On 18 February 2019, ERN-RND organised a multi-stakeholder workshop (clinicians, industry, patients, regulators, and registry administrators) in Amsterdam on trial readiness and registries in rare neurological diseases.
The workshop pursued the following objectives:
- Bring together leading persons of the different stakeholder groups (see proposed list of invitees) and form a RND trial readiness network at European level
- Map clinical status of trial readiness for the different RND
- Discuss and agree minimal quality requirement for trial-ready registries
- Define areas in which ERN-RND can facilitate trial readiness for RNDThe following action points were consented:
- Team up for a pilot studies as regards FAIRification of a RND registries and natural history studies
- Recommendation on Informed Consent Form to be used for RND registries
- Invite and communicate with key opinion leader from the neuromuscular field to benefit from their experiences
- Start structured dialogue with EMA on therapeutic biomarkersThe workshop was attended by the following participants:
Clinical experts
- Ludger Schoels, University Hospital Tübingen
- Thomas Klockgether, University Hospital Bonn
- Matthis Synofzik, University Hospital Tübingen
- Alexandra Durr, APHP Paris
- Gunter Hoeglinger, DZNE Munich
- Rebecca Schüle-Freyer, University Hospital TübingenData sharing / FAIR data
- Eleni Mina, Leiden UMC
- Marco Roos, Leiden UMCERN-RND coordination
- Holm Graessner, University Hospital Tübingen
- Carola Reinhard, University Hospital TübingenRegulators
- Pavel Balabanov, EMA
Patient representatives
- Cathalijne van Doorne, euro-Ataxia and European Federation of Neurological Associations
- Tsveta Schyns-Liharska, European Network for Research on Alternating Hemiplegia (ENRAH)
- Astri Arnesen, European Huntington Association
- Gulcin Gumus, Eurordis
Registry hosting organisations
- Torsten Illmann, 2mt
- Michael Wallner, 2mt
- Reinhard Schneider, LCSB Luxembourg
- Ruth Herberz, DZNE Bonn
Data protection experts
- Johan Wisenborn, Novartis
- Annika Spottke, DZNE Bonn
Pharma industry
- Paul Goldberg, Ionis
- Baltazar Gomez-Mancilla, Novartis
- Petra Kaufmann, Avexis/Novartis