RARE Advocacy Exchange Session 5 : Interacting with FDA: PFDD and Listening Sessions

June 10, 2025

If your rare disease is likely to have a clinical trial for a drug or treatment come before the FDA in the next few years, a key tool in your advocacy efforts for your patient community can be a listening session or an Externally-led Patient-Focused Drug Development (EL PFDD) session with the FDA. This can feel like a daunting undertaking. You should anticipate spending nearly one year to plan for your EL PFDD session. But watching the session below and the recordings of the EL PFDD sessions that other rare disease groups have held (links below) will make the path ahead easier to manage.

Engaging with the FDA requires a blend of policy knowledge, strategic timing, and strong collaboration. This session provides a roadmap for organizing Patient-Focused Drug Development (PFDD) and listening sessions, offering insights into maximizing ROI and structuring discussions. Participants will leave with practical tips, a guide for speakers, and the confidence to effectively advocate for their communities.

This session of the RARE Advocacy Exchange from Thursday, June 5, 2025 includes:

Moderator: Charlene Son Rigby, Rare mom (STXBP1); CEO of Global Genes

And panelists:
Robyn Bent, Director of the Patient-Focused Drug Development (PFDD) Program in the Center for Drug Evaluation and Research (CDER), FDA
Larry Bauer, Regulatory Drug Expert, Hyman, Phelps & McNamara, P.C.
Dorothea Lantz, Rare mom PWS, Prader-Willi Syndrome Association | USA

Key Themes

Regulatory engagement (like PFDD sessions) is a critical tool as new therapies are being considered for rare patients.
The difference between listening sessions (shorter, private) and EL PFDDs (longer, public
Patient and caregiver voices are important at every stage of drug development: from when first starting to plan the development of a new treatment, during the planning and conduct of clinical trials, as well as through every step of the regulatory process with the FDA.
Patient and caregiver voices can have a very large impact on the pathway to developing new treatments.
When it comes to rare diseases, progress doesn’t happen by accident. It happens because families, advocates, and organizations stand up and speak up – together

Highlights

1. Growing Importance of PFDD: PFDD continues to gain traction as an essential tool to integrate patient perspectives into drug development and regulatory processes.
2. Shift Toward Externally-Led Models: While the FDA still hosts PFDD meetings, patient groups now increasingly lead them—over 100 ELPFDDs to date.
3. Strategic Use of Patient Voice: Voice of the Patient (VoP) reports are widely used not only by FDA reviewers, but also by industry and researchers to inform drug development.
4. Emphasis on Inclusivity: Multilingual access, hybrid formats, and incorporating nonverbal or developmentally delayed individuals and siblings are critical to equity in patient engagement.
5. Early and Ongoing Engagement: A strong emphasis was placed on engaging the FDA—and industry—early and often, not just once.

“We learned so much [from a PFDD Meeting] —things we thought were one-
off experiences turned out to be shared across the community.”
— Robin Bent, FDA

Key Takeaways

1. Components of PFDD Success:

Start early (12+ months lead time).
Submit a Letter of Intent.
Invest in community preparation and panelist coaching.
Use multimedia tools (e.g., QR codes, polls, graphics, word clouds to augment stories).
Ensure diverse patient and caregiver representation—including nonverbal individuals and siblings.

2. Regulatory and Research Impact:

Voice of the Patient (VoP) reports are viewed as scientific resources.
Developers and regulators actively use them to design protocols and select endpoints.
They offer long-term utility, posted on the FDA’s website and referenced years later.

3. Downstream Benefits:

PFDDs can unlock industry interest, drive trial design, and mobilize community voices—e.g., in the Prader-Willi case, it contributed to the first approved hyperphagia treatment.
Adult and sibling engagement increased dramatically post-PFDD.

“Some FDA reviewers keep the Voice of the Patient report [from a PFDD
meeting]on their desks.”
— Larry Bauer, Hyman, Phelps & McNamara

Recommended Next Steps for Advocacy Groups

1. Determine Readiness:

Assess where your community is: learning, planning, or ready to execute.
Evaluate timing, disease understanding, and availability of clinical research partners.

2. Leverage Tools & Resources

Review previous VoP reports and PFDD examples.
Use FDA’s PFDD website, Patient Affairs resources, and stakeholder engagement teams.

3. Plan Strategically:

Create a planning team (consider hiring a consultant).
Build in ample time for prep and practice with speakers.
Fundraise early—engage sponsors, foundations, and industry.

4. Consider Alternative or Supplemental Options:

Patient Listening Sessions (easier lift).
Scientific Workshops with FDA.
Publish white papers if a full PFDD isn’t feasible.

5. Document & Share:

Record sessions and generate a strong VoP report.
Consider publication in peer-reviewed journals for broader scientific impact.
Use visuals (graphic recordings, story submissions, polling results) to deepen the narrative.

“That [the PFDD] meeting gave us hope—and more than that—it gave our
loved ones a voice they’d never had before.”
— Dorothea Lance, PWSA

Related Resources:

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