Module232025

12/05/2025

A study performed by EFGCP Children’s Medicines Working Party sought to understand how adolescent inclusion in adult trials is positioned in regulatory guidance documents as they set critical expectations for trial design and regulatory decision-making.* Characterisation of FDA and EMA Regulatory Guidance (RG)

This study utilised a qualitative analysis approach

Review of the FDA and EMA guidelines in view of statement regarding the inclusion of adolescents into adult clinical trials

In total, 96 Food and Drug Administration (1977 to 2019) and 106 European Medicines Agency (1987 to 2019) guidance documents were identified for analysis. The guidance contained explicit or implicit recommendations

RG were adjudicated to identify FDA & EMA recommendations related to adolescent patient participation utilizing a standardized categorization

Three categories for RG characterization:

1. Permissive – RG includes any language permitting adolescent inclusion (either explicitly or implicitly) 2. Exclusionary – RG includes any language prohibiting adolescent inclusion (either explicitly or implicitly) 3. Silent – RG makes no reference (explicit or implicit) to the adolescent population

EFGCP = European Forum for Good Clinical Practice; CMWP = Children’s Medicines Working Party *Ref: Strategies to facilitate adolescent access to medicines: Improving regulatory guidance

* C.Bucci-Rechtweg et al , Strategies to facilitate adolescent access to medicines: Improving regulatory guidance , Clinical TrialsVolume 20, Issue 1, February 2023, Pages 13-21

The Organisation for Professionals in Regulatory Affairs

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M Dehlinger-Kremer, 14 May 2025

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Source Document Identification for the Characterization of FDA and EMA Regulatory Guidance EMA database 2 FDA database 1

*Guidelines included all ‘Guidelines’, ‘Reflection papers’, ‘Concept Papers’ & ‘Points to Consider’, excluded ‘Biostatistics’ & ‘General’ guidelines **Out of scope guidance included: ‘Core summary for product characteristics’, ‘Warning on’ guidelines, ‘ICH’, ‘Position statements’, ‘product-specific core SmPC and product leaflet guidance’ 2 EMA database 12 DEC 2019 https://www.ema.europa.eu/en/human-regulatory/research development/scientific-guidelines/clinical-efficacy-safety-guidelines

*‘Topics’ included ’Antimicrobial resistance’, ‘Cellular & Gene Therapy’, ‘Clinical Antimicrobial’, ‘Clinical Medical’, ‘Clinical Pharmacology’, ‘Combination Products’, ‘Microbiology’, ‘Rare Diseases’, and ‘Real World Data / Real World Evidence’

1 FDA (accessed 11 NOV 2019); https://www.fda.gov/drugs/guidances-drugs/all-guidances-drugs

* C.Bucci-Rechtweg et al , Strategies to facilitate adolescent access to medicines: Improving regulatory guidance , Clinical TrialsVolume 20, Issue 1, February 2023, Pages 13-21

The Organisation for Professionals in Regulatory Affairs

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M Dehlinger-Kremer, 14 May 2025

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