"Expanded Access" in ClinicalTrials.gov
[Editor's Note: This is a reprint of two announcements published on the NLM® Web site on October 25, 2009. To be notified of announcements like this subscribe to NLM-Announces e-mail list.]
FAQ: What is "expanded access"?
Answer: 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.FAQ: How do I find information on Expanded Access Studies in ClinicalTrials.gov?
Answer: Expanded Access Studies can be found by using the Advanced Search feature at ClinicalTrials.gov.
- Select "Search for Clinical Trials" from the ClinicalTrials.gov homepage
- Select "Advanced Search" on the Search Page.
- Find the field titled "Study Type" and select “Expanded Access Studies” from the pull-down menu on the “Advanced Search” page. Note: Additional search terms (e.g., condition) may be added to the Advanced Search page.
- Select "Search" to see a list of Expanded Access studies.
For additional information on expanded access programs, please see the FDA website.
"Expanded Access" in ClinicalTrials.gov. NLM Tech Bull. 2009 Nov-Dec;(371):e5.