Rare disease research presents challenges that extend beyond scientific complexity. Small and geographically dispersed patient populations, delays in diagnosis, limited disease knowledge and the need for meaningful outcomes can all influence how research is designed and conducted. Understanding the experiences and priorities of patients and families can help identify research gaps and shape more meaningful, practical approaches.
This panel discussion brings together leaders representing Creutzfeldt-Jakob disease (CJD), Pompe disease, CDKL5 deficiency disorder (CDD) and fibrodysplasia ossificans progressiva (FOP) communities to explore shared challenges and disease-specific needs that influence research priorities and patient participation.
Panelists will explore how patient advocacy organizations can identify unmet needs, connect researchers with affected communities and bring patient and caregiver perspectives into research earlier. The discussion will also highlight opportunities for stronger collaboration across advocacy, research, healthcare and industry to improve research readiness, patient engagement and study design.
Attendees will gain insight into:
How research needs and barriers can differ across rare disease communities
How patient and caregiver perspectives can help identify research priorities and unmet needs
How advocacy organizations can support research readiness and connect researchers with rare disease communities
How patient input can inform study feasibility, participation and meaningful outcomes
How collaboration across advocacy, research, healthcare and industry can strengthen rare disease research
This session offers a unique opportunity to hear directly from leaders representing four rare disease communities and understand how advocacy perspectives can help shape research that better reflects the needs and realities of patients and families.
Register for this webinar to learn how patient advocacy and community insights can help identify research gaps, strengthen research strategies and support more patient-centered approaches to rare disease research.
Who Should Attend
This session is designed for professionals and stakeholders involved in rare disease research, drug development, clinical research, healthcare and patient advocacy, including:
Rare Disease Nonprofit and Patient Advocacy leaders
Clinical Research and Clinical Development professionals
Drug Discovery and Development teams
Medical and Scientific Affairs professionals
Patient Engagement and Patient Experience teams
Researchers and Academic Scientists
Healthcare professionals working with rare disease communities
Professionals involved in study design, research strategy and patient recruitment
Industry professionals collaborating with rare disease patient organizations
Register free on Xtalks: https://xtalks.com/webinars/improve-rare-disease-research-through-advocacy-and-patient-input/
