- Planned Treatment Uses with sufficient time to secure prospective IRB review
- Emergency Uses when there is insufficient time to secure prospective IRB review
Treatment Uses (Expanded Access or Compassionate Uses) of a Drug or Device:
Nature and timing of Submission and Review:
Treatment uses of investigational or restricted drugs or devices must receive initial review by the convened IRB. Please call the IRB office when preparing the submission because the IRB can make exceptions to the deadline for urgent reviews of treatment applications. If there are life-threatening circumstances, an emergency IRB meeting can be convened or the physician can pursue an emergency exemption from prospective IRB review.
The following documents must be included in all applications involving treatment use of a drug or device:
- Treatment Use Application (updated 06 Jun 2013)
- Treatment Protocol. A separate treatment protocol must be included. The application form was not designed to capture treatment plans.
- Conflict of Interest and Scientific Misconduct (updated 24 Dec 2012)
- Current (within 3 years) CVs for all physicians (if not on file with the IRB)
- If available, written, audio, or video information related to the drug or device to be provided to, or meant to be seen or heard by patients.
The following documents must also be included for Humanitarian Device Applications:
- Patient information using one or both of the following:
- FDA Summary of Safety and Probable Benefit document
- Description of the device, such as device labeling
The following documents must also be included for Treatment Use/Expanded Access Drug or Device Applications:
- Consent form for Treatment Use of a Drug (posted 23 May 2013)
- Description of the drug, such as an Investigator's Brochure, Package Insert, or Prescription Information
- Description of the device, such as device specifications
Number of copies:
Submit the signed original to IRB@NJHealth.org.
The physician is asked to call the IRB office immediately (303.398.1477) when a patient is faced with a life-threatening or severely debilitating condition and where a restricted-access drug or device is needed before the IRB can be convened to review the request.
The IRB office will determine whether the drug or device was used on an emergency basis in the past. With that information, the physician and IRB office will determine the best strategy for IRB review.
- If there is sufficient time for IRB review, the physician is asked to follow the procedures for planned Treatment Uses with sufficient time to secure prospective IRB review.
- If there is insufficient time to convene a meeting and the drug/device was not used on an emergency basis in the past, the institution may invoke the Emergency Exemption from Prospective IRB Approval (21 CFR 56.104(c)). The exemption allows for one emergency use of a test article at an institution without prospective IRB review. FDA regulations require that any subsequent use of the investigational product at the institution have prospective IRB review and approval.
Steps for pursuing an Emergency Exemption from Prospective IRB Approval:
Before administering the test article:
After the administering the test article:
- Prepare a memo describing the following conditions in detail to meet the conditions of an emergency exemption. The memo should describe why:
- The patient is confronted by a disease or condition that is life threatening.
- The situation necessitates the use of the drug/device.
- No standard acceptable treatment is available.
- There is not sufficient time to obtain IRB approval.
Submit the memo to the IRB office by email at IRB@NJHealth.org, Please call the IRB office (303.398.1477) to notify staff that the memo was sent so that staff can take immediate action.
- The IRB Chairperson (serving as an independent physician) will review the memo and determine whether the conditions are met for an emergency exemption.
If conditions are met, an IRB staff member will prepare a letter for the IRB Chairperson’s signature acknowledging awareness of the emergency use. This letter also serves as a reminder that any subsequent use of the test article requires prospective IRB review and approval.
- Prepare a treatment consent form. You can modify the manufacturer's consent template or you can customize the treatment use of a drug consent template or humanitarian use device consent template (by removing references to humanitarian use). It is not necessary to receive IRB review of this consent form.
- Obtain written informed consent from the patient. If consent is granted, you may administer the drug or device. If the patient is unable to provide consent, contact the IRB office for options.
- Submit a written follow-up report to the IRB within five (5) days of the emergency use. This report should include:
- Name of the test article
- Copy of the signed informed consent document
- Conditions under which the test article was administered
- Date and time administered
- Subject protection measures
- Any adverse events or unanticipated problems to recipient or others
- Outcomes, if known.
- An IRB Chairperson will review the notification and evaluate the appropriateness of the request and the nature of informed consent (if any).
- If the IRB Chairperson finds the information satisfactory, an IRB staff member will issue a letter acknowledging receipt of the notification and offer a reminder that medical information obtained from the emergency use of a test article cannot be claimed as research.
- Please evaluate the likelihood of a similar need for the test article. If future use is likely, we encourage you to initiate efforts to obtain IRB and FDA approval for the test article’s subsequent use.
Exception to the Requirement for Informed Consent:
Even for an emergency use, the treating physician is required to obtain informed consent from the patient or patient's legally authorized representative unless both the treating physician and another (independent) physician certify in writing all of the following [21 CFR 50.23(a)]:
- The patient is confronted by a life-threatening situation necessitating the use of the test article.
- Informed consent cannot be obtained because of an inability to communicate with, or obtain legally effective consent from, the patient.
- Time is not sufficient to obtain consent from the patient's legal representative.
- No alternative method of approved or generally recognized therapy is available that provides an equal or greater likelihood of saving the patient's life.
If, in the treating physician's opinion, immediate use of the test article is required to preserve the patient's life, and if time is not sufficient to obtain an independent physician's determination that the four conditions above apply, the treating physician should make the determination. Within 5 working days after the use of the article, have the determination reviewed and evaluated in writing by an independent physician. The investigator must notify the IRB within 5 working days after the use of the test article [21 CFR 50.23(c)].
Policy and guidance:
FDA guidance on Treatment Uses of Drugs: http://www.fda.gov/RegulatoryInformation/Guidances/ucm126495.htm
FDA IND application process: http://www.fda.gov/drugs/developmentapprovalprocess/howdrugsaredevelopedandapproved/approvalapplications/investigationalnewdrugindapplication/default.htm
FDA guidance on obtaining an IND for single patient use or Emergency Use: http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/ApprovalApplications/InvestigationalNewDrugINDApplication/ucm107434.htm
FDA guidance on obtaining an Emergency IND: http://www.fda.gov/RegulatoryInformation/Guidances/ucm126491.htm
FDA guidance on Treatment and Emergency Uses of Devices: http://www.fda.gov/MedicalDevices/DeviceRegulationandGuidance/HowtoMarketYourDevice/InvestigationalDeviceExemptionIDE/ucm051345.htm
FDA guidance on preparing an IDE: http://www.fda.gov/MedicalDevices/DeviceRegulationandGuidance/HowtoMarketYourDevice/InvestigationalDeviceExemptionIDE/ucm046706.htm (but the manufacturer will customarily submit the IDE for FDA review).