This section of the Investigator Manual covers a range of information related to expanded access, including when an expanded access request is appropriate, requirements for drugs/biologics and devices, as well as the steps for submitting an expanded access request.

Expanded Access Overview

Expanded access is a potential pathway allowed by the FDA for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available. Expanded access can involve an individual patient, or a group of patients.

The University of Wisconsin-Madison IRB is committed to using expanded access programs and the procedures for obtaining access to human investigational drugs (including biologics) and medical devices.

Wherever possible, a patient’s use of an investigational medical product as part of a clinical trial is preferable because clinical trials can generate data that may lead to the approval of products and, consequently, to wider availability. However, when a patient cannot be enrolled in a clinical trial (e.g., the patient is not eligible or there are no ongoing clinical trials), the patient may be able to receive the product, when appropriate, through expanded access.

For emergency expanded access requests (i.e. there is not enough time to obtain prospective IRB approval for the use), see Emergency Use (One Patient) Guidance.

When is an expanded access request appropriate?

FDA guidance states that using an investigational medical product under expanded access regulations (including emergency uses) may be appropriate when all the following apply:

  • Patient has a serious disease or condition, or whose life is immediately threatened by their disease or condition.
  • There is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition.
  • Patient enrollment in a clinical trial is not possible.
  • Potential patient benefit justifies the potential risks of treatment.
  • Providing the investigational medical product will not interfere with investigational trials that could support a medical product’s development or marketing approval for the treatment indication.

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Expanded Access for Drugs/Biologics

There are three different types of expanded access for drugs/biologics:

  • Expanded access for individual patients, including for emergency use
  • Expanded access for intermediate-size patient groups
    • For use by more than one patient, but generally fewer patients than are treated under a typical treatment IND or protocol
  • Expanded access for widespread treatment use
    • Access to an investigational drug (including a biologic) for treatment use by a large (widespread) population under a treatment IND. The investigational product must be under active development for marketing.

See HRP-324 for additional information.

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Expanded Access for Devices


There are three different mechanisms for expanded access of an unapproved device:

  • Emergency Use for an individual patient
  • Compassionate Use (or Individual Patient/Small Group Access)
    • Used for devices that are being studied in a clinical trial under an IDE 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 or diagnosing their disease or condition. It can also be used for devices that are not being studied in a clinical investigation. This provision is typically approved for individual patients but may be approved to treat a small group, if the small group request is under an IDE.
  • Treatment Investigational Device Exemption (IDE)
    • An approved IDE specifies the maximum number of clinical sites and the maximum number of human subjects that may be enrolled in the study. During the course of the clinical trial, if the data suggest that the device is effective, then the trial may be expanded under a new IDE to include additional patients with life-threatening or serious diseases. This is called a treatment IDE.

See HRP-325 for additional information.

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Expanded Access Request Process

The following steps should be completed to obtain approval for Expanded Access requests:

Step 1: Contact the Manufacturer

  • For all types of investigational medical products, the FDA requires that the clinician contact the manufacturer to see if they will provide the investigational medical product for expanded access use. Investigational medical products may be provided through a supplement to a manufacturer’s existing IND or IDE, or through an IND or IDE specific to the use (e.g., under a Treatment IND or Treatment IDE).

Step 2: Submit Request to the FDA

Step 3: Obtain IRB approval and informed consent from the patient(s)

  • Once the appropriate expanded access route has been identified and the FDA and drug/biologic/device manufacturer contacted, treating teams will need to submit an application through ARROW for prospective IRB approval, using the Expanded Access Application type. Expanded access requests for the use of a drug for a single patient may be reviewed by the Chair or designee in lieu of convened board review if the study team has requested a waiver from the FDA by checking box 10(b) on FDA Form 3926. Expanded access requests for the use of a device in an individual patient may be reviewed by the Chair or designee in lieu of convened board review.

    Study teams are encouraged to use the expanded access consent template for drugs and devices.

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