possible applications.
Proposed solution: Educating and involving PWS patient partners in the development of RWD research and tools will strengthen the RWD project design and the impact of research outcomes. Shared lessons learned and outcome resources will benefit the greater rare disease community. A workshop on RWD in patient-centric research is a critical step to ensure that patient and stakeholder partners collaborate to capitalize on RWD to the benefit of those with PWS.
Objectives: This project aims to bring together patient and stakeholder partners in a co-learning environment to: 1) educate patient partners on RWD research and tools; 2) develop prioritized RWD research questions for PWS, including challenges and solutions to implementation; 3) promote dialogue among the PWS community and developers of RWD research, devices, and analytics; 4) prepare PWS patient partners to engage in planning, conducting, and disseminating RWD research; and 5) provide lessons learned and resources to the greater rare disease community on engaging in RWD research.
Activities: An in-person workshop and an interactive web meeting will bring together patient and stakeholder partners to achieve these objectives. Both events will incorporate expert talks in RWD followed by facilitated discussion. Topics include types of RWD, RWD research and analytics, mobile health applications, and wearable devices.
Outcomes/outputs: The outcomes are: an in-person workshop and web-based meeting; video recordings for education in the PWS community and organizations seeking to engage in RWD for other diseases; identification of priority RWD research and tools in PWS; potential challenges and avenues for advancement; a workshop report; a foundation for continued bidirectional information flow between those developing RWD tools for research, and those that are using RWD tools for research.
Engagement plan: Participants include a spectrum of PWS patient and stakeholder partners including patients, families, caregivers, NORD, physicians, researchers, pharmaceutical companies, and companies developing RWD tools, software, and analytics for PWS and other rare diseases. Co-learning will involve presentations by experts on real-world data (RWD), and discussions around RWD research priorities, methods, and feasibility in the PWS community.