DMD/BMD – OUTCOME MEASURES: EP.118 The TREAT NMD DMD Global Registry development

Abstract

The previous TREAT-NMD Duchenne muscular dystrophy (DMD) dataset was designed mainly to support clinical trial feasibility and recruitment. Therapeutic developments, anticipation of post-marketing surveillance and interest in better understanding the disease natural history, meant the dataset needed to be updated to meet these new requirements. To reach consensus on a new version of data items that can be shared amongst the stakeholder community, and used by all registries collecting data from DMD patients, a working group (WG) including clinical experts, patient representatives and registry curators met in 2019 to workshop an updated dataset. Using an established TREAT-NMD project methodology, a consensus was achieved for new data items including for hospitalisations and comorbidities, nutrition, therapies and others. The feasibility of implementing the new dataset into existing registries was evaluated in a pilot exercise with 14 registries. The results of the pilot exercise were then shared with the WG and discussed in a further dataset development workshop to finalise the new DMD dataset. The updated dataset includes items. Based on the pilot exercise feedback, 49% of the new data items were already collected. 74% of new items were rated as very easy to moderately difficult to collect. 39 items rated as challenging were taken to the WG for re-evaluation. Version 1.1 of the new DMD dataset for clinically reported registries was published in February 2021. A separate focus group is reviewing a proposed dataset for patient reported registries. Collaborating with its global alliance of registries, leading clinicians and other partners, TREAT-NMD is ideally positioned to advance the expansion of the DMD dataset, to enable registries to be a reliable source of data in the collection of real-world evidence and to capture valuable information for regulatory purposes and post marketing activities.