By: Amy Abernethy, M.D., Ph.D., Principal Deputy Commissioner
When a patient is in need of an important medicine, paperwork and forms are the last thing their physician wants to deal with. In some cases, it’s necessary, but the U.S. Food and Drug Administration is doing everything we can to keep it to a minimum. Our expanded access program is one example.
Expanded access is a pathway for a patient with a serious or immediately life-threatening disease or condition, and no comparable or satisfactory alternative therapy, to gain access to an investigational drug or biological product (i.e., one that is still being studied for safety and effectiveness to treat that disease or condition). Many patients may be able to obtain the investigational product by participating in a clinical trial testing the drug or biologic, but expanded access helps those patients who do not qualify for a clinical trial or otherwise cannot participate.
There are some administrative aspects involved in expanded access – but we’re making them easier. Until now, for non-emergency situations for individual patients, physicians had to fill out forms online and submit their written request for their patient to obtain an investigational drug – by “snail mail” or, after checking with the FDA, by email or fax. For busy physicians, filling out an application in writing can be an extra burden.
To make expanded access easier, the Reagan-Udall Foundation for the FDA, with input from experts at the FDA, has launched “Expanded Access eRequest” that provides users a streamlined and convenient experience for applying for expanded access for individual patients in non-emergency settings.
Expanded Access eRequest:
- Allows physicians to request expanded access for individual patients in non-emergency settings;
- Helps physicians determine whether expanded access is appropriate for their patients;
- Walks physicians through the process of electronically completing and signing Form FDA 3926 and submitting it to the FDA;
- Provides a single site for uploading relevant documents;
- Offers useful links and resources.
Expanded access for emergency (and non-emergency) situations may be appropriate when:
- The patient has a serious or immediately life-threatening disease or condition;
- There is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition;
- Patient enrollment in a clinical trial is not possible;
- Potential patient benefit justifies the potential risks of treatment and these risks are not unreasonable in the context of the disease or condition to be treated;
- 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.
In an age where time is critical and a health care provider’s focus needs to be primarily on the clinical aspects of a patient’s care, we wanted to provide an easier, more streamlined approach to apply for expanded access.
The Reagan-Udall Foundation for the FDA is an independent 501(c)(3) organization created by Congress to advance the many missions of the FDA. Expanded Access eRequest is one of many ways the foundation is accomplishing that goal.