Registry to Speed a Cure
Patient registries can be a powerful tool. They can help researchers:
- follow the course of a disease and the effects of therapies by providing real world data
- learn more about how patients are diagnosed and treated and how they respond to treatments
- examine factors that affect outcomes and quality of life
- support research collaboration among investigator
- facilitate multiple avenues of research on a disease
Registries can provide a natural history of a disease(natural history registry), showing when certain things happen to a patient. They can be used to compare people with APBD who are treated with those who are untreated. The aim is to speed up the development of drugs and treatments and to improve the quality of life for those living with APBD.
The APBD patient registry will consist of a secure database to store contact information and basic clinical data on APBD patients. APBD patients can be confident that the registry is maintained in an anonymous and protected way, ensuring confidentiality. Patients who would like to share their stories and learn from one another can also choose to make their contact information available to other patients.
In discussions with NIH's Office of Rare Diseases Research (ORDR) on establishing an APBD patient registry, here is a recap of their thoughts:
- The process lends itself to developing a comfort level with a registry provider that will give the best product
- The selected solution should provide access to de-identified information and the ability to search and analyze the data
- The setup should facilitate the ability to provide de-identified data to the Global Rare Diseases Patient Registry and Data Repository (GRDR) established by NIH. This may result in leading other potential patients to our organization.
- Finalized patient registry questions can be shared with NIH ORDR.
- ORDR has developed an informed consent template that can be used in establishing APBD registry
What your donation will fund:
Annual maintenance fee $15,000
Naming opportunity available