Treating a Patient with an FDA Unapproved Drug or Device

Use of Unapproved Drug for Treatment of a Patient

The guidance below isfor use of an unapproved drug to treat a patient or patients, outside of a clinical trial. The FDA must authorize any type of expanded access use in advance, even Emergency use for individuals, via an IND (Investigational New Drug). The Emory IRB also must approve in advance any Expanded Access use, except for emergency use situations in which it is not possible to obtain prospective IRB review/IRB chair concurrence. 

In addition, FDA may approve a waiver of full IRB review in response to a request for alternative IRB review when submitting Form FDA 3926 to the FDA. If this process is approved, you will only need IRB chair concurrence before treatment begins. This could be possible for both, emergency and non-emergency single, expanded access uses. If the FDA does not approve the process or you do not request the full IRB  review waiver during the Form FDA 3926 submission, you will be required to submit a new study in eIRB. We could assist you with that process by giving you a link to a submission with pre-populated information. 

  • For information about what would qualify as drug treatment use, follow this link.
  • For instructions on how to submit an Expanded Access use request to the FDA, please review the IRB P&Ps, or this FDA webpage
  • The FDA also has a Q&A at this link and specific guidance about requests using Form FDA 3926 at this link.

If you need assistance with the expanded accesssubmission to the FDA, please contact the Office of
Compliance at compliance@emory.edu or at (404) 727-2398. 

Please follow the relevant instructions below, based on the type of Expanded Access: whether the use is
considered a non-emergency “compassionate use”, or an emergency use.

Use of Unapproved Device for Treatment of a Patient

This guidance is for use of an unapproved medical device to treat a patient outside an IDE protocol, or use of an unapproved device not under an IDE. Please follow the relevant instructions below, based on whether the use is considered a non-emergency treatment “compassionate” use, or an emergency use (both are types of Expanded Access uses).

  • For information about expanded access including what would qualify and submission to FDA, follow this link
  • For additional instructions on how to submit an Expanded Access use request to the FDA, please review the IRB P&Ps.

If you need assistance with the submission to the FDA, please contact the Office of Compliance at compliance@emory.edu or at (404) 727-2398.

Please follow the relevant instructions below, based on the type of Expanded Access: whether the use is
considered a non-emergency “compassionate use”, or an emergency use.

Emergency use of an unapproved drug

The treatment use in an emergency situation does not need IRB approval. FDA needs to authorize the use before treatment occurs. The IRB should receive a report within 5 business days of the expanded access emergency use. This could be done via email to an IRB Education and QA team member or via the eIRB submission is one is required.

Submission process if alternative waiver of full IRB review was selected

If you submitted Form FDA 3926 to the FDA and selected the box under the Field 10.b (request for authorization to use alternative IRB review procedures), you will need to contact a member of the IRB Education and QA team to help you with obtaining IRB Chair concurrence. In order to secure IRB chair concurrence, please email us the following information:

  • A copy of all information submitted (or that you need to submit) to the FDA in connection with the Expanded Access use request, including Form FDA 3926
  • Informed consent form used (or to be used) or information demonstrating qualification for Emergency Use exception from informed consent if consent was not secured before See the IRB P&P entitled: Waiver or Alteration of Informed consent for Research, subsection entitled Emergency Medical Care Exception.

Exception to the Requirement to Obtain Informed Consent for the Use of a FDA-Regulated Item in Emergency Medical Care Situations. Use the IRB consent document for expanded access use when creating the consent document.

  • Documentation of FDA authorization for the Expanded Access Use This could be an email communication.

Our staff will email our IRB chair this information and let you know if there are any changes required before the IRB concurrence letter can be issued. The IRB staff will create a submission shell with this information for reference.

If alternative waiver of full IRB review was not requested or not approved by FDA

For assistance with the eIRB submission process, you may want to review this instructional video or contact the IRB Education and QA team. We can provide you will a pre-populated submission template to use. You will find that several sections do not apply to your situation. Please fill in these fields with “N/A” and the IRB will alert you if additional information is required.

The eIRB submission should include the following: In the “Informed Consent Process” section:

  • A copy of the informed consent that was used, if Please use our template, which can be found here.
  • If consent was not obtained before use, please let us know why, and upload in the informed consent process section instead the informed consent form. The physician and a licensed physician who is not participating in the medical care protocol must certify in writing that:
    • The patient was under a life-threatening
    • There was an inability to communicate with or obtain legally effective informed consent from the patient.
    • There was not sufficient time to obtain informed consent from the subject or legally authorized
    • There was no available alternative method of FDA-approved or generally recognized therapy that provided an equal or greater likelihood of saving the patient’s

In the “Research Design” section:

A copy of all information submitted to the FDA in connection with the Expanded Access use request, under the protocol section, especially:

  • Reason for intended treatment use of the Investigational New
  • List of available therapeutic options that would usually be tried before using the Investigational New Drug OR an explanation of why the Investigational New Drug is preferable to other available
  • Criteria for patient selection for Expanded Access if the use is for more than one individual OR a description of patient’s disease/condition, medical history and previous treatment for Expanded Access for an individual
  • Dose and method of administration for the Investigational New Drug and duration of
  • Description of the clinical procedures, laboratory test or other monitoring necessary to evaluate the effects for the drug and minimize its

In the “Drugs” section:

  • Drug information under the “Drugs” This is triggered by selecting “Investigational drug or

investigational use of an approved drug” in the “Biomedical Research” section of the smartform.

  • Documentation of FDA authorization for the Expanded Access Use

NOTE: If you are not collecting information for research, the initial HIPAA related questions in the smartform may be answered “no,” allowing you to skip further HIPAA questions.

Non-Emergency Treatment or "Compassionate" use of an Unapproved Drug

For non-emergency treatment/compassionate use, you are required to secure both IRB approval and FDA authorization before use.

Submission process if waiver of full IRB review was selected

 If you submitted Form FDA 3926 to the FDA and selected the box under the Field 10.b (request for authorization to use alternative IRB review procedures), you will need to contact a member of the IRB Education and QA team to help you with obtaining IRB Chair concurrence before the use. In order to secure IRB chair concurrence, please email us the following information:

 A copy of all information submitted (or that you need to submit) to the FDA in connection with the Expanded Access use request, including Form FDA

  • A clean copy of the informed consent form on the required template. Please, do not tamper with the header, but please do add a version date at the bottom of the
  • Documentation of FDA authorization for the Expanded Access Use request

Our staff will email our IRB chair this information and let you know if there are any changes required before the IRB concurrence letter can be issued. The IRB staff will create a submission shell with this information for reference.

After the use, you are required to notify us within 5 days of use about the status of the patient. The FDA will need a similar report.

If the alternative IRB review was not requested or FDA has requested IRB review

You will need to create a new submission in eIRB. For assistance with the eIRB submission process,, you may want to review this instructional video or contact the IRB Education and QA team for assistance. You will find that several sections do not apply to your situation. Please fill in these fields with “N/A” and the IRB will alert you if additional information is required.

The eIRB submission should include the following: In the “Informed Consent Process” section:

  • A copy of the informed consent that was used, if Please use our template, which can be found here.

In the “Research Design” section:

A copy of all information submitted to the FDA in connection with the Expanded Access use request, under the protocol section, especially:

  • Reason for intended treatment use of the Investigational New
  • List of available therapeutic options that would usually be tried before using the Investigational New Drug OR an explanation of why the Investigational New Drug is preferable to other available
  • Criteria for patient selection for Expanded Access if the use is for more than one individual OR a description of patient’s disease/condition, medical history and previous treatment for Expanded Access for an individual
  • Dose and method of administration for the Investigational New Drug and duration
  • Description of the clinical procedures, laboratory test or other monitoring necessary to evaluate the effects for the drug and minimize any side effect.

In the “Drugs” section:

  • Drug information under the “Drugs” This is triggered by selecting “Investigational drug or investigational use of an approved drug” in the “Biomedical Research” section of the smartform.
  • Documentation of FDA authorization for the Expanded Access Use

NOTE: If you are not collecting information for research, the initial HIPAA related questions in the smartform may be answered “no,” allowing you to skip further HIPAA questions.

Treatment or "Compassionate" use of an unapproved medical device (non-Emergency)

FDA approval and IRB chair concurrence are required before the use of the device. The submissions to the FDA and email to IRB can occur simultaneously, but both are needed before use.

You are no longer required to create a new submission in eIRB. Instead, you need to contact a member of the Education and QA team to help you with obtaining IRB Chair concurrence before the use. In order to secure IRB chair concurrence, please email us the following information:

  • A consent form. Please use our current template.
  • A description of the circumstances necessitating the use (do not include patient identifiers)
  • IDE protocol with description of device and name of IDE
  • Copy of uninvolved physician’s assessment of
  • Copy of authorization from IDE
  • Timeline for FDA submission

If you are already working with a staff member, please email it to them.  Our staff leadership or any member of our QA and Education team can help.  Our staff will email our IRB chair this information and let you know if there are any changes required before the IRB concurrence letter can be issued. After receiving this letter, you will be required to:

  • Notify the sponsor (if you do not hold the IDE) or FDA about the sucess or failure of the procedure, whether any problems ocurred with the use of the device, a summary of the patient outcome and the patient protections meansures that were followed within 45 days after treatment took place.
  • Provide a copy of the FDA communication agreeing with the use for our records.

The IRB staff will create a shell in eIRB with this information for reference.

Emergency use of Unapproved Medical Device

Unlike treatment (“compassionate”) use, the use under an emergency situation does not need IRB or FDA approval before use. Emergency use of an unapproved device to save a patient’s life may occur when:

  • An IDE does not exist (IDE: Investigational Device Exemption. The FDA requires an IDE when doing research with an unapproved medical device. For more information, please follow this link) OR
  • Patient does not qualify for the IDE protocol or the physician wants to use the device in a way not approved under an IDE OR
  • Physician is not an investigator under an

In order for the use of an unapproved device to qualify as Emergency use, the following conditions must exist:

  • The patient has a life-threatening or serious disease or condition AND
  • There is no generally acceptable alternative treatment AND
  • There is no time to obtain FDA approval for the use because the unapproved medical device needs to be used in the patient immediately

Before using the device, the physician should take as many of the following patient protection measures as possible, and provide the following information in the submission:

  • Obtain a written independent assessment of the use of the device by an uninvolved
  • Obtain documented informed consent from the patient or patient’s legally authorized. Use our template to create this document.
  • If consent was not obtained before use, please let us know why, and upload in the informed consent process section instead the informed consent form. The physician and a licensed physician who is not participating in the medical care protocol must certify in writing that:
    • The patient was under a life-threatening
    • There was an inability to communicate with or obtain legally effective informed consent from the
    • There was not sufficient time to obtain informed consent from the subject or legally authorized
    • There was no available alternative method of FDA-approved or generally recognized therapy that provided an equal or greater likelihood of saving the patient’s
  • Obtain documented authorization from the holder of the IDE for the Investigational Medical Device, if an IDE exists.
  • Notify the Emory IRB (via call or email), and provide the Emory IRB with a written description of the circumstances necessitating the use of the device, along with copies of the uninvolved physician’s assessment, informed consent, and the IDE holder’s

Prior FDA approval for shipment or use of the device is not necessary but emergency use needs to be reported to the FDA and IRB. The information to the FDA should be submitted by the sponsor (IDE holder). If the unapproved medical device is not under an IDE, the investigator should submit the information directly to the FDA.

Report the emergency use to the Emory IRB in writing within five business days. In this case, you need to create a new submission in eIRB. We can provide you with a pre-populated submission if requested. If you want assistance, you may want to review this instructional video or contact the IRB Education and QA team for assistance.

NOTE: If you are not collecting information for research, the initial HIPAA related questions in the smartform may be answered “no,” allowing you to skip further HIPAA questions.

Please include the following information in your submission:

  • In the Informed Consent Process section:
    • A copy of the informed consent that was If a consent form was not used, please let us know why consent was not obtained (as explained above), and upload your justification under the informed consent process section instead of the informed consent. For example, if there was not time to consent the subject, please explain how the subject (or legally authorized representative) was informed after the use. You may submit an unsigned copy of the informed consent, so you can obtain documentation of consent after the IRB reviews the use
  • In the “Medical Devices” section:
    • Device information (including device manual)
    • Copy of authorization from IDE holder (sponsor).
  • In the “Research Design” section:
    • A description of the circumstances necessitating the Don’t include patient identifiers, but do describe the medical condition of the patient and why the device is needed.
    • IDE protocol with description of device and name of IDE holder, if this
    • Copy of an uninvolved physician’s assessment of
    • Information submitted to the FDA (including FDA acknowledgement if already obtained).

 

 

Expanded Access Mechanism

 

Criteria for Use

 

When Can It Be Used?

 

Number of Patients to be Treated

 

FDA

Approval Needed?

 

How is FDA Approval Obtained?

 

Patient Protection Measures

 

Emergency Use

1.  Life- threatening condition‡;

2.  No alternative; and

3.  No time to obtain FDA approval.

Before or after initiation of clinical trial

Limited to few patients

No; submit report to FDA

following device use

N/A

1.  Independent assessment by uninvolved doctor;

2.  IRB chairperson's concurrence;

3.  Institutional clearance; and

4.  Informed consent

Compassionate Use

1.  Serious disease or condition and

2.  No alternative.

During clinical trial

Individual patient or small groups of patients

Yes

IDE supplement with:

1.  Explanation of circumstances constituting need for the device;

2.  Reasons alternatives not acceptable;

3.  Deviations from protocol, if any; and

4.  Patient protection

measures.

1.  Independent assessment by uninvolved doctor;

2.  IRB chairperson's concurrence;

3.  Institutional clearance; and

4.  Informed consent.

From FDA website

References