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Expanded Access: Information for Patients

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What is expanded access?

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Sometimes called “compassionate use”, expanded access is a potential pathway for a patient with a serious or life-threatening disease or condition to try an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when there are no comparable or satisfactory therapies available.

Investigational medical products are investigational drugs, biologics or medical devices that are being tested to see whether they are safe and effective. This means that these products have not yet been approved or cleared by the FDA and the FDA has not found these products to be safe and effective for their specific use. Furthermore, the investigational medical product may, or may not, be effective in the treatment of the condition, and use of the product may cause serious side effects that were not expected.

When seeking expanded access to an investigational medical product, it is critical that you and your licensed physician (typically your doctor, but not all doctors or healthcare providers are licensed physicians) consider all possible risks.

Whenever possible, an investigational medical product should be used as part of a clinical trial. However, there are times when it is not possible for a patient to be a part of a clinical trial (e.g., that there are no ongoing trials, a patient may not have access to a clinical trial or may not be eligible for the clinical trials, distance to get to a trial prevents access). In those circumstances, expanded access may be the only way for a patient to receive an investigational medical product.

FDA is committed to increasing awareness about the expanded access process and the procedures for getting access to investigational medical products. It is important to note that, even if you meet the criteria under the law and FDA regulations, the licensed physician, the Institutional Review Board (IRB), and the company all need to agree that expanded access is appropriate for you in order for you to receive the investigational medical product. In addition, there may be costs not covered by third-party payers such as private insurance or Medicare.

Learn more about expanded access categories for drugs and biologics.

Learn more about expanded access categories for medical devices.

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  1. Patient
    Consults with a licensed physician to explore alternate approved treatment options, available and clinical trials or whether to decide if expanded access may be anotheris the only option.
  2. Licensed Physician
    Agrees to oversee the patient's treatment and works with a company (industry), files paperwork with FDA and Institutional Review Board (IRB), and is responsible for patient care and reporting.
  3. Company (Industry)
    Willing to provide the investigational medical product, and work with FDA and yourthe patient’s licensed physician to provide required information for the expanded access request or submits the requests to the FDA.
  4. Investigational Review Board (IRB)
    Assures that appropriate steps are taken to protect the rights and welfare of people participating as subjects in a research study. For expanded access, the IRB reviews the expanded access plan, including the informed consent to help ensure the patient understands the nature of the investigational medical product for the proposed treatment.
  5. FDA
    Reviews the expanded access request and determines whether the expanded access request may proceed.

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A licensed physician requests the investigational medical product under the expanded access program for you. If your healthcare is managed by anyone other than a licensed physician, you must find a licensed physician who is willing to do the following:

  • Oversee and monitor your treatment.
  • Work with the company to submit, or have the company submit, the necessary paperwork to an IRB and FDA on the expanded access request.
  • Obtain the investigational medical product if the company is willing and able to provide the investigational medical product for treatment under expanded access.

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  1. Talk to your licensed physician before deciding if an investigational medical product is right for you.

    You and your healthcare provider should consider several issues, including the kind of illness you have, the stage of disease, and other conditions you may be experiencing, among other factors.

     

    Points to discuss with your licensed physician:
     How much information is known about the investigational medical product?
     What are the possible risks and side effects of using the investigational medical product?
     What is the severity of your condition?
     Are other therapies available?
     What is the likelihood that the investigational medical product will work?

     

    Other points for consideration:
      Cost. An important area to consider is the cost of the investigational medical product and the medical services associated with its use. The costs related to investigational medical products are usually not covered by third-party payers such as private insurance or Medicare. In addition, costs could be incurred related to IRB review, which may be your responsibility.
      Available trials. You may qualify for a clinical trial to address your disease or condition. Participation in a clinical trial can help with the development process of a medical product so it can be used in other people who have the same disease or condition. You can search for possible clinical trials by using FDA’s clinical trials search tool or visiting www.clinicalTrials.gov.
      Patient advocacy organizations. Contact patient advocacy organizations to see if they have information on expanded access programs for your disease or condition.

     


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  2. Have your licensed physician visit FDA's Expanded Access: Information for Physicians page.

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  3. Have your licensed physician contact the company to request the investigational medical product for expanded access use.

    It is important to remember that the company provides the investigational medical product. Without their cooperation, you will not be able to get expanded access to the investigational medical product.


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  4. For sponsor-investigator single patient expanded access requests, if the company is willing to provide the investigational medical product for treatment under expanded access, the expanded access request can be submitted to FDA.

    Several things must happen before the request is sent to FDA:

    • You and your licensed physician agree that expanded access is a good option for you, and your licensed physician agrees to manage the process, monitor your treatment, and keep records and reports.*
    • The company agrees to provide their investigational medical product for treatment under expanded access.
    • Your licensed physician submits the appropriate paperwork to FDA and the IRB.

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  5. Treatment can begin once everyone agrees to expanded access to the medical product.

    Expanded access to the investigational medical product generally requires five key decisions:

    1. Your licensed physician agrees to oversee your treatment and submit records and reports.
    2. The company agrees to provide the investigational medical product for treatment under expanded access.
    3. The IRB reviews and approves the expanded access plan.
      • Your licensed physician may or may not have access to an IRB, which is usually connected with a hospital or research institution, but there are independent IRBs as well. Depending on the IRB, you may have to pay for the review.
    4. FDA reviews and determines if the expanded access request may proceed.
    5. You carefully read the informed consent form*, ask questions, and make sure that you understand the risks associated with the investigational medical product before signing. You may opt to discontinue your participation at any time, even after signing the informed consent.

      * As part of this process, your licensed physician is required to obtain informed consent from you or your legal representative.

    Learn more about expanded access categories for drugs and biologics.

    Learn more about expanded access categories for medical devices.

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On average, FDA determines that 99% of all expanded access requests may proceed.

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The FDA must determine the following:

  • The patient (or patients) to be treated has a serious or life-threatening disease or condition.
  • There is no comparable or satisfactory therapy to diagnose, monitor, or treat the disease or condition.
  • The patient cannot get the medical product under another investigational medical product study or protocol.
  • The possible benefits to the patient justify the possible risks of the treatment, and those possible risks are not unreasonable given the disease or condition to be treated.
  • Providing the investigational medical product will not interfere with the clinical investigations that could support marketing approval of the expanded access use or otherwise compromise the pontential development of the expanded access product.
  • FDA will determine whether the expanded access request may proceed. If the investigational medical product treatment is not allowed to proceed, FDA will inform the licensed physician or the company (whoever submitted the request) of this decision. FDA will communicate what issues need to be resolved in order for treatment to move forward.

Learn more about drug and biologic expanded access program data. Learn more about medical device expanded access/compassionate program data.

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Not necessarily. Even if you meet the criteria under the law and FDA regulations, the licensed physician, the IRB, or the company may not agree that expanded access is appropriate for you.
  • Your licensed physician may not be able to obtain expanded access for you because of your medical history or the risks associated with taking an investigational medical product. Your licensed physician must determine that the possible risks from the investigational medical product are not greater than the possible risks from the disease. It is also possible that your licensed physician is not willing or able to oversee your treatment of an investigational medical product.
  • The company may not be willing or able to offer the investigational medical product outside of their clinical trials. There is no law or policy that requires a company to provide access to investigational medical products outside of the clinical trial. They may not have enough of the investigational medical product for all patients requesting expanded access. Some companies will start a lottery system to give some patients access to the investigational medical product. Other companies make the decision on a case-by-case basis.
  • The IRB may not agree with the expanded access plan or may have questions.

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All the costs involved with the investigational medical product and the medical services associated with its use must be considered. These are often not covered by third-party payers such as private insurance or Medicare.

  1. A company may decide to cover the cost of the investigational medical product for expanded access. However, if a company decides to charge for the investigational medical product, the costs must be justified and limited to those necessary to recover costs of manufacture, research, development and handling.
  2. It is important to remember that there may be additional or other costs associated with the use of an investigational medical product, that do not require FDA authorization:
    • Your healthcare provider (or the facility or hospital) may charge you for giving you the investigational medical product and for monitoring the treatment.
    • Additional costs could be incurred related to IRB review, which may be your responsibility.

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Additional resources

 

Page Last Updated: 11/08/2018
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