Expanded Access Mechanism

Regulatory Authority

Criteria for Use

When Can It Be Used?

Number of Patients to be Treated

FDA Approval Needed?

How is FDA Approval Obtained?

Patient Protection Measures

Emergency Use

"Guidance for the Emergency Use of Unapproved Medical Devices"

50 FR 42866
21 CFR 812.35(a)

1. Life-threatening
condition^‡;
2. No alternative;
and
3. No time to obtain
FDA approval.

Before or after initiation of clinical trial

Limited to few patients

No; submit report to FDA following device use

Not applicable

1. Independent
assessment by
uninvolved doctor;
2. IRB chairperson's
concurrence;
3. Institutional
clearance; and
4. Informed consent

Compassionate Use

21 CFR 812.35(a)

1. Serious disease
or condition and
2. No alternative.

During clinical trial

Individual patient or small groups of patients

Yes

IDE supplement with:
1. Explanation of
circumstances
constituting need for
the device;
2. Reasons alternatives not
acceptable;
3. Deviations from
protocol, if any; and
4. Patient protection
measures.

1. Independent
assessment by
uninvolved doctor;
2. IRB chairperson's
concurrence;
3. Institutional
clearance; and
4. Informed consent.

Treatment IDE

21 CFR 812.36

1. Life-threatening
or serious
disease;
2. No alternative;
3. Controlled
clinical trial; and
4. Sponsor pursuing
marketing
approval.

During clinical trial

Wide access; depends on patient/physician need

Yes

Trt IDE supplement with:
1. Intended Use, protocol,
and patient selection
criteria;
2. Rationale for trt use
3. Methods used to
evaluate device use and
minimize risks;
4. Monitoring plan;
5. Summary of S&E data
6. Instructions for use and
device labeling;
7. Commitment to
patient protection;
8. Investigator agreement; and
9. Price, if will be sold.

1. IRB approval and
2. Informed consent.

Continued Access

"Continued Access to Investigational Devices During PMA Preparation and Review"

ODE Blue Book IDE Memorandum #D96-1

1. Public health
need; or
2. Preliminary
evidence that
device will be
effective and
no significant
safety concerns.

After completion of clinical trial

Same rate of enrollment as study

Yes

IDE supplement with:
1. Justification for
extended study;
2. Summary of S & E
data and risks posed by
the device;
3. Proposed enrollment
rate;
4. Clinical protocol; and
5. Progress towards
marketing approval.

1. IRB approval and
2. Informed consent.