The International Rare Diseases Research Consortium (IRDiRC) is pleased to participate in this year’s World Orphan Drug Congress Europe, taking place from 27–29 October 2025 in Amsterdam, Netherlands.
We warmly invite all attendees to visit the IRDiRC booth (10.510) to learn more about our mission, collaborative initiatives, and how we are accelerating the development of diagnostics and therapies for people living with rare diseases worldwide.
IRDiRC members will be actively contributing to several key sessions throughout the congress. We encourage you to attend the following presentations and discussions:
Programme
Monday, 27 October
14:00–16:30
Pre-Congress Workshop: Rare Disease Public-Private Partnerships
Organized by: Sanofi
Speaker: Vinciane Pirard, Scientific Advocacy and Insights, Global Medical Affairs Rare Diseases at Sanofi; Chair of IRDiRC Companies Constituent Committee (CCC)
The workshop will present the work of the consortium in partnership with the Rare Disease Moonshot and The European Federation of Pharmaceutical Industries and Associations (EFPIA). Read the report here: https://irdirc.org/wp-content/uploads/2025/04/Report_The-contribution-of-private-in-PPP.pdf
Tuesday, 28 October
11:30–12:30
Track 6: Competition of Regulatory Ecosystems in Approving Medicines – Policy Implications in the Case of Europe
Speaker: Violeta Stoyanova-Beninska, Senior Scientific Specialist, European Medicines Agency (EMA); Chair of IRDiRC Regulatory Scientific Committee (RSC)
14:20
Fondazione Telethon Not-for-Profit Model: From Research to Distribution of ATMPs for Ultra-Rare Conditions
Speaker: Stefano Benvenuti, Public Affairs Manager, Fondazione Telethon; Chair, IRDiRC Funders Constituent Committee (FCC)
14:50
IRDiRC Task Force on Prevention: Opportunities and Challenges in the Shift from Treatment to Prevention
Speaker: Dan O’Connor, Director, Regulatory Policy and Early Access, ABPI; Chair of IRDiRC Therapies Scientific Committee (TSC)
More information about the IRDiRC Task Force on Preventive Medicines: https://irdirc.org/facilitating-the-development-of-preventive-medicines-at-scale-for-rare-diseases-2/
16:40
Engagement of young people in rare diseases research: a roadmap for action
Speaker: Anneliene Jonker, Scientific Director, Duchenne Parent Project and Vice Chair of IRDiRC Therapies Scientific Committee (TSC)
Wednesday, 29 October
09:45
Keynote Panel: New Treatments on the Block – 2025’s Rare Disease Pipeline
Topics:
- Breakthroughs in rare disease and gene therapies
- Evolving policy and regulatory frameworks
- Ensuring sustainability in RD drug development
Speakers:
- PJ Brooks, Program Director, Office of Rare Disease Research at NCATS; Member, IRDiRC Funders Constituent Committee (FCC)
- Violeta Stoyanova-Beninska, EMA; Chair of IRDiRC Regulatory Scientific Committee (RSC)
14:25
Orphan Drugs Development: Productivity and Probability of Success
Speaker: Samantha Parker, Patient Engagement Lead Rare Diseases, Italfarmaco; Vice Chair of IRDiRC
Event details:
- Dates: Pre-congress workshops: 27 October, 2025; Conference and Exhibition: 28-29 October, 2025
- Venue: RAI Congress Centre, Amsterdam, Netherlands
- More information: here
