Joint Heads of Medicines Agencies (HMA)/European Medicines Agency (EMA) multistakeholder workshop on real-world data collection in duchenne muscular dystrophy
EventHumanCorporateMedicines
- The original event time is:
- Friday, September 25, 2026 - 09:00 - 17:30 Amsterdam time (CEST)
Date
- , -
Location
- OnlineEuropean Medicines Agency, Amsterdam, the Netherlands
Event summary
This joint HMA/EMA hybrid workshop is anchored in the Network Data Steering Group workplan and follows on stakeholders’ interactions to support medicines regulation and access in the field of Duchenne Muscular Dystrophy (DMD).
Drug development in DMD remains complex. Despite existing and past efforts on several levels, the challenges persist, including the generation of relevant (as defined in the Data Quality Framework for EU medicines regulation: application to Real-World Data) and high-quality evidence to support regulatory decisions.
This workshop, supported by the World Duchenne Organisation, aims to discuss current gaps and hurdles in data collection, and to explore how we can all collaborate to enable the generation of meaningful evidence on the effectiveness and safety of medicines.
The event brings together representatives from patient organisations, regulatory agencies, healthcare professional organisations, data holders, academia, non-for-profit research institutions, pharmaceutical companies, health technology assessment bodies and payers with the following objectives:
- Taking stock on current challenges from the perspectives of different stakeholders and lessons learnt from past experiences in using Real-World Data (RWD) in DMD
- Discussing how to leverage the output of the EMA-funded project on key data elements for regulatory use
- Aligning on actionable recommendations and collaboration to support the collection and use of fit-for-purpose RWD for regulatory decision-making in this disease area, with a focus on data quality, patient consent, governance for data access and sharing, and registry interoperability
Note: The definition of relevant evidence can be found in 'Data Quality Framework for EU medicines regulation: application to Real-World Data, under 'Related documents'.
Registration
In-person attendance will be organised via EU associations and direct invitations to allow a broad range of stakeholders to participate.
A registration form for in person and online participation will be published in the next few weeks.
A video recording of the plenary sessions will be available after the event.
Documents
Video recording
A video recording of the plenary sessions will be available after the event.