TEDDY’s active participation in the Workshop on 27 October: “Innovative Methodological Approaches to Accelerating Rare Disease Drug Development: Bridging Data, Innovative Design, and Decision-Making”
World Orphan Drug Congress
From October 27-29, 2025, the World Orphan Drug Congress (WODC), organized by TERRAPINN, will return to Amsterdam at the RAI Congress Centre. As the leading global event dedicated to orphan drugs and rare diseases, WODC brings together experts, innovators, and key stakeholders from around the world. The full event agenda is available via the World Orphan Drug Congress Europe 2025 programme: LINK.
Workshop “Innovative Methodological Approaches to Accelerating Rare Disease Drug Development: Bridging Data, Innovative Design, and Decision-Making”
On October 27th at 10:00 AM, ERDERA, INVENTS and RealiseD will co-host an interactive workshop titled “Innovative Methodological Approaches to Accelerating Rare Disease Drug Development: Bridging Data, Innovative Design, and Decision-Making.” This workshop will cover topics such as disease progression models, simulation technologies, the importance of co-creation with health authorities, regulatory harmonization, and patient involvement in orphan drug development.
ERDERA, INVENTS and RealiseD aim to transform research and development in rare diseases. ERDERA strives to make a significant impact on rare diseases by supporting patient-driven research to develop new treatments and diagnostic pathways, leveraging health data, Artificial Intelligence, and digital technologies. INVENTS focuses on the safe and effective inclusion of small patient populations in clinical trials, optimizing study designs through innovative approaches like in silico trials and Real-World Data (RWD). Meanwhile, RealiseD works on setting new standards for clinical trials in rare diseases, bringing together a wide network of partners from academia, industry, and patient organizations.
TEDDY’s role in INVENTS
Dr. Donato Bonifazi on behalf of TEDDY, will be presenting TEDDY’s active contribution to the INVENTS project.
TEDDY has played a key role in establishing the Patients Expert Group (PEG), contributing its expertise in ethical and regulatory issues and ensuring that the perspectives of young patients are included in decision-making processes. Through these efforts, TEDDY aims to empower adolescents living with chronic conditions such as Juvenile Idiopathic Arthritis (JIA) and Multiple Sclerosis (MS), promoting their active involvement in innovative methodologies based on in silico models and on the secondary use of health data.
The PEG launched its work with a two-day intensive training course (Bari, 2024), focusing on the scientific and methodological foundations of the project. Following this, the PEG participated in six monthly online sessions (2025), where leading experts provided in-depth insights into clinical trial methodologies, ethical and regulatory aspects, data extrapolation, and simulation strategies tailored to rare diseases.
During the workshop, an active young patient from the PEG will share her experience working with the INVENTS Project.
It is essential to promote the active involvement of young people in decision-making processes, ensuring that their participation is appropriate to their age and level of maturity. This approach guarantees that their voices are not only heard, but also meaningfully integrated into the research, enabling them to effectively participate in decisions that affect their lives and wellbeing.