Determine Patients’ Needs

Planning an Externally-Led PFDD

The FDA has multiple resources to help your group plan a successful externally-led PFDD. FDA’s Center for Drug Evaluation and Research (CDER ) PFDD Program Staff leads the externally-led PFDD program.

Submit a Letter of Intent (LOI) at least 1 year in advance to the planned meeting date.
- Refer to Guidelines for Developing a Letter of Intent (LOI) for Externally Led Patient Focused Drug Development Meetings
  - These guidelines state the LOI can be approximately 5 pages long, but current information on the externally-led PFDD webpage states approximately 3 pages long.
- LOIs are reviewed 4 times per year by the CDER PFDD Program Staff.
After an LOI is accepted by the FDA, CDER PFDD Program Staff will reach out to you and upon request provide specific recommendations on the planning of the meeting including:
- Development of agenda.
- Discussion/polling questions.
FDA cannot provide a meeting space, facilitator/moderator, or financial assistance.
Previous FDA-led PFDD meetings provide examples of agendas, slides, recordings, transcripts, and reports that can help guide the planning process.
Meeting should not require hiring event planners, consultants, scientific writers, or other external resources on your team, especially when resources may be limited.
If resources are limited, consider web-based/virtual meetings or including as a session or workshop during an already planned event or conference.
Successful meetings include other patient groups and stakeholders in your disease community when possible.
Seeking outside funding, for example, from therapy developers or large umbrella groups, is encouraged.
- Disclose any known funding resources in LOI.
- If established after LOI submission, include in the planning materials and deliverables.
- Therapy developers, whether academic or pharmaceutical, should not be involved in the development of the meeting agenda, discussion/polling questions, or selection of patient panelists.
If you have questions, contact FDA’s CDER Patient-Focused Drug Development Program Staff at patientfocused@fda.hhs.gov
Externally led Patient-Focused Drug Development Meetings , an FDA webpage, provides more information and links to resources in easy to read format and language.
For even more detail, you may consider referring to Patient-Focused Drug Development: Collecting Comprehensive and Representative Input Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders (2018). However, this resource is written in a more formal style and contains high levels of technical language.
- This draft guidance (Guidance 1) is the first of a series of four PFDD FDA guidance documents.
- Guidance 1 answers the questions:
  - Whom do you get input from, and why?
  - How do you collect the information?

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)