Determine Patients’ Needs


Your group may wish to start a patient registry or support a natural history study for your disease. Patient registries and natural history databases are proving to be invaluable tools for stimulating research. Documenting the experiences of patients and their caregivers provides valuable information for researchers, industry partners, and regulators. The type of data collected will vary depending on the purpose of the registry. These databases are helping engage patients in the therapy development process early, often, and effectively.

In addition, your group may consider organizing an Externally-led Patient-Focused Drug Development Meeting (PFDD). The U.S. Food and Drug Administration (FDA) encourages patient groups to coordinate and run meetings to capture patient perspectives on specific diseases and current treatments using the process established through the FDA’s PFDD Initiative. The format of the meetings was developed through the 24 FDA-led PFDD public meetings. Your patient group can submit a Letter of Intent (LOI) to the FDA and receive assistance to adapt the process for your disease(s).


Natural History Study
FAQ Orphan Products Natural History Grants Program U. S. Food and Drug Administration (FDA) (link)
OOPD Grant Application Overview U. S. Food and Drug Administration (FDA) (link)
Building a Registry
Rare Diseases Registry Program (RaDaR) National Center for Advancing Translational Sciences (NCATS) (link)