# Unapproved Use Dossier — editorial working template

> **For drafting only.** This original worksheet aligns to the C section family in AMCP Format 5.0 without reproducing AMCP text. It is not an AMCP form. Use it only for a use of an FDA-approved product for which FDA approval is being sought, and obtain internal legal/regulatory/compliance review before communication.

## Dossier control

| Field | Complete |
|---|---|
| Approved product / sponsor | |
| Existing approved indication(s) and label version | |
| Specific unapproved use seeking FDA approval | |
| Development / regulatory milestone status | |
| Draft version / evidence cutoff / owner | |
| Intended HCDM audience / communication pathway | |
| Link to common instructions / evidence trace / change log | |

**Scope rule:** Keep this dossier limited to the unapproved use(s) seeking FDA approval. Present factual, objective evidence. Do not characterize or conclude on safety or effectiveness for the unapproved use.

## 1.0C — Highlights and overview
- Approved product context (clearly distinct from the unapproved use):
- Proposed unapproved use and development status:
- Population, disease context, and anticipated care pathway:
- Key factual clinical/economic information:
- Projected price/access information, basis, and uncertainty:
- Unapproved-use limitation:
- Linked evidence IDs:

## 2.0C — Product information and disease description
### 2.1C Product information
| Topic | Current approved-product information | Proposed-use information (factual / status labeled) | Evidence ID |
|---|---|---|---|
| Product / class / mechanism | | | |
| Approved use(s) / label context | | | |
| Unapproved use under review | | | |
| Administration / site of care | | | |
| Development program / milestones | | | |

### 2.2C Disease description
| Topic | Target population / unmet need | Evidence ID |
|---|---|---|
| Epidemiology / burden | | |
| Current treatment pathway | | |
| Comparator landscape | | |
| Equity / access considerations | | |

## 3.0C — Clinical evidence
| Evidence ID | Study / status | Population / comparator | Endpoint / result | Limitation / maturity |
|---|---|---|---|---|

- Completed, ongoing, and planned studies (separate and status-labeled):
- RWE relevance and limitations:
- ITC/NMA record: review provenance, network, assumptions, certainty, and claim boundary:
- What evidence is unavailable:

## 4.0C — Economic value and modeling report

Include cost-effectiveness or budget-impact models only when available and suitable for preapproval communication. Such models may be infeasible to construct or communicate before approval because they depend on assumptions about unapproved safety or effectiveness. If unavailable or unsuitable, record that status and the reason; do not manufacture a result. Model inclusion requires the applicable legal, regulatory, and compliance review.

| Topic | Input / result | Approved/current vs projected/pending status | Uncertainty / scenario | Evidence ID |
|---|---|---|---|---|
| Disease / treatment cost context | | | | |
| Cost-effectiveness / value model | | | | |
| Budget-impact / uptake assumptions | | | | |
| Model validation and technical documentation | | | | |

## 5.0C — Additional supporting evidence
- Clinical guidelines, HTAs, and systematic reviews:
- Other outcomes/economic evidence:
- PRO, patient-experience, health-disparity, and quality-measure evidence:
- Information held unavailable and reason:

## 6.0C — Dossier appendices
- [ ] References and evidence tables
- [ ] Study summaries / protocols / SAPs as permitted
- [ ] Economic-model documentation and input provenance
- [ ] Current product information, clearly distinct from proposed-use material
- [ ] Change log and distribution record

## Lifecycle action
- On FDA approval of the use: preserve this version, update the Approved Product Dossier, and retire this C dossier for the use (or revise a multi-use C dossier).
- If the use remains unapproved: update factual evidence/status only through documented change control.

## Final internal check
- [ ] The unapproved use, not the approved product generally, is the subject of each claim.
- [ ] Projected/pending information is separated from approved/current product information.
- [ ] No safety/effectiveness conclusion is made for the unapproved use.
- [ ] The complete dossier was reviewed against official AMCP Format 5.0.