I've heard that compassionate use is a way to get access to experimental treatments. How does it work?

Answers from Timothy J. Moynihan, M.D.

In certain situations, the Food and Drug Administration (FDA) allows companies to provide their experimental drugs to people outside of clinical trials. This is referred to as compassionate use. But getting access to not-yet-approved drugs through a compassionate use request can be a long and challenging process.

If you're interested in trying an experimental treatment, talk to your doctor about your options. For you to receive an experimental drug through the compassionate use program, your doctor must contact the drug company and then submit an application to the FDA. For the FDA to consider your request, you must meet certain criteria:

  • Your disease is serious or immediately life-threatening.
  • No treatment is available or you haven't been helped by approved treatments for your disease.
  • You aren't eligible for clinical trials of the experimental drug.
  • Your doctor agrees that you have no other options and may benefit from the experimental treatment.
  • The company that makes the drug agrees to provide it to you.

To find out more about the rules regarding compassionate use, visit the FDA website and search for "access to investigational drugs."

Another way to get access to experimental treatments is through expanded access studies. In these studies, experimental drugs in the later stages of clinical trials are offered to people who don't qualify for the clinical trials. To find out if a drug is available this way, contact the drug's manufacturer. Or go to ClinicalTrials.gov and search for "expanded access studies."

As you consider whether to try to obtain an experimental treatment, it's important to keep a few things in mind:

  • You aren't guaranteed to benefit. Experimental drugs haven't been approved by the FDA, and their efficacy may not yet be proved.
  • The risks of the drug may be unknown. Experimental drugs may not have been fully tested, so the range of side effects may be unknown.
  • Some companies don't give access to experimental drugs. Drug companies aren't required to comply with your request for an experimental drug. The company you ask could refuse your request.
  • Your doctor may not agree with your request. Your doctor might be unwilling to pursue your request if he or she thinks an experimental drug is dangerous or ineffective for your condition. You can ask for a second opinion from another doctor or seek advice from groups that advocate for people with your disease.
  • You may pay out of pocket for experimental treatment. The drug company may charge you for the experimental drug. Also, your insurance company is unlikely to pay associated costs of your treatment, such as fees for your doctor to administer the experimental drug and monitor side effects.
  • Getting an answer may take time. Unless your situation is an emergency, the review process may take some time. Because each compassionate use application is decided on a case-by-case basis, there is no set timeline and no one can predict how long you'll wait for an answer.
Jan. 28, 2012 See more Expert Answers