Empowering Young Patients: TEDDY’s Role at WODC 2025 Workshop
The World Orphan Drug Congress (WODC), organized by TERRAPINN, held from October 27 to 29, 2025, at the RAI Congress Centre in Amsterdam, saw great success with the workshop titled “Innovative Methodological Approaches to Accelerating Rare Disease Drug Development: Bridging Data, Innovative Design, and Decision-Making”, co-organized by ERDERA, INVENTS and RealiseD on October 27.
The session “What Matters Most: Ensuring Patient Voice Drives Methodological Innovation and Outcome Selection — Focus on INVENTS WP6” presenting TEDDY’s contribution to the INVENTS project, which focuses on the inclusion of small patient populations in clinical trials, optimizing study designs through innovative approaches like in silico trials and Real-World Data (RWD). TEDDY has played a key role in INVENTS in the set-up of the Patients Expert Group (PEG), defining its structure and participation framework for adolescents aged 12 to 18, and fostering the empowerment of patients living with chronic conditions, particularly in rare diseases such as Juvenile Idiopathic Arthritis (JIA) and Multiple Sclerosis (MS). The PEG began its work with a two-day intensive training course (Bari, 2024), focused on the scientific and methodological foundations of the project. This was followed by six monthly online sessions (2025), where leading international experts provided detailed insights into clinical trial methodologies, ethical and regulatory considerations, simulation strategies, and the secondary use of health data.
An emotional highlight of the workshop was the intervention of Valentina Jalby, a 12-year-old patient with Juvenile Idiopathic Arthritis (JIA), who shared her experience and feedback of involvement in the INVENTS project and the PEG program. Accompanied by her mother, Olivia Jalby, Valentina enthusiastically recounted her active participation, which led to numerous collaboration requests after the workshop from other organizations and professionals in the field to include her voice in the decision-making processes. Her testimony underscored the fundamental value of actively involving young patients in research, as well as the crucial role of family support in the process. Without the constant support and active participation of family members, it would be difficult for young patients to fully contribute to research projects. Check out the video of Valentina Jalby’s presentation on the TEDDY YouTube channel: LINK.
This WODC workshop highlighted the importance of listening to the needs of young patients, bringing forth ideas and solutions that might otherwise be overlooked. The WODC 2025 proved to be an event where innovation, collaboration, and patient inclusion, especially involving young people and their families, alongside the presence of experts, are essential in accelerating progress in therapies for rare diseases.