Expanded Access Programs

To be considered for expanded access, all the following must be in place:

• You have a serious or life-threatening condition.
• There are no approved therapies available that meet your needs.
• You cannot take part in a clinical trial either because you do not qualify or no suitable trial is available.
• The potential benefits outweigh the risks of using the investigational treatment.
• Providing the treatment will not interfere with any ongoing clinical trials.

Expanded access requires several people and groups to work together:

• The patient and licensed physician decide if the treatment is appropriate and the physician agrees to oversee the treatment.
• The company developing the product provides the investigational treatment and supports any necessary documentation.
• An Institutional Review Board (IRB) reviews the request to protect the patient and ensure informed consent.
• The U.S. Food and Drug Administration (FDA) reviews and provides authorization.

To see if an EAP is right for you, follow these steps:

Step 1 Talk with Your Healthcare Provider

Your licensed physician will assess whether expanded access is appropriate for your condition.

Step 2 Physician and Manufacturer Involvement

Your physician must be willing to oversee your treatment. The manufacturer of the therapy must agree to provide the investigational product.

Step 3 Review by an Institutional Review Board (IRB)

An IRB is a group of people who review research studies to help make sure they are safe and fair for patients. For most expanded access requests, an IRB reviews the treatment plan and consent form to help protect your safety and rights.

Step 4 FDA Review and Authorization

The FDA reviews the request to decide if the expanded access use can move forward.

Starting a conversation about expanded access can feel overwhelming, but your healthcare provider is there to help you understand all your options. These tips can help you prepare.

Questions You May Want to Ask

• Could expanded access be an option for my condition?
• Are there any clinical trials I might qualify for?
• What are the possible risks and benefits of trying an investigational product?
• What would my care plan look like if I move forward?
• What other treatments should I consider?

Information to Bring to Your Appointment

• A list of all treatments you have already tried and how you responded
• Any recent test results and/or medical records
• Your current symptoms and how they affect your daily life
• A full list of medications, including any over the counter medications and supplements you use
• Questions or concerns you want to discuss

Most of the time, yes. Investigational treatments are rarely covered by insurance, so the cost of the treatment and oversight is usually an out-of-pocket cost for the patient. In some situations, the manufacturer may decide to cover the cost of the investigational product. Because there may be a cost, it is important for you to talk with your physician to understand any potential costs related to receiving the investigational product, as well as any medical services that will be associated with receiving it.