Wednesday 4th July

9.00 – Biomarkers
Pietro Spitali and Andreas Roos

Objective: to explain why types of biomarkers exist and how they can be used in trial planning and as outcome measures, the regulatory perspective on biomarkers, highlighting ongoing networking efforts (ENMC workshops)


10.00 – Showcase: validation of MRI as a biomarker in clinical trials
Volker Straub

Objective: introduce MRI as an outcome measure in trials, ongoing efforts to validate this biomarker for assessment of muscle quality in neuromuscular diseases


11.00 – Coffee/tea


11.30 – Showcase: PROM development
Nathalie Goemans

Objective: explain what happens after a drug is approved, marketing, postmarketing studies, reimbursement and challenges around drug access


12.15 – Lunch


13.15 – After EMA approval: lessons learned, challenges
Ria Broekgaarden

Interactive workshop with Ria Broekgaarden (VSN Netherlands, PAB member EURO-NMD) to discuss challenges around drug access, start stop critera and drug migration


14.15 – Role of patient organisations in research: examples, achievements from start up to registries
Ria Broekgaarden

Objective: to learn how patients are equal partners in drug development and can be instrumental in the processes involved in drug development


15.30 – Clinical trial practicality forum
moderated by Teresinha Evangelista

Participants: trial nurse, clinician involved in clinical trials, patients involved in clinical trial, patient representatives
In this panel session participants will briefly introduce themselves and their personal experience with being involved in a clinical trial as a health care professional or a patient. What is it like? What is challenging? What is burdensome? Where things going as expected?
This is followed by an interactive panel discussion where participants can ask questions and contribute to the discussion


16.30 – After approval: Proms and HTA – TBC



17.30 – End of day 3

The ERNs are co-funded by the
European Union (Health Programme and CEF)

EU Commission

“EURO-NMD is one of the 24 European Reference Networks (ERNs) approved by the ERN Board of Member States. The ERNs are co-funded by the European Union (Health Programme and CEF).
For more information about the ERNs and the EU health strategy,
please visit