US FDA Expanded Access for Medical Devices

Expanded access pathway allows the manufactures or physician to use an investigational medical product to use the patients with a serious or life-threatening disease or condition when there are no available alternative options.

There are three below options are available,

• Emergency Use
• Compassionate Use (or Individual Patient/Small Group Access)
• Treatment Investigational Device Exemption (IDE)

Emergency use

During emergency, investigational device can be used immediately, to treat an individual patient life-threatening situation (there are no alternative options and no time to use existing procedures to get FDA approval for the use).

Physician shall follow the patient protection procedures as possible. Some of the protection procedures are getting concern from patient (Informed consent process), Concurrence from Institutional Review Board (IRB) chairperson and Authorization from the device manufacturer.

Reporting of Emergency use

• Criteria: Devices with IDE    
• Who report: IDE sponsor
• Timelines: within 5 days
• Information to be submitted: IDE Report

• Criteria: Devices without IDE
• Who report: physician 
• Timelines: within 5 days
• Information to be submitted: a description of device used, details of the case, and the patient protection measures that were followed

Compassionate Use (or Individual Patient/Small Group Access)

If there is investigational device is the only option to treat a patient faced with a serious or life-threatening disease or condition.

First device manufacturer shall agree to provide the device under compassionate use.

Approval process for devices with IDE

IDE sponsor should submit an IDE supplement requesting approval for a compassionate use.

IDE supplement contents,

• Patient conditions and circumstances necessitating treatment
• why alternative therapies are unsatisfactory
• deviations from approved clinical protocol to treat the patient
• Patient protection measures

Approval process for devices without IDE

Physician or manufacturer shall submit the compassionate use request to FDA with above information and along with a description of the device provided by the manufacturer

Timelines for review

 IDE supplements have the same statutory 30-day review cycle. Average timelines are within 15 days of receipt.

FDA Actions: Approve, Approve with conditions, or Disapprove the request.

Follow-up Report: Within 45 days, the person who submitted the request shall provide the below details to FDA,

• Patient outcome
• If any problems occurred because of device use

Treatment Investigational Device Exemption (IDE)

If an approved IDE is extended under new IDE to include additional patients with life-threatening or serious diseases. This is called a treatment IDE.

Treatment IDE Application contents are listed below,

1. Name, address, and telephone number of the sponsor
2. Intended use of the device, the criteria for patient selection, and a written protocol describing the treatment use
3. Rationale for use of the device
4. Description of clinical procedures, laboratory tests, or other measures that will be used to evaluate the effects of the device and to minimize risk
5. Procedures for monitoring the treatment use
6. Name and address of the monitor
7. Instructions for use for the device and all other Labeling 
8. Information that is relevant to the safety and effectiveness of the device for the intended treatment use
9. A statement of the sponsor's commitment to meet all applicable responsibilities under the IDE regulations
10. Example of the agreement to be signed by all investigators participating in the treatment IDE and certification that no investigator will be added to the treatment IDE before the agreement is signed
11. Device price (if sold) and a statement indicating that the price is based on manufacturing and handling costs only

FDA actions:Approve, Disapprove or withdrawal of approval of treatment IDEs

Follow-up Report:

• To whom reported: IRB and FDA    
• Who report: Sponsor
• Timelines: Semi-annual basis
• Information to be submitted: Progress reports      

References:

Expanded Access

Expanded Access for Medical Devices