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U.S. Department of Health and Human Services

For Consumers

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Deciding Whether to Seek Access to an Investigational Drug

It can often be difficult to determine what medical treatment will be right for you, particularly if you have been diagnosed with a serious or life-threatening illness. 

  1. Before you decide to pursue treatment with an investigational (experimental) drug, talk with your healthcare professional about whether an FDA-approved medication or other therapy could be used to treat your condition. Your healthcare professional may be able to adjust the dose of a given medication or try different medications to find the best treatment for you.

    In general, drugs that have been approved by the FDA have been studied carefully to assess whether they are effective and to identify their potential side effects. Most drugs are tested in people through studies called clinical trials.

    FDA approval does not guarantee that you will not experience side effects from a particular drug or that you will benefit from using that drug. Many drugs have side effects that can range from mild to severe.  Drugs used for serious conditions often result in more severe side effects. Clinical testing helps to determine whether the benefits of a drug are likely to outweigh any negative effects. Testing can also help doctors find ways to minimize or manage side effects that their patients may experience.

  2. If you and your doctor have considered available FDA-approved treatment options, and you still want to seek access to an investigational drug, you should consider whether you want to explore the possibility of participating in a clinical trial.



Contact FDA

Office of Health and Constituent Affairs

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Silver Spring, MD 20993