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FDA Designations for Rare Disease Products, Part 1

Comparing Orphan Drug, Rare Pediatric Disease, and Humanitarian Use Device Designations

The US FDA offers sponsors a variety of special designation programs to incentivize them to develop and deliver therapies to treat unmet patient needs, such as Fast Track Designation (FTD), Breakthrough Therapy Designation, and Qualified Infectious Disease Product Designation. Today, we are kicking off a four-part blog series highlighting those designation programs available specifically for products with rare disease indications: Orphan Drug Designation (ODD), Rare Pediatric Disease Designation (RPDD), and Humanitarian Use Device (HUD) designation.

The table below gives a side-by-side overview and comparison of each of these programs, and we will explore each one in more detail in subsequent blog posts. Subscribe to our mailing list at the bottom of this page to be notified when new content is published.

Orphan Drug Designation

Rare Pediatric Disease Designation

Humanitarian Use Device Designation

Where is the regulation found? 21 Code of Federal Regulations (CFR) §316;
Orphan Drug Act
Section 529 of the Federal Food, Drug, and Cosmetic Act (FD&C Act)/ 21 United States Code (USC) §360ff 21 CFR §814 Subpart H
What type of products qualify? Drugs and biological products Drugs and biological products Medical devices
What are the major criteria? The product must be intended for use in a rare disease or condition.
The disease or condition must affect fewer than 200,000 people in the US.
There must be strong scientific rationale supporting the use of the product in the proposed indication.
The product must be intended for the prevention or treatment of a rare disease that is serious or life-threatening and primarily affects patients 0-18 years old.
The disease or condition must affect fewer than 200,000 people in the US.
Supportive data must suggest that the product may be effective in the indication (with a lower bar than for ODD).
Additional criteria for the marketing application must be met to obtain the benefits.
The device must be intended to benefit patients in the treatment or diagnosis of a rare disease or condition.
The disease or condition must affect or be manifested in no more than 8,000 individuals in the US per year.
 There must be scientific rationale supporting the use of the device in the proposed indication.
When can a sponsor submit the request? Any time prior to NDA or BLA submission Preferably concurrent with the ODD or FTD request, but any time prior to NDA or BLA filing Any time prior to submission of the Humanitarian Device Exemption (HDE) or other device marketing application
To which FDA office or division is the request submitted? Office of Orphan Products Development Office of Orphan Products Development Office of Orphan Products Development
How long does it take for the FDA to respond to the request?* 90 days 60 days (This does not apply if the request is submitted later than the ODD or FTD request.) 45 days
What are the benefits? Pre-approval Benefits

Potential Pediatric Research Equity Act (PREA) requirements exemption
Waiver of user fee for NDA or BLA
Tax credits and grants
Protocol assistance

Post-approval Benefits

Seven years of orphan drug market exclusivity upon NDA or BLA approval

Note: This benefit may not be available if the active moiety is already approved.

Pre-approval Benefits

No significant pre-approval benefits

Post-approval Benefits

Receipt of a Priority Review Voucher (PRV) for use on another product or application**:

The voucher is received upon NDA or BLA approval of the RPDD product.
PRVs can be used to expedite the review of another NDA or BLA from the 10‑month standard review time to the 6‑month priority review time.
The PRV can be either used by the sponsor who initially received it or sold to another sponsor.

Pre-approval Benefits

Eligibility for an HDE application

Note: The HDE application has its own separate benefits, but HUD designation is required to submit an HDE:

Exemption from the effectiveness requirements in Sections 514 & 515 of the FD&C Act
 Shorter review time compared to a premarket approval (PMA) application (75 days instead of 180 days)
No user fees (marketing application submission fee)

Post-approval Benefits

If approved via HDE, no user fees (marketing application maintenance/ periodic reporting fee)

Are there any required activities for maintaining it? An annual report must be submitted (distinct from an IND annual report). No, but a post-marketing 5-year report is required after approval. No, but post-approval requirements exist, including post-marketing reporting to both the FDA and the Institutional Review Board (IRB) and IRB approval for use of HUDs in the clinical care of patients at a facility.
Can the designation be rescinded later in product development? Yes, if the product no longer meets the designation-specific qualifying criteria. Yes, if the product no longer meets the designation-specific qualifying criteria. (The FDA may also revoke the PRV if the product is not marketed in the US within 1 year of approval.) Yes, if the product no longer meets the designation-specific qualifying criteria.
Can it be obtained in conjunction with other rare disease designations? Yes, sponsors can request both ODD and RPDD for the same product. Yes, sponsors can request both ODD and RPDD for the same product. No, since the program is intended for medical devices and not drugs or biological products, it is highly unlikely that it could be obtained for the same product as ODD or RPDD.

*The timelines provided are goals for the FDA but may not be strictly enforced.
**For more information about Priority Review Vouchers and the ability to sell or transfer them, please see these other Camargo Blog posts: Shortening the Review Clock: the Latest on Priority Review Vouchers | Tropical or Rare Pediatric Disease Priority Review Vouchers | Priority Review Vouchers are a Big Carrot for Hungry Companies


With rare disease therapies as one of our focus areas, Camargo has deep experience obtaining special designation status for orphan drugs and other products meeting unmet patient needs. Contact us to find out whether your product could qualify for one or more of these programs.

Author:

Marissa Berry, PhD, RAC
Regulatory Scientist

Sources:

CFR – Code of Federal Regulations Title 21

The United States Code

Designating an Orphan Product: Drugs and Biological Products

FDA’s Orphan Drug Modernization Plan

Clarification of Orphan Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases

User Fee Waivers, Reductions, and Refunds for Drug and Biological Products: Guidance for Industry

Rare Pediatric Disease Priority Review Vouchers: Guidance for Industry

Humanitarian Use Device (HUD) Designations: Guidance for Industry

Humanitarian Device Exemption (HDE) Program: Guidance for Industry

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