Expanded access (compassionate use) electricity wikipedia simple english

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 application (IND).

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 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

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.

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.

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.

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 complying.

Important Information – When accessing FDA Form 3926 you may need to open the page in Internet Explorer. If you see a "please wait" message, right click (or control-click on Mac) and select the "save as" option to download the document as a PDF to your computer before opening it.

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.

Obtain IRB review and approval, consistent with 21 CFR part 56. A physician submitting an individual patient expanded access IND using Form FDA 3926 may select the appropriate box on that form to request authorization to obtain concurrence by the IRB chairperson or by a designated IRB member before the treatment use begins, in lieu of obtaining IRB review and approval at a convened IRB meeting at which a majority of the members are present. Although Form FDA 1571 does not include a specific field for making such a request, a physician submitting an individual patient expanded access IND using Form FDA 1571 may include a separate request with the application.

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.

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.

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.

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 see "Please wait," right-click (or control-click on Mac) and select the “save as” option to save the PDF file to your computer.

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.