Compassionate Use

What Is Compassionate Use?

Compassionate use is delivering an investigational agent (drug, device or biologic) to treat a serious condition or disease. Investigational agents do not yet have Food and Drug Administration (FDA) approval.

Compassionate use is not research. Rather, it is a way of treating someone who might be eligible for clinical trials but can’t participate for some reason. It is one part of Aurora Health Care’s expanded access program. Expanded access also includes emergency use.

How Does Expanded Access Approval Work?

Expanded access requires advance IRB review and approval. It also requires FDA approval before treatment can proceed.

The FDA grants expanded access approval for compassionate use only when the treatment is unlikely to cause greater harm than the disease or condition. However, the FDA approves 99 percent of individual expanded access requests.

For compassionate use of medications:

• The FDA must approve the request.
• The drug manufacturer must agree to provide the medication.
• The manufacturer may charge for the medication. Insurance companies usually do not pay for investigational drugs. Sometimes, they also exclude related medical costs from coverage.

The FDA website also includes extensive information about expanded access.

Aurora & FDA Policies for Compassionate Use

Our guidance and policies can help you understand how Aurora’s human subject research program reviews and approves compassionate use of drugs, devices or biologics.

Authorization for compassionate use requires approval from our IRB and the FDA. It also requires patient consent for treatment. Learn how to initiate your request for IRB review of compassionate use by reading:

• Aurora SOPs: See our standard operating procedures to understand how our IRB reviews compassionate use requests:
  • SOP 1 – Initial Submission Requirements & Processes [PDF]
  • SOP 2 – Review of Initial Submission [PDF]
• FDA guidance: The FDA provides guidance on compassionate use to patients, physicians, IRBs, clinical investigators and FDA staff. Learn more about expanded access from the FDA.

Compassionate use requires continuing review. The RSPP Office will notify you when you need to complete your review. Remember to complete a Final Report [DOCX] when you complete the use and patient monitoring.