Introduction

TREAT-NMD is a neuromuscular network that aims to ensure that the most promising new therapies reach patients as quickly as possible. The TREAT-NMD Global Network of Spinal Muscular Atrophy (SMA) Registries include 50 national registries that collect a common core dataset and are governed by the TREAT-NMD Global Database Oversight Committee (TGDOC). Researchers and industry can request anonymised and aggregate data, offering a single point of access to this extensive dataset.

Results

The core dataset was established 10 years ago when the main purpose of the registries was clinical trial readiness and recruitment. In the current SMA landscape, with emerging treatments and new therapeutic approaches in development, there is a need for more widespread longitudinal data collection to support future research and post marketing surveillance (PMS) requirements for emerging therapies. To support this, TREAT-NMD are reviewing and expanding the core dataset for their SMA Registries.

A workshop was held in May 2017 involving expert clinicians, physiotherapists, registry curators, patient representatives and other stakeholders from across the world, who developed a proposed expanded dataset containing 38 data items. A pilot feasibility study with the new dataset will be run in a sub-group of SMA registry sites (n=12).

Conclusions

Feedback from the pilot sites has been collated and discussed during a second workshop in June 2018, to make recommendations on (a) the content and structure of the expanded core dataset, and (b) the timescales, costs, and considerations for the full-scale roll-out to all 50 TREAT-NMD SMA Registries.