Expanded Access
Expanded Access is a way for patients with immediately life-threatening conditions or serious diseases to access investigational medical products (drugs, biologics, or medical devices) for treatment outside of clinical trials, especially when no comparable or satisfactory alternative therapy options are available.
Expanded Access is a way for patients with immediately life-threatening conditions or serious diseases to access investigational medical products (drugs, biologics, or medical devices) for treatment outside of clinical trials, especially when no comparable or satisfactory alternative therapy options are available.
As the Principal Investigator (PI), you must obtain FDA approval before using an investigational product under the FDA expanded access regulations. If the product is a drug or biologic under an IND (Investigational New Drug application), you need to get a Letter of Authorization from the sponsor to reference the IND.
Please note that the expanded access use of a drug, biologic, or medical device is treatment. NIH investigators are not allowed to perform additional research interventions or collect or analyze data for research purposes under an expanded access protocol.
You must obtain informed consent from the subject or their legally authorized representative (LAR), unless the requirements at 21 CFR 50.23(a) are met.
Patient eligibility for expanded access use of an investigational product
For a patient to be eligible for expanded access treatment, the treating physician must determine and document that the following criteria are met:
- The patient must have a serious or immediately life-threatening disease or condition with no comparable or satisfactory alternative therapy available to diagnose, monitor, or treat the disease or condition.
- The potential benefit to the patient justifies the potential risks of the treatment, and these risks are not unreasonable in the context of the disease or condition being treated.
- The expanded use of the investigational test article for the requested treatment will not interfere with the initiation, conduct, or completion of clinical investigations that could support marketing approval of the product.
This documentation should be recorded in the medical record and included in the submission to the FDA.
Additionally, to be enrolled in an NIH expanded access protocol, the person must already be enrolled in an NIH research protocol.
IRB review and approval for expanded access protocols
Prospective IRB review and approval must be obtained for all expanded access protocols, except in certain cases of individual patient use of investigational drugs, biologics, or devices.
If you are a physician submitting a new single patient expanded access IND, you may request a waiver from the FDA for full IRB review. To do this, you need to obtain concurrence from the IRB Chair before the treatment use begins. This option is available only for single patient non-emergency and single patient emergency use.
Reporting to FDA and the IRB
Reports that must be submitted to FDA include:
- Initial and follow-up safety reports
- Annual Reports (for INDs) and semi-annual reports (for IDEs) and
- Final report at the completion of treatment
Reporting to the IRB:
- NIH Investigators are responsible for reporting events to the IRB per Policy 801.
- Submit a Continuing Review application for protocols that will exceed a 12-month period.
- Notify the IRB when the protocol is complete.