Core Dataset for TREAT-NMD SMA Registries
The core dataset for TREAT-NMD registries collecting data on SMA patients was expanded in September 2018, following a pilot study. This work was done so that the TREAT-NMD Global SMA Registry can:
- better inform on the natural history of SMA
- provide context to understand the safety and effectiveness of new treatments
- support post marketing surveillance (PMS) for those new treatments.
Expanded SMA Dataset (Current Version 1: September 2018 to present)
Please be sure to read the ‘Points to note’ below, in conjunction with the dataset.
We are preparing suggested wording for those items in the Dataset which are applicable to patient reported registries (or to the patient reported elements of dual-reported registries). This document is now open for consultation and will be confirmed in conjunction with Version 2 of the dataset (June 2020, see below). All feedback is welcome; if you would like to review please contact Project Manager Jo Bullivant.
Dataset Revisions and Stakeholder Input
The first formal revision of the SMA Dataset will begin with a wide stakeholder consultation in March 2020, and version 2 will be finalised in June 2020. Please send any suggestions or feedback on the Dataset to Project Manager Jo Bullivant.
If you have any questions or feedback on this revision process please contact Project Coordinator Joanna Das.
The Original Core Dataset (March 2014 to August 2018)
Prior to September 2018, the previous SMA Core Dataset had been in place since March 2014:
- Original SMA Core Dataset (March 2014 to August 2018)
Points to note
- TREAT-NMD does not expect all registries to be able to collect the expanded SMA dataset straight away. Please contact Jo Bullivant if you would like to discuss timescales or support needed for your registry.
- The dataset is split into ‘mandatory’ and ‘highly encouraged’ items. In addition, some items are only applicable to clinician-reported registries, and others only to patient-reported registries. Registries are required to include all relevant mandatory items in their case report forms (or identify actions to work towards their inclusion).
- The TREAT-NMD network aims to be inclusive. If there are any mandatory items that registries are not easily able to implement, they are encouraged to identify any support needs, and/or a plan to work towards their inclusion, and discuss with us.
- This is a minimum standard dataset; registries may choose to collect additional items of local interest or relevance.
- A dataset revision process will support continued harmonisation with other data collection initiatives, and allow responsiveness to emerging consensus or developments around outcome measures and other key issues. This will also allow suggested revisions to be accepted throughout the year, and considered and implemented (if appropriate) in a streamlined way.
Project Deliverables and Funding
This project is funded by Biogen. The deliverables are:
- Expanded dataset collected by all active TREAT-NMD SMA Registries.
- Dataset manual: data dictionary, wording for patient-reported registries, outcome measure toolkits, and standardised text for consent etc.
- Financial bursaries and 3 x annual workshops to support registry curators with implementation.
- Formal revision process to gather stakeholder feedback, harmonise with other initiatives and support continuous improvement.