In some cases, the Food and Drug Administration (FDA) allows pharmaceutical companies to provide their experimental drugs to people outside of clinical trials. This practice is known as compassionate use, or expanded access.
The FDA created compassionate use in 1987 to allow people with life-threatening diseases to request access to investigational new drugs outside of clinical trials. Investigational drugs have not been approved by the FDA, and the FDA has not established that they are safe and effective for their specific use.
Gaining access to experimental medications through a compassionate use request can be a long and difficult process. However, it is a reasonable option for some people with rare diseases. It is important to know that the cost of these medications can be high. Also, few insurance companies will cover the cost of these drugs due to their experimental classification. A physician and other medical resources are required to administer these drugs, which further increases out-of-pocket costs for patients.
Talk to your doctor if you are interested in gaining access to an experimental medication through compassionate use dispensation. To be approved for the program, your doctor must contact the pharmaceutical company and submit a request to the FDA. The FDA requires the following criteria to be met to consider your request:
- Your disease is serious or immediately life-threatening.
- No treatment is available, or treatments approved for your disease have not helped.
- You are not eligible for clinical trials of the experimental drug.
- Your doctor verifies that you have no other options, and the experimental treatment may help.
- Your doctor believes the benefit outweighs the potential risks of the treatment.
- The pharmaceutical company that makes the drug agrees to provide it.
Expanded Access Studies
Participation in expanded access studies is another way to gain access to experimental treatments. In these studies, experimental drugs in the later stages of clinical trials are offered to people who do not qualify for the clinical trials.
To determine if an experimental drug is available through an expanded access study, contact the drug’s manufacturer or visit clinicaltrials.gov and search “expanded access studies.”
