Expanded Access: Information for Physicians
Expanded access is the use of an investigational drug outside of clinical trials to diagnose, monitor, or treat patients with serious diseases or conditions for which there are no comparable or satisfactory approved therapy options available.
Wherever possible, use of an investigational medical product by a patient as part of a clinical trial should be considered. However, when patient enrollment in a clinical trial is not possible (e.g., patient ineligibility, lack of ongoing clinical trials), expanded access may be an option to gain access to an investigational medicinal product.
FDA is committed to increasing awareness about its expanded access process and the procedures for obtaining access to investigational drugs, biologics and medical devices. This page provides important information for physicians about their role in seeking expanded access for individual patients, how to submit a request for emergency use and non-emergency use of an investigational new drug or biological product, which forms to use, FDA contacts and more.
Visit the medical devices page to learn about expanded access requirements for medical devices and investigational device exemptions (IDEs).
On this page you will find information about:
Physician Role When a Patient is Seeking Expanded Access to Drugs and Biologics
You must determine that your patient’s current disease or condition is 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.
If your patient meets the criteria for expanded access, you must speak with the drug manufacturer to see if they will provide the investigational medical product for expanded access use. After you get the agreement from the manufacturer to provide the investigational medical product for use outside of a clinical trial, you will be responsible for managing the use of the investigational medical product and the patient's medical care. This includes:
- Reviewing the requirements for Expanded Access with the patient.
- Evaluating potential risks and potential benefits with the patient.
- Identifying the appropriate FDA review division prior to submitting the necessary paperwork.
- Obtaining informed consent, consistent with Federal requirements under 21 CFR 50.
- Submitting FDA Form 3926 and the necessary paperwork to FDA.
- Submitting the expanded access protocol to an Institutional Review Board (IRB) that complies with the Federal IRB requirements under 21 CFR 56. The IRB will be responsible for initial and continuing review and approval of the protocol.
Ensuring patient safety is a priority; FDA must determine that the potential benefit justifies the potential risk of the expanded access use of the investigational product. Even with safeguards, there may be significant unknowns about safety and effectiveness.
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Expanded Access Categories and Regulatory Submission Types
Under FDA’s current regulations for investigational drugs and biologics, there are three categories of expanded access:
- Expanded access for individual patients, including for emergency use;
- Expanded access for intermediate-size patient populations (generally smaller than those typical of a treatment IND or treatment protocol); and
- Expanded access for widespread treatment use through a treatment IND or treatment protocol (designed for use in larger patient populations).
Information on individual patient expanded access to investigational drugs (including biologics) is discussed below, along with more detailed information on submitting individual patient investigational new drug (IND) requests.
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How to Submit a Request for Individual Patient Expanded Access IND
Important Information - When accessing FDA Form 3926 you may need to open the page in Internet Explorer or save the document as a PDF to your desktop before opening it up.
You may need to get a letter of authorization (LOA) from a representative of the company that makes the investigational medical product. This person is typically the regulatory affairs official of the company. FDA may be able to help identify the contact. It is important to remember that for drugs and biologics, a patient may obtain access to an investigational drug for treatment use only through a licensed physician. Under FDA's regulations, a physician who submits an investigational new drug application (IND) for Individual Patient Expanded Access is considered the sponsor of that application.You will need to complete the necessary paperwork.
- FDA Form 3926 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. (You may need to open this form in a different browser i.e. Internet Explorer or save the form to your desktop.) The form may be printed out and responses may be handwritten. Alternatively, responses may be typed in to the electronic form, then saved, signed and either printed or attached to an email. It may be sent to the CDER or CBER Document Room at the below address, or sent directly to the relevant FDA review division. For more information, including instructions, please visit the guidance Individual Patient Expanded Access Applications: Form FDA 3926.
- Physicians will still be able to use Form 1571 and Form 1572 for single patient expanded access submissions; however, Form 3926 is developed specifically for these requests and is easier to complete. Form FDA 1571 and 1572 are still required for other expanded access submissions (e.g., intermediate access or treatment INDs) and for IND submissions by commercial sponsors or drug manufacturers.
- If you are not familiar with the process for submitting a request for EA, you may want to speak with the appropriate review division prior to submitting your request for Expanded Access.
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Application Requirements for a Protocol Submission to an Existing IND
The application requirements and process are different if the request for expanded access is being submitted to an existing IND as a protocol submission. Only the company or individual who holds an IND may make a protocol submission to that IND. Learn more about submitting protocols to existing INDs.
When Can Treatment Begin?
- For non-emergency requests, treatment may begin 30 days after FDA receives the IND application, unless the treating physician (also referred to as a sponsor) of the IND receives earlier notification from FDA that the treatment may proceed. The FDA authorizes over 99% of expanded access requests it receives.
- In the case of an emergency request, once the FDA provides authorization you may begin treatment without prior IRB approval, provided the IRB is notified of the emergency treatment within 5 working days of beginning treatment.
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Emergency Requests for Individual Patient Expanded Access
- FDA may authorize expanded access for an individual patient without a written submission if there is an emergency that requires the patient to be treated before a written submission can be made.
- Emergency requests may be submitted over the phone or via email by a licensed physician after receiving agreement from the drug company to provide the product. Please follow the instructions on FDA's Expanded Access Contact Information page.
- The licensed physician or sponsor must explain how the expanded access use will meet the requirements of FDA’s regulations and agree to submit an expanded access IND or protocol within 15 working days of FDA’s authorization of the use.
- All questions about and requests for expanded access for emergency use for drugs, biologics and medical devices should be directed to the FDA Emergency Call Center, telephone: 866-300-4374 after 4:30 pm EST weekdays and all day on weekends.
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Questions?
If you have questions about requirements, need assistance with completing an application or submitting a request for expanded access for a specific investigational drug, biologic, or device contact:
- CDER's Division of Drug Information at 301-796-3400 or druginfo@fda.hhs.gov.
- CBER’s Office of Communication, Outreach and Development at 240-402-8020 or 800-835-4709 or industry.biologics@fda.hhs.gov.
- CDRH’s Division of Industry and Consumer Education at 301-796-7100 or DICE@fda.hhs.gov.
Additional Resources
- Form FDA 3926 for requests for individual patient expanded access to investigational drugs, including emergencies. It can also be used for certain 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
- 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.
- 21 CFR 312 Subpart I
Learn more about FDA’s current expanded access regulations for investigational drugs (including biologics). - Expanded Access Video for Physicians
FDA Drug Info Rounds pharmacists discuss the requirements that must be met before FDA can authorize Expanded Access and discuss the safeguards in place to avoid exposing patients to unnecessary risks. - For Physicians: A guide to Non-emergency single patient expanded access submissions. This website provides a reference chart for initiating single patient expanded access and for follow-up submissions.
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