- University of Michigan (lead site)
- Duke University
- University of Rochester
- University of Texas Southwestern
Frequently Asked Questions
Expanded Access is a program in which the United State Food and Drug Administration (FDA) allows a qualified physician to access investigational medical products for patients in need. By “investigational” this means that the drug, device or biologic is NOT already cleared or approved by the FDA and therefore is not generally available for purchase.
In order for patients to receive treatment under Expanded Access, the manufacturer must agree to provide access to the investigational product and the following criteria must be met:
(1) The patient must have a serious or immediately life-threatening disease or condition with no comparable or satisfactory alternative to diagnose, monitor, or treat the disease or condition;
(2) The potential patient benefit must justify the potential risks of the treatment use and the potential risks cannot be unreasonable in the context of the disease or condition to be treated; and
(3) Providing the investigational drug for the requested use cannot interfere with the initiation, conduct, or completion of clinical investigations that could support marketing approval of the Expanded Access use or otherwise compromise the potential development of the Expanded Access use.
To qualify for Expanded Access, a patient must have a serious condition, have exhausted standard of care therapies, and be ineligible for a clinical trial. A product must also be available that has a reasonable chance to benefit the patient, and the risks and possible benefits of the use must be appropriate.
In addition to these standards, doctors and patients should discuss:
- In the best case scenario that the therapy works as intended, how much benefit would the patient expect to receive? Some therapies might be curative, while others may only help manage symptoms or extend time to disease progression for a little while.
- In the worst case scenario, what side effects might the patient expect? Could they be serious or even deadly?
- What are the patient’s goals for care? An investigational therapy may have side effects which could impact quality of life. Other therapies may be available not to treat the disease, but to improve quality of life and manage symptoms.
- How long could it take for the patient to start therapy? Some manufacturers do not allow for emergency requests, and some take a significant amount of time to review requests and get contracts in order. For non-emergency cases, the FDA can take up to 30 days to review the request, although it is unusual for review to take that long.
- What would treatment with the drug look like? Would it require hospitalization, or frequent visits to a clinic or lab?
The therapeutic is not currently on the market in the United States so there is no way for the treating physician to purchase the product to supply it to the patient for the treatment of their illness or condition.
The purpose of traditional research is to learn more about the safety and effectiveness of the therapeutic. The primary purpose of Expanded Access is to treat a patient’s disease rather than to gain information about the safety and effectiveness of the medical product. FDA must approve the use of investigational products under Expanded Access, and federal regulations require oversight by an institutional review board (IRB). This is not always that much different from traditional research, and in many cases they do follow similar paths. However, the final goal of Expanded Access is treatment of a patient’s serious or life-threatening condition.
An informed consent explains what is known and what is unknown about the investigational drug. It helps ensure the patient understands the nature of the investigational medical product for the proposed treatment. An Informed Consent document must be reviewed by an Institutional Review Board (IRB) before being provided to the patient for them to sign.
A consultation between a physician and patient (and/or caregiver team) is the first step in determining whether it is appropriate or not to seek out use of an investigational product for treatment.
There are several ways to find out if a drug is available:
- Contact the manufacturer. Per the 21st Century Cures Act, manufacturers are required to post their Expanded Access policy and contact information online.
- Use the Company Directory on the Expanded Access Navigator, maintained by the Reagan Udall Foundation for the FDA.
- Look through entries in the www.ClinicalTrials.Gov database. Companies with Expanded Access programs for specific products will often post these on ClinicalTrials.Gov. Search for the drug name under “Other terms.” Results can be limited to Expanded Access programs through the “Status” or “Study Type” filters. These listings will contain the requirements for Expanded Access as well as contact information for the program.
Below are several groups that may be involved obtaining Expanded Access to an investigational product:
- Food and Drug Administration (FDA)
- Institutional Review Board (IRB)
- Treating physician who agrees to serve as the sponsor
- Company who is supplying the investigational product
- Regulatory Affairs Office
- Contracts and/or legal department at the treating physician’s hospital
- Pharmacy to assist with preparing and dispensing the investigational product
Expanded Access can be requested through your physician who will file the paperwork with the FDA.
There is no way to be sure an investigational drug or device will help you. These therapies are, by definition, still being studied, and doctors have limited experience with their use.
In order to request a drug or device through Expanded Access, your doctor must have a reason to believe that it might work, usually because of how it is thought to work (called a “mechanism of action.”) However, these therapies may not always work properly or work enough for a patient to benefit. Keep in mind that, less than 10% of drugs that enter clinical trials are eventually found to provide enough benefit to enough patients to be approved. Even in late stage (phase 3) trials, only about half of drugs go on to be approved.
If you are a patient who has a serious condition, has exhausted all treatment options, and is not eligible for clinical trials, you may consider discussing drugs available through Expanded Access with your doctor.
Not all doctors are familiar with Expanded Access, the process or the evolving requirements. You may want to use resources like the FDA webpage for Expanded Access or this FAQ as resources to help with this discussion.
Keep in mind, not all doctors are comfortable requesting Expanded Access, and Expanded Access is not right for all patients. This should be a discussion between you and your doctor to determine what is best for your situation.
In some cases, you may be charged for a drug or device provided under Expanded Access, but in many cases it will be provided at no cost. When a manufacturer does charge, the cost is tightly restricted and must be approved by the FDA.
You or your insurance company may be charged for the other costs of care associated with your use of the investigational product, including clinic visits, hospitalizations, infusions and injections, or lab monitoring. You may also be responsible for any costs incurred if you have a bad reaction to the therapy.
An Emergency Use Authorization or EUA is a process by which the FDA allows an unapproved product to be used for serious or life-threatening conditions when there are no adequate, approved, and available alternatives. These emergency authorizations are typically used for conditions associated with chemical, biological, radiological, nuclear (CBRN) threats, or emerging infectious diseases. The unapproved product is “considered approved” only for the length of time that the EUA stays valid.
Emergency Use Authorization is a process that allows a medical product to be temporarily brought onto the market like an approved product. It can be bought or sold just like any other approved medical therapeutic. However this approval only lasts as long as the public health emergency for which it has been specifically declared to be used for.
While an EUA gives a medical product a temporary “authorization” to be bought and sold on the market in the US, Expanded Access applies to investigational products that are NOT approved by the FDA. Under Expanded Access, the product is still considered investigational and IRB oversight is required for treatment of the patient. Once an EUA has been given for a product, it can be used like an approved drug, e.g. an IRB does not need to be involved. The product can be used with the accompanying Patient and Health Care Provider information Sheets.
An Emergency Use Authorization is not the same as an Emergency Use request which can be processed through the Expanded Access pathway.
Clinical trials are divided up according to how much research has already been conducted on the investigational drug.
- Phase 1 studies are early, small-scale studies and mostly focus on determining the safety (and safe dosages) of the product in humans.
- Phase 2 studies increase the number of people involved and look at both efficacy of the drug and continue to look at side effects.
- Phase 3 studies enroll many more participants and continue to evaluate efficacy while also monitoring frequency of side effects.
Right to Try is legislation passed in May of 2018 that creates a secondary route for patients to gain access to investigational medical products. Right to Try is a parallel pathway that coexists with FDA’s Expanded Access program. Right to Try does not guarantee that a therapeutic will be available for the patient, it does not follow the FDA pathway, nor require regulatory oversight. For more on Right to Try, please refer to the additional resource: https://www.fda.gov/patients/learn-about-expanded-access-and-other-treatment-options/right-try