Pediatric Drug Development: Regulatory Considerations — Complying With the Pediatric Research Equity Act and Qualifying for Pediatric Exclusivity Under the Best Pharmaceuticals for Children Act

Description

This guidance is intended to assist industry developing drug products to comply with the pediatric study requirements under the Pediatric Research Equity Act (PREA), and to describe the process for qualifying for pediatric exclusivity and the protections that pediatric exclusivity offers under the Best Pharmaceuticals for Children Act (BPCA). In 2010, the Biologics Price Competition and Innovation Act of 2009 extended provisions of the BPCA to biological products.

Scope & Applicability

Regulatory Context

• 21CFR601.51§ 601.51 Confidentiality of data and information in applications for biologics licenses.

  (a) For purposes of this section the biological product file includes all data and information submitted with or incorporated by reference in any application for a biologics license, IND's incorporated into any such application, master files, and other related submissions. The availability for publiRead full regulation →

• 21CFR314.430§ 314.430 Availability for public disclosure of data and information in an application or abbreviated application.

  (a) The Food and Drug Administration will determine the public availability of any part of an application or abbreviated application under this section and part 20 of this chapter . For purposes of this section, the application or abbreviated application includes all data and information submitted wRead full regulation →

• 21CFR99.3§ 99.3 Definitions.

  (a) Agency or FDA means the Food and Drug Administration.Read 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 →

• 21CFR314.101§ 314.101 Filing an NDA and receiving an ANDA.

  (a) Filing an NDA.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 →

• 21CFR601.70§ 601.70 Annual progress reports of postmarketing studies.

  (a) General requirements. This section applies to all required postmarketing studies (e.g., accelerated approval clinical benefit studies, pediatric studies) and postmarketing studies that an applicant has committed, in writing, to conduct either at the time of approval of an application or a suppleRead full regulation →

• 21CFR314.81§ 314.81 Other postmarketing reports.

  (a) Applicability. Each applicant shall make the reports for each of its approved applications and abbreviated applications required under this section and section 505(k) of the act.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 →

• 21CFR314.93§ 314.93 Petition to request a change from a listed drug.

  (a) The only changes from a listed drug for which the agency will accept a petition under this section are those changes described in paragraph (b) of this section. Petitions to submit ANDAs for other changes from a listed drug will not be approved.Read full regulation →

• 21CFR601.12§ 601.12 Changes to an approved application.

  (a) General.Read full regulation →

• 21CFR314.70§ 314.70 Supplements and other changes to an approved NDA.

  (a) Changes to an approved NDA.Read full regulation →

• Clinical Investigator

  Americo F. Padilla, M.D.

  2025-03-25

• Preparation and Public Availability of Information Given to Advisory Committee Members: Guidance for Industry (Status: Final)
• PDUFA Pilot Project: Proprietary Name Review - Concept Paper (Status: Final)
• Comparability Protocols for Postapproval Changes to the Chemistry, Manufacturing, and Controls Information in an NDA, ANDA, or BLA (Status: Final)
• Providing Regulatory Submissions in Electronic Format — Certain Human Pharmaceutical Product Applications and Related Submissions Using the eCTD Specifications Guidance for Industry (Status: Final)
• Reference Guide for the Nonclinical Toxicity Studies of Antivial Drugs Indicated for the Treatment of N/A Non-Life Threatening Disease Evaluation of Drug Toxicity Prior to Phase I Clinical Studies (Status: Final)
• Implementation of Section 120 of the Food and Drug Administration Modernization Act of 1997-Advisory Committees (Status: Final)
• Disclosure of Materials Provided to Advisory Committees in Connection with Open Advisory Committee Meetings Convened by the Center for Drug Evaluation and Research Beginning on January 1, 2000 (Status: Final)
• Harmonizing Compendial Standards With Drug Application Approval Using the USP Pending Monograph Process Guidance for Industry (Status: Draft)