Emergency Use Requests:
Access to Investigational Drugs, Devices and Biologics for Patients
FDA regulations ordinarily require prior approval from FDA and an IRB for research use of an unapproved or "investigational" drug, biologic, or device. Exceptions to this rule apply in very limited cases, including life-threatening emergencies. This webpage describes the circumstances when emergency use is appropriate and the process and procedures that must be followed before or immediately following an emergency use.
When is an Emergency Use Appropriate?
Emergency use of an unapproved or investigational drug, device or biologic may occur only in those circumstances where it is not feasible to secure prospective FDA and IRB approval. Emergency use is permitted only if all of the following criteria are met:
- The patient faces a life-threatening condition (e.g., a serious disease or condition such as a sight-threatening or limb-threatening condition, as well as other situations involving risk of irreversible morbidity) that requires immediate treatment.
- The patient is likely to benefit from treatment with the unapproved drug, biological, or device.
- No generally acceptable alternative for treating the patient is available.
- There is no time to use existing procedures to secure prospective FDA and IRB approval and/or informed consent.
- The drug, device or biologic has not previously been used in the past for emergency use for another patient at FH
FH Investigational Drug Service in consultation with the MCW HRPP office developed a process map which outlines this decision making process. In addition, the map identifies who treating physicians should contact if they are interested in pursuing access for an investigational drug, device or biologic for the treatment of 1 of their patients.
Absent an emergency, an unapproved or investigational drug, device or biologic may not be used without prior IRB (and sponsor/FDA) approval of a protocol or any necessary amendments.
What is the Procedure for Reporting an Emergency Use to the MCW HRPP Office?
Call (955-8337 or 955-4415) and email the MCW HRPP office (firstname.lastname@example.org) as soon as the decision to use an unapproved or investigational drug, device or biologic is made, prior to the treatment or procedure if at all possible.
- During after office hours, call 955-8439 and leave a message with call back number.
Treating physicians/principal investigators must file a formal report of the emergency use with the IRB within eBridge within 5 calendar days of the use. An additional 90 day follow up report is required to provide the IRB an update on the patient's outcome post-treatment.
When Must FDA Be Notified?
Even in an emergency where it is impossible to secure an IND or IDE in advance, FDA must be consulted prior to use. Contact information (24/7) for drugs/biologics and for devices is available on FDA’s website.
Note: if sponsor approval is secured, the treating physician/principal investigator is strongly encouraged to request written assurance from the sponsor that FDA approval also has been secured.
What Other Procedures are Required?
Upon confirmation the emergency use criteria have been met by the MCW HRPP office; treating physicians/principal investigators must notify and coordinate with FH Investigational Drug Services (IDS) and Office of Clinical Research & Innovative Care Compliance (OCRICC) to facilitate the securing of the drug, device or biologic and treatment of the patient. This workflow has been outlined in the below process map:
If time permits prior to treatment with an investigational agent, the treating physician/principal investigator should secure the following:
- An independent assessment by a physician uninvolved with the investigation, as required in 21 CFR 50.23. The assessment must certify the physician’s agreement that all of the criteria have been met:
- The subject was confronted by a life-threatening situation (including one involving risk of serious, irreversible morbidity), necessitating use of the product.
- At the time of the procedure, there was no available alternative method of approved or generally recognized therapy that would have provided equal or greater likelihood of saving the subject’s life or avoiding serious, irreversible morbidity.
- Informed consent from the recipient or recipient’s legally authorized representative,
- OR an outside physician’s certification that:
- (1) it was not feasible to secure the recipient’s legally effective consent (for example because the recipient was unconscious or sedated or legally incompetent); and
- (2) time was insufficient to secure consent from the recipient’s legal representative.
- Concurrence from the MCW HRPP office that there is an emergency or, preferably, prospective IRB approval (in which case the use is not an emergency use) exists.
- Permission from the IND or IDE holder of the drug, device or biologic (i.e., the sponsor of a trial/drug or device manufacturer).
- Contact FH Investigational Drug Service (IDS) to alert them of the emergency use and to begin their workflow process
- Contact FH Office of Clinical Research & Innovative Care Compliance to alert them of the emergency use.
Required activities for Treating Physician/Principal Investigator Following an Emergency Use
Promptly following an emergency use (and in any event within 5 calendar days), the PI must:
- Obtain an independent physician assessment if time did not permit prior to the treatment or procedure.
- Write or dictate a note or report including a statement of who administered the drug or placed the device and why it was used.
- Submit a report via eBridge.
- Submit a report to the sponsor (IND/IDE holder).
- If you are the sponsor, notify the FDA of the emergency use and provide FDA with a written summary of the conditions constituting the emergency, subject protection measures, and results.
- Evaluate the likelihood of a similar need for the drug/device occurring again.
- Refrain from further emergency use until any necessary approvals have been secured (e.g., opening of a new “high risk” arm on the trial), even if the conditions for an emergency use otherwise exist.
- Other than mandatory reports to FDA or the sponsor, do not use data related to the emergency use in any publication or report on a related research project.
Note: subject to VERY limited exceptions, FDA prohibits additional emergency uses that depart from approved investigational plans and IRB protocols.
Is it emergency use? A decision tree (PDF)
Emergency Use Workflow (PDF)
Cost Recovery Workflow/decision tree (PDF). This workflow outlines the process and decisions regarding the costs of the drug or device and if the costs will need to be recovered.