# Section 6: PFDD Meetings Inventory & Timing Analysis

*The FDA's Patient-Focused Drug Development (PFDD) initiative — FDA-led public
meetings (a defined 24-disease series under PDUFA V, 2013–2017) and the ongoing
Externally-Led PFDD (EL-PFDD) program — is the formal mechanism by which patient
experience enters drug development. This section inventories PFDD activity in
each relevant disease area and, per your request, positions each event
**before / during / after** the corresponding program's development. Details in
pfdd_inventory.csv; timing is visualized in the PFDD-overlay figure.*

> **Accuracy note:** the FDA-led PFDD 24-meeting series (2013–2017) did **not**
> include dedicated type 1 diabetes or celiac meetings. Patient input in those
> areas came primarily through externally-led meetings and advocacy channels.
> Where a specific date could not be verified to the day it is marked
> approximate.

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## 6.1 The central timing finding

**No patient-voice event demonstrably *preceded* the program it might have
shaped.** Across every disease area in this landscape, formal patient-experience
input arrived *contemporaneously with or after* the pivotal development work —
never before it. The programs were designed on scientists' and sponsors'
hypotheses about what mattered; patient priorities were, at best, consulted
mid-stream. This is the structural gap an EoE patient-led program can close.

---

## 6.2 Type 1 diabetes (Tzield, GAD-alum)

- **PFDD status:** No dedicated T1D meeting in the FDA-led 24-disease series.
  Patient input entered through **advocacy organizations (JDRF / Breakthrough
  T1D)** and diabetes clinical-outcome-assessment work, not a formal PFDD.
- **Timing vs. program:** Teplizumab's science was set in 2002–2019; the
  advocacy that shaped its *use* (autoantibody-screening infrastructure) matured
  around and after approval (2022). **Patient voice shaped access more than
  design, and did so late.**
- **What patients had said mattered:** hypoglycemia fear, relentless daily
  management burden, and a strong desire for disease-modifying rather than
  purely glycemic therapies — priorities that the delay-of-onset indication
  happens to serve, but that were not the original design driver.

## 6.3 Celiac disease (Nexvax2, TAK-101, KAN-101)

- **PFDD status:** An **externally-led PFDD** for the celiac community
  (~2020, date approximate) plus a mature patient-powered research
  infrastructure (iCureCeliac).
- **Timing vs. program:** This is the sharpest lesson. The celiac EL-PFDD came
  **after Nexvax2 had already failed (2019)** and **during** the
  nanoparticle-platform trials. Had the patient-articulated priorities —
  persistent symptoms despite diet, the specific symptom concepts, the burden of
  vigilance — been formally captured *before* Nexvax2's Phase 2, the endpoint
  and reactogenicity problems that sank it might have been anticipated.
- **Patient priorities recorded:** non-dietary therapy that genuinely relaxes
  the gluten-free-diet burden; symptom concepts that map to what patients feel
  (the nausea/vomiting finding); trust and safety of an antigen-based approach.

## 6.4 Eosinophilic esophagitis — the whitespace

- **PFDD status:** **No dedicated EoE FDA-led or externally-led PFDD meeting was
  identified** as of the data cutoff. Instead, EoE patient voice is codified in
  two other ways: (i) **FDA's EoE drug-development guidance**, which mandates a
  patient-reported dysphagia **symptom COA as a co-primary endpoint** alongside
  histology; and (ii) **APFED-led advocacy** (Global EoE Position Paper 2025,
  ICD-10 codes, World EoE Day).
- **Timing vs. a pMHC program:** there is no pMHC EoE program yet — so **a
  patient-led EoE pMHC effort could, uniquely in this landscape, put patient
  voice FIRST**, before the trial is designed. The adjacent **HES EL-PFDD
  (~2018, APFED)** is the closest existing template and a natural partner.

## 6.5 Multiple sclerosis (ATX-MS-1467)

- **PFDD status:** MS patient input via community/advocacy channels (~2017,
  approximate); ATX-MS-1467's Phase 2 read out in 2018 — approximately
  contemporaneous. The program stopped before patient priorities could
  meaningfully iterate its design.

---

## 6.6 Implication for the EoE program

The timing analysis converts directly into a strategic recommendation: **because
EoE has no dedicated PFDD and no pMHC program, an EoE pMHC vaccine effort has the
rare opportunity to invert the field's historical sequence — capturing patient
needs, interest, and concerns *before* designing the trial.** The follow-on
patient survey (Section 10) is the first instrument of that inversion, and
partnering with APFED/CURED to convene an EoE (or eosinophilic-GI) EL-PFDD would
formalize it.