Dear Reader,

Imagine a world where every child, regardless of their condition, has access to safe, effective, and child-friendly therapies. This vision has been the driving force behind CVBF since its inception in 2000. Our journey truly gained momentum in 2005 with our first EU funded project, the FP6 “TEDDY Task-force in Europe for the Drug Development for the Young”, an innovative initiative coordinated by Adriana Ceci even before the Paediatric Regulation came into effect, and still active as a Network of Excellence for Paediatric Research.

This pioneering initiative (TEDDY) laid the foundation for subsequent endeavours in the following EU Framework Program for Research, with CVBF contributing to several FP7 Projects: the multinational paediatric clinical trials CloSed, DEEP, GAPP, NeoMero and NeoVanc, and the network GRiP (Global Research in Paediatrics), coordinated by Carlo Giaquinto, aimed at implementing an infrastructure to stimulate and facilitate the development and the safe use of medicines in children.

Based on these achievements the IMI2 conect4children project started and is still operating as the European paediatric clinical trials network, coordinated by Mark Turner, with CVBF playing an active role in the trainings (Paediatric GCP, Audit & Inspection Readiness, Trial Start Up, Trial Monitoring, and the Advanced Course on Paediatric Clinical Trials), in the standardization and sharing of paediatric clinical trial data, and in the provision of CRO services for all the selected proof of viability not for profit studies.

Meanwhile, under the Horizon 2020 the Paediatric Clinical Research Infrastructure Network (PedCRIN) started as a project, coordinated by Jaques Demotes, bringing together the European Clinical Research Infrastructure Network (ECRIN) and the founding partners of the European Paediatric Clinical Trial Research Infrastructure (EPCT-RI), including CVBF to develop the necessary tools and capacity to enhance the quality, safety, efficacy and ethical standards of multinational, non-commercial paediatric clinical trials.

Notwithstanding these initiatives, children and young patients are still left behind an acceptable level of available treatments. Most of the drugs used today have not been specifically developed for, or adequately tested on, or authorised for children of all the different ages, considering their drugs reactions change radically with growth and development.

As it is well known, the anatomical and metabolic differences between children and adults, along with their distinct responses to therapeutics and the unique types and manifestations of pediatric diseases, make it clear that pediatric trials based on adult medicines are insufficient. This is particularly true for rare conditions and neonates.

To address this critical gap CVBF took the initiative to focus on increasing knowledge of human development and on developing tailored drugs and devices specifically for children with the Horizon 2020 project EPTRI, European Paediatric Translational Research Infrastructure, coordinated by Donato Bonifazi, which is now established as a scientific international association (EPTRI AISBL) based at the University of Leuven, Belgium, and composed of 14 members from 7 countries, engaged in basic, preclinical, translational, and clinical paediatric research.

EPTRI is also actively promoting the enhancement of paediatric research at institutional level, by promoting its inclusion in the ESFRI National and European Roadmaps, by creating consensus among the stakeholders with its Manifesto on Paediatric Research, and participating to all the public consultations affecting paediatric research. Recently, the proposal from the EC of the new general pharmaceutical legislation, including the orphan and paediatric regulations, gave the way to the amendments from EPTRI approved by the EU Parliament on 11 April 2024.

With your support, we can continue making a meaningful difference in the lives of all the children and especially the young patients affected by rare conditions. Together, we can pave the way for a future where no child is left behind and hope prevails over adversity.

We are dedicated to increasing visibility and comprehension of rare and paediatric diseases through ongoing awareness and education initiatives. Within the activities of the European Joint Program on Rare Diseases, we recently released an informative video featuring insights from CVBF Clinical Projects Director Mariagrazia Felisi, who also serves as the TEDDY Network Board Chair, detailing the challenges and strategies involved in conducting the international DEEP-2 paediatric clinical trial for a rare disease. We also published two lay summaries explaining the key findings of the DEEP-2 paediatric study in a kid-friendly manner, one tailored for children aged 6-12 and another for adolescents aged 13-17. Recent publications from our researchers involved in the c4c project underscore their commitment to advancing efforts in paediatric data standardization, further supporting research into conditions affecting children.

There are several major paediatric research and advocacy events taking place in Bari, Italy in July 2024.

The 2024 iCAN Advocacy and Research Summit, scheduled for July 15-19, promises to be an extraordinary gathering of kids members from iCAN chapters from around the world. For five days the summit will spotlight groundbreaking paediatric research, healthcare innovation, collaboration, and advocacy.

Recently, we have actively participated in several key events focused on advancing paediatric and rare disease research. In March 2024, we presented an abstract on the innovative computational approach involving virtual patient cohorts and in silico trials at the conference hosted by the Italian Society of Pharmacology at the University of Trieste. Earlier that month, we took part in the kick-off meeting for ERAMET, coordinated by the University of Namur with the participation of 16 other partners which has the objective to provide an integrated approach for developers and regulators decision-making for paediatric and orphan drugs and implement a framework for establishing the credibility of Modelling & Simulation methods and related results as sources of evidence within regulatory procedures. Additionally, our Scientific Director Giovanni Migliaccio, who also serves on the EPTRI Board, spoke at the international Rare Diseases and Orphan Drugs Congress organized by Rare Diseases Greece in Athens in late February, promoting collaboration across the rare disease community.

The CVBF CEO, Donato Bonifazi, representing TEDDY, is a member of the Coordinating Group of the Enpr-EMA (European Network of Paediatric Research at the European Medicines Agency), and in December 2023 he was selected as a permanent member of the Advisory Group of the ACT EU Multistakeholder Platform, allowing the paediatric research community to have a voice in shaping policies and strategies. The ACT EU platform is an initiative launched in 2022 by the European Commission, the European Medicines Agency, and the Heads of Medicines Agencies to accelerate clinical trials in the EU.

CVBF participated in the European Joint Programme on Rare Diseases (EJP RD) Final Conference, held on 27-28 May 2024 in Bari, Italy. This two-day conference brought together experts, professionals, and advocates from around the world who are involved in efforts surrounding rare diseases. The event provided a valuable platform for discussing the challenges and advancements in the rare disease space. Attendees were able to share insights, explore potential solutions, and facilitate new collaborations aimed at improving research and care for individuals affected by rare conditions.

The FDA claims that the Research Acceleration for Cure and Equity (RACE) for Children Act, enacted in 2017, has significantly increased the number of planned studies to test drugs for pediatric cancers. Between 2020-2023, the FDA issued 131 investigational Pediatric Study Plans under the RACE Act, with most (86%) including waivers or deferrals for pediatric study requirements under the previous Pediatric Research Equity Act (PREA).

Read more