How Does a Physician Request Single-Patient Expanded Access?
The FDA requires that a licensed physician apply for single-patient EA on your behalf. Your treating physician must complete each of the following steps to request single-patient expanded access for you:
- Request permission from the pharmaceutical company
- Request permission from the FDA
- Obtain approval from an IRB
- Obtain the patient’s informed consent
- Administer the treatment and report serious and unexpected adverse events and outcomes during the treatment
If your physician needs support in submitting an expanded access request specifically for a cancer therapy, he/she can contact Project Facilitate, a service offered through FDA's Oncology Center of Excellence.
Requesting EA from the Pharmaceutical Company
First, your physician will need to contact the pharmaceutical company to request use of the treatment. The pharmaceutical company will review the request, ask questions of the physician, and decide whether to grant access based on a number of considerations, including whether you are able to participate in a clinical trial, there is evidence to suggest you will benefit and that the potential risks do not outweigh the potential benefits. If the company does grant access to the treatment, they may work with your physician to develop a treatment plan. The company will also issue required documentation such as a Letter of Authorization (LOA) to your physician.
Requesting Permission from the FDA
After your physician’s request for EA has been approved by the pharmaceutical company, he or she must apply to the FDA for expanded access by submitting a completed form FDA 3926 and a copy of the required documentation. The FDA has up to 30 days to review the application but often reviews much more quickly – typically within two business days. During this time, the FDA will consider several factors, with an emphasis on weighing the potential risks against the potential benefits. The FDA may reach out to your physician with additional questions or to clarify the information provided. If the FDA allows the investigational treatment to proceed, they will provide the physician with an Investigational New Drug Application (IND) number.
Requesting Approval from an Institutional Review Board (IRB)
An IRB is a committee of medical and ethics experts (and community or legal representatives) who are charged with reviewing and approving biomedical research involving patients. Although EA is considered a treatment option and not formal research, because it involves investigational products, all patient safeguards must be adhered to, especially when it comes to conveying expectations and possible beneficial and harmful outcomes.
Your physician will present FDA forms, a treatment protocol, required documentation from the pharmaceutical company and the informed consent document to the IRB, which will review the application using a number of criteria.
Informed consent is critical to patient welfare and your physician is required by the IRB to obtain your informed consent for the proposed investigational therapy and protocol. Informed consent provides you with information about a protocol and clearly defines expectations and foreseeable outcomes and risks. Well-written informed consent includes an explanation of procedures, benefits and alternative treatment options (if they exist), as well as confidentiality of records and a clear explanation of your rights as a patient.
To ensure you understand all the information that your physician has provided:
- Ask your physician to clarify medical terms that you do not understand
- Ask for potential best- and worst-case scenarios and most likely outcomes
- Summarize what you have heard and ask your physician to confirm your summary
- Take notes and request written materials
Treatment and Next Steps
Once your physician has received informed consent from you and necessary permissions from the pharmaceutical company, the FDA and the IRB, the investigational treatment will be shipped from the pharmaceutical company for treatment. You will be expected to follow the treatment plan that your physician has outlined in his or her application process. During and after the treatment, your physician will report results and adverse events to the pharmaceutical company, the FDA and the IRB.