The Children's Tumor Foundation
10/21/2025 | Press release | Archived content
CTF Europe Takes the Stage at Brussels Conference to Advance Orphan Drug Innovation
CTF Europe Takes the Stage at Brussels Conference to Advance Orphan Drug Innovation
October 21, 2025Featured, Featured EU, Press Release
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On October 22, Children's Tumor Foundation (CTF) Europe Managing Director Dariusz Adamczewski will represent the Foundation at theOn October 22, Children's Tumor Foundation (CTF) Europe Managing Director Dariusz Adamczewski will represent the Foundation at the European Forum for Good Clinical Practice's Better Medicines for Children Conference in Brussels.
Dr. Adamczewski will speak on the panel "Optimising the Clinical Development Path of Orphan Medicines," alongside representatives from the European Commission, European Medicines Agency, Sanofi, Newcastle University, and Necker Enfants Maladies University Hospital, to explore how collaboration and innovation can improve the clinical development process for rare disease treatments.
Orphan medicines face distinct scientific and logistical challenges-from small patient populations to limited clinical trial data-that can slow progress and increase costs. The session will focus on strategies to overcome these barriers, emphasizing how initiatives like EU-PEARL are pioneering new ways to accelerate and optimize development through platform trial models.
"As Director of CTF Europe and implementation lead for EU-PEARL, I look forward to contributing practical insights on streamlining orphan-medicine trials and sharing lessons learned across our public-private partnership in designing rare-disease platform trials," said Dariusz Adamczewski. "By rethinking how we design and conduct clinical studies, we can make research more efficient, inclusive, and impactful, ultimately accelerating the development of therapies in the high unmet need areas, such as NF and other rare diseases."
With EU-PEARL and the NF1 platform trial, CTF Europe is helping to redefine how rare disease research is conducted, fostering collaboration among patients, clinicians, academia, industry, and regulators. This work reflects CTF's broader commitment to driving innovation in rare disease drug development, ensuring that patients and families across Europe and around the world benefit from faster, more efficient access to life-changing therapies. By championing platform trial models and patient-centered collaboration, CTF Europe continues to lead the way toward a stronger, more connected ecosystem for NF and other rare diseases, insights that Dariusz will bring directly to the orphan medicines panel.
"Optimising the clinical development path of orphan medicines"
October 22nd, 2025 | 10:35am
Moderators:
Christina Kyriakopoulo, EU Commission
Kristina An Haack, Sanofi
Panelists:
Dariusz Adamczewski, Children's Tumor Foundation Europe
Kristina An Haack, Sanofi
Michela Guglieri, Newcastle University
Rima Nabbout, Necker Enfants Malades University Hospital
Dominik Karres, European Medicines Agency
The Children's Tumor Foundation published this content on October 21, 2025, and is solely responsible for the information contained herein. Distributed via Public Technologies (PUBT), unedited and unaltered, on October 23, 2025 at 14:43 UTC. If you believe the information included in the content is inaccurate or outdated and requires editing or removal, please contact us at [email protected]