Did you know?
With more than 400 million people worldwide living with a rare disease, and 10,000 rare diseases known to exist today, rare diseases are not as rare as we may think.1
rare diseases that we know of
Our Impact In Rare
Living with Rare Disease
Patient-Focused Innovators
Cultivating deep connections to integrate their insights and lived experience into everything we do.
Co-creating Community Solutions
Understanding the challenges of a rare diagnosis and the journey that follows is critical to the work we do. That's why we partner so closely with patients and caregivers to ensure it's their stories and experiences that inform our research and development.
Our partnerships with the rare disease community are essential to addressing the unique burdens facing the rare disease community. Together we co-create solutions and amplify awareness, while shaping policies and ecosystems needed to advance access to innovation.
Rare Disease by the Numbers
References.1-5
on average, and often more, to get an accurate rare disease diagnosis
patients diagnosed with a rare disease is a child
of rare diseases are genetic
of rare diseases do not have an approved treatment
Striving for Equity in Rare
People living with rare diseases can face significant challenges like lack of treatment options and access to care.
These challenges weigh even more heavily on racial and ethnic minorities and marginalized groups, leading to economic hardship, difficulty accessing care, and poorer outcomes for both patients and caregivers.6-8
References
- Rare disease facts [Internet]. 2024 [cited 2024 Jan 17]. Available from: https://globalgenes.org/rare-disease-facts
- Fermaglich LJ, et al. A comprehensive study of the rare diseases and conditions targeted by orphan drug designations and approvals over the forty years of the Orphan Drug Act. Orphanet J Rare Dis. 2023;18(1):163.
- U.S. Department of Health & Human Services. About GARD. Available here. Accessed December 2024.
- National Organization for Rare Disorders. Barriers to rare disease diagnosis, care and treatment in the US: a 30-year comparative analysis. Available here. Accessed December 2024.
- National Human Genome Research Institute. Rare genetic diseases. Available here. Accessed December 2024.
- EveryLife Foundation for Rare Diseases. Challenges to diversity in rare diseases. Available here. Accessed December 2024.
- Rare Disease Diversity Coalition. Inequities in the rare disease community. Available here. Accessed December 2024.
- Office of the Assistant Secretary for Planning and Evaluation. Participant diversity by race, ethnicity, and sex in rare disease clinical trials: a case study of eight rare cancers. Available here. Accessed December 2024.
Veeva ID: GL/ALL/0039