Humanitarian Use Device (HUD)

Program Overview

Katie Chowdhury, MS

Biomedical Engineer/Health Science Administrator

Office of Orphan Products Development

Office of Special Medical Programs

U.S. Food and Drug Administration
Learning Objectives 

• To understand why Congress established the 

Humanitarian Use Device (HUD) designation and 

Humanitarian Device Exemption (HDE) programs

• To define the term “Population Estimate” and how it 

applies to different categories of devices

• To understand how HUDs apply to pediatric patients

• To know where to submit an HUD application 

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21 CFR 814.100(c): Two-Step Process 

• Step 1: Submit a HUD designation request and 

receive approval from the FDA’s Office of 

Orphan Products Development

• Step 2: After HUD designation is granted, submit 

an HDE application to the Center for Devices and 

Radiological Health (CDRH) or Center for 

Biologics Evaluation and Research (CBER)

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Humanitarian Use 

Device (HUD) Program

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Why Humanitarian Use Devices?

• In 1990, Congress established the HUD designation and 

HDE marketing pathway program

– encourage development of devices intended for rare diseases

• General Requirement for a new device to enter the 

market: 

– reasonable assurance that the device is safe and effective 

• Under the HUD/HDE pathway: – device is safe and provides a probable benefit

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HUD Definition

A Humanitarian Use Device (HUD) is a medical 

device intended to benefit patients in treatment 

or diagnosis of a disease or condition that 

affects or is manifested in not more than 8,000 

individuals per year in the United States.

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21 CFR 814.3(n) 
Guidance Document

• “Guidance for Industry and FDA Staff: Humanitarian 

Use Device (HUD) Designations” 

– Issued on January 24, 2013

www.fda.gov/downloads/RegulatoryInformation/Guidances/UCM336515.pdf 

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HUD Designation by OOPD

• Sponsor submits a HUD Designation Request to FDA’s 

Office of Orphan Products Development (OOPD)

– Two copies

• FDA sends the Sponsor an acknowledgement letter 

with an assigned HUD number for each application

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Content of HUD Request • Cover Letter

– requesting OOPD consider the device for HUD designation for a rare disease or condition or an ‘orphan subset’ of a 

disease or condition

• Description of the Disease or Condition 

– device intends to treat or diagnose

• Population Estimate

– Calculation of the number of patients affected or manifested with the disease/condition in the United States per year

• Description of the Device and Scientific Rationale for its Proposed Use

– written description and engineering diagrams

– supporting use of the device; preclinical, clinical, and/or proof-of-principle data pertaining to the device

• Supporting Documentation with Appended References

– demonstrate the device is designed to treat/diagnose a rare disease or condition (or orphan subset) that affects or 

is manifested in not more than 8,000 individuals per year in the United States 9 
How OOPD Reviews a HUD Request

1. Evaluate disease or condition based upon device functionality

2. Evaluate target population that may benefit from device

– May broaden (or reduce) scope of patient population as appropriate

– Target population must be not more than 8,000 individuals/per year

45 day review  FDA issues Decision letter to applicant

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HUD Decisions

• Approval: Designate the device for the disease or condition

– Population estimate must be not more than 8,000/year in the U.S.

– Disease or condition may not always be identical to what was 

requested by the applicant

• Disapproval

• Request Additional Information

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Disease/Condition

• Evaluate how device works to treat/diagnose the disease or 

condition

• Evaluate how it performs in the patient population

• Factors examined such as:

 disease/condition pathogenesis, course, or prognosis; or

 resistance to treatment

• Designate device for a disease/condition, and not intended use

• May designate the disease/condition to be broader than eventual 

HDE approved indications for use/label

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Prevalence vs. Incidence

• Prevalence: 

– Total number of patients with a disease/condition in the population at a given time

– example: number of patients with Cystic Fibrosis on April 1, 2015 in the United States

• Incidence:

– Number of new patients diagnosed with a disease/condition during a particular time 

period

– example: number of new patients diagnosed with Cystic Fibrosis from January 1 – 

December 31, 2015 in the United States

• Generally, FDA relies on Incidence for HUD designation

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Population Estimate:

Therapeutic Devices

Number of:

• new patients per year; and

• diagnosed with the relevant disease or condition; and

• potentially eligible for treatment with the device.

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Population Estimate: 

Diagnostic Devices

Number of:

• new patients per year; and

• subjected to diagnosis with device, regardless of test result (e.g., 

positive or negative).

• example: screening of all newborns for a rare disease/condition – does not 

qualify

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Population Estimate: Devices for 

Repeat or Multiple Use

Number of:

• new patients who would be eligible for, or subjected to, the 

device in a given year

• not on the total number of expected uses of a device in a 

given year by all patients.

– For example, a device to treat both eyes in a given year would count 

as one patient instead of two.

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“Orphan Subset” - Definition

• Is a subset of individuals with a non-rare disease or 

condition on whom use of device is appropriate

– where use of the device on the remaining individuals with that disease 

or condition would be inappropriate

– For example, some intrinsic feature of the device prevents it from 

being used in some patients who have a disease or condition and as 

such those patients are not excluded in the population estimate

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“Orphan Subset” Factors - Considered

• Adverse Event Profile

– Device is shown to have a serious adverse event profile and should be 

restricted only to certain patients

• Mechanism of Action

– Device characteristics limit its use

• For example, a blood pump that is too large to fit in the chest cavity of a small 

person

• Prior Clinical Experience

– May show that the device has no significant activity in some patients

– Should only be used in the remaining population with the disease

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“Orphan Subset” Factors - Not Considered

• Eligibility of Patients in Proposed Clinical Trial

– Inclusion/exclusion criteria of a study does not qualify patients as an 

“orphan subset”

• Sponsor’s Choice to Narrow Indication for Use

– May not exist just because of a specifically chosen patient population

• Unmet Medical Need

• Standard of Care

• Price

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Device Description/Scientific Rationale

• Adequate information to describe the function of the 

device

• Adequate information to support the use of the 

device or justify use of the device for an “orphan 

subset” 

– e.g., preclinical, clinical, and/or proof-of-principle data

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Pediatrics

• Younger than 22 years of age

• Device may be eligible if the pediatric population 

affected by the disease or condition is not more than 

8,000 individuals per year

• OOPD will designate a device solely for pediatric use 

if it qualifies based upon the population estimate

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Where to Submit

HUD Designation Request

Send two signed and dated submissions. One 

hardcopy (original) and copy (paper or eCopy):

Office of Orphan Products Development

Food and Drug Administration

10903 New Hampshire Ave.

WO32, Room 5295

Silver Spring, MD 20993

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How to Reach OOPD

Email: orphan@fda.hhs.gov 

Phone Number: 301-796-8660

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Resources

For additional assistance regarding the HUD Program:

• OOPD website

www.fda.gov/orphan

• HUD guidance

www.fda.gov/downloads/RegulatoryInformation/

Guidances/UCM336515.pdf

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Summary

• A HUD designation allows for a device intended for use in 

not more than 8,000 patients per year with a rare 

disease or condition to be eligible for an alternative 

pathway to market 

• The HUD/HDE Marketing Pathway is a Two-Step Process 

• The Sponsor should submit specific documentation to 

support the HUD request 

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Summary

• OOPD designates devices for a disease or condition 

and not for a specific indication or label

• Disease Incidence and Population Estimates are 

important factors in determining HUD eligibility

• Where appropriate, a device for a non-rare disease 

or condition may qualify for an orphan subset

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Providing Industry Education

1. CDRH Learn – Multi-Media Industry Education

 over 80 modules - videos, audio recordings, power point presentations, software-based 

“how to” modules

 accessible on your portable devices: www.fda.gov/Training/CDRHLearn 

2. Device Advice – Text-Based Education

 comprehensive regulatory information on premarket and postmarket topics:

www.fda.gov/MedicalDevices/DeviceRegulationandGuidance

3. Division of Industry and Consumer Education (DICE) 

 If you have a question - Email: DICE@fda.hhs.gov 

 Phone: 1(800) 638-2041 or (301) 796-7100 (Live Agents 9am – 12:30 pm; 1-4:30 pm EST)

 Web Homepage: 

www.fda.gov/MedicalDevices/DeviceRegulationandGuidance/ContactUs-- DivisionofIndustryandConsumerEducation/default.htm 

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