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)
Objective: introduce MRI as an outcome measure in trials, ongoing efforts to validate this biomarker for assessment of muscle quality in neuromuscular diseases
Objective: explain what happens after a drug is approved, marketing, postmarketing studies, reimbursement and challenges around drug access
Interactive workshop with Ria Broekgaarden (VSN Netherlands, PAB member EURO-NMD) to discuss challenges around drug access, start stop critera and drug migration
Objective: to learn how patients are equal partners in drug development and can be instrumental in the processes involved in drug development
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