Background

The U.S. Food and Drug Administration’s Office of Pediatric Therapeutics (OPT), Center for Drug Evaluation and Research (CDER), and Center for Biologics Evaluation and Research (CBER) are announcing a public meeting entitled "Interested Parties Meeting: Implementation of the Best Pharmaceuticals for Children Act and Pediatric Research Equity Act." The purpose of the public meeting is to seek input from interested parties including, patient/parent/caregiver groups, consumer groups, regulated industry, academia, and others. This input will enable FDA to obtain any recommendations or information relevant to the report to Congress that FDA is required to submit concerning pediatrics, including pediatric drug and biologic development and labeling, as outlined in section 508 of the Food and Drug Administration Safety and Innovation Act (FDASIA). 

FDASIA (Pub. L. 112-144) section 508 directs the HHS Secretary to submit a report to Congress every 5 years on the implementation of sections 505A and 505B of the FD&C Act, which are commonly known as the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. FDASIA also requires FDA to obtain recommendations or relevant information from interested parties on the report.

Topics for Discussion at the Public Meeting

Some of the issues to be discussed at the meeting will include, but not be limited to:

  • Hearing from patients/parents/caregivers and patient/parent/caregiver groups, consumer groups, industry, academia and other interested parties about the public health impact that pediatric legislation may have had on them or their communities, including treatment advances for children resulting from the legislation, as well as areas of continued unmet medical need.
  • Understanding the effects of the requirement of pediatric studies under PREA or the incentives under BPCA on drug/biologic development plans, including issues related to the balance of incentives and requirements and progress toward international alignment on pediatric drug development to the extent practicable.
  • Understanding if there are any barriers or resource issues preventing undertaking or completing studies under PREA and BPCA, including issues related to clinical trial infrastructure and enrollment and ensuring pediatric clinical trial populations reflect the diversity of children most likely to use and benefit from the therapeutic treatments.
  • Understanding successes and challenges with leveraging scientific advances in product development, including, but not limited to, use of pediatric extrapolation, adaptive trial designs, biomarkers as surrogates, and real-world data to facilitate more timely evidence-generation for pediatric populations.

Registration

To register for in-person or online attendance, go to https://fda.zoomgov.com/webinar/register/WN_L_jvkxo7RKGmfx9JilBK_Q. Please be prepared to provide complete contact information for each attendee, including name, email address, and affiliation (if any).

Registration is free and based on space availability, with priority given to early registrants. Persons interested in attending this public meeting in person must register by May 1, 2025, 11:59 p.m. ET. Early registration is recommended because seating is limited; therefore, the FDA may limit the number of participants from each organization. If time and space permit, onsite registration on the day of the public meeting will be provided beginning at 8:00 a.m. We will post a notice on this meeting web page if registration for in-person attendance closes before the day of the public meeting.

If you need special accommodations due to a disability, please contact OPT@fda.hhs.gov no later than May 8, 2025, 11:59 p.m. ET. 

Streaming Webcast of the Public Meeting

This public meeting will also be webcast. The link to view the virtual Zoom webinar will be sent to registered participants prior to the meeting.

Requests for Oral Comment

During the meeting registration process you may indicate if you wish to present during the public comment session, and which topic(s) you wish to address. We will do our best to accommodate requests to make public comments. Individuals and organizations with common interests are urged to consolidate or coordinate their comments. 

All requests to make oral comments (whether virtual or in-person) must be received by May 1, 2025, 11:59 p.m. ET.  Based on the number of requests we receive, we will determine the amount of time allotted to each presenter and will notify participants making an oral comment by May 5, 2025, 11:59 p.m. ET. If making an oral comment, any presentation materials must be emailed to OPT@fda.hhs.gov no later than May 9, 2025, 11:59 p.m. ET.  No commercial or promotional material will be permitted to be presented or distributed at the public meeting. 

Submitting Public Comment

Regardless of attendance at the public meeting, you can submit electronic or written comments to the public docket number FDA-2024-N-5784. The https://www.regulations.gov electronic filing system will accept comments on this docket until 11:59 p.m. ET, June 13, 2025. Comments received by mail/hand delivery/courier (for written/paper submissions) will be considered timely if they are received on or before that date. Please note that comments filed after the due date will not be considered.

Electronic Submissions

Submit electronic comments through the Federal eRulemaking Portal: https://www.regulations.gov. Follow the instructions for submitting comments. Comments submitted electronically, including attachments, to https://www.regulations.gov will be posted publicly to the docket unchanged. Because your comment will be made public, you are solely responsible for ensuring that your comment does not include any confidential information that you or a third party may not wish to be posted, such as medical information, your or anyone else's Social Security number, or confidential business information, such as a manufacturing process. Please note that if you include your name, contact information, or other information that identifies you in the body of your comments, that information will be posted on https://www.regulations.gov. Comments with confidential information should be submitted only as a written or paper submission.

Written/Paper Submissions

Send written comments by mail, hand delivery, or courier to:

Dockets Management Staff (HFA-305)
Food and Drug Administration
5630 Fishers Lane, Rm. 1061
Rockville, MD 20852

FDA will post all comments, as well as any attachments, at https://www.regulations.gov or make them publicly viewable at the Dockets Management Staff office between 9 a.m. and 4 p.m. ET, Monday through Friday, 240-402-7500, unless the information is submitted, marked and identified as confidential as detailed in the instructions.

Instructions

All submissions must include the Docket No. FDA-2024-N-5784 for “Interested Parties Meeting: Implementation of the Best Pharmaceuticals for Children Act and Pediatric Research Equity Act." Comments received by the due date will be placed in the docket and, except for those submitted as "Confidential Submissions," be made public.

Confidential Submissions

To submit a comment with confidential information that you do not wish to be made public, submit your comments only as a written/paper submission. You should submit two copies total. One copy will include the information you claim to be confidential with a heading or cover note that states "THIS DOCUMENT CONTAINS CONFIDENTIAL INFORMATION." The Agency will review this copy, including the claimed confidential information, in its consideration of comments. The second copy, which will have the claimed confidential information redacted/blacked out, will be available for public viewing and posted on https://www.regulations.gov. Submit both copies to the Dockets Management Staff. If you do not wish your name and contact information to be made publicly available, you can provide this information on the cover sheet and not in the body of your comments and you must identify this information as "confidential."  Any information marked as "confidential" will not be disclosed except in accordance with 21 CFR 10.20 and other applicable disclosure law. For more information about FDA's posting of comments to public dockets, see 80 FR 56469, September 18, 2015, or access the information at: https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf.

Access Public Comments

For access to the docket to read background documents or the electronic and written/paper comments received, go to https://www.regulations.gov and insert the docket number FDA-2024-N-5784 into the "Search" box and follow the prompts, or go to the Dockets Management Staff, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852, 240-402-7500.