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Pluristem Therapeutics, Inc. Message Board

  • allo_g allo_g Jul 30, 2014 8:22 PM Flag

    Access to Investigational Drugs Outside of a Clinical Trial (Expanded Access)


    Expanded access, sometimes called "compassionate use," is the use of an investigational drug outside of a clinical trial to treat a patient with a serious or immediately life-threatening disease or condition who has no comparable or satisfactory alternative treatment options.

    FDA regulations allow access to investigational drugs for treatment purposes on a case-by-case basis for an individual patient, or for intermediate-size groups of patients with similar treatment needs who otherwise do not qualify to participate in a clinical trial. They also permit expanded access for large groups of patients who do not have other treatment options available, once more is known about the safety and potential effectiveness of a drug from ongoing or completed clinical trials.

    Just as in clinical trials, these investigational drugs have not yet been approved by the FDA as safe and effective. They may be effective in the treatment of a condition, or they may not. They also may have unexpected serious side effects. It is important for you to consider the possible risks if you are interested in seeking access to an investigational drug.

    Can any individual patient qualify for expanded access to any drug?
    No. In order for a patient to gain access to an investigational drug outside of a clinical trial, the patient must have a serious or immediately life-threatening disease or condition and no comparable or satisfactory therapeutic alternatives. Additionally, the drug manufacturer and the patient’s doctor must make special arrangements to obtain the drug for the patient. These arrangements must be authorized by the FDA. These safeguards are in place to avoid exposing patients to unnecessary risks.

    Manufacturers may not always be willing or able to provide access to a drug outside of their clinical trials. Physicians may not always be able to seek expanded access for patients, depending on a patient’s medical history and the risks associated with taking an investigational drug. The physician must determine that the probable risk from the drug is not greater than the probable risk from the disease. Not all physicians are willing to manage the use of an investigational drug for patients in their care.

    Companies are not required to make their drug available through expanded access, or to make more of a drug for that purpose. Companies manufacture investigational drugs for the purpose of testing them in clinical trials, since that is the most effective and efficient way to determine whether the drugs work, and whether they are safe to use. Once the drug is FDA-approved, it may be marketed and made more widely available.

    Sometimes, even when an expanded access program has been established, there may not be enough of a drug available for all patients requesting access. Some companies establish lotteries to determine which patients will have treatment access, while others make the determination on a case-by-case basis.

    Are there costs associated with expanded access?
    Investigational drugs are expensive to manufacture. Some companies provide the drug for free to patients. Other companies charge patients costs associated with the manufacture of the drug. Most insurance companies will not pay for access to an investigational drug. In addition, there may be additional costs associated with administration and monitoring of the investigational drug by healthcare professionals.

    Am I protected from risks?
    When a drug is investigational, federal law requires that its use be reviewed by an Institutional Review Board (IRB) to protect the individuals receiving the drug, including assuring that, in general, the risks are reasonable in light of the potential benefit. However, there may be significant unknown risks. The IRB will require and review an informed consent document to ensure that patients are aware of potential risks and are willing to accept the level of possible risk associated with the drug.

    How can I find out whether I can access a particular investigational drug outside of a clinical trial?
    Some companies have established expanded access programs. You can use ClinicalTrials.gov1 to view a list of expanded access studies2 on the web, or search for specific expanded access programs, or you or your healthcare provider can call a drug company directly to inquire about their policies. Patient advocacy groups can sometimes help patients find and explore expanded access options.

    If you are interested in seeking expanded access to a particular drug, talk with your healthcare professional to see if it might be a reasonable option for you.

    Your and your healthcare professional can contact the Office of Special Health Issues3 for information and assistance.

    Additional information for healthcare professionals can be found at Physician Request for a Individual Patient IND for Compassionate or Emergency Use4., and at IND Applications for Clinical Treatment: Treatment of a Single Patient in Emergency Setting5. For more information specific to biologics, contact the Consumer Affairs Branch at the Center for Biologics Evaluation and Research at ((800) 835-4709, or (240) 402-8010, or via email at

    Information is also available about expanded access to unapproved medical devices6, explaining emergency use, single patient/small group access, treatment use, and continued access after a controlled clinical trial under an Investigational Device Exemption (IDE) has been completed.

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