domingo, 24 de mayo de 2015

Expanded Access (Compassionate Use)

Expanded Access (Compassionate Use)



Expanded Access (Compassionate Use)

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.
Doctor advising patient
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 (including biologics), referred to as a single patient investigational new drug (IND).

Statutory Background for All Expanded Access Uses

Under the Federal Food, Drug, and Cosmetic Act, a patient may seek individual patient expanded access (sometimes called single patient access) to investigational products for the diagnosis, monitoring, or treatment of a serious disease or condition if the following conditions are met.
  • 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; and
  • The sponsor (generally the company developing the investigational product for commercial use) or the clinical investigator 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.
Also under FDA’s statute, a sponsor or a physician may submit a protocol intended to provide widespread access to an investigational product. 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 IND or treatment IDE.

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.

Investigational Drugs (Including 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 (including 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.

Expanded Access Requirements For Individual Patient Access to Investigational Drugs (Including Biologics): What You Need to Know

Participation

  • The patient and his or her licensed physician must both be willing to participate.
  • The patient must:
    • Have a serious or immediately life-threatening disease or condition;
    • Have no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition; and
    • Be unable to obtain the investigational drug under another IND or to participate in a clinical trial.

Safety

  • 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.
  • Patients should understand and be willing to accept the possible risks and unknown effectiveness associated with the investigational medical product. Patients generally will need to sign an informed consent form in order to receive the investigational drug.
  • The patient’s physician must determine that the probable risk to the patient from the investigational drug is not greater than the probable risk from the disease or condition.

Single Patient Expanded Access Submissions

  • 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.
  • Currently, Form FDA 1571 is the approved form for submission of all INDs, including individual patient expanded access submissions. FDA also is developing an alternative form, draft Form FDA 3926, that licensed physicians will be able to use (when it is finalized) for submitting requests for individual patient expanded access to investigational drugs, including in emergencies. Draft Form FDA 3926 is shorter than Form FDA 1571 and should be easier to complete when it is used in the future.

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.

How to Apply for Expanded Access to an Investigational Drug (Including a Biologic) Under a Single Patient IND

  1. Applications for expanded access to an investigational drug under a single patient IND are made by physicians.
  2. The physician should review the requirements for expanded access with the patient and obtain informed consent.
  3. The physician should 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.
  4. The physician must complete the necessary paperwork and submit the request to FDA.
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

  • 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.
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 a an example of wording that could be used for a Letter of Authorization, FDA contacts and more.

Questions?

If you have questions about requirements, need assistance with completing an application or submitting a request for expanded access for a specific investigational medical product (drug, biologic, or medical device),contact the appropriate FDA review division or office.
If you have general questions about expanded access to investigational drugs, contact the CDER’s Division of Drug Information.
If you have general questions about FDA’s expanded access program, contact the Office of Health and Constituent Affairs’ Expanded Access Team at 301-796-8460 or PatientNetwork@fda.hhs.gov.

FDA Resources on Expanded Access

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