Compassionate Use (Expanded Access) of Test Articles
Expanded access, sometimes called "compassionate use," is the use of an investigational drug/ biologic outside of a clinical trial to treat a patient with a serious or immediately life-threatening disease or condition who has no comparable or satisfactory alternative treatment options.
The compassionate use provision allows access for patients who do not meet the requirements for inclusion in the clinical investigation but for whom the treating physician believes the device may provide a benefit in treating and/or diagnosing their disease or condition.
FDA also recognizes that there are circumstances in which an investigational device is the only option available for a patient faced with a serious, albeit not life-threatening, disease or condition. In these circumstances, FDA uses its regulatory discretion in determining whether such use of an investigational device should occur.
Additional Information About Compassionate Use of Drugs/ Biologics
FDA regulations allow access to investigational drugs for treatment purposes on a case-by-case basis for an individual patient, or for intermediate-size groups of patients with similar treatment needs who otherwise do not qualify to participate in a clinical trial. They also permit expanded access for large groups of patients who do not have other treatment options available, once more is known about the safety and potential effectiveness of a drug from ongoing or completed clinical trials.
In order for a patient to gain access to an investigational drug outside of a clinical trial, the patient must have a serious or immediately life-threatening disease or condition and no comparable or satisfactory therapeutic alternatives. Additionally, the drug manufacturer and the patient’s doctor must make special arrangements to obtain the drug for the patient. These arrangements must be authorized by the FDA. These safeguards are in place to avoid exposing patients to unnecessary risks. Note: The manufacturer of the drug is not required by regulations to provide the drug outside of a clinical trial.
Additional Information About Compassionate Use of Devices
Prior FDA approval is needed before compassionate use occurs. In order to obtain Agency approval, the sponsor should submit an IDE supplement requesting approval for a compassionate use under section §812.35(a) in order to treat the patient.
The physician should not treat the patient identified in the supplement until FDA approves use of the device under the proposed circumstances. In reviewing this type of request, FDA will consider the above information as well as whether the preliminary evidence of safety and effectiveness justifies such use and whether such use would interfere with the conduct of a clinical trial to support marketing approval. Note: The manufacturer of the device is not required by regulations to provide the device outside of a clinical trial.
After the compassionate use occurs: The responsible physician will devise an appropriate schedule for monitoring the patient, taking into consideration the limited information available regarding the potential risks and benefits of the device and the specific needs of the patient.
IRB Review and Guidance
When a drug/ device is investigational, federal law requires that its use be reviewed by an Institutional Review Board (IRB) to protect the individuals receiving the drug, including assuring that, in general, the risks are reasonable in light of the potential benefit. The IRB will require and review an informed consent document to ensure that patients are aware of potential risks and are willing to accept the level of possible risk associated with the drug.
UT Southwestern IRB requires submission of a compassionate use of a test article application using eIRB application. Please see study submission instructions on the Electronic Application (eIRB).
Please use “Compassionate Use Consent Form” template found in IRB Forms.
Note: Because the use of a “compassionate use” drug/ device is for diagnosis or treatment purposes only, the HIPAA regulations for research are not applicable. Therefore, a HIPAA Research Authorization and/or Waiver of Authorization are not required.
The FDA has also released an educational module titled “Institutional Review Board: Compassionate and Emergency Use” specific to the use of unapproved devices.
References and Additional Guidance
- U.S. Food and Drug Administration (FDA): 21 CFR 812.36
- FDA Guidance: Access to Investigational Drugs Outside of a Clinical Trial (Expanded Access)
- FDA Guidance: IDE Early/Expanded Access
- FDA Presentation: “Institutional Review Board: Compassionate and Emergency Use”