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.