Improve Rare Disease Research Through Advocacy and Patient Input
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About this webinar
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
- 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
What you will learn
- 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
Featured Speakers
Teresa Wong
More information coming soon.
Alice Anane
More information coming soon.
Michelle Davis
More information coming soon.
Heather Shorten
More information coming soon.
Partners for this event
Xtalks Life Sciences United (XLSU)
Xtalks Life Sciences United (XLSU) is a free, global alliance developed to connect life science organizations in a virtual and collaborative environment. Through XLSU, member associations, societies, advocacy groups and research networks come together to share success stories, tackle common challenges and build meaningful relationships.
CDKL5 CANADA
CDKL5 Canada is a non-profit patient advocacy group for the rare CDKL5 disorder. It provides community support and funding for affected families.
Ceutzfeldt-Jakob Foundation Israel
Ceutzfeldt-Jakob Foundation Israel is a nonprofit organization working to accelerate research and find a cure for genetic CJD, a rare and fatal brain disease. We lead national efforts in sample collection through Israel’s first gCJD biobank, foster international collaborations, and advocate for patients and families by providing information, guidance, and support.
International Fibrodysplasia Ossificans Progressiva (FOP) Association (IFOPA)
The IFOPA supports and connects the global fibrodysplasia ossificans progressiva (FOP) community while driving research toward treatments and a cure.
Pompe Alliance
Here at Pompe Alliance, we see the value in everyone. We want to be a catalyst for positive change, and since our beginnings in 2018, we’ve been driven by the same ideas we initially founded our Public Services Organization upon: support, empowerment, and progress. Our mission is “To provide supportive services, education and information to patients, caregivers, medical professionals and community stakeholders.