EMA finalizes pediatric trial preparedness framework
The European Medicines Agency (EMA) has released the final version of a framework for pediatric clinical trial preparedness. The final document aims to “increase the likelihood of a smooth and timely course of a paediatric clinical trial, integrating information from multiple stakeholders on what is possible within individual studies and therefore also for the overall drug development plan within which a trial is embedded,” according to EMA.This framework’s release follows a September 2019 draft version that was open for a 2-month public consultation period. Considerations posed by stakeholders during that period were included in the final document, said EMA. (Related: EMA consults on pediatric study preparedness framework, Regulatory Focus 12 September 2019)
The preparedness framework tops a list of priorities for EMA’s European Network of Pediatric Research (Enpr-EMA), whose overarching goal is to facilitate studies that increase the availability of medicines for children.
Preparedness for clinical trials involving children can avoid trial delays and even failure, noted the agency, adding that the complexities of pediatric trials means that foresight and planning are key. “For the majority of pediatric clinical trials problems can be addressed by using all available data to estimate what is possible using a structured approach,” said EMA in the framework.
The framework includes recommendations on data collection, the use of a structured approach for designing and conducting research, the use of appropriate resources for pediatric trials, and the best approaches to prepare trial plans and studies. The framework also provides advice about how best to involve other contributors, including individual clinical trial sites, expert groups and societies, patients and families, and regulators.
“Other actions are needed beyond the preparation of individual clinical trials,” said EMA in the framework, calling for the entire pediatric research community to improve site selection and management, address practicalities such as participant travel, and to look at the big picture when designing data collection strategies. The framework also asks those involved in pediatric research to “lobby for greater recognition of the importance of research and readiness to participate in research amongst healthcare professionals and across society.”
Other Enpr-EMA ongoing work addresses the need to include young people and their families in all aspects of pediatric research; how best to collaborate with ethics committees; how to obtain informed consent in pediatric clinical trials; and how to interact with pharmaceutical companies. International collaboration, best practices for public consultation, and collaboration with pediatric research nurses are also priorities for the working group.
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