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Expanded Access Submissions

For Emergency Use, Compassionate Use, Expanded Access, and Humanitarian Use Device (HUDs), please contact irb-orp@psu.edu prior to beginning any paperwork.

 

Use the following format for a proposed Expanded Access, Compassionate or Emergency Use submission of an FDA-regulated drug or device and email it as instructed below.

Expanded Access/Compassionate Use/Humanitarian Use Device: 

Non-emergency individual patient expanded access/compassionate use of an investigational drug or device when a patient has a life-threatening or serious disease or condition. 

Complete a submission in CATS-IRB (irb.psu.edu), See “Expanded Access/Compassionate Use/HUD instructions”.  

 

Institutional approvals: (treating physician’s Department Chair), Chief Medical Officer, Dr. Kenneth Wood, kwood5@pennstatehealth.psu.edu, written notification to the Department of Health by contacting Matthew Dean, mdean@pennstatehealth.psu.edu, and if a Confidential Disclosure Agreement is provided by the Sponsor, the Office of Research Affairs, E-Contracts@pennstatehealth.edu (will need to be consulted prior to signing any documents), an email/letter from a Penn State Health unaffiliated physician are needed and can be uploaded into CATS-IRB as emails, See “Expanded Access CATS IRB Instructions”.

 

Emergency Use (Rare situation) 

To use a drug, biologic, or device under the emergency use category it must be a true emergency, e.g., a life threatening or severely debilitating situation. FDA (Food and Drug Administration) (Food and Drug Administration) guidance where these terms are clearly defined:  https://www.fda.gov/regulatory-information/search-fda-guidance-documents/emergency-use-investigational-drug-or-biologic.

  1. Confirm in the email that the circumstances meet the following criteria for emergency use:
    • The patient is confronted by a disease or condition that is life-threatening or severely debilitating, or the patient is confronted by a life-threatening disease or a serious condition requiring immediate use of an unapproved device.
    • The situation necessitates the use of the test article (drug, biologic, device).
    • No generally acceptable alternative for treating the patient is available.
    • There is insufficient time to obtain IRB approval. (Or if there is time for review at the next IRB meeting, indicate this and the information will be added to the agenda.)
  2. Attach the following materials to the email as supporting documentation:
    • Document or email that describes the patient’s medical condition and the reason for the use of the test article. (Do not include any identifiers in documents.)
    • Correspondence to and from the FDA with the investigational new drug (IND) number or investigational device exemption (IDE) number, if available. (For a single-patient IND or compassionate IDE, submit the number when it is available).
    • Correspondence to and from the sponsor regarding use of the test article.
    • Protocol or detailed treatment plan for this patient.
    • Investigator’s brochure or drug package insert, or device information.
    • Emergency use consent form to be used. (Use “HRP-582 – HSPO Consent Form” in CATS IRB.)
  3. Send the email and all subsequent communications about the emergency use to irb-orp@psu.edu or to staff member contacted; also copy other persons involved in coordinating the use.
  4. Keep the HRPP informed about the status of a proposed use to proceed or if circumstances change.
  5. By close of business within five days after the use of the device or first administration of the drug/biologic, submit to the IRB written notification of the emergency use of a test article in a life-threatening situation, and include a copy of the signed consent document used (or provide certification by the treating physician and an uninvolved physician that the criteria for the exception from informed consent were met).

 

Guidance documents and templates are available in CATS IRB, including:

  • HRP-023 - SOP All Emergency Use, Expanded Access for an Individual Patient (Drug Only), and Compassionate Use (Device Only) Review
  • HRP-027 - SOP  All Emergency Use, Expanded Access for an Individual Patient (Drug Only) and Compassionate Use (Device Only) Post Review
  • HRP-322 - Worksheet – Emergency Use
  • HRP-325 - Worksheet – Compassionate Use of an Unapproved Medical Device
  • HRP-582 - HRPP Consent Form for Emergency Use/Individual Patient Expanded Access/Compassionate Use
  • HRP-593 - Protocol for Humanitarian Use Device

 

 

HRP-593: Template Protocol for Individual Expanded Access/Humanitarian Use Device (HUD) 

Use this form for all situations. If the sponsor includes a protocol document, include both protocol documents under "IRB Protocol" to the submission in CATS-IRB.

Do not remove the grey instruction boxes

  1. ​Update to reflect this is an Individual Expanded Access of an Unapproved Drug/Compassionate Use of an Unapproved Medical Device OR HUD --Can include the medication/device name   
  2. Title Page:   
    • ​​Update with treating physician name   
    • ​Update with treating physician’s department, phone number and email address
    • ​Version Date (update with every change made/clarification submission)
  3. Section 1.0 - Description of the Unapproved Test Article
    • Section 1.1
      • Add Manufacturer/Sponsor information
    • Section 1.2
      • Provide the generic and trade name 
    • Section 1.3 
      • Add either IND, IDE or HDE number 
    • Section 1.4
      • Describe the indication for use
  4. Section 2.0 - Consent Process 
    • Describe the consent process and documentation, if applicable (not required with a HUD). Address how, when, and that the treating physican will obtain consent.  
  5. Section 3.0 - Risks and Discomforts 
    • Describe the potential risks and discomforts to patients and methods of minimizing risks. 
      • This should match the risk section within the consent document. *Sponsor will typically send a list of risks associated with the investigational item.  This may be found in a sponsor’s consent document. 
  6. Section 4.0 - Financial Information  
    • Describe what the patient will be told about the cost of the device/biologic/drug and how insurance/Medicare will handle billing for this intervention. *This should match the cost section in the local and sponsor's consent document. 
  7. Section 5.0 - Benefits 
    • Describe the potential benefits to patients.  This should match the benefits section in the local and sponsor's consent document.  
  8. Section 6.0: Device Storage Plan (Update title to reflect type of access/compassionate use requested within the protocol)
    • Describe how/where the device or drug will be stored prior to implantation/provided to the patient.

Documents to be submitted in all situations (Exception - no consent required for HUD, but can be used):

HRP-582 Template Consent Document: Remove the grey instructions boxes before submitting in CATS-IRB

Note: Use of a sponsor’s consent is allowed if one is provided but must include the treating physician’s and IRB’s contact information, along with the HRP-582, local consent language.

  1. Title: 
    • Update to reflect the type of request, delete other 2 permission statements 
  2. Title Page:
    • Update with treating physician name
    • Update Test Article- drug or device 
    • Update with treating physician phone number and 24-hour contact number. Typically, this is the on-call MD service. (Must match Section: ‘Who can I talk to?’) 
    • Update to reflect the subject taking part in the intervention (e.g., if it is ‘you,’ ‘your child’ or ‘your representative’) 
    • Update to include name of drug or device 
  3. Section: ‘What you should know about this experimental treatment’ 
    • Add any information that is not already provided
  4. Section: ‘How long will this experimental treatment last?’ 
    • Provide the length of time the entire course of treatment will last, (e.g., until it is not effective in relieving symptoms, etc.) 
  5. Section: ‘What happens if I get this experimental treatment?’ 
    • Provide in lay language what the process will be for the subject, include the drug/biologic/device used and if applicable how it is approved, how this will be ingested/infused/implanted, and frequency (e.g., infused day 1-15 of a 28-day cycle, implanted after completion of cardiac catheterization, etc.). 
  6. Section: ‘Is there any way this experimental treatment could be bad for me?’ 
    • Add all risks associated with the intervention, in lay language. *This is provided by the sponsor providing the drug/biologic/device. In cases where this drug or device is approved for other indications, the package inserts, or attachment should include a risk section.  This will also be stated in HRP-593, Section 3.0 and should match. 
  7. Section: ‘Can this experimental treatment help me?’  
    • The key to this section is to add the goal (e.g., alleviate symptoms related to your disease, keep your infection under control and stop it from getting worse, etc.). This will also be stated in HRP-593, Section 5.0 and should match. 
  8. Section: ‘What else will I need to know?’ 
    • Confidentiality risks 
    • Costs (add the information regarding drug/biologic/device will be provided free of charge. Sponsors will sometimes provide information, like this example: “There will be no cost to you for the investigational drug. Getting this treatment may lead to added costs to you. You and your insurance company will be charged for the health care services that you would ordinarily be responsible to pay. Insurance may not pay for this treatment because it is considered experimental.” This should match the cost section 4.0 in the HRP-593, Protocol.
    • Typically, only the injury language provided in the template is stated.  
    • Do not add HIPAA (Health Insurance Portability and Accountability) template language as this does not meet the definition of research.  
  9.  Section: ‘Who can I talk to?’ 
    • Update with treating physician phone number. This should match the treating physician phone number listed on the title page.  
    • Occasionally the sponsor will add an additional section: ‘Other options or choices do I have if I do not receive emergency/expanded/compassionate use treatment?’ 
    • Including all viable options for subjects outside of the emergency use/expanded/compassionate use treatment.  
    • You may also contact the Penn State Human Research Protection Program (HRPP) at (814) 865-1775 or visit the HRPP website at https://www.research.psu.edu/irb/particiapnts if you:
      • Have concerns, complaints or general questions
      • You may also call this number if you cannot reach the clinical team or wish to offer input or to talk to someone else about any concerns.
  10. Section: Signatures 
    • Update the signature lines to correctly indentify the individual taking part in the intervention (e.g., patient, LAR (Legally Authorized Representative), parent or guardian), and child (over the age of 7 and if applicable).

 
 

CATS - IRB Documentation - irb.psu.edu

Basic information Page:

  1. Question #1-Title of study:
    • Identify type of request: Expanded Access/ request for (drug/device) to treat (condition)
  2. Question #2-Short title:
    • Identify type of request:  Expanded Access
  3. Question #3-Brief description in lay language:
    • Identify type of request: Expanded Access – (drug/device company name)
  4. Question #4- What kind of study is this?
    • This should always be single-site study
  5. Question #5- Will an external IRB act as the IRB of record for this study?
    • This should always be answered ‘NO.’
  6. Question #6- Will your IRB act as the single IRB or record for other participating sites?
    • This should always be answered ‘NO.’
  7. Question #7- Principal Investigator:
    • Add treating physician
  8. Question #8- Does the investigator have a financial interest related to this research?
    • Answer as applicable – as this is not research, most times this should be no. 
  9. Question #9-Attach the protocol:
    • Add HRP-593

 

Study Funding Sources Page:

  • No funding should be added 

 

Study Team Members Page: 

  1. Question #1- Add all individuals that would be involved with this submission:
    • Co-investigator(s) (physicians that could in the future obtain access for their patients)
    • Project Coordinator(s) (involved in the IRB submission process)
  2. Question #2- Do not add anything to this question.

 

Study Scope Page: 

  1. Question #1-
    • Select Yes, if this is a drug or biologic,
    • Select no, if a device
  2. Question #2-
    • Select Yes, if this is a device or HUD,
    • Select no, if a drug or biologic 

 

Drugs Page:

Drug or Biologic (Expanded Access)

  1. Question #1-Add name of drug (Generic and Brand)
  2. Question #2-Select Yes
  3. Question #3-Add IND number and Investigator 
  4. Question #4-Attach any/all the following:
    • Drug or Investigator information/pamphlets/IBs 

 

Devices Page:  

Device (Expanded Access) HUD

  1. Question #1-Add name of drug or HUD 
  2. Question #2-Choose IDE or HUD
  3. Question #3-Add IDE or HDE
  4. Question #4-Attach any/all of the following: 
    • Device or Investigator information/pamphlets

 

Local Site Documents Page:

  1. Question #1-Consent forms: Add HRP-582 Consent Template and any sponsors consent template provided (may use only sponsors consent template, local consent requirements apply, e.g., contact information)
  2. Question #2-Recruitment materials: Do not add any items as there should not be any recruitment
  3. Question #3- Other Attachments: Add all the following:
    1. Category: Collaborating Approval Materials
      • Letter of support by unaffiliated (PSH) physician providing support for use of medication/device
      • Letter of support by treating physician’s department chair
      • FDA – IND/ide Correspondence/Approval (for each occurrence)  
      • Sponsor Correspondence/Approval (for each occurrence)
      • Chief Medical Officer Approval 
        • Any formal training requirements deemed necessary by the CMO
      • Department of Health notification email 
    2. Category: Other
      • Initial email from treating physician to HRPP requesting use
      • Subject facing instructions from sponsor 

 

Study Finder Recruitment Page: 

  1. Question #1-Select Not Applicable, the study does not involve active recruitment of participants 

 

Clinical Trials Page: 

  1. Question #1-Select No

 

Create STAR (Study Tracking and Analysis for Research) Submission Page:

  1. Leave the box unchecked 

 

Training Summary Page: 

  1. Nothing required

 

Final Page: 

  1. Click Save & Exit 
  2. Then, Click Submit