TREAT-NMD joined the Joint Heads of Medicines Agencies (HMA) and European Medicines Agency (EMA) Multistakeholder workshop on Patient Registries, held in Amsterdam on February 12th and 13th. The workshop brought together a diverse array of stakeholders, including representatives from patient registries, regulatory agencies, pharmaceutical companies, patients, healthcare professionals, academics, and health technology assessment bodies.
The primary objectives of this significant event were twofold:
- EMA Qualification Procedure Clarification: The workshop aimed to facilitate discussions on the EMA qualification procedure for patient registries. Stakeholders collaborated to clarify the benefits, identify current limitations, and propose measures to optimise the qualification process.
- Value of Patient Registries in Regulatory Decision-Making: The second objective sought to establish the value of patient registries for regulatory decision-making. This involved exploring the contexts in which registry data are ‘fit for purpose’ and examining tools to support data discoverability and assessment.
As part of the workshop, TREAT-NMD’s Head of Real World Evidence, Neil Bennett, and Project Manager, Seung Yeon Lee, presented highlights of the organisation’s recent Spinal Muscular Atrophy (SMA) natural history study conducted in collaboration with the EMA and Aetion. The project was presented as a successful case study to foster the use of registries for regulatory purposes, and TREAT-NMD were able to share their experience regarding the challenges faced during the study and imparting key learnings related to data quality improvement, feasibility assessment, and the promotion of data interoperability.
TREAT-NMD were glad to be invited to take part in this workshop and given the opportunity to discuss the SMA natural history study as a model for the effective use of patient registries in regulatory decision-making.
