MSM #6 Paediatric Chronic Kidney Disease

The sixth and final c4c International Multi-Stakeholder Meeting (MSM) took place on 28–29 April 2025 in Heidelberg, Germany, ahead of the ESCAPE Network Meeting. Organised in collaboration with ERKNet, the European Rare Kidney Disease Reference Network, the meeting focused on the unmet needs and treatment development landscape for children and adolescents living with chronic kidney disease.

Held in a hybrid format, the event brought together 128 participants, including 50 online attendees, and marked a significant conclusion to the c4c MSM series. Stakeholders included patients, patient representatives, clinicians, academics, regulators from EMA and FDA, and representatives from the pharmaceutical industry.

The meeting was co-chaired by Professor Franz Schaefer, ERKNet Coordinator, and underscored the critical value of multi-stakeholder collaboration in shaping research and regulatory strategies that better address the needs of paediatric patients with rare diseases. Notable contributions from regulators, including Lynne Yao (FDA) and Gunter Eggert (EMA), highlighted the global commitment to accelerating innovation in this field.

Over the two days, participants engaged in meaningful discussions to outline a strategic pathway for improving the development and access to new therapies for paediatric chronic kidney disease. Outcomes of the meeting will be captured in a forthcoming publication, and ERKNet is committed to transforming the meeting’s conclusions into concrete actions.

This meeting concluded a successful series of c4c Multi-Stakeholder Meetings, which since inception have created an invaluable platform for collaborative dialogue in the development of medicines for children and young people. The model has proven essential in defining unmet needs and informing prioritisation processes – a foundation that will be critical as the revised EU pharmaceutical package takes shape.

c4c warmly thanks all contributors and partners involved in this impactful series.

MSM #5 Paediatric Irritability

conect4children is pleased to announce that the fifth c4c International Multi-Stakeholder Meeting (MSM) on paediatric Irritability was successfully held on 18 and 19 March 2024 in Nice and virtually. The meeting was held immediately after the European College of Neuropsychopharmacology (ECNP) New Frontiers Meeting 2024 in Nice to maximise participation and interaction.

More than 100 participants from 22 countries worldwide attended the event, evenly split between on site and virtual. Participants included International academic experts in child and adolescent psychiatry, representatives from pharmaceutical companies interested in developing relevant assets or having relevant assets, representatives from European regulators (EMA and National Agencies), young patients, parents and their advocates.

The overarching objective of the meeting was to discuss and propose a strategy to improve the timely development of and access to relevant therapeutic interventions, properly addressing paediatric unmet needs, introducing innovative development pathways in the regulatory environment and increasing accessibility for all patients.

Want to know more? CLICK HERE 

MSM #4 Perinatal Asphyxia

 Call for Expression of Interest

conect4children is pleased to announce that the fourth c4c International Multi-Stakeholder Meeting (MSM) will address the topic perinatal asphyxia. The meeting will be a hybrid meeting that will take place on 18 and 19 September 2023, ahead of the 5th jENS (joint European Neonatal Societies) conference in Rome. 

Who can participate?

International academic experts in perinatal asphyxia, experts in neonatal preclinical and biomarker research, representatives from pharmaceutical companies developing relevant assets, representatives from regulators (EMA, FDA) and patients, parents and their advocates. The meeting will be on invitation only. Interested participants can apply by filling the Expression of Interest form available here.

Deadline for submission: 30 July 2023 !       Want to know more? CLICK HERE

READ ABOUT THE OUTCOMES FROM THE 4TH MSM HERE!

MSM #3 - Feb 21st 2023

A 3rd c4c Multistakeholder Meeting (MSM) was held on 21 February in Berlin; the topic was medical devices for children with Type 1 diabetes. The aim of the meeting was to define the unmet medical needs, understand the regulatory environment in the EU and US and learn about the challenges faced by industry when developing devices for children. The strategic discussion identified actions to promote access to medical devices for all children of all ages. MSMs are designed to facilitate discussion and interaction among relevant stakeholders on the development of medicines in the best interest of children and adolescents. More details of the outcome of this MSM will soon be available on the c4c website.

Read the meeting report here!

MSM #2 - Mar 1-2nd 2022

On 1-2 March 2022, c4c hosted a successful 2nd online c4c Multi-Stakeholder Meeting on paediatric Atopic Dermatitis (AD). Many thanks to speakers, the Programme Committee and to the 100 participants from academia, regulatory bodies, young patients, advocates and industry representatives. Presentations included biology/immunology of AD in children, perspective from young patients and families and description of industry assets. Over 60 drugs in clinical development or approved were identified.  Panel discussions focused on paediatric unmet medical needs and improvement of the evaluation of new medicines for children and adolescents. A scientific manuscript for a specialized journal is currently in development.


Read the meeting report here

MSM #1 - April 14-15th 2021

More than 100 stakeholders from academia, industry, regulators and patients/patient associations attended our first Multi-Stakeholder Meeting (MSM) on April 14 and 15, 2021. Our first c4c MSM was held online and focused on Paediatric inflammatory Bowel Disease (IBD). There are multiple drugs in development for IBD, however several clinical trials, as part of agreed pediatric development plans, are attempting to enroll patients with limited success. Please find the report on the meeting here.

You can also read the resulting publication: “Paediatric Inflammatory Bowel Disease: A Multi-Stakeholder Perspective to Improve Development of Drugs for Children and Adolescents“.