Mandatory Core Dataset for TREAT-NMD SMA Registries

Introduction

From May 2017 to August 2018, a project was carried out to expand the mandatory core dataset for the SMA Registries affiliated to the TREAT-NMD network. This work was done so that the TREAT-NMD Global SMA Registry can:

a. better inform on the natural history of SMA

b. provide context to understand the safety and effectiveness of new treatments

c. support post marketing surveillance (PMS) for those new treatments.

The Expanded SMA Dataset

 

Please 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.

 

To support the dataset, a Dataset Manual is in development which will include detailed guidance on each data item, outcome measure toolkits, and suggested standardised text to include in your patient consent and ethical approval applications. Please contact Jo Bullivant with any other requests for content.

Points to note

  • The dataset is split into 'mandatory' and 'highly encouraged' items. In addition, some items are only applicable to clincian-reported registries, and some 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 dataser for TREAT-NMD-affiliated registries; registries may choose to collection additional items of local interest of relevance.
  • An annual revision plan will support continued harmonisation with other data collection initiatives, and allow responsiveness to emerging developments around outcome measures and other key issues. This will allow suggested revisions to be accepted througout the year, and conisdered and implemented (if appropriate) on an annual basis.

Please direct any questions or comments about this work to Jo Bullivant.

 
22 Nov 2018