This is a ready-to-use filing-readiness checklist for a marketing application (NDA under FD&C Act 505 / 21 CFR 314, or BLA under PHS Act 351 / 21 CFR 601). The purpose is to cross the 60-day filing review without a Refuse to File and to be ready for the inspections that gate approval. Work each item to Yes, N/A with rationale, or a documented gap. Replace every <<FILL: ...>> placeholder. A filled specimen follows.
Checklist control
| Field | Entry |
|---|---|
| Checklist number | <<FILL: CHK-ID>> |
| Application type | NDA 505(b)(1) / NDA 505(b)(2) / BLA 351(a) |
| Product / candidate | <<FILL>> |
| Target submission date | <<FILL>> |
| PDUFA goal date (once assigned) | <<FILL>> |
| Completed by | <<FILL>> |
Module 1: Regional / administrative
| # | Item | Status (Y / N / NA) | Location |
|---|---|---|---|
| 1.1 | Application form (356h) complete and signed | <<FILL>> | <<FILL>> |
| 1.2 | Proposed labeling (prescribing information) in required format | <<FILL>> | <<FILL>> |
| 1.3 | REMS proposal or rationale for none | <<FILL>> | <<FILL>> |
| 1.4 | Financial disclosure (Part 54) for clinical investigators | <<FILL>> | <<FILL>> |
| 1.5 | Patent and exclusivity information (NDA) | <<FILL>> | <<FILL>> |
| 1.6 | Pediatric plan (PREA) / rare-disease considerations addressed | <<FILL>> | <<FILL>> |
| 1.7 | User fee paid or waiver requested (PDUFA) | <<FILL>> | <<FILL>> |
Module 2: Summaries
| # | Item | Status | Location |
|---|---|---|---|
| 2.1 | Quality Overall Summary (QOS) complete and consistent with Module 3 | <<FILL>> | <<FILL>> |
| 2.2 | Nonclinical overview and summaries | <<FILL>> | <<FILL>> |
| 2.3 | Clinical overview and summaries, including integrated summaries of safety and efficacy | <<FILL>> | <<FILL>> |
Module 3: Quality (CMC)
| # | Item | Status | Location |
|---|---|---|---|
| 3.1 | Drug substance: manufacture, control, characterization, container-closure | <<FILL>> | <<FILL>> |
| 3.2 | Drug product: composition, manufacture, control, container-closure | <<FILL>> | <<FILL>> |
| 3.3 | Analytical method validation reports complete | <<FILL>> | <<FILL>> |
| 3.4 | Process validation (or process performance qualification) summary | <<FILL>> | <<FILL>> |
| 3.5 | Stability data supporting the proposed shelf life and storage statement | <<FILL>> | <<FILL>> |
| 3.6 | (BLA) potency assay, comparability, impurity profile, viral safety, cell-bank characterization | <<FILL>> | <<FILL>> |
| 3.7 | Comparability between clinical and commercial material demonstrated | <<FILL>> | <<FILL>> |
Module 4: Nonclinical study reports
| # | Item | Status | Location |
|---|---|---|---|
| 4.1 | Pharmacology, PK/ADME, and toxicology study reports complete | <<FILL>> | <<FILL>> |
| 4.2 | GLP compliance statements present | <<FILL>> | <<FILL>> |
Module 5: Clinical study reports
| # | Item | Status | Location |
|---|---|---|---|
| 5.1 | Pivotal-trial clinical study reports (ICH E3) complete | <<FILL>> | <<FILL>> |
| 5.2 | Datasets in required standard (CDISC SDTM/ADaM) and define files | <<FILL>> | <<FILL>> |
| 5.3 | Database lock and statistical analysis plan finalized before unblinding | <<FILL>> | <<FILL>> |
| 5.4 | Integrated safety and efficacy summaries reconcile with study reports | <<FILL>> | <<FILL>> |
| 5.5 | Case report form and source-data traceability intact | <<FILL>> | <<FILL>> |
Inspection readiness (gates the approval)
| # | Item | Status | Location |
|---|---|---|---|
| 6.1 | Pre-Approval Inspection (PAI) readiness: named site(s), process validated, state of control demonstrable | <<FILL>> | <<FILL>> |
| 6.2 | BIMO readiness: pivotal-site data verifiable, source documents retrievable, audit trails intact | <<FILL>> | <<FILL>> |
| 6.3 | Data-integrity self-check across the modules (no unreviewed audit trails, no unreconciled queries) | <<FILL>> | <<FILL>> |
| 6.4 | Mock inspection completed with findings closed | <<FILL>> | <<FILL>> |
Sign-off
| Role | Name | Signature | Date |
|---|---|---|---|
| Regulatory lead | <<FILL>> | ||
| CMC / quality lead | <<FILL>> | ||
| Clinical / biostatistics lead | <<FILL>> | ||
| QA | <<FILL>> |
Filled specimen
Illustrative extract for a BLA 351(a).
| # | Item | Status | Location |
|---|---|---|---|
| 2.1 | QOS complete, reconciled to m3 | Y | m2/2.3, cross-check log QC-2026-071 |
| 3.6 | Potency relative-bioassay validated; HCP and viral-safety packages complete | Y | m3/3.2.S and 3.2.A |
| 3.7 | Comparability clinical vs commercial process demonstrated | Y | m3/3.2.S.2.6 comparability report CMP-2026-004 |
| 5.3 | Database locked 30 Apr 2026, SAP final 12 Apr 2026 (pre-unblinding) | Y | DM lock memo DM-2026-019 |
| 6.2 | BIMO readiness: two pivotal sites pre-audited, source retrievable | Y | GCP audit reports AUD-2026-030/031 |
Items 3.7 and 6.2 are the classic delay points: a comparability gap between clinical and commercial biologic material, or a BIMO data-integrity finding at a pivotal site, can hold an otherwise approvable BLA.
Common Refuse-to-File and delay triggers this checklist prevents
- Missing or mis-formatted labeling, forms, or integrated summaries (administrative RTF).
- Module 3 gaps: incomplete method or process validation, stability that does not support the claimed shelf life.
- For biologics, comparability between clinical and commercial process not demonstrated.
- Database lock or SAP finalized after unblinding, undermining the statistical evidence.
- Data-integrity findings surfacing at PAI or BIMO that were never self-identified.
How to adapt this checklist
- Set the application type and candidate in the control block.
- Trim Module 3 rows to NDA or expand for BLA-specific biologics content.
- Point each location cell to the actual eCTD leaf.
- Run the inspection-readiness section against a real mock inspection, not on paper.
- Confirm the current regulations and ICH references before relying on them; this is educational guidance, not regulatory advice.