Definition: FDA regulations at 21 CFR 312.300 (subpart I) allow certain individuals not enrolled in clinical trials to obtain expanded access to investigational drugs through various expanded access programs (EAPs). All expanded access programs must meet the basic criteria in 21 CFR 312.305(a). Specifically the FDA must determine:
1) The patient or patients to be treated have a serious or immediately life-threatening disease or condition, and there is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition;
2) The potential patient benefit justifies the potential risks of the treatment use and those potential risks are not unreasonable in the context of the disease or condition to be treated; and 3) Providing the investigational drug for the requested use will not interfere with the initiation, conduct, or completion of clinical investigations that could support marketing approval of the expanded access use or otherwise compromise the potential development of the expanded access use.
Steps to Prepare a Compassionate Use Submission:
- Contact the sponsor of the investigational drug/device to determine ability to use for patients. Find out from the sponsor who will be submitting the Investigational New Drug/Device (IND) application.
- Go to fda.gov and type in “Compassionate Use IND” in the Search box. This will bring up guidance on the submission of an Investigational New Drug application.
- Collect the following documents to be submitted to the Pediatric Research Office:
- Email approval from IRB for emergency use
- Letter to FDA. (Download the FDA letter template and complete all sections highlighted in red. After completed, sign and submit with other requested documents.)
- Signed and dated curriculum vitae
- If investigator to submit IND, collect the following documents from sponsor to be submitted to Pediatric Research Office:
- Contact name for representative at pharmaceutical company
- Letter of authorization
- Sponsor agreement/contract
- Investigational brochure and/or pharmacy manual
- Sponsor’s consent template
- Any other documents that sponsor is requiring to be completed
- Upon completion of all tasks, please contact Ashley Servais, PRO manager, by email or at 402-559-2511.