U.S. flag An official website of the United States government

On Oct. 1, 2024, the FDA began implementing a reorganization impacting many parts of the agency. We are in the process of updating FDA.gov content to reflect these changes.

U.S. Food and Drug Administration
  1. Home
  2. For Patients
  3. Learn About Expanded Access and Other Treatment Options
  4. Understanding Investigational Drugs
  1. Learn About Expanded Access and Other Treatment Options

Understanding Investigational Drugs

pills, pill bottle and beaker

Have you ever thought about joining a clinical trial that is trying to find out if an investigational drug works in treating your disease or medical condition?  Or maybe your healthcare provider has talked to you about treating you with an investigational drug through expanded access. 

An investigational drug can also be called an experimental drug and is being studied to see if your disease or medical condition improves while taking it. Scientists are trying to prove in clinical trials:

  • If the drug is safe and effective.

  • How the drug might be used in that disease.

  • How much of the drug is needed.

  • Information about the potential benefits and risks of taking the drug.

When to consider using an investigational drug

Not every person’s disease or medical condition responds the same way to approved drugs. Your healthcare provider might have talked to you about using an investigational drug if you have:

  • Experienced side effects that are too severe to continue taking

  • Limited treatment options available.

  • Heard about promising early study results for a specific investigational drug.

  • No approved drugs available to treat your disease or medical condition.

Questions you may want to consider

If your healthcare provider thinks that using an investigational drug is an option for you, then consider asking questions like those listed below before deciding whether it is right for you.

  • What is this drug being studied for?

  • Is there a clinical trial site near you?

  • How much is already known about the investigational drug?

    • What basis is there for thinking that the drug will work better for your medical condition than using an approved treatment?

    • What scientific evidence is available to support the use of this investigational drug?

    • What are the potential risks of using this investigational drug?

  • Are there other drugs that are already approved to treat your disease or condition?

    • Have you already tried them?

    • Why or why not?

Many of these questions may be answered for you when you read the informed consent document. Before you can be given an investigational drug either through a clinical trial or through expanded access, your healthcare provider must give you additional information about the potential risks and potential benefits of the drug.

As promising as an investigational drug may sound. It is still being tested in clinical trials to determine if it can be used to treat a disease or medical condition. And not everyone who wants to enroll in a clinical trial will be able to participate.  Clinical trials have strict inclusion and exclusion criteria about who can participate. The criteria can be based on such factors as:

  • Having a certain type or stage of disease.

  • Having received (or not received) a certain kind of therapy in the past.

  • Being in a certain age group.

  • Your medical history.

  • Your current health status.

Criteria like those listed above can help researchers understand the medical differences among people in the trial and help them answer the questions they are studying about the drug. 

Finally, remember that approved drugs have completed extensive testing in clinical trials and there is scientific proof that they are safe and effective for treating the particular disease or medical condition that has been studied.  It is important to talk with your healthcare provider on a regular basis about the medicines you are taking and talking about the side effects you may be experiencing.
 

Back to Top