Treating a Patient with an FDA Unapproved Drug or Device

If you are seeking concurrence (as an alternative to full IRB approval), follow this guidance as we require the submission of an RNI to process your request.

 

Please see the sections below for additional information and instructions.

 

Email communication with the FDA

Emory LITS has established an encrypted, secured email connection with the FDA to send emails containing PHI for the purposes of an expanded access request or IND/IDE communication. This only applies when sending emails from an email address with these domains:

  • @emory.edu or
  • @emoryhealthcare.org 

You do not need to establish an individual account for secure email with the FDA as long as you are using an Emory email with the above-described domains.

More information can be found here:

Drug: Emergency use

Treatment use in an emergency situation does not need prior IRB approval unless you're seeking alternative IRB review procedures (see bolded section below). FDA needs to authorize the use before treatment occurs. The IRB should receive a report within 5 business days of the emergency use. This should be done via the eIRB submission (more info and how-to below).

Submission process if the alternative waiver of full IRB review was selected

If you submitted Form FDA 3926 to the FDA and checked the box under 10.b (request for authorization to use alternative IRB review procedures), you will need to obtain IRB Chair concurrence before the use. In order to secure IRB chair concurrence, please submit the following information using an RNI submission:  

  • 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, a subsection entitled Emergency Medical Care Exception.
  • 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. 

If the use already took place or an alternative waiver of IRB review was not requested or not approved by the FDA

You will need to submit a new study submission.  For assistance with the eIRB submission process, contact the IRB Education and QA team.  

In the “attach the protocol” section:

Upload a copy of all information submitted to the FDA in connection with the Expanded Access use request, including:

  • The reason for the emergency use of the investigational drug
  • A 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 options
  • A description of patient’s disease/condition, medical history and previous treatment for Expanded Access for an individual OR if more than one individual was treated, the criteria for patient selection
  • Dose and method of administration for the Investigational New Drug and duration of treatment
  • Description of the clinical procedures, laboratory tests or other monitoring necessary to evaluate the effects for the drug and minimize its side effects

In the “Drugs” section:

  • Drug information including drug's investigational brochure.
  • Documentation of FDA authorization for the emergency use

In the “Local Study Documents” section:

  • A copy of the informed consent that was used (unsigned). We prefer that you use our template, which can be found here.
  • If consent was not obtained before use, please let us know why, and upload that information in the informed consent process section instead the informed consent form. The treating 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 emergency
    • 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 representative
    • 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 life

NOTE: If you are not collecting information for research, answer the HIPAA questions as appropriate.  The HIPAA attestation and waiver document is not required if data is not being collected for research purposes.

Once Treatment is Complete

You should follow all reporting obligations and then close your IND with FDA. After the IND is closed, you can close your submission with the IRB.

For Oncology Patients

On June 3, 2019, the U.S. Food and Drug Administration Oncology Center of Excellence announced a new pilot program to assist oncology health care professionals in requesting access to unapproved therapies for patients with cancer. A new call center called Project Facilitate will be a single point of contact where FDA oncology staff will help physicians treating patients with cancer through the process to submit an Expanded Access request for an individual patient, including follow-up of patient outcomes.

The Project Facilitate phone number is 240-402-0004 and the email address is OncProjectFacilitate@fda.hhs.gov. Health care professionals may call during regular business hours, 9 a.m. to 5 p.m., Eastern Standard Time, Monday through Friday. Patients and families with questions can call 301-796-3400 or email druginfo@fda.hhs.gov.

Drug: Non-emergency treatment use

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 checked the box under 10.b (request for authorization to use alternative IRB review procedures), you will need to obtainin IRB Chair concurrence before the use. In order to secure IRB chair concurrence, please submit the following information using an RNI submission:  

  • A copy of all information submitted to the FDA in connection with the Expanded Access use request, including Form FDA 3926
  • Informed consent form  to be used 
  • Documentation of FDA authorization for the Expanded Access Use. This could be an email communication.
  • 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. 

After the use, you are required to follow all reporting requirements of FDA and IRB.

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

You will need to submit a new study submission.  For assistance with the eIRB submission process, contact the IRB Education and QA team.  

In the “attach the protocol” section:

Upload a copy of all information submitted to the FDA in connection with the Expanded Access use request, including:

  • The reason for the emergency use of the investigational drug
  • A 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 options
  • A description of patient’s disease/condition, medical history and previous treatment for Expanded Access for an individual OR if more than one individual was treated, the criteria for patient selection
  • Dose and method of administration for the Investigational New Drug and duration of treatment
  • Description of the clinical procedures, laboratory tests or other monitoring necessary to evaluate the effects for the drug and minimize its side effects

In the “Drugs” section:

  • Drug information including drug's investigational brochure.
  • Documentation of FDA authorization for the emergency use

In the “Local Study Documents” section:

  • A copy of the informed consent that was used (unsigned). We prefer that you use our template, which can be found here.
  • If consent was not obtained before use, please let us know why, and upload that information in the informed consent process section instead the informed consent form. The treating 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 emergency
    • 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 representative
    • 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 life

NOTE: If you are not collecting information for research, answer the HIPAA questions as appropriate.  The HIPAA attestation and waiver document is not required if data is not being collected for research purposes.

Once Treatment is Complete

You should follow all reporting obligations and then close your IND with FDA. After the IND is closed, you can close your submission with the IRB.

For Oncology Patients

On June 3, 2019, the U.S. Food and Drug Administration Oncology Center of Excellence announced a new pilot program to assist oncology health care professionals in requesting access to unapproved therapies for patients with cancer. A new call center called Project Facilitate will be a single point of contact where FDA oncology staff will help physicians treating patients with cancer through the process to submit an Expanded Access request for an individual patient, including follow-up of patient outcomes.

The Project Facilitate phone number is 240-402-0004 and the email address is OncProjectFacilitate@fda.hhs.gov. Health care professionals may call during regular business hours, 9 a.m. to 5 p.m., Eastern Standard Time, Monday through Friday. Patients and families with questions can call 301-796-3400 or email druginfo@fda.hhs.gov.

Device: Emergency use

Treatment use of a device in an emergency situation does not need prior IRB approval nor FDA approval. However, the emergency use is required to be reported to the FDA and IRB after treatment occurs. The report to the FDA should be submitted by the sponsor (IDE holder), if one exists, or by the treating physician if there is no IDE.

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
  • The patient does not qualify for the IDE protocol or the treating physician wants to use the device in a way not approved under an IDE OR
  • The physician is not an investigator under an IDE protocol

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 treating physician should take as many of the following patient protection measures as possible, (which should be documented in the follow-up submission to the IRB):

  • Obtain a written independent assessment of the use of the device by an uninvolved medical doctor
  • Obtain documented informed consent from the patient or the 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 of 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 legally authorized representative
    • 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 life.
  • 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 authorization

Report the emergency use to the Emory IRB in writing within five business days. You will need to submit a new study submission.  For assistance with the eIRB submission process, contact the IRB Education and QA team.  

Please include the following information in your submission:

In the “attach the protocol” section:

  • IDE protocol with a description of the device and name of IDE holder, if one exists
  • Copy of an uninvolved physician’s assessment of this use
  • Information submitted to the FDA (including FDA acknowledgment if already obtained).

In the "Local study documents" section:

  • A copy of the informed consent that was  used
    • If a consent form was not used, please let us know why consent was not obtained (as explained above), and upload your justification. For example, if there was not time to consent to 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
  • A description of the circumstances necessitating the use
    • don’t include patient identifiers but do describe the medical condition of the patient and why the device is needed.
In the “Device” section:
  • Device information (including device manual)
  • Copy of authorization from IDE holder (sponsor).

NOTE: If you are not collecting information for research, answer the HIPAA questions as appropriate.  The HIPAA attestation and waiver document is not required if data is not being collected for research purposes.

 

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

Device: Non-Emergency treatment use

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.

In order to secure IRB chair concurrence, please submit the following information using an RNI submission

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

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 success or failure of the procedure, whether any problems occurred with the use of the device, a summary of the patient outcome and the patient protections measures that were followed within 45 days after treatment took place.
  • Provide a copy of the FDA communication agreeing with the use for our records.

Required Reports for all treatment uses

You are required to follow all reporting requirements of sponsors, FDA, and the IRB. Failure to complete the required reports may result in determinations of non-compliance. The IRB also makes unanticipated problem determinations for serious adverse events related to treatment use under expanded access. Please contact a member of Team Q if you have any questions about expanded access reports.

References