Determine Patients’ Needs

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)

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 Rare Patient Advocacy Summit 2016 (link)

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

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)

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

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

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

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