Module232025

12/05/2025

Other Identified Aspects

When both FDA and EMA had Regulatory Guidances specific to a disease or condition, they contained similar permissive or exclusionary language

Silent Regulatory Guidances

● Often failed to mention any paediatric cohort ● Differences in disease between populations were discussed but did not state whether the degree of “dissimilarity” leads to exclusion from study ● When paediatric extrapolation was acceptable there was no mention of the opportunity for inclusion ● Used clauses such as “generally not acceptable” without examples to clarify when inclusion could be acceptable ● Includes language on the need to generate “additional safety and effectiveness data” in paediatric cohorts, but did not discuss if a separate study is required

The Organisation for Professionals in Regulatory Affairs

41

M Dehlinger-Kremer, 14 May 2025

41

Summary

What to remember

Call for Action

− To present knowledge, this study represents the first comprehensive analysis of age-inclusive language within regulatory guidance for two globally important health agencies (FDA & EMA) − Important opportunities for the enhancement of guidance were identified − For example, contextualization of developmental factors influencing adolescent disease progression provides insights into the role of adolescent inclusion. If addressed, guidance documents can facilitate broader acceptance of age-inclusive trial methodologies and accelerate adolescent access to medicines

To address the gap of lack of age-inclusive language , following proposals were made − Silent regulatory guidance should be prioritized for review and revision − Existing text of longstanding exclusionary regulatory guidance should be scrutinized for continued applicability − Stakeholders (adult and paediatric trialists, industry, ethics committees, regulators) should work together to facilitate the evolution of regulatory thinking on adolescent inclusion in adult trials and/or the conduct of adolescent trials in parallel to the adult program

The Organisation for Professionals in Regulatory Affairs

42

M Dehlinger-Kremer, 14 May 2025

42