Paediatric Update - What evidence is necessary for decision making?

Recent discussions within the paediatric community around ethical concerns and practical issues with randomized clinical trials in children show the need to find better methodological study designs that will spare children from unnecessary research.

This need has triggered clinicians, statisticians and regulators to discuss new ways of evidence generation in children that can be used for paediatric product development. Modelling and extrapolation designs are being discussed that allow the usage of data from other population segments for new indication development and regulatory decision-making.

The International Council of Harmonisation (ICH) has already established a new expert group to develop international guidance. Regulators in Europe and around the world are discussing how to use these new concepts in practice.

If such new designs are successfully implemented into product development, even less RCT data than today will be available at the licensing point of a new paediatric medicine.

This might create significant methodological issues at the level of the health technology assessment (HTA) review to determine the added therapeutic value of a newly licensed paediatric indication and impact its reimbursement status in a healthcare system.

We will hear from Regulators, Industry and patients about the ongoing debate that will help raising awareness amongst stakeholders for further dialogue and potential solutions. 

This webinar will follow-up on the discussion at a breakout session during the EFGCP/DIA Paediatric conference last October in Brussels.