Expanded Access (Compassionate Use)
Expanded access (also called "compassionate use") allows patients with serious or life-threatening conditions to access investigational medical devices outside of a clinical trial when no comparable or satisfactory alternative therapy is available.
For medical devices, expanded access is governed by 21 CFR Part 812 and FD&C Act § 520(g).
When expanded access applies
Expanded access for a device is appropriate when:
- A patient has a serious or life-threatening condition
- No comparable or satisfactory alternative therapy is available (no cleared/approved device for that indication)
- The patient cannot participate in a clinical trial (e.g., no trial is open, the patient does not meet trial criteria)
- The treating physician believes the potential benefit justifies the risk
Two types of device expanded access
1. Emergency access (individual patient — emergency)
When there is no time to file a formal IDE or expanded access request, a physician may request emergency access from FDA by phone or other rapid means:
- Contact the appropriate CDRH Division
- FDA may authorise use verbally with follow-up written submission
- IRB notification required as soon as possible
2. Non-emergency access (individual patient — non-emergency)
For a non-emergency individual patient access request:
- Treating physician submits request to FDA (can be informal)
- Manufacturer concurrence required
- IRB review and approval required (or concurrence under expedited review)
- FDA typically responds within a few days to weeks
3. Expanded access IDE (treatment use)
When multiple patients need access, a Treatment IDE (21 CFR § 812.36) may be appropriate:
- Applies when the device is under active investigation or awaiting FDA review
- Requires a formal IDE submission or supplement
- Used to make a promising device widely available before final approval
Expanded access vs IDE clinical trial
| Feature | Expanded Access | IDE Clinical Trial |
|---|---|---|
| Purpose | Provide individual patient access to therapy | Generate safety/effectiveness data |
| Data collection | Required but secondary | Primary objective |
| Number of patients | One or a few | Defined trial population |
| IRB | Required | Required |
| FDA submission | Simplified request | Full IDE application |