Innovative therapies – can it be smarter and faster?

The world of rare diseases is developing rapidly. Innovative therapies, such as mRNA, gene therapy and CAR-T cell therapy, offer unprecedented opportunities for children and adults with rare diseases. At the same time, these innovations also raise social debate and questions about responsibility, effectiveness and affordability.

During this symposium, we will discuss various questions with you, such as:

• Who should develop these therapies – academia, the pharmaceutical industry, or both?
• Who are we doing it for – are we focusing on the individual patient or on society as a whole?
• How much should it cost – and who decides that?

Below you will find the registration link and the programme with contributions from leading speakers from the clinical, scientific, industrial and policy fields. By combining knowledge and experience, we can make a difference for patients together.

Programme

12.45 – 13.30 Lunch in the Accelerator atrium
13.30 – 13.35 Welcome and opening by chairperson
13.35 – 14.00 Keynote speech

Innovative therapies – who should develop them?

14:00 – 14:45 Two pitches & panel discussion

Innovative therapies – who are we doing it for?

15:15 – 16:00 Two pitches & panel discussion

Innovative therapies – what should they cost?

16:10 – 16:55 Two pitches & panel discussion
16:55 – 17:10 Closing remarks by chairperson
17:10 – 18:00 Drinks reception

Speakers and panellists

• Teun van Gelder, Professor of Clinical Pharmacology, Leiden University Medical Centre (LUMC)
• Carla Hollak, Internist and Professor of Medicines and Society, University of Amsterdam (UvA)/Amsterdam University Medical Centre (Amsterdam UMC) and Chair of the Board of RARE-NL
• Marc Jansen, Paediatrician, Immunologist-Rheumatologist, UMC Utrecht/WKZ
• Chris van Lieshout, Assistant Professor of Health Economic Evaluations, UMC Utrecht
• Ludo van der Pol, Professor and Head of SMA Expertise Centre, UMC Utrecht/WKZ
• Raymond Schiffelers, Professor and Head of CDL, UMC Utrecht
• Frank Staal, Professor of Molecular Stem Cell Biology at Leiden, LUMC Leiden

More speakers to follow.

Symposium location

Accelerator Utrecht (Science Park)
Uppsalalaan 17
3584 CT UTRECHT

Accreditation is requested from

• NIV Dutch Society of Internal Medicine
• NVK Dutch Society for Paediatrics

Contact: childhealth@umcutrecht.nl