Access to Investigational Drugs
Investigational or experimental drugs are new drugs that have not yet been approved by the FDA or approved drugs that have not yet been approved for a new use, and are in the process of being tested for safety and effectiveness.
Patients may decide to seek access to investigational drugs for different reasons. Some patients with serious or life-threatening illnesses seek treatment with investigational drugs if FDA-approved therapies are not working or if their side effects are too severe. Others may have heard about promising early study results for a specific investigational drug, and they might want to learn more.
Investigational drugs are available through two pathways designed to protect patients, because an investigational drug may pose unknown risks to patients and we do not know if it is effective. Patients may be eligible to receive an investigational drug as (1) a participant in a clinical trial, or (2) as part of an expanded access program.
Access to Investigational Drugs, For Consumers, U.S. Food & Drug Administration, August 12, 2009.
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.
Access to Investigational Drugs Outside of a Clinical Trial (Expanded Access), For Consumers, Food & Drug Administration, August 8, 2009.
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