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201509247

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UWHC,

Policies,Clinical,UWHC Clinical,UWHC-wide,Legal Affairs

Compassionate Use of an Investigational Agent or Use of an Investigational Agent from a Second Institution (4.27)

Compassionate Use of an Investigational Agent or Use of an Investigational Agent from a Second Institution (4.27) - Policies, Clinical, UWHC Clinical, UWHC-wide, Legal Affairs

4.27

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Administrative (Non-Clinical) Policy
Category:
 UWHC only (Hospital Administrative-entity wide)  UWMF only (entity wide)
 UWHC Departmental (indicate name)  UWMF Departmental (indicate name)
 UWHC and UWMF (shared)
Policy Title: Compassionate Use of an Investigational Agent or Use of an
Investigational Agent from a Second Institution (policy used by UWHC and
UWMF)
Policy Number: 4.27
Effective Date: September 1, 2015
Chapter: Legal Affairs
Version: Revision
I. PURPOSE

To coordinate and facilitate the “compassionate use” of investigational drugs in accordance with federal
regulations, applicable accreditation organizations, institutional and professional standards, sponsor
stipulations and UW Health Hospital Administrative policy-4.11(Investigational and Study Drug
Control). Even though not a term used within the FDA regulations governing the use of investigational
drugs, “compassionate use” is commonly understood to describe a way to provide an investigational
therapy to a patient who is not eligible to receive that therapy under a clinical trial but who has a serious
or life-threatening illness for which other treatments are not available.

This policy also summarizes procedures involved when a patient is treated at the University Wisconsin
Hospital and Clinics (UWHC) with an investigational drug in research conducted at another institution.

II. POLICY

Uses of non FDA approved (investigational) drugs at UWHC fall into four categories:

Category:

1.0 Investigational drugs used in a clinical drug research protocol approved by the UW Health Sciences
Institutional Review Board (HS-IRB) or any other IRB that the UW and UWHC have agreed may serve
as an IRB of record on the UW Health Entity’s behalf ("Other Approved IRB"). See UW Health Hospital
Administrative policy-4.11(Investigational and Study Drug Control) for information about this category.

2.0 Investigational drugs obtained from a manufacturer for compassionate use (e.g., a drug available
under a treatment IND (Investigational New Drug Application)) approved by the HS-IRB. See section
IV.A for appropriate procedures.

3.0 Investigational drugs obtained from a manufacturer for compassionate use where the FDA has waived
IRB approval (for example Group C drugs provided by the National Cancer Institute). Although IRB

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approval is not required for federal purposes, HS-IRB approval is still required per local institutional
policy. See section IV.A for appropriate procedures.

4.0 Investigational drugs obtained from a patient or another institution through the patient's participation
in a research study at that institution. See section IV.B for appropriate procedures.

III. FORMS

The HS-IRB submission form ("Application for Emergency or One-Time Use of an Investigational Drug
or Device Form") is available within the HS-IRB’s electronic submission system, ARROW, which can be
accessed via https://kb.wisc.edu/hsirbs/17466. For submission and consent form guidance contact
the HS-IRB office (263-2362).

IV. PROCEDURE
A. INVESTIGATIONAL DRUGS OBTAINED FOR COMPASSIONATE USE REQUIRING
IRB APPROVAL (CATEGORY 2 and 3 above) at UWHC
1. The compassionate use of investigational drugs requiring HS-IRB approval (category 2)
is regulated by the FDA and falls into two classifications: Non-Emergency Use (under a
Treatment IND) and Emergency Use. Emergency use is defined as the use of an
investigational drug or biological product with a human subject in a life-threatening
situation in which no standard treatment is available and in which there is not sufficient
time to obtain (prior) IRB approval, or in some cases, an IND.
2. Both situations require drug handling by the Department of Pharmacy's Pharmaceutical
Research Center (PRC) and similar regulatory processes but differ in the timing of HS-
IRB approval as summarized in the table below (see points 4 and 5 below for detailed
description of non-emergency and emergency procedures). The PRC program will report
emergency compassionate use activities to the HS-IRB (immediately or on the first
available business day) and the Pharmacy and Therapeutics Committee (on a monthly
basis as part of the PRC/HS-IRB Drug Studies report).
Classification HS-IRB Requirements Informed Consent
Non-Emergency Prior HS-IRB submission and
Approval
Yes
Emergency Use Prior HS-IRB Notification
(whenever possible) and HS-
IRB submission within 5
days
Yes, but see IV.A.3
3. The investigator is required to obtain informed consent of the subject or the subject's
authorized representative, even for emergency use, unless both the investigator and a
physician who is not otherwise participating in the clinical investigation certify in writing
all of the following:
a. The subject is confronted by a life-threatening situation necessitating the use of
the investigational agent.
b. Informed consent cannot be obtained because of an inability to communicate
with, or obtain legally effective consent from, the subject.
c. Time is not sufficient to obtain consent from the subject's representative.
d. No alternative method of approved or generally recognized therapy is available
that provides an equal or greater likelihood of saving the subject's life.

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4. Non-Emergency Use:
a. The requesting physician or designee will contact the PRC pharmacist (24/7 on-
call pager #2717) and alert him/her of the interest in obtaining an investigational
drug on a non-emergency "compassionate use" basis (usually under a treatment
IND).
b. The physician or designee will contact the drug manufacturer and determine if
the investigational drug is available for use in the presenting patient and
situation. If use is permitted by the manufacturer, continue with remaining steps
below.
c. The physician will submit an HS-IRB Application for Emergency or One-Time
Use of an Investigational Drug or Device Form and the proposed consent form to
the HS-IRB via the ARROW submission system.
d. The physician must re-contact the PRC program (24/7 on-call pager #2717) to
arrange drug-handling procedures. PRC will provide shipment address
information, prepare physician order template(s), preparation and/or dispensing
instructions and inform appropriate internal and external parties as applicable.
e. Once HS-IRB approval has been received and the appropriate procedures are in
place, the investigational drug can be released by PRC for administration.
5. Emergency Use:
a. The requesting physician or designee will contact the PRC pharmacist (24/7 on-
call pager #2717) and alert him/her of the interest in obtaining an investigational
drug on an emergency basis for a single patient.
b. The physician will contact the manufacturer and determine if the investigational
drug is available for use in the presenting patient and situation. If use is permitted
by the manufacturer, continue with the remaining steps below.
c. The physician will then immediately contact the HS-IRB whenever possible and
notify them of the situation.
d. The physician will then immediately re-contact the PRC pharmacist (24/7 on-call
pager #2717). PRC will contact the manufacturer and arrange for appropriate
shipment*, assist with the development of physician order(s), and if required,
create preparation and dispensing procedures for involved personnel.
i. If weekday, send to:
Pharmacy Services Department
ATTN: Pharmaceutical Research Center
University of Wisconsin Hospital and Clinics
600 Highland Ave, E5/125-1530
Madison WI 53792
ii. If weekend specific addresses will be provided by the PRC Pharmacist.
e. The physician will obtain consent from the patient or the patient's authorized
representative prior to administering the drug, unless criteria for consent
exception are met as described under IV.A.3 above. For assistance with consent
form development see section III. above.
f. Within 5 days after the initial use of the investigational drug, the physician will
submit via ARROW, an Application for Emergency or One-Time Use of an
Investigational Drug or Device and a copy of the signed consent form (patient
name/signature should be concealed to preserve confidentiality - all dates of
signatures are to remain visible); to the HS-IRB.
B. INVESTIGATIONAL DRUGS OBTAINED FOR COMPASSIONATE USE REQUIRING
IRB APPROVAL (CATEGORY 2 and 3 above) – locations not under the purview of the
UWHC Pharmacy Department’s Pharmaceutical Research Center (PRC).

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1. The compassionate use of investigational drugs requiring HS-IRB approval (category 2)
is regulated by the FDA and falls into two classifications: Non-Emergency Use (under a
Treatment IND) and Emergency Use. Emergency use is defined as the use of an
investigational drug or biological product with a human subject in a life-threatening
situation in which no standard treatment is available and in which there is not sufficient
time to obtain (prior) IRB approval, or in some cases, an IND.
2. The investigator is required to obtain informed consent of the subject or the subject's
authorized representative, even for emergency use, unless both the investigator and a
physician who is not otherwise participating in the clinical investigation certify in writing
all of the following:
a. The subject is confronted by a life-threatening situation necessitating the use of
the investigational agent.
b. Informed consent cannot be obtained because of an inability to communicate
with, or obtain legally effective consent from, the subject.
c. Time is not sufficient to obtain consent from the subject's representative.
d. No alternative method of approved or generally recognized therapy is available
that provides an equal or greater likelihood of saving the subject's life.
3. Non-Emergency Use:
a. The physician or designee will contact the drug manufacturer and determine if
the investigational drug is available for use in the presenting patient and
situation. If use is permitted by the manufacturer, continue with remaining steps
below.
b. The physician will submit an HS-IRB Application for Emergency or One-Time
Use of an Investigational Drug or Device Form and the proposed consent form to
the HS-IRB via the ARROW submission system.
c. An investigator is responsible for the control of study drugs used in a research
project in accordance with federal, sponsor, and institutional policy.
4. Emergency Use:
a. The physician will contact the manufacturer and determine if the investigational
drug is available for use in the presenting patient and situation. If use is permitted
by the manufacturer, continue with the remaining steps below.
b. The physician will then immediately contact the HS-IRB whenever possible and
notify them of the situation.
c. The physician will obtain consent from the patient or the patient's authorized
representative prior to administering the drug, unless criteria for consent
exception are met as described under IV.A.3 above. For assistance with consent
form development see section III. above.
d. An investigator is responsible for the control of study drugs used in a research
project in accordance with federal, sponsor, and institutional policy.
e. Within 5 days after the initial use of the investigational drug, the physician will
submit via ARROW, an Application for Emergency or One-Time Use of an
Investigational Drug or Device and a copy of the signed consent form (patient
name/signature should be concealed to preserve confidentiality - all dates of
signatures are to remain visible); to the HS-IRB.
5. The Investigator shall comply with all other polices applicable to that location.
C. INVESTIGATIONAL DRUGS OBTAINED FROM A PATIENT OR ANOTHER
INSTITUTION THROUGH THE PATIENT'S PARTICIPATION IN A RESEARCH
STUDY AT THAT INSTITUTION.
1. The procedures outlined here apply when UWHC is NOT a performance site and is NOT
responsible for the research (no UW faculty sub-investigators nor systematic monitoring,
collecting or reporting of data to the other institution except as it relates to the care of the

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patient). There are two likely scenarios in which investigational drugs from another
institution will be encountered at UWHC:
a. When the treatment or hospitalization at UWHC is incidental to the research and
unanticipated.
b. When the patient needs to leave the research institution (for example to return
home), continue investigational or study drug administration and requires
medical monitoring.
2. In both of the above scenarios, UW/UWHC is providing incidental medical care in
relation to use of the investigational drug and is not a research site. UW/UWHC staff are
not investigators and HS-IRB does not need to review the protocol. Even though the
investigational drug is being administered at UWHC, only routine medical monitoring is
conducted with little or no reporting to the second institution.
3. Prior to continuing or administering the investigational or study drug at UWHC, the
physician should:
a. Contact the PRC program (24/7 on-call pager #2717) for instructions on drug
handling procedures.
b. Obtain necessary information to safely continue the investigational drug from the
clinical investigator. This information might include a description of treatment
procedures, warnings of possible adverse reactions, emergency procedures or a
copy of the signed informed consent.
D. COMPLIANCE AUDIT/MONITORING AND REPORTING MECHANISM FOR NON-
COMPLIANCE WITH COMPASSIONATE USE PROCEDURE
1. Compliance Monitoring Procedures:
a. PRC will notify the HS-IRB upon receipt of an emergency "compassionate use"
request and provide physician name, agent being requested and estimated
treatment initiation date; if request received after HS-IRB hours, PRC will notify
the HS-IRB at the start of the next HS-IRB business day.
b. PRC will update the HS-IRB with actual treatment initiation date or if the
emergency use agent was abandoned prior to treatment initiation.
c. Acting on the behalf of the HS-IRB, the PRC will audit consent compliance for
high risk "compassionate use" applications (e.g. situations with no dedicated
study coordinator) within 24 business hours of "compassionate use".
Discrepancies within the consent process will be forwarded immediately to the
HS-IRB.
2. Non-Compliance Procedures - Failure to submit to the HS-IRB within the required time
limits and, for non-emergency use, to await HS-IRB approval before instituting
"compassionate use" therapy will result in the HS-IRB taking appropriate action in
accordance with Federal Regulations. Additional information on non-compliance can be
obtained from HS-IRB website: https://kb.wisc.edu/hsirbs/.
V. REFERENCES

FDA Information Sheets 1998 Update (Use Of Investigational Products When Subjects Enter A Second
Institution)
Federal Register
21 CFR Parts 312.34
21 CFR Parts 56.102
21 CFR Parts 56.104
21 CFR Parts 50.23


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VI. COORDINATION

Sr. Management Sponsor: SVP, Chief Operating Officer (COO)
Author: Manager of Pharmaceutical Research Center

Approval Committee: Administrative Policy and Procedure Committee

SIGNED BY

Ronald Sliwinski
President of University of Wisconsin Hospitals and Chief of Clinical Operations


Revision Detail:

Previous revision: December 2014
Next revision: September 2018