EMA Consults on Pediatric Study Preparedness Framework

Regulatory NewsRegulatory News | 12 September 2019 |  By 

The European Network of Pediatric Research at the European Medicines Agency (Enpr-EMA) on Thursday opened a public consultation on a draft framework for pediatric clinical trial preparedness.
Specifically, the 23-page framework provides recommendations for a structured assessment to determine preparedness for conducting pediatric studies to “increase the likelihood of a smooth and timely course of a pediatric clinical trial.”
Within the framework, EMA recommends steps to collect relevant information that could increase preparedness; involve contributors including expert groups, patients and regulators; and set out a structured process to follow “to avoid a futile trial.”
EMA says it is looking for feedback from stakeholders including clinical trial sites, investigators, networks, sponsors and patients by 15 November to identify gaps that will be addressed when the framework is finalized.
The draft framework explains that such an assessment should include activities that go beyond study feasibility assessments. “Design needs to take account of the specificities of infants, children and young people while maximizing the use of extant data (including preclinical data such as toxicity) and minimizing the burden of research in these populations,” the paper states.
Preparation for pediatric studies, according to the framework, should begin before and carried out alongside the design of clinical development plans.
The need for increased preparedness stems from the difficulties sponsors of pediatric studies often encounter, including small patient populations and the burden research can place on participants and their families.
“According to the results of our survey … sites often over-estimate what is possible and it was reported that sponsors, regulators and ethics committees can disagree about what should be done or is possible during drug development,” EMA writes.
The paper also argues that factoring in patient perspectives, needs and experiences during early preparations can influence development plans and reduce study complications such as amendments and delays.


© 2023 Regulatory Affairs Professionals Society.

Discover more of what matters to you

No taxonomy