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Interested Parties Meeting: Implementation of the Best Pharmaceuticals for Children Act and Pediatric Research Equity Act

The Food and Drug Administration's (FDA, Agency, or we) Office of Pediatric Therapeutics, the Center for Drug Evaluation and Research, and the Center for Biologics Evaluation an...

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Department of Health and Human Services

Food and Drug Administration

[Docket No. FDA-2024-N-5784]

AGENCY:

Food and Drug Administration, HHS.

ACTION:

Notice of public meeting; request for comments.

SUMMARY:

The Food and Drug Administration's (FDA, Agency, or we) Office of Pediatric Therapeutics, the Center for Drug Evaluation and Research, and the Center for Biologics Evaluation and Research are announcing a public meeting entitled “Interested Parties Meeting: ( printed page 323) 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 pediatric drug and biologic development and labeling, as outlined in section 508 of the Food and Drug Administration Safety and Innovation Act (FDASIA).

DATES:

The public meeting will be held on May 15, 2025, from 9 a.m. to 4:30 p.m. Eastern Time. Regardless of attendance at the public meeting, you can submit electronic or written comments to the public docket. Either electronic or written comments on this public meeting must be submitted by June 13, 2025. See the SUPPLEMENTARY INFORMATION section for registration date and information.

ADDRESSES:

The public meeting will be held in-person at the FDA White Oak Campus, 10903 New Hampshire Ave., Building 31 Conference Center, the White Oak Great Room, Silver Spring, MD 20993-0002 and virtually using the Zoom platform. Entrance for the in-person public meeting participants (non-FDA employees) is through Building 1 where routine security check procedures will be performed. For parking and security information, please refer to https://www.fda.gov/about-fda/visitor-information.

You may submit comments as follows. Please note that late, untimely filed comments will not be considered. The https://www.regulations.gov electronic filing system will accept comments until 11:59 p.m. Eastern Time at the end of 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.

Electronic Submissions

Submit electronic comments in the following way:

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 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.
If you want to submit a comment with confidential information that you do not wish to be made available to the public, submit the comment as a written/paper submission and in the manner detailed (see “Written/Paper Submissions” and “Instructions”).

Written/Paper Submissions

Submit written/paper submissions as follows:

Mail/Hand Delivery/Courier (for written/paper submissions): Dockets Management Staff (HFA-305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.
For written/paper comments submitted to the Dockets Management Staff, FDA will post your comment, as well as any attachments, except for information submitted, marked and identified, as confidential, if submitted as detailed in “Instructions.”

Instructions: All submissions received 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.” Received comments, those filed in a timely manner (see ADDRESSES ), will be placed in the docket and, except for those submitted as “Confidential Submissions,” publicly viewable at https://www.regulations.gov or at the Dockets Management Staff between 9 a.m. and 4 p.m., Monday through Friday, 240-402-7500.

Confidential Submissions—To submit a comment with confidential information that you do not wish to be made publicly available, 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 onhttps://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.

Docket: 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, found in brackets in the heading of this document, into the “Search” box and follow the prompts and/or go to the Dockets Management Staff, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852, 240-402-7500.

FOR FURTHER INFORMATION CONTACT:

Cindy Tworek, the Office of Pediatric Therapeutics, Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, MD 20993-0002, 301-796-9234, OPT@fda.hhs.gov.

SUPPLEMENTARY INFORMATION:

I. Background

On July 9, 2012, the President signed into law the Food and Drug Administration Safety and Innovation Act (FDASIA) (Pub. L. 112-144). Section 508 of FDASIA directs the Secretary of HHS to submit a report to Congress on the implementation of sections 505A and 505B of the Federal Food, Drug, and Cosmetic Act, which are commonly known as the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA), respectively.

The first report was required to be submitted to Congress by July 9, 2016, and subsequent reports are required every 5 years thereafter, with the next report due to be submitted in July 2026. FDASIA also requires FDA to obtain, at least 180 days prior to submission of the report, input from interested parties, including: patient groups (including pediatric patient groups), consumer groups, regulated industry, academia, and any other interested parties to obtain any recommendations or information relevant to the report including suggestions for modifications that would improve pediatric drug research and pediatric labeling of drugs and biological products. In addition, on August 18, 2017, the FDA Reauthorization Act of 2017 (Pub. L. ( printed page 324) 115-52) was signed into law, which outlined additional requirements to be included in the report.

II. 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 the 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.

III. Participating in the Public Meeting

Registration: For more information and to register for the public meeting, please visit: https://www.fda.gov/news-events/fda-meetings-conferences-and-workshops/interested-parties-meeting-implementation-best-pharmaceuticals-children-act-and-pediatric-research. Please provide complete contact information for each attendee, including name, email address, and affiliation. Registration is free and based on space availability for in-person attendance, 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. Eastern Time. Early registration is recommended because seating is limited; therefore, 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 a.m. We will post a notice on the 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. Eastern Time.

Requests for Oral Comment: During online registration you may indicate if you wish to present an oral comment during a public comment session, and which topic(s) you wish to address. We will do our best to accommodate requests to make oral comments. Individuals and organizations with common interests are urged to consolidate or coordinate their comments. All requests to make oral comments, for both virtual and in-person attendees, must be received by the close of in-person registration on May 1, 2025, 11:59 p.m. Eastern Time. Following the close of registration, we will determine the amount of time allotted to each presenter and the approximate time that the oral comment session is to begin, and will notify participants making an oral comment by May 5, 2025, 11:59 p.m. Eastern Time. If making an oral comment, any presentation materials must be emailed to OPT@fda.hhs.gov (see FOR FURTHER INFORMATION CONTACT ) no later than May 9, 2025, 11:59 p.m. Eastern Time. No commercial or promotional material will be permitted to be presented or distributed at the public meeting.

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. The meeting web page link is: https://www.fda.gov/news-events/fda-meetings-conferences-and-workshops/interested-parties-meeting-implementation-best-pharmaceuticals-children-act-and-pediatric-research.

Although FDA verified the website addresses in this document, please note that websites are subject to change over time.

Transcripts: Please be advised that as soon as a transcript of the public meeting is available, it will be accessible at https://www.regulations.gov, https://www.fda.gov/news-events/fda-meetings-conferences-and-workshops/interested-parties-meeting-implementation-best-pharmaceuticals-children-act-and-pediatric-research, or the Dockets Management Staff, Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852, between 9 a.m. and 4 p.m., Monday through Friday, 240-402-7500.

Notice of this meeting is given pursuant to 21 CFR 10.65.

Dated: December 20, 2024.

P. Ritu Nalubola,

Associate Commissioner for Policy.

[FR Doc. 2024-31312 Filed 1-2-25; 8:45 am]

BILLING CODE 4164-01-P

Legal Citation

Federal Register Citation

Use this for formal legal and research references to the published document.

90 FR 322

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Suggested Web Citation

Use this when citing the archival web version of the document.

“Interested Parties Meeting: Implementation of the Best Pharmaceuticals for Children Act and Pediatric Research Equity Act,” thefederalregister.org (January 3, 2025), https://thefederalregister.org/documents/2024-31312/interested-parties-meeting-implementation-of-the-best-pharmaceuticals-for-children-act-and-pediatric-research-equity-act.