Office of Orphan Products 

Development

Eric Chen, MS

Director, Humanitarian Use Device Program

Office of Orphan Products Development

Office of Special Medical Programs

U.S. Food and Drug Administration
Learning Objectives 

• To understand the mission of Office of Orphan 

Products Development (OOPD)

• To review the programs managed by OOPD

• To understand the legislative basis and evolution of 

the Humanitarian Use Device (HUD)/Humanitarian 

Device Exemption (HDE) program

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Office of the 

Commissioner

(OC) 

Office of Medical 

Products and 

Tobacco

OMPT

OF

Office of Foods

Center for 

Biologics 

Evaluation and 

Research

Center for 

Drug 

Evaluation 

and Research

For Complete FDA Organizational Chart see:http://www.fda.gov/downloads/AboutFDA/CentersOffices/OrganizationCharts/UCM288864.pdf

OOPD

Office of Orphan 

Products 

Development

CDER CBER

Center for 

Devices and 

Radiological 

Health

OSMP

Office of Special 

Medical Programs

RDP 

Rare Diseases 

Program

“The Review Divisions”

CDRH

CFSAN

Center for Food 

Safety and 

Applied 

Nutrition 

Office of 

Nutritional Labeling 

and Dietary 

Supplements

(Medical Foods)

OEXA

OHCA

Office of Health and 

Constituent Affairs

Office of 

External 

Affairs

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Rare Diseases and Challenges 

• ~7,000 known rare diseases

• Only a portion have approved 

treatments 

• >500 orphan drug approvals 

(drugs & biologics)

• 69 Humanitarian Use Device 

approvals

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OOPD Mission Statement

To promote the development of products, including drugs, 

devices, biologics, and medical foods, for the treatment, 

diagnosis, and prevention of rare diseases and conditions. 

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DESIGNATION 

PROGRAMS

GRANT 

PROGRAMS

Orphan Drug Designation Orphan Products Grants

Rare Pediatric Disease Designation Orphan Products Natural History Grants

Humanitarian Use Device Designation Pediatric Device Consortia Grants
OOPD Programs

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DESIGNATION 

PROGRAMS

GRANT 

PROGRAMS

Orphan Drug Designation Orphan Products Grants

Rare Pediatric Disease Designation Orphan Products Natural History Grants

Humanitarian Use Device Designation Pediatric Device Consortia Grants
Orphan Drug Designation

The Orphan Drug Act (ODA) in 1983 

was created to motivate industry to 

develop drugs and biologics for rare 

diseases by providing financial 

incentives:

o Tax Credits for 50% of Clinical 

Trial Costs

o Waiver of Marketing 

Application User Fee

o Eligibility for 7-Year Marketing 

Exclusivity

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Orphan Drug Act

• Defined “rare disease” for 

drugs and biologics

– Disease/condition that affects 

< 200K people in the U.S.; or

– Drug that will not be profitable 

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OOPD Programs

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DESIGNATION 

PROGRAMS

GRANT 

PROGRAMS

Orphan Drug Designation Orphan Products Grants

Rare Pediatric Disease Designation Orphan Products Natural History Grants

Humanitarian Use Device Designation Pediatric Device Consortia Grants
Rare Pediatric Disease Designation

• Created under Food and Drug Administration Safety and Innovation 

Act (FDASIA) of 2012 to encourage development of drugs and 

biologics for “rare pediatric diseases (RPD)”

• Basic Idea: 

– If a sponsor receives approval of a “rare pediatric disease product application” for 

a “rare pediatric disease,” the sponsor is eligible to receive a Priority Review 

Voucher (RPV) which can be redeemed, or transferred to another sponsor, to 

obtain priority review of another application that would otherwise be ineligible for 

priority review

• Website: 

www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/ 

RarePediatricDiseasePriorityVoucherProgram/default.htm

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OOPD Programs

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DESIGNATION 

PROGRAMS

GRANT 

PROGRAMS

Orphan Drug Designation Orphan Products Grants

Rare Pediatric Disease Designation Orphan Products Natural History Grants

Humanitarian Use Device Designation Pediatric Device Consortia Grants
Orphan Products (OPD) Grants

• Encourage clinical development of products for use in rare diseases or 

conditions

• Applies to drugs, biologics, devices or medical foods 

• Grants funding for clinical trials

– Usually 3 to 4 years 

– 60 to 85 ongoing grant-funded projects

– Fund 10 to 15 new grants per fiscal year

• Website: 

www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/ 

WhomtoContactaboutOrphanProductDevelopment/ucm2005538.htm

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OOPD Programs

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DESIGNATION 

PROGRAMS

GRANT 

PROGRAMS

Orphan Drug Designation Orphan Product Grants

Rare Pediatric Disease Designation Orphan Products Natural History Grants

Humanitarian Use Device Designation Pediatric Device Consortia Grants
OPD Natural History Grants Program

The ultimate goal is to support 

marketing approvals

• Help characterize the natural history 

of a rare disease or condition

• Identify subpopulations based on 

clinical signs and symptoms or 

biomarkers

• Develop and/or validate clinical 

outcome measures, biomarkers, and 

diagnostics

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www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesCondition/

OrphanProductsNaturalHistoryGrantsProgram/default.htm
OOPD Programs

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DESIGNATION 

PROGRAMS

GRANT 

PROGRAMS

Orphan Drug Designation Orphan Product Grants

Rare Pediatric Disease Designation Orphan Products Natural History Grants

Humanitarian Use Device Designation Pediatric Device Consortia Grants
Pediatric Device Consortia (PDC) Grants

• Goal is to support the development of nonprofit 

consortia designed to stimulate projects for pediatric 

devices 

• Types of Services and Referrals Provided:

– Intellectual Property / Legal; Business Planning; Funding Advice; 

Regulatory Consulting; Preclinical and Clinical Study Planning; 

Prototyping and Design Services 

• Consortia websites:

www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/

PediatricDeviceConsortiaGrantsProgram/default.htm

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OOPD Programs

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DESIGNATION 

PROGRAMS

GRANT 

PROGRAMS

Orphan Drug Designation Orphan Product Grants

Rare Pediatric Disease Designation Orphan Products Natural History Grants

Humanitarian Use Device Designation Pediatric Device Consortia Grants
HUD/HDE Program

• Humanitarian Use Device (HUD)

– a medical device intended to benefit patients in the treatment or 

diagnosis of a disease or condition that affects or is manifested in not 

more than 8,000 individuals in the United states per year. 

• Humanitarian Device Exemption (HDE)

– the premarket submission that allows a HUD to be marketed once a 

demonstration of safety and probable benefit has been satisfied by 

FDA. 

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Section 520(m) of the FD&C Act

Humanitarian Device Exemption

“To the extent consistent with the protection of the public 

health and safety and with ethical standards, it is the purpose of 

this subsection to encourage the discovery and use of devices 

intended to benefit patients in the treatment and diagnosis of 

diseases or conditions that affect not more than 8,000 

individuals in the United States.”

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Law (FD&C Act) Regulation 

• 21 CFR 814.100(a):

“...to encourage the discovery and use of devices 

intended to benefit patients in the treatment or 

diagnosis of diseases or conditions that affect or are 

manifested in fewer than 4,000 individuals in the 

United States per year.”

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HUD/HDE Program:

Legislative History

• Safe Medical Device Act (SMDA) [1990]

– HDE program was created

• Food and Drug Administration Modernization Act (FDAMA) 

[1997]

– HDE program was revised

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HUD/HDE Program:

Legislative History

• Food and Drug Administration Amendments Act (FDAAA) 

[2007]

– intended to motivate development of pediatric devices

– removed profit restriction if labeled for pediatric use

– annual distribution number (ADN)

– requires annual device review by Pediatric Advisory Committee 

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HUD/HDE Program:

Legislative History

• Food and Drug Administration Safety and Innovation Act 

(FDASIA) [2012]

– expanded exemption from profit prohibition

– modified ADN definition

• 21st Century Cures Act [2016]

– increased population threshold from 4,000 to 8,000

– modified IRB oversight

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21 CFR 814.100(c): Two Step Process 

• Step 1: Obtain designation of the device as a 

HUD 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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Summary 

• OOPD’s mission is ‘to promote the development of products, 

including drugs, devices, biologics, and medical foods, for the 

treatment, diagnosis, and prevention of rare diseases and 

conditions’ 

• OOPD oversees three designation and three grant programs

• The HUD/HDE program, created in 1990, established a 

pathway to market for medical devices intended to treat or 

diagnose diseases that occur in small (rare) populations
How to Reach OOPD

Email: orphan@fda.hhs.gov 

Phone Number: 301-796-8660

OOPD Website:

www.fda.gov/forindustry/

developingproductsforrarediseasesconditions/default.htm

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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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