Dariusz Adamczewski, MD, is Managing Director of Children's Tumor Foundation Europe (CTF Europe), the Brussels-based patient foundation dedicated to neurofibromatosis and schwannomatosis (NF). CTF Europe combines patient advocacy with an active role in drug development and research. He leads the development of EU-PEARL NF1, a multinational adaptive basket platform trial in neurofibromatosis type 1, and Atlas NF, a European mapping of NF clinical centres and trial-site capability, while overseeing the foundation's research grant portfolio, pharmaceutical partnerships and health policy engagement. A physician by training, he has a career spanning clinical medicine, government and industry. He held senior positions at the Polish Ministry of Health, where he led major health system reforms, and brings industry experience from Johnson & Johnson and Pfizer. His work focuses on the infrastructure rare disease research needs to deliver for patients in Europe: regulatory science, platform and basket trial design, health economics, and sustainable funding models that bring patient organisations, academic networks, regulators and companies together around shared goals. He is a committed advocate for patient-led foundations acting as partners in research and development ecosystem.
- Patient led solutions to orphan drug development and access bottlenecks
- How to solve the issue of small patient populations through statistical innovation, regulatory flexibility and patient-centricity
- Utilising Real-World Data and natural history studies
- Creating meaningful endpoints that improves patients’ quality of life