Determine Patients’ Needs

Building a Registry

It is important that the information in your registry database will be helpful to the stakeholders in the therapy development process. You also want to collect the information using the correct methods so that it can be used by medical researchers, industry partners, and the FDA. Because this process is crucial to laying the foundation for the therapy development process, especially for rare and genetic diseases, the National Center for Advancing Translational Sciences (NCATS) established a website devoted to building your registry.

NCATS Rare Diseases Registry Program (RaDaR) includes step by step guidance to help you develop your registry. It is not a registry platform, but rather a collection of best practices, templates, and resources to help you achieve your goal. So if you want to learn more about building a registry or you are ready to begin, we encourage you to explore RaDaR:

Rare Diseases Registry Program (RaDaR)

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)