Presented by Fabienne San

Presented by Fabienne San

Institutional Review Bo

and 

Humanitarian Use Device 

Institutional Review Bo

Institutional Review Bo

and

Humanitarian Use Device 

Humanitarian Use Device 

Division of Bioresearch Monitoring 

Office of Compliance Center for Devices and Radiological Health Food and Drug Administration 

Division of Bioresearch Monitoring 

Office of Compliance Center for Devices and Radiological Health Food and Drug Administration
Learning objectives Learning objectives

z Define Humanitarian Use Devz Describe FDA’s approval z Identify the HDE-holder respoz Identify the physician responsz Describe the IRB responsibilit
Topics

• Humanitarian Use Device (H• Humanitarian Device Exemp• FDA review• HDE-holder responsibilities• Physician responsibilities• IRB responsibilities • FDA concerns
Humanitarian Use Device 

Humanitarian Use Device 

z HUD definition

– Title 21 CFR 814.3(n) – A device intended to benefit pa

treatment or diagnosis of a disecondition affecting fewer than 4individuals in the US per year 
Humanitarian Use Device 

Humanitarian Use Device 

z Office of Orphan Products De

designates a device as a HumUse Device (HUD)

– Verifies that the device is desig

or diagnose a disease or conditfollowing the parameters in the 

– Reviews a description of the de– Reviews a description of the ra

or condition
Humanitarian Device Exemptio

Humanitarian Device Exemptio

z HDE Definition

– Title 21 CFR 814.2– A premarket approval applicatio

submitted to FDA seeking a HuDevice Exemption from the effe

requirements of sections 514 athe Food, Drug, Cosmetic Act.
Humanitarian Device ExempApplication to FDA 

Humanitarian Device ExempApplication to FDA

z FDA approval of HDE applica

– HUD does not pose unreasona

injury to patients

– That the probable benefit outwe

injury from its use 
Humanitarian Device ExempApplication to FDA 

Humanitarian Device ExempApplication to FDA

z FDA approval of the HDE

HDE label states – The device is a Humanitarian U– The device, to treat or diagnose 

disease or condition, is authorizfederal law

– The effectiveness of this device 

has not been demonstrated
Humanitarian Device ExempApplication to FDA 

Humanitarian Device ExempApplication to FDA

z FDA approval of a HDE applic

allows the HUD to be markete
HDE-Holder HDE-Holder

z The HDE-holder must

– Have both HUD designation 

approved HDE from FDA bedevice is shipped to institutiInstitutional Review Board (oversight
HDE-Holder HDE-Holder

z The HDE-holder is responsibl

ensuring initial and continuing review

z The HDE-holder may wish to 

this by not shipping HUDs to iuntil after receiving documentIRB approval
HDE-Holder HDE-Holder

z The HDE-holder is respon

ensuring

– the HUD is not administered 

implanted in a patient prior obtaining IRB approval at thcare facility
HDE-Holder HDE-Holder

z The HDE-holder must

– Maintain IRB corresponden– Report clinical experience, 

safety information, to FDA ireports
Physician Responsibilities Physician Responsibilities

z Obtain IRB approval and cont

approval

z Follow IRB requirementsz Give patients HUD informatioz Report serious adverse event

deaths using the Medical DevReporting system at 21 CFR 8
IRB initial review IRB initial review of HUDs of HUDs

z Use approval criteria at 21 CF

– Consideration of the patient

for the HUD

– Likelihood that device is ap

for the patient’s condition or state 
IRB continuing review IRB continuing review of HU

of HU

z Follow written procedures for 

review

– Convened meeting, or– Expedited review is acceptable 

is an approved device
IRB review of HUDs IRB review of HUDs

z An IRB may approve the use 

HUD

– In general– For groups of HUD patients tha

certain criteria

– Under a HUD treatment protoco– On a case-by-case HUD basis 
IRB limitations on HUD use 

IRB limitations on HUD use 

z One or more measures of dise

progression

z Prior use and failure of alterna

treatment modalities
IRB limitations on HUDs use 

IRB limitations on HUDs use 

z Reporting requirements to IRB 

Chair

z Appropriate follow-up precaut

evaluations

z Any other criteria it determine

appropriate
IRB withdrawal of approv

IRB withdrawal of approv

z IRBs must be constituted and 

accordance with the agency’s regulations and withdraw app

– Failure to follow IRB or FDA req– Unexpected serious harm or de

z Questions to ask at continuing 

– Reporting serious adverse even

deaths

– Following IRB conditions of app

limitations
Medical Device Reporting (M

Humanitarian Use Devic

Medical Device Reporting (M

Humanitarian Use Devic

z Applies to all FDA approved dz Serious adverse events and d

must be reported to FDA and using the Medical Device Repsystem at 21 CFR 803

z HDE-holders and IRBs should 

that physicians know about threquirement
FDA Concerns FDA Concerns

z Off label use of an HUD

– IRB should ensure that physicians a

aware of any restrictions or limitationuse at the time of initial review.

– FDA recommends informed consent 

reasonable patient protections meas

• Monitoring and considering the specific 

patient and limited information about ri

effectiveness of the HUD

– Summary report to IRB and HDE-ho

the use
FDA Concerns FDA Concerns

z Research for HDE-approved i

– No Investigational Device Exem

required

– IRB review and informed conse

recommeded

z Research outside approved in

– Requires an (IDE) 21 CFR 812– IRB review and informed conse
References References

z Regulation

- 21 CFR 814 Subpart H- 21 CFR 56 Institutional Revi- 21 CFR 803 Medical Device 

z Guidance

www.accessdata.fda.gov/scripts/cfHDE/HDEInformation.cfm

- List of all HUDs- Frequently asked questions and 
Summary Summary

z Humanitarian Use Device and 

Humanitarian Device Exempt

z FDA’s approval criteriaz HDE-holder responsibilitiesz Physician responsibilitiesz IRB responsibilities