Rare diseases present unique challenges for researchers and companies working towards treatments and cures:
- Small patient populations;
- Hard to diagnose or delayed diagnosis due to lack of education and understanding among medical professionals;
- Poor understanding of the natural history of the disease and its progression without intervention;
- No prior pathways to follow; clinical endpoints that are often unclear; and
- Enrollment and retention challenges.
To address the special needs of those developing treatments for rare diseases, NORD has created a natural histories patient registry platform as part of the IAMRARE Registry Program with extensive input from FDA, NIH, patients, organizations and experts in the field.
NORD’s platform is an easy to use system that allows patients and organizations to inform and shape medical research and translational science for rare diseases by launching high-quality, customized registries to collect the data needed to define the natural progression of their disease – ultimately advancing product development.
What is a Registry?
Modern Powerful Features
- NORD’s Registry Platform utilizes a cutting-edge, cloud-based design that is mobile-friendly, secure and easy-to-use.
- Responsive and adaptive survey design enhances the participant experience and enables natural history data tracking.
- Automated survey reminders encourage long-term participant engagement.
- Role-based permissions allow for flexible project management.
- Data visualization tools provide participants with real-time de-identified data for comparison to other patients.
- Sub-studies encourage multi stakeholder collaboration and minimize community fragmentation.
- Study resources include standardized surveys and access to rare disease Institutional Review Board (IRB) expertise.
About IAMRARE
Additional Resources
NORD has assembled the following resources and links to help patients, organizations, researchers and companies interested in rare disease registries and natural history studies.