Towards a Framework for N-of-1 Individualized Therapies in the Netherlands
Recent developments in individualized medicine have challenged traditional approaches to the development, evaluation, and regulation of therapies. High-profile examples such as milasen and other N-of-1 treatments, including the case of Baby KJ, illustrate both the potential of highly tailored therapies and the complexity of bringing them responsibly to patients within existing regulatory and healthcare frameworks.
On 20 May 2026, the MEB and CCMO jointly welcome you to the Science Day 2026, dedicated to exploring these challenges under the theme “Towards a Framework for N-of-1 Individualized Therapies in the Netherlands.” This event brings together regulators, clinicians, researchers, and other key stakeholders to reflect on international experiences and on current developments in the Netherlands including N-of-1 approaches for antisense oligonucleotides and advanced therapy medicinal products (ATMPs).
A highlight in the programme will be the launch of the European Medicines Regulatory Database (EMRD), co-developed by Utrecht University and the MEB. By making regulatory information on European medicines findable, accessible, interoperable, and reusable, the web-based EMRD platform aims to support transparency, learning, and the responsible use of regulatory data.
Through presentations, poster sessions, and a panel discussion, the Science Day will examine what can be learned from international initiatives, which challenges researchers and clinicians encounter in the Netherlands, and how collaboration across the therapeutic and regulatory chain can be strengthened. Together, we will reflect on the elements needed to move towards a roadmap for N-of-1 therapies in the Netherlands with the goal of bringing these therapies to patients.
We look forward to welcoming you on 20 May 2026 for a day of discussion, reflection, and networking.
Register now
Registration is now open. You can register via the link below.
Not able to attend in person? The Science Day will also be live broadcasted. A link to the broadcast will be shared on this website a few days in advance.
For in-person attendance, registration is required. For the livestream, registration is not required.
The programme
12.00 – 13.00
Registration and lunch
13.00 – 13.05
Opening and welcome (Dr. Marjon Pasmooij, Head Science Department, MEB & Associate Professor Drug Regulatory Science, Utrecht University)
13.05 – 13.20
Regulatory Science at the CCMO and MEB (Prof. Frits Rosendaal, Chair Science Committee CCMO & Professor of Clinical Epidemiology, LUMC)
13.20 – 13.40
Launch of the European Medicines Regulatory Database – making regulatory data more accessible (Dr. Lourens Bloem, Assistant professor Clinical Therapeutics, Utrecht University)
13.40 – 13.45
Introduction to the topic
13.50 – 14.10
From the story of Milasen to current n-of-1 developments (Dr. Tim Yu, Physician-Scientist at Boston Children's Hospital and associate professor of Pediatrics, Harvard Medical School)
14.10 – 14.30
Poster pitches
14.30 – 15.15
Break & Poster session
15.15 – 15.35
Antisense oligonucleotide treatment development for N-of very few patients with brain disorders (Prof. Willeke van Roon-Mom, Professor and co-lead of the Dutch Center for RNA Therapeutics, Leiden University Medical Center, department of Human Genetics)
15.35 – 15.55
Prime Time for patients with metabolic diseases (Prof. Sabine Fuchs, Professor Metabolic Diseases and Innovative Therapies, University Medical Center Utrecht)
15.55 – 16.15
Learnings on Rare Disease: A pilot program for rare therapies and individualised medicines in the United Kingdom (Julian Beach, Int. Executive Director Healthcare Quality and Access, Medicines and Healthcare Products Regulatory Agency, United Kingdom)
16.15 – 16.35
A roadmap for N-of-1 treatment in the Netherlands (speaker tbc)
16.35 – 16.55
Panel discussion
Panel members: Dr. Tim Yu, Prof. Willeke van Roon-Mom, Prof. Sabine Fuchs, Julian Beach, other members tbc
16.55 – 17.00
Reflections of the day
17.00 – 18.00
Drinks & networking