FDA’s Expanded Access program
When seriously ill patients have exhausted all standard of care treatment options, the use of investigational agents may be warranted. However, not all patients qualify for clinical trials.
In these instances, FDA’s Expanded Access program (also sometimes referred to as “Compassionate Use”), may be allowed for use of investigational therapies outside of the traditional clinical trial space. However, this is a fairly complex and tightly regulated process whereby physicians and drug companies coordinate the allowable disposition of these treatments for particular patient uses through the Federal Drug Administration (FDA).
Expanded Access may be an appropriate avenue to pursue when ALL of the following conditions apply:
1. The patient has a serious disease or condition or the patient’s life is immediately threatened by their disease or condition;
2. There is no comparable or satisfactory alternative therapy to diagnose, monitor or treat the disease or condition;
3. Patient enrollment in a clinical trial is not possible;
4. Potential patient benefit justifies the potential risks of treatment;
5. Providing the investigational medical product will not interfere with investigational trials that could support a medical product’s development or marketing approval for the treatment indication.
More detailed information on procedures for requesting Expanded Access use of an investigational medicine and additional information can be found on FDA’s website.
Expanded Access Categories
There are three main categories of Expanded Access submissions:
1) Expanded Access for the individual patient
2) Expanded Access for intermediate-size patient populations
3) Expanded Access for widespread treatment use
Expanded Access for individual patients can be further subdivided into two categories:
In a non-emergency situation, a written request for single patient use of an investigational new drug (IND) must be submitted to the FDA. The investigational drug may be shipped and treatment of the patient may begin 30 days after the application is received by FDA or earlier if notified by the FDA that treatment may proceed. These non-emergency requests are known as individual patient INDs, or single patient expanded access requests.
In a life-threatening situation in which no acceptable standard treatment is available and in which there is not sufficient time to obtain full institutional review board (IRB) approval, the request may be made via the telephone or other rapid means of communication.