Expanded access is a means by which manufacturers make
investigational new drugs available,
under certain circumstances, to treat a patient(s) with a serious disease or condition who cannot
participate in a
controlled clinical trial.
Most human use of
investigational new drugs
takes place in controlled clinical trials conducted to assess the safety and
efficacy of new drugs. Data from these trials
are used to determine whether a drug is safe and effective, and serve as the basis for the drug marketing
application. Sometimes, patients do not qualify for these controlled trials because of other health
problems, age, or other factors, or are otherwise unable to enroll in such trials (e.g., a patient
may not live sufficiently close to a clinical trial site).
For patients who cannot participate in a clinical trial of an investigational drug, but have a
serious disease or condition that may benefit from treatment with the drug,
FDA regulations enable manufacturers of such drugs
to provide those patients access to the drug under certain situations, known as "expanded access."
For example, the drug cannot expose patients to unreasonable risks given the severity of the disease
to be treated and the patient does not have any other satisfactory therapeutic options (e.g., an
approved drug that could be used to treat the patient's disease or condition). The manufacturer must
be willing to make the drug available for expanded access use. The primary intent of expanded access
is to provide treatment for a patient's disease or condition, rather than to collect data about the
study drug.
Some investigational drugs are available for treatment use from pharmaceutical manufacturers through
expanded access programs listed in ClinicalTrials.gov. If you or a loved one is interested in treatment
with an investigational drug under an expanded access protocol listed in ClinicalTrials.gov, review the
protocol eligibility criteria and inquire at the Contact Information number. If there is not an expanded
access protocol listed in ClinicalTrials.gov, you or your health care provider may contact a manufacturer
of an investigational drug directly to ask about expanded access programs.
For additional information on expanded access programs, please see the FDA website at
Access to Investigational Drugs.
Expanded Access Studies can be found by in ClinicalTrials.gov:
- go to the Advanced Search page,
- select "Expanded Access Studies" from the "Study Type" pull-down menu, and
- press Search.
or use these prepared links:
