What is an Investigational HIV Drug?
- An investigational drug is an experimental drug that is being studied to see whether it is safe and effective.
- Investigational HIV drugs are studied in medical research studies called clinical trials. Once an investigational HIV drug has been proven safe and effective in a clinical trial, the U.S. Food and Drug Administration (FDA) may approve the drug for general use or sale in the United States.
- Investigational HIV drugs being studied 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 an experimental drug that is being studied to see whether it is safe and effective. Investigational HIV drugs are studied in medical research studies called clinical trials. Once an investigational HIV drug has been proven safe and effective in a clinical trial, the U.S. Food and Drug Administration (FDA) may approve the drug for general use or sale in the United States.
What types of investigational HIV drugs are being studied?
Investigational HIV drugs being studied include drugs to treat HIV and prevent HIV. Some types of investigational HIV drugs being studied include microbicides, immune modulators, latency-reversing agents, gp120 attachment inhibitors, and rev inhibitors.
HIV researchers are also studying investigational vaccines to prevent HIV and treat HIV. The goal of a preventive HIV vaccine is to prevent HIV in people who do not have HIV but who may be exposed to the virus. A safe and effective HIV treatment vaccine (also called a therapeutic HIV vaccine) could prevent HIV from advancing to acquired immunodeficiency syndrome (AIDS), replace the daily use of HIV medicines, and help prevent HIV transmission.
To learn more, read the HIVinfo 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 are conducted in phases. Each phase has a different purpose and helps researchers answer different questions about the investigational drug.
- Phase 1 trial: Initial testing in a small group of people (20–80) to evaluate the drug’s safety and to identify side effects.
- Phase 2 trial: Testing in a larger group of people (100–300) to determine the drug’s effectiveness and to further evaluate its safety.
- Phase 3 trial: Continued testing in large groups of people (1,000–3,000) to confirm the drug’s effectiveness, monitor side effects, compare it with standard or equivalent treatments, and collect information to ensure that the investigational drug can 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 the FDA for sale in the United States. (However, some drugs go through the FDA’s accelerated approval process and are approved before a Phase 3 clinical trial is complete.)
- Phase 4 trial: Ongoing tracking that occurs after a drug is approved by the FDA for sale in the United States. The purpose of the tracking is to seek more information about the drug’s risks, benefits, and optimal use.
For more information, read the HIVinfo HIV and AIDS Clinical Trials fact sheet.
How can a person find a clinical trial that is studying an investigational HIV drug?
To find an HIV and AIDS clinical trial that is studying an investigational HIV drug, use the find a study search feature on ClinicalTrials.gov.
For help with your search, call a Clinicalinfo health information specialist at 1-800-448-0440 or email ContactUs@HIVinfo.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.
Are investigational HIV drugs available for use outside of a clinical trial?
In some cases, an investigational HIV drug may be available through an expanded access program. Expanded access allows for the use of an investigational drug outside of a clinical trial to treat a person who has a serious or immediate life-threatening disease and who has no FDA-approved treatment options. Drug companies must have permission from the FDA to make an investigational drug available for expanded access.
People seeking expanded access to an investigational HIV drug should talk to their health care provider to see if they may qualify to take part in an expanded access program.
Is it safe to use an investigational HIV drug?
One goal of HIV research is to identify safer, more effective HIV medicines. Researchers try to make clinical trials as safe as possible. However, taking an investigational HIV drug can involve both benefits and risks. Risks may include unexpected side effects from the drug, which can be unpleasant, serious, or even life-threatening.
The benefits and possible risks of participating in a clinical trial or an expanded access program are explained to people before they decide whether to participate.
How can a person find more information on investigational HIV drugs?
To find more information on investigational HIV drugs, use the Clinicalinfo Drug Database, which includes up-to-date information on many investigational HIV drugs.
This fact sheet is based on information from the following sources:
From the National Institutes of Health (NIH):
- NIH Clinical Research Trials and You: The Basics
- NIH Clinical Research Trials and You: Finding a Clinical Trial
From the National Institute of Allergy and Infectious Diseases:
From the U.S. Food and Drug Administration (FDA):
Also see the HIV Source collection of HIV links and resources.