Determine Patients’ Needs

Overview

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).

Resources

Patient Registries

From Molecules to Medicine: How Patients Can Their Voices Throughout The Drug Development Process Section 5 and 6 Global Genes (link)

Rare Disease Registries: Advancing Disease Understanding, Treatments, and Cures Global Genes (link)

List of Registries National Institutes of Health Clinical Center (link)

Natural History Study

FAQ Orphan Products Natural History Grants Program U. S. Food and Drug Administration (FDA) (link)

Find Funding Opportunities through Office of Orphan Products and Development Development (OOPD) U. S. Food and Drug Administration (FDA) (link)

OOPD Grant Application Overview U. S. Food and Drug Administration (FDA) (link)

Natural History Clinical Trials Global Genes (link)

Building a Registry

Rare Diseases Registry Program (RaDaR) National Center for Advancing Translational Sciences (NCATS) (link)

PFDD Initiative

The Voice of the Patient: A Series of Reports from FDA's Patient-Focused Drug Development Initiative U. S. Food and Drug Administration (FDA) (link)

FDA-led Patient-Focused Drug Development (PFDD) Public Meetings U. S. Food and Drug Administration (FDA) (link)

Implementation Manual: How to Operationalize the National Health Council's Patient Information Tool National Health Council (link)

Patient-Focused Drug Development Meetings: Smart Practices from Community Leaders FasterCures (link)

External Resources or Information Related to Patients’ Experience U. S. Food and Drug Administration (FDA) (link)

Planning an Externally-Led PFDD

Guidelines for Developing a Letter of Intent (LOI) for Externally-Led Patient Focused Drug Development Meetings U. S. Food and Drug Administration (FDA) (link)

FDA-led Patient-Focused Drug Development (PFDD) Public Meetings U. S. Food and Drug Administration (FDA) (link)

FDA’s CDER Patient-Focused Drug Development Program Staff U. S. Food and Drug Administration (FDA) (link)

Externally-led Patient-Focused Drug Development Meetings U. S. Food and Drug Administration (FDA) (link)

Patient-Focused Drug Development: Collecting Comprehensive and Representative Input Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders U. S. Food and Drug Administration (FDA) (link)