How to Comply with the Pediatric Research Equity Act

Description

This draft guidance provides recommendations on how to interpret the pediatric study requirements of the Pediatric Research Equity Act (Public Law 108-155) (PREA). PREA amends the Federal Food, Drug, and Cosmetic Act (the Act) by adding section 505B (21 U.S.C. 355B). PREA requires the conduct of pediatric studies for certain drug and biological products.2Specifically, PREA requires new drug applications (NDAs) and biologics licensing applications (BLAs) (or supplements to applications) for a new active ingredient, new indication, new dosage form, new dosing regimen, or new route of administration to contain a pediatric assessment unless the applicant has obtained a waiver or deferral (see section 505B(a) of the Act). It also authorizes FDA to require holders of applications for previously approved marketed drugs and biological products who are not seeking approval for one of the changes enumerated above (hereinafter "marketed drugs and biological products") to submit a pediatric assessment under certain circumstances (see section 505B(b) of the Act).

Scope & Applicability

Regulatory Context

• 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 →

• 21CFR314.55§ 314.55 Pediatric use information.

  (a) Required assessment. Except as provided in paragraphs (b) , (c) , and (d) of this section, each application for a new active ingredient, new indication, new dosage form, new dosing regimen, or new route of administration shall contain data that are adequate to assess the safety and effectivenessRead full regulation →

• 21CFR601.27§ 601.27 Pediatric studies.

  (a) Required assessment. Except as provided in paragraphs (b) , (c) , and (d) of this section, each application for a new active ingredient, new indication, new dosage form, new dosing regimen, or new route of administration shall contain data that are adequate to assess the safety and effectivenessRead 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 →

• 21CFR312.23§ 312.23 IND content and format.

  (a) A sponsor who intends to conduct a clinical investigation subject to this part shall submit an “Investigational New Drug Application” (IND) including, in the following order:Read full regulation →

• 21CFR312.32§ 312.32 IND safety reporting.

  (a) Definitions. The following definitions of terms apply to this section:Read full regulation →

• 21CFR312.33§ 312.33 Annual reports.

  A sponsor shall within 60 days of the anniversary date that the IND went into effect, submit a brief report of the progress of the investigation that includes: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 →

• Clinical Investigator (Sponsor)

  Angela D. Ritter, M.D.

  2024-06-18

• Evaluating the Risks of Drug Exposure in Human Pregnancies (Status: Final)
• Reports on the Status of Postmarketing Study Commitments — Implementation of Section 130 of the Food and Drug Administration Modernization Act of 1997 (Status: Final)
• Postmarketing Studies and Clinical Trials—Implementation of Section 505(o)(3) of the Federal Food, Drug, and Cosmetic Act Guidance for Industry (Status: Final)
• Postmarketing Studies and Clinical Trials—Implementation of Section 505(o)(3) of the Federal Food, Drug, and Cosmetic Act Guidance for Industry: Draft Guidance for Industry (Status: Draft)
• Pediatric Drug Development: Regulatory Considerations — Complying With the Pediatric Research Equity Act and Qualifying for Pediatric Exclusivity Under the Best Pharmaceuticals for Children Act (Status: Draft)
• Postmarketing Studies and Clinical Trials: Determining Good Cause for Noncompliance with Section 505(o)(3)(E)(ii) of the Federal Food, Drug, and Cosmetic Act: Draft Guidance for Industry (Status: Draft)
• Postmarketing Approaches to Obtain Data on Populations Underrepresented in Clinical Trials for Drugs and Biological Products (Status: Draft)
• Annual Status Report Information and Other Submissions for Postmarketing Requirements and Commitments: Using Forms FDA 3988 and FDA 3989 Guidance for Industry: Guidance for Industry (Status: Final)