On 31 August 2020, the Enpr-EMA (European Network of Paediatric Research at the European Medicines Agency) working group on trial preparedness published recommendations on preparedness of medicines’ clinical trials in paediatrics. Enpr-EMA held a two-month public consultation on the draft document from September 2019 until November 2019, with the aim of identifying potential gaps and presenting a more comprehensive view. The feedback received during the consultation period has informed the final version of this document.
Donato Bonifazi, TEDDY Representative in the Enpr-EMA Coordinating Group, explained the meaning and the importance of trial preparedness in paediatric clinical trials: “The trial preparedness is defined as a structured assessment of the key factors that could 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. To identify the main barriers in paediatric clinical trials leading to delays, or impairment of clinical trial feasibility as well as the good practice and lessons learned, the Enpr-EMA Working group on trial preparedness developed a survey covering questions about four different areas of the planning and conduction of a paediatric clinical trial: planning phase, preparation of the study, study conduct, and the post-study aspects.”
Moreover, he clarified how TEDDY Network provided its contribution in the working group activities and in the development of the document: “TEDDY Network has actively participated in Enpr-EMA Working Group on Trial Preparedness, contributing to review the current regulatory guidance and academic publications in relation to the conduct of trials in the paediatric population to identify discussion on preparedness. Moreover, TEDDY contributed to the development of the questionnaire and to the dissemination of the survey, collecting the experience and suggestions from different stakeholders, allowing a brainstorming session on trial preparedness with stakeholder groups.” TEDDY obtained globally 31 answers from 5 different categories of stakeholders:
- CRO representatives: 7 answers
- Patient Associations: 8 answers
- Study Coordinators: 3 answers
- European Reference Networks: 6 answers
- EnprEMA Networks: 7 answers