Description
This guidance provides information on the implementation of section 908 of the Food and Drug Administration Safety and Innovation Act (FDASIA), which added section 529 to the Federal Food, Drug, and Cosmetic Act (the FD&C Act). Under section 529, FDA will award priority review vouchers to sponsors of certain rare pediatric disease product applications that meet the criteria specified in that section.
Key Topics
Terms and concepts identified from this document
Scope & Applicability
Product Classes
5Drug intended for prevention or treatment of rare pediatric diseases
Eligibility of drug-drug combinations for vouchers
Defined as a serious or life-threatening disease primarily affecting individuals aged birth to 18 years
Regulated under section 351(i) of the PHS Act; Virus, therapeutic serum, toxin, vaccine, or protein applicable to prevention or treatment; Alternative regulation category for products meeting device definition
Products eligible for designation and priority review vouchers.; Product application seeking a priority review voucher; Applications for products treating rare pediatric diseases
Stakeholders
1Entity responsible for submitting applications under section 524B
Regulatory Context
Regulatory Activities
10Process for designating a drug for a rare pediatric disease; Eligibility for a priority review voucher requires this designation granted by September 30, 2020.; Process of requesting FDA to recognize a drug for a rare pediatric disease; Prerequisite for receiving a voucher; Request for designation as a drug for a rare pediatric disease
A master protocol sponsor should request a pre-IND meeting to discuss the protocol.
Expedited review goal of 6 months for marketing applications
Voucher awarded to sponsors of rare pediatric disease product applications; Issued to sponsors of rare pediatric disease product applications upon approval.; Voucher issued under section 529
Status providing financial incentives for rare disease drug development; Regulatory status related to disease prevalence.
Expedited program for serious conditions; Designation program to facilitate development and expedite review
Expedited program for serious conditions; Designation for products demonstrating substantial improvement over available therapies
Pathway for drugs and biologics for serious conditions; Regulatory pathway supported by surrogate or intermediate endpoints; Regulatory pathway based on surrogate or intermediate endpoints; Pathway for products based on surrogate or intermediate clinical endpoints
Alternative submission pathway to ANDA
New Drug Application
Document Types
7Cybersecurity information should be included in device labeling
Published to seek public comment on withdrawal proposals
Commits FDA to performance goals for priority reviews
Contains information related to the priority review voucher
Official letter from FDA granting rare pediatric disease designation
FDA communication requesting additional information
Report submitted to FDA no later than 5 years after approval
Attributes
2Estimated number of people with a disease, used to determine clinical investigation enrollment.
Disease affecting children and meeting rare disease criteria
Technical Details
Substances
1Basis for determining drug sameness
Processes
1Explanation of how the drug works in the rare pediatric disease
Clinical Concepts
3A serious or life-threatening disease primarily affecting individuals aged 0 to 18 years.; Disease or condition affecting pediatric patients; Defined in section 529(a)(3)
Criteria for rare pediatric disease designation
A subset of a non-rare disease that affects less than 200,000 persons.
Standards & References
External Standards
1Publication for notices and draft guidance availability
ICH References (1)
Clinical Investigation of Medicinal Products in the Pediatric Population.
Related CFR Sections (7)
- 21CFR316.21§ 316.21 Verification of orphan-drug status.
(a) So that FDA can determine whether a drug qualifies for orphan-drug designation under section 526(a) of the act, the sponsor shall include in its request to FDA for orphan-drug designation under § 316.20 either:Read full regulation →
- 21CFR316.3§ 316.3 Definitions.
(a) The definitions and interpretations contained in section 201 of the act apply to those terms when used in this part.Read full regulation →
- 21CFR201.57§ 201.57 Specific requirements on content and format of labeling for human prescription drug and biological products described in § 201.56(b)(1) .
The requirements in this section apply only to prescription drug products described in § 201.56(b)(1) and must be implemented according to the schedule specified in § 201.56(c) , except for the requirement in paragraph (c)(18) of this section to reprint any FDA-approved patient labeling at the end oRead full regulation →
- 21CFR50.52§ 50.52 Clinical investigations involving greater than minimal risk but presenting the prospect of direct benefit to individual subjects.
Any clinical investigation within the scope described in §§ 50.1 and 56.101 of this chapter in which more than minimal risk to children is presented by an intervention or procedure that holds out the prospect of direct benefit for the individual subject, or by a monitoring procedure that is likely tRead full regulation →
- 21CFR50.53§ 50.53 Clinical investigations involving greater than minimal risk and no prospect of direct benefit to individual subjects, but likely to yield generalizable knowledge about the subjects' disorder or condition.
Any clinical investigation within the scope described in §§ 50.1 and 56.101 of this chapter in which more than minimal risk to children is presented by an intervention or procedure that does not hold out the prospect of direct benefit for the individual subject, or by a monitoring procedure that is Read full regulation →
- 21CFR314.50§ 314.50 Content and format of an NDA.
NDAs and supplements to approved NDAs are required to be submitted in the form and contain the information, as appropriate for the particular submission, required under this section. Three copies of the NDA are required: An archival copy, a review copy, and a field copy. An NDA for a new chemical enRead full regulation →
- 21CFR601.2§ 601.2 Applications for biologics licenses; procedures for filing.
(a) General. To obtain a biologics license under section 351 of the Public Health Service Act for any biological product, the manufacturer shall submit an application to the Director, Center for Biologics Evaluation and Research or the Director, Center for Drug Evaluation and Research (see mailing aRead full regulation →
Enforcement Impact
Deficiencies cited in Warning Letters referencing the same regulations
Related Warning Letters (2)
See Also (8)
- Interpreting Sameness of Monoclonal Antibody Products Under the Orphan Drug Regulations (Status: Final)
- Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations: Guidance for Industry (Status: Final)
- Clinical Development Programs for Drugs, Devices, and Biological Products for the Treatment of Rheumatoid Arthritis (RA) (Status: Final)
- Prussian Blue Drug Products — Submitting a New Drug Application (Status: Final)
- Calcium DTPA and Zinc DTPA Drug Products-Submitting a New Drug Application (Status: Final)
- Meetings with the Office of Orphan Products Development: Guidance for Industry, Researchers, Patient Groups, and Food and Drug Administration Staff (Status: Final)
- Tropical Disease Priority Review Vouchers (Status: Final)
- Questions and Answers on Biosimilar Development and the BPCI Act Guidance for Industry: Guidance for Industry (Status: Final)