Expanded Access: Information for Patients
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Expanded access, also called “compassionate use,” provides a pathway for patients to gain access to investigational drugs, biologics and medical devices for serious diseases or conditions.
Investigational drugs and devices have not yet been approved by the FDA and they have not been proven to be safe and effective. Therefore, they may be effective in the treatment of a condition, or they may not. It is important to remember that the drug/biologic/medical device may have unexpected serious side effects and that patients need to consider all the possible risks when seeking access to an investigational medical product.
Visit the Center for Devices and Radiological Health to learn more information about expanded access to unapproved medical devices.
On this page, you will find information about expanded access for investigational drugs and biologics.
- What are the Different Types of Expanded Access/Compassionate Use?
- What Does the FDA Consider When Reviewing my Request?
- Am I Protected From Risks?
- If I Meet the Criteria, Will I Qualify for Expanded Access/Compassionate Use to a Drug?
- How to Submit an Expanded Access Application
- What my Physician and I Can Expect After my Physician Submits the Expanded Access Application
- Questions?
Talk with your healthcare provider to see if using an investigational treatment is right for you. Be sure to consider how much is known about the investigational medical product, the severity of your condition, the availability of other therapies, and the likelihood that the therapy will be effective. You and your healthcare provider should consider the kind of illness you have, the stage of disease, and other conditions you may be experiencing, among other factors.
If your healthcare is managed by anyone other than a physician you will need to find a licensed physician who is willing to:
- oversee your treatment,
- work with the manufacturing company and FDA
- obtain the drugs or biologics,
- monitor you during the course of treatment, and
- file the necessary paperwork.
Only a licensed physician is able to apply for expanded access. Form FDA 3926 can be used by physicians when submitting requests for individual patient expanded access to investigational drugs, including emergencies. This form is designed specifically for single patient investigational new drug (IND) requests. It can also be used for submissions to FDA after the initial application is filed. For more information including instructions, please visit the guidance Individual Patient Expanded Access Applications: Form FDA 3926.
It is important to remember that the company that is developing the drugs (including biologics) will need to provide the medicine, and without the company’s cooperation, you will not be able to access the investigational product.
What are the Different Types of Expanded Access/Compassionate Use?
Under FDA’s current regulations for investigational drugs (including biologics), FDA has three categories that expanded access can be approved under. They are:
- Expanded access for individual patients, including for emergency use;
- Expanded access for intermediate-size patient populations; and
- Expanded access for widespread treatment use.
What does the FDA Consider When Reviewing my Request?
When a treating physician requests expanded access to an investigational drug , the FDA must determine if the patient or patients to be treated have a serious or immediately life-threatening disease or condition. The Code of Federal Regulations (21 CFR Part 312.300) details how expanded access can be granted and what steps the FDA needs to follow.
It is important to have a discussion with your physician or healthcare provider to see if your current disease or condition can be defined as either:
- A serious disease/condition: a disease or condition associated with morbidity that has substantial impact on day-to-day functioning. Short-lived and self-limiting morbidity will usually not be sufficient, but the morbidity need not be irreversible, provided it is persistent or recurrent. Whether a disease or condition is serious is a matter of clinical judgment, based on its impact on such factors as survival, day-to-day functioning, or the likelihood that the disease, if left untreated, will progress from a less severe condition to a more serious one; or
- Immediately life threatening: a disease or condition is a stage of disease in which there is reasonable likelihood that death will occur within a matter of months or in which premature death is likely without early treatment.
The FDA must also determine that:
- There is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition.
- The patient cannot obtain the drug under another IND or protocol.
- The potential patient benefit justifies the potential risks of the treatment use and those potential risks are not unreasonable in the context of the disease or condition to be treated.
- Providing the investigational drug will not 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.
Am I Protected From Risks?
When a drug is investigational, Federal law requires that its use be reviewed by an institutional review board (IRB), or ethics committee. The IRB works to protect the people receiving the drug and ensures that the risks are reasonable given the potential benefits. The IRB will require and review an informed consent document to make sure patients are aware of potential risks and are willing to accept the level of potential risk associated with the drug. However, significant unknown risks may exist.
Carefully read the informed consent presented to you and make sure that you understand the risks associated with the drug before signing.
Your physician may or may not have access to an IRB, which is usually associated with a hospital or research institution, but there are independent IRBs as well. Depending on the IRB, a cost may be associated with the review, which may be your responsibility.
If I Meet the Criteria, Will I Qualify for Expanded Access/Compassionate Use to a Drug?
Not necessarily. Even if you meet the criteria, there may still be obstacles.
- Your physician may not be able to obtain expanded access for you because of your medical history or the risks associated with taking an investigational drug. Your physician must determine that the probable risk from the drug is not greater than the probable risk from the disease.
- Your physician may not be willing to manage the use of an investigational drug.
- The company that makes the drug is not required to offer it outside their clinical trials, and they may not be willing or able to do so.
- The company may not have enough of the drug available for all patients requesting expanded access. Some companies establish a lottery system to determine which patients will have treatment access. Others make the decision on a case-by-case basis.
Investigational drugs are expensive to make. The drug company may request authorization from FDA to chargeyou the direct costs of making the drug available, such as manufacturing and shipping, and indirect costs, such as reporting and IRB approval, or it may elect to cover the cost. Remember that most insurance companies will not pay for access to an investigational drug and that there may be additional cost for administering them and then for monitoring of the IND by your hospital or healthcare provider. These costs do not require FDA authorization. It is important that you and your physician consider the cost of the investigational drug and the medical services associated with its use that are not covered by third-party payers such as insurance or Medicare.
How to Submit an Expanded Access Application
It is important to remember that individual patients are not able to apply for expanded access, only a licensed physician who is overseeing your care may do so. Patients still have an important role in applying for expanded access and it is important that patients understand the application process. You should:
- Discuss with your physician if expanded access is appropriate for you.
- Have your physician visit FDA's Expanded Access: Information for Physicians page.
- Have your physician contact the manufacturer to obtain permission from the manufacturer to administer the drug to you.
- If the manufacturer grants you permission to continue with expanded access, have your physician submit the paperwork to FDA to apply for expanded access.
What My Physician and I Can Expect After My Physician Submits the Expanded Access Application
- The FDA will either authorize the expanded access request or not allow it to proceed; however, the FDA authorizes over 99% of expanded access requests it receives.
- If the treatment use is not allowed to proceed, FDA will notify the physician of this decision. The call will be followed by a written letter that provides the reasons for FDA's denial of the request.
- If approved, a number will be assigned to the application.
- The IND sponsor (treating physician) should provide this IND number to the drug supplier, so the supplier may ship the drug to the treating physician.
- The IND is considered active (treatment with the drug may proceed) 30 days after FDA receives the IND submission or if the physician is notified by FDA that they can begin treatment earlier.
Physicians can visit Expanded Access: Information for Physicians page to learn more about submitting an application for Expanded Access
Questions?
Before you or your healthcare provider contact FDA to request Expanded Access to an IND, you may wish to:
- Search for possible clinical trials you may qualify for by using our clinical trials search tool or visiting www.clinicalTrials.gov.
- Search for specific expanded access programs through an online search engine.
- Call drug companies directly to ask about their policies.
- Contact patient advocacy organizations to see if they have information on expanded access programs for your disease condition.
Resources on Expanded Access
- Individual Patient Expanded Access Applications: Form FDA 3926 Guidance for Industry provides a streamlined method for submitting an Investigational New Drug Application (IND) for use in cases of individual patient expanded access. It can also be used for certain submissions to FDA after the initial application is filed. Form FDA 3926 (if you are having trouble opening this form you may need to open it in another web browser or by saving it as a PDF first.
- Form FDA 1571 and 1572 are required for other expanded access submissions (e.g., intermediate access or treatment INDs) and for IND submissions by commercial sponsors or drug manufacturers.
- FDA's Final Guidance: Expanded Access to Investigational Drugs for Treatment Use — Questions & Answers (PDF - 75KB)
- FDA's Guidance: Charging for Investigational Drugs Under an IND — Questions & Answers (PDF - 57KB)
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