This section of our website provides information about FDA’s current expanded access policies, requirements for enrolling in expanded access programs, and steps you can take to get more information.
Expanded access, sometimes called "compassionate use," is the use outside of a clinical trial of an investigational medical product (i.e., one that has not been approved by FDA). FDA is committed to increasing awareness of and knowledge about its expanded access programs and the procedures for obtaining access to human investigational drugs (including biologics) and medical devices.
Wherever possible, use of an investigational medical product by a patient 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 patient enrollment in a clinical trial is not possible (e.g., a patient is not eligible for any ongoing clinical trials, or there are no ongoing clinical trials), patients may be able to receive the product, when appropriate, through expanded access.
On this page, you will find general information about expanded access, followed by more detailed information on individual patient access to investigational drugs and biologics, referred to as a single patient investigational new drug (IND).
Requirements for All Expanded Access Uses
Under the Federal Food, Drug, and Cosmetic Act (FD&C Act), a patient may seek individual patient expanded access to investigational products for the diagnosis, monitoring, or treatment of a serious disease or condition if the following conditions are met.
- The patient and his or her licensed physician are both willing to participate and the person’s physician determines that there is no comparable or satisfactory alternative therapy available to diagnose, monitor, or treat the person’s disease or condition, and that the probable risk to the person from the investigational product is not greater than the probable risk from the disease or condition;
- FDA determines that there is sufficient evidence of the safety and effectiveness of the investigational product to support its use in the particular circumstance;
- FDA determines that providing the investigational product will not interfere with the initiation, conduct, or completion of clinical investigations to support marketing approval;
- The sponsor (generally the company developing the investigational product for commercial use) or the clinical investigator (or the patient’s physician in the case of a single patient expanded access request) submits a clinical protocol (a document that describes the treatment plan for the patient) that is consistent with FDA’s statute and applicable regulations for INDs or investigational device exemption applications (IDEs), describing the use of the investigational product; and
- The patient is unable to obtain the investigational drug under another IND or to participate in a clinical trial.
Also under the FD&C Act statute, a sponsor or a physician may submit a protocol intended to provide widespread access to an investigational product for multiple patients. In this scenario, FDA will permit the investigational product to be made available under a treatment IND or treatment IDE if certain criteria are met. Learn more about treatment INDs and treatment IDEs.
Ensuring patient safety is a priority - FDA must determine that the potential patient benefit justifies the potential risk of the expanded access use of the investigational drug, and that the potential risk is not unreasonable in the context of the disease or condition to be treated. Even with safeguards, there may be significant unknowns about safety and effectiveness.
Expanded Access to Investigational Medical Devices
There may be circumstances under which a health care provider may wish to use an unapproved device to save the life of a patient or to help a patient suffering from a serious disease or condition for which there no other alternative therapy exists. Learn more about IDEs and the expanded access requirements for medical devices.
Expanded Access to Investigational Drugs and Biologics
21 CFR part 312 subpart I provides general requirements, describes criteria that must be met to authorize expanded access, lists requirements for expanded access submissions, and describes safeguards that will protect patients and preserve the ability to develop meaningful data about the use of the investigational product.
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; and
Expanded access for widespread use.
This web page primarily addresses single patient expanded access INDs.
Investigational Product Availability and Costs
- The medical product company must agree to provide the investigational drug for expanded access use. FDA cannot require a company to provide an investigational drug for expanded access use to proceed.
- A company may decide to turn down a request if, for example it is not able or willing to provide access to an investigational drug outside of clinical trials intended to support marketing approval.
- In some cases, patients may have to pay for using the investigational drug and/or for medical care associated with the use of the investigational drug. In others, pharmaceutical companies may elect not to charge.
How to Apply for Expanded Access to an Investigational Drug or Biologic Under a Single Patient IND
Individual patient expanded access submissions made by individual physicians are submitted as new INDs. If a licensed physician is making the individual patient expanded access submission, he or she also must be willing to manage the use of the investigational drug and the patient’s medical care. This includes discussing risks and benefits, obtaining all required informed consent and Institutional Review Board (IRB) approval, reporting adverse events and outcomes, and submitting the necessary paperwork to FDA.
- Apply for expanded access to an investigational drug under a single patient IND. Form FDA 3926 can be used by physicians when submitting requests for individual patient expanded access to investigational drugs, including in emergencies. This form is designed specifically for single patient IND requests. 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
- Ask the medical product company for a Letter of Authorization (LOA), if applicable. An LOA from a company allows the physician submitting the single patient IND to satisfy some of the submission requirements by relying on information in the company’s existing IND. It also authorizes FDA to refer to the company’s IND when reviewing the single patient IND.
- Complete the necessary paperwork and submit the request to FDA.
- Request approval from the IRB.
- Review the requirements for expanded access with the patient and obtain informed consent.
Emergency requests for expanded access use under a single patient IND may be submitted over the phone or electronically by a licensed physician, provided the physician explains how the expanded access use will meet the requirements in FDA’s regulations and agrees to submit an expanded access submission within 15 working days of FDA’s initial authorization of the expanded access use.
Visit the Expanded Access: Information for Physicians page for more detailed information about the expanded access requirements, application process, and what to do in the case of emergency requests.
What to Expect After Submitting a Request for Expanded Access to an Investigational Drug (Including a Biologic) Under a Single Patient IND
- FDA authorizes over 99% of expanded access requests it receives.
- Treatment may begin 30 days after FDA receives the IND, or earlier if FDA notifies the treating physician that the expanded access use may begin. The treating physician must ensure that IRB review is obtained in accordance with FDA’s regulations.
- The treating physician may need to provide the IND application number to the medical product company prior to the company shipping the investigational drug. This number will be provided upon FDA authorization of the expanded access request.
The process is different if the request for expanded access is being submitted to an existing IND as a protocol submission.
Information for Patients, Physicians and Industry
Learn about what your physician should do before submitting a request for individual patient expanded access use of an investigational medical product, who may be eligible for expanded access, associated costs, FDA contacts and more
Learn about your responsibilities under the expanded access pathway, how to submit a request for expanded access for an individual patient (including for emergency use), which forms to use, FDA contacts and more.
Learn about current regulations, what information is required when you provide access to investigational medical products under an individual patient expanded access IND, and view an example of wording that could be used for a Letter of Authorization, FDA contacts and more.
If you have general questions about FDA’s expanded access program, or you are a patient, contact the Office of Health and Constituent Affairs’ Expanded Access Team at 301-796-8460 or PatientNetwork@fda.hhs.gov.
If you are a physician and have questions about completing an application or submitting a request for expanded access for an investigational drug or biologic, contact the Division of Drug Information at 301-796-3400 or
If you have questions about an investigational device exemption, contact the Division of Industry and Consumer Education at 301-796-7100 or DICE@fda.hhs.gov
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.
FDA Resources on Expanded Access
Updated table “Expanded Access Submission Receipt Reports 2010 – 2015.
- Individual Patient Expanded Access Applications: Form FDA 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.
- Investigational New Drug Application: Form FDA 1571 and 1572 are no longer required for individual patient expanded access to investigational drugs and biologics. 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.
- FDA's Guidance: Charging for Investigational Drugs Under an IND - Questions & Answers (PDF - 57KB)
Expanded Access Information for Medical Devices
Learn more about IDEs and the expanded access requirements for medical devices.
Expanded Access INDs and Protocols 2009 - 2015
Data tables on the number of CBER and CDER expanded access submissions and FDA's Expanded Access performance.