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What is an Investigational HIV Drug?

Last Reviewed: August 25, 2017

Key Points

  • An investigational HIV drug is a drug that is being tested and is not approved by the U.S. Food and Drug Administration (FDA) for general use or sale in the United States.
  • Medical research studies—also called clinical trials—are done to evaluate the safety and effectiveness of an investigational HIV drug.
  • Investigational HIV drugs include drugs to treat or prevent HIV and vaccines to treat or prevent HIV.
  • Investigational HIV drugs can only be accessed through clinical trials and expanded access programs.

What is an investigational HIV drug?

An investigational HIV drug is a drug that is being tested to treat or prevent HIV infection and is not approved by the U.S. Food and Drug Administration (FDA) for general use or sale in the United States. Medical research studies—also called clinical trials—are done to evaluate the safety and effectiveness of an investigational HIV drug. 

What types of investigational HIV drugs are being studied?

Currently, there are investigational drugs for treating HIV and preventing HIV. There are also investigational drugs for treating HIV-related opportunistic infections. (Opportunistic infections are infections and infection-related cancers that occur more frequently or are more severe in people with weakened immune systems than in people with healthy immune systems.) 

Although no HIV vaccines exist yet, researchers are studying investigational preventive vaccines and treatment vaccines. The goal of a preventive HIV vaccine is to prevent HIV in people who don’t have HIV but who may be exposed to the virus. The goal of an HIV treatment vaccine, also called a therapeutic vaccine, is to slow the progression of HIV infection or delay the onset of AIDS in people with HIV. To learn more, read the AIDSinfo What is a Preventive HIV Vaccine? and What is a Therapeutic HIV Vaccine? fact sheets.

How are clinical trials of investigational drugs conducted?

Clinical trials, which are medical research studies, are conducted in phases. Each phase has a different purpose and helps researchers answer different questions about the investigational drug.

  • Phase 1 trials: Researchers test the investigational drug in a small group of people (20–80) for the first time. The purpose is to evaluate its safety and identify side effects.
  • Phase 2 trials: The investigational drug is administered to a larger group of people (100–300) to determine its effectiveness and to further evaluate its safety.
  • Phase 3 trials: The investigational drug is administered to large groups of people (1,000–3,000) to confirm its effectiveness, monitor side effects, compare it with standard or equivalent treatments, and collect information that will allow the investigational drug to be used safely.

In most cases, an investigational drug must be proven effective and must show continued safety in a Phase 3 clinical trial to be considered for approval by FDA for sale in the United States. (Some drugs go through FDA’s accelerated approval process and are approved before a Phase 3 clinical trial is complete.) After a drug is approved by FDA and made available to the public, researchers track its safety in Phase 4 trials to seek more information about the drug’s risks, benefits, and optimal use. For more information, read the AIDSinfo HIV/AIDS Clinical Trials fact sheet.

How can I get access to an investigational HIV drug?

One way to get access to an investigational HIV drug is by enrolling in a clinical trial that is studying the drug. Another way is through an expanded access program. Expanded access involves using an investigational drug outside of a clinical trial to treat a person who has a serious or immediately life-threatening disease and who has no FDA-approved treatment options. Drug companies must have permission from FDA to make an investigational drug available for expanded access. Talk to your health care provider to see if you may qualify to take part in an expanded access program.

How can I find a clinical trial on an investigational HIV drug?

To find an HIV/AIDS clinical trial on an investigational HIV drug, use the AIDSinfo clinical trial search. For help with your search, call an AIDSinfo health information specialist at 1-800-448-0440 or email ContactUs@aidsinfo.nih.gov. 

You can also join ResearchMatch, which is a free, secure online tool that makes it easier for the public to become involved in clinical trials. 

Is it safe to use an investigational HIV drug?

One goal of HIV research is to identify new drugs that are less toxic and have fewer side effects. Researchers also try to make HIV/AIDS clinical trials as safe as possible. But investigational HIV drugs may have side effects that are not well known yet. Although this risk of poorly understood side effects is explained to you before you start taking the investigational drug, this makes it hard to know your actual risk. As testing of an investigational HIV drug continues, additional information on possible side effects is collected.

How can I find more information on investigational HIV drugs?

To find more information on investigational HIV drugs, use the AIDSinfo Drug Database, which includes up-to-date information on many investigational HIV drugs.

This fact sheet is based on information from the following sources:

 

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