(Last updated 12/13/2016; last reviewed 9/15/2016)
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.
Currently, there are investigational drugs for treating HIV and preventing HIV. There are also investigational drugs for treating HIV-related opportunistic infections. (Examples of opportunistic infections include pneumonia and tuberculosis.)
Although no HIV vaccines exist yet, researchers are studying investigational preventive vaccines and treatment vaccines. A preventive HIV vaccine is a vaccine to prevent HIV infection in people who do not have HIV. An HIV treatment vaccine, also called a therapeutic vaccine, is a vaccine to slow the progression of HIV infection or delay the onset of AIDS.
In most cases, an investigational drug must be proven effective and must show continued safety in a Phase III 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 III clinical trial is complete.) After a drug is approved by FDA and made available to the public, researchers track its safety in Phase IV trials to seek more information about the drug’s risks, benefits, and optimal use.
Some HIV clinical trials enroll HIV-infected people who have never taken HIV medicines before or who have taken HIV medicines for only a very short time—this is called being treatment-naive. Other HIV clinical trials enroll HIV-infected people who have already taken HIV medicines for a period of time—this is called being treatment-experienced. Some HIV clinical trials enroll people who are not infected with HIV.
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.
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 registry that makes it easier for the public to become involved in clinical trials.
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.
To find more information on investigational HIV drugs, use the AIDSinfo Drug Database, which includes up-to-date information on many investigational HIV drugs.