Rules and Regulations. Notice of availability

Agency: Food and Drug Administration, HHS
Action: Notice of availability
Citation: FR Doc. 2024-31026 · Docket No. FDA-2022-D-1562

Summary

The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “E11A Pediatric Extrapolation.” The guidance was prepared under the auspices of the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH). The guidance provides a comprehensive and systematic approach to pediatric extrapolation during drug development. Notably, the guidance discusses approaches to safety extrapolation and defining extrapolation as a continuum. The guidance also includes approaches to study designs and statistical methodologies, including modeling and simulation, for developing and implementing pediatric extrapolation. The guidance is intended to provide approaches that can increase the efficiency of pediatric drug development and accelerate the availability of safe and effective drugs approved for use in children. The guidance replaces the draft guidance “E11A Pediatric Exploration” issued on August 29, 2022.

Dates

The announcement of the guidance is published in the Federal Register on December 30, 2024.

Supplementary Information

I. Background FDA is announcing the availability of a guidance for industry entitled “E11A Pediatric Exploration.” The guidance was prepared under the auspices of ICH. ICH seeks to achieve greater regulatory harmonization worldwide to ensure that safe, effective, high-quality medicines are developed, registered, and maintained in the most resource-efficient manner. By harmonizing the regulatory requirements in regions around the world, ICH guidelines enhance global drug development, improve manufacturing standards, and increase the availability of medications. For example, ICH guidelines have substantially reduced duplicative clinical studies, prevented unnecessary animal studies, standardized the reporting of important safety information, and standardized marketing application submissions. The six Founding Members of the ICH are FDA; the Pharmaceutical Research and Manufacturers of America; the European Commission; the European Federation of Pharmaceutical Industries Associations; the Japanese Ministry of Health, Labour, and Welfare; and the Japanese Pharmaceutical Manufacturers Association. The Standing Members of the ICH Association include Health Canada and Swissmedic. ICH membership continues to expand to include other regulatory authorities and industry associations from around the world (refer to https://www.ich.org/ ). ICH works by engaging global regulatory and industry experts in a detailed, science-based, and consensus-driven process that results in the development of ICH guidelines. The regulators around the world are committed to consistently adopting these consensus-based guidelines, realizing the benefits for patients and for industry. As a Founding Regulatory Member of ICH, FDA plays a major role in the development of each of the ICH guidelines, which FDA then adopts and issues as guidance for industry. FDA's guidance documents do not establish legally enforceable responsibilities. Instead, they describe the Agency's current thinking on a topic and should be viewed only as recommendations, unless specific regulatory or statutory requirements are cited. In the Federal Register of August 29, 2022 (87 FR 52788), FDA published a notice announcing the availability of a draft guidance entitled “E11A Pediatric Extrapolation.” The notice gave interested persons an opportunity to submit comments by October 28, 2022. After consideration of the comments received and revisions to the guideline, a final draft of the guideline was submitted to the ICH Assembly and endorsed by the regulatory agencies in August 2024. This guidance finalizes the draft guidance issued on August 29, 2022. The final guidance includes clarification and minor formatting changes to its recommendations on pediatric extrapolation concepts and approaches. The guidance was restructured for easier navigability and includes additional details on key concepts like study designs, modeling approaches, and the role of biomarkers. Additionally, the final guidance includes updates to the figure on the continuum of pediatric extrapolation. This guidance is being issued consistent with FDA's good guidance practices regulation (21 CFR 10.115). The guidance represents the current thinking of FDA on “E11A Pediatric Extrapolation.” It does not establish any rights for any person and is not binding on FDA or the public. You can use an alternative approach if it satisfies the requirements of the applicable statutes and regulations. II. Paperwork Reduction Act of 1995 While this guidance contains no collection of information, it does refer to previously approved FDA collections of information. The previously approved collections of information are subject to review by the Office of Management and Budget (OMB) under the Paperwork Reduction Act of 1995 (PRA) (44 U.S.C. 3501-3521). The collections of information relating to submission of investigational new drug applications, including efficient approaches to clinical trial design and study protocols, have been approved under OMB control number 0910-0014. The collections of information relating to submission of new drug applications have been approved under OMB control number 0910-0001. The collections of information relating to submission of biologics license applications have been approved under OMB control number 0910-0338. III. Electronic Access Persons with access to the internet may obtain the guidance at https://www.regulations.gov, https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs, https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances, or https://www.fda.gov/regulatory-information/search-fda-guidance-documents. Dated: December 16, 2024. P. Ritu Nalubola, Associate Commissioner for Policy. [FR Doc. 2024-31026 Filed 12-27-24; 8:45 am]