The ICH E11 Addendum on clinical investigation in children now enters into implementation

Recommendation

5-7 November 2024
Hamburg, Germany

GMP meets Development
GMP and FDA Compliance in Pharmaceutical Development and IMP Manufacturing

The International Council for Harmonisation, ICH, recently informed that the "E11(R1) Addendum on Clinical Investigation of Medicinal Products in the Pediatric Population reached Step 4 of the ICH Process in August 2017 and now enters into the implementation period (Step 5)". The "Addendum is proposed to address new scientific and technical knowledge advances in pediatric drug development and update the ICH E11 Guideline adopted in 2000", the council says.

This topic was endorsed by the ICH Steering Committee in August 2014. The 17-page document targets "scientific and technical issues relevant to pediatric populations, regulatory requirements for pediatric study plans, and infrastructures for undertaking complex trials in pediatric patient populations that have been considerably advanced in the last decade, without a parallel development of harmonised guidance in these areas", the council notes.

Therefore, the addendum outlines current regulatory perspectives on various topics in pediatric drug development, including extrapolation and the use of modeling and simulation. Its implementation by the ICH regulatory members is expected to "reduce the likelihood that substantial differences will exist among regions for the acceptance of data generated in pediatric global drug development programs and ensure timely access to medicines for children," the council says.

The following three key practical factors should be considered before deciding which types of methodological approaches are to be used in clinical trial design and execution of pediatric clinical trials:

• feasibility,
• outcome assessments,
• long-term clinical aspects, including safety.

As reported before, this is also in line with FDA & EMA´s collaborative Clinical Trials Approach for rare Diseases in Children.

Furthermore, the development of a separate ICH guideline on pediatric extrapolation is expected, which is in line with the updated version of EMA´s draft Reflection paper on the use of extrapolation in the development of medicines for paediatrics published on October 13, 2017 (deadline for comments is January 14, 2018).

For more information please see the ICH E11(R1) Addendum and further documents on the Efficacy Guideline page of the ICH website.