TGDOC Annual Curators Meeting 2020

Support Available! Last Opportunity to Participate in the TREAT-NMD SMA Core Dataset Implementation Project

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All TGDOC member registries for SMA are now asked to comply with the TREAT-NMD SMA Core Dataset, or begin working towards it. If this applies to you and you have not yet been part of the SMA Core Dataset implementation project, there is a range of support available including:

• A one-off bursary of €8,000 (some eligibility criteria apply).
• Monthly project support drop-ins to access information and guidance from the project team.
• Annual SMA Dataset Workshop.
• Access to and support from a network of registries and curators who have already implemented the dataset.
• Direct linkage with other TREAT-NMD Dataset projects (DMD and LGMD).

The final year of this support will run from 1st June 2021 to 31st May 2022. Previous participating registries have found:

‘Our impression regarding expanded dataset is very positive. Information it collects are valuable as well as the whole process of acquiring the information. It points to the potential weakness in data we collect, especially concerning patient history, and draws out issues that require more focus in the future thanks to their potential significance in improving the care for the patients as well as their academic and scientific value. Opportunities for improving were identified in the dataset workshop through discussions which hold great value for research.’

‘First we noticed an improvement in the quality of the data. More patients have been entered and data fields are more complete compared to previous collections for which we had a TREAT-NMD SMA section (using the v0 core dataset) in our general registry. Further, we received several requests for data from pharmaceutical companies within the context of a drug reimbursement application. Without the [SMA Core Dataset] we would not be able to answer these requests.’

’We know that working on an expanded SMA registry is very difficult, but at the same time very, very useful.’

If you are interested in participating please contact Project Coordinator Joanna Das to discuss.  

TGDOC Registry Membership Process Update

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As you will now be aware, the TGDOC Membership Process was launched in October 2020 following extensive consultation with the Global Registries Network. The original deadline for existing TGDOC registries to complete the new TGDOC Membership Pack was the end of December 2020, but due to the ongoing complex situation with covid 19 the deadline was extended until the end of March 2021 to allow more time for registry colleagues to complete the required documentation.

We are pleased to announce that 39 registries have now completed this process and are now confirmed as TGDOC Member Registries. A further 20 registries are still finalising their documents and will be welcomed as TGDOC Member Registries once these are completed, and so far, 8 newly identified registries are in the process of joining the TGDOC Community.

Please note, if your registry has not yet received a TGDOC Membership Certificate and TGDOC Welcome Pack this means you have not yet completed the Membership Process. We are now not legally permitted to send any information about new industry enquiry data requests until the new Confidential Disclosure Agreement is finalised (see Membership Pack or contact Helen Walker for further information).

If you have any questions about the new TGDOC Membership Process, please don’t hesitate to get in touch with Helen or Emma.

TREAT-NMD Dataset Workshop

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The TREAT-NMD Dataset workshop for SMA, DMD and LGMD, will be taking place on 19th and 20th May. 

The purpose of the workshop is to explain the newest versions of the SMA, DMD and LGMD datasets and to clarify the new technical specifications. Based on in depth research undertaken by the SMA dataset project, common challenges expressed by the curators have already been identified and will be addressed as part of the workshop. 

To accommodate all registries participating in the dataset roll out, registries are invited to attend one of the following three sessions: 

• Wednesday 19th May: 11:00 – 14:00 (BST)
• Thursday 20th May: 11:00 – 14:00 (BST)
• Thursday 20th May: 18:00 – 21:00 (BST)

We are looking to expand the dataset roll out project across registries worldwide. If you are working with registries and you are interested in the newest version of the SMA, DMA and LGMD Core Dataset please contact:

Joanna Das, project coordinator of TREAT-NMD SMA Core Dataset

John McKenna, project manager of TREAT-NMD DMD Core Dataset

Sonia Segovia, project manager of TREAT-NMD LGMD Core Dataset

For further information about the dataset projects please go to the TREAT-NMD website.

TREAT-NMD Global Registries Platform Update

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It is a busy time for the TREAT-NMD Global Registries Platform project as we move into the next phases of the project before the launch at the end of May. The build of the patient portal is now moving into the beta testing phase, where we have several testers engaged to test and provide feedback. The platform team is currently engaging with TGDOC Member Registries to discuss the implementation of the platform and the datasets. This process is also helping the platform team to understand how each registry may plan to use the platform after the launch date.

Registries using the platform will be identified as one of the following:

• Option 1 Registry, which are registries that wish to use the platform as their main data repository, and where data transfers to the Central Data Warehouse are completed within the platform,
• Option 2 Registry is a registry that intends to remain with their current/existing platform and will be able to provide data via secure transfer to the Central Data Warehouse using a data transfer toolkit.

While we encourage and support all TGDOC Member Registries to share data via the platform, we acknowledge that this may not be possible for all registries or might take some time to implement. These registries will of course continue to participate in our usual TGDOC Registry Enquiries. As we progress with our roll out we will be working closely with these registries to understand their challenges or reasons for not participating via the platform to determine how we can best work together to safely and securely capture and share data, in a unified and standardised manner that allows for efficient and effective high quality data analysis and reporting.

The LGMD module build is also progressing with the clinician portal almost complete and will soon be ready for the feasibility study phase of the dataset project. The patient portal is also nearing the end of its development phase and will soon be ready to be built within the platform software, where we will be alpha testing during its update releases.

We are continuing to update our stakeholders for the project, but if you would like to be involved in testing, offer support or advice in relation to the platform or would like to see a demonstration of the platform please feel free to contact Caroline directly using the email below or the link on our webpage. We are keen to engage with as many people as possible.

For further information on the TREAT-NMD Global Registries Platform or to participate as a ‘test registry’ then please contact Caroline Ogden, Project Manager or visit our website at to learn more.

TREAT-NMD Core Dataset for DMD Project Rolled Out to TGDOC DMD Registries

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We are pleased to announce that since our last update in March, the TREAT-NMD Core Dataset for DMD v1.1 for clinician reported registries has been approved by the TGDOC Chairs.  

During discussions with our Working Group it became apparent that the dataset for patient reported registries required additional clarifications and a focus group of representatives from current patient reported registries met to implement these. The patient reported dataset has now been finalised and is planned for publication in May 2021.

As a part of the dataset roll out process, the project team has now contacted the majority of TGDOC DMD Clinically Reported registries with information about the new dataset release and will continue over the summer. The project team offers one to one meetings to discuss the dataset and to understand the unique requirements and challenges of implementing the dataset within individual registries. During the project, 26 registries have indicated their interest in using the new dataset with their existing platform. Following the project team’s recent engagements, 13 registries have now expressed an initial preference to implement the dataset in this way. 14 more registries wish to use the Global Registries Platform, which has the new dataset already implemented within.

TREAT-NMD is in the process of developing tools which will standardise dataset implementation. Newly released datasets will be accompanied by a web-based ‘dataset specification’, similar to the one successfully developed for the TREAT-NMD Core Dataset for SMA. As well as guiding registries on how best to implement the dataset, this web-based specification application also offers a feedback function, so registry curators can report issues or provide feedback directly to TREAT-NMD. Our dataset team will collect the feedback and respond with advice and will also gather the information received, review it and evaluate the need for dataset updates which are planned every 18-24 months.

The TREAT-NMD Core Dataset for DMD Version 1.1 can be found on the TREAT-NMD website.

If you would like further information on the project, please contact the DMD Core Dataset Project Manager John McKenna or Project Coordinator Janet Wilkins

TGDOC Strategy in Development

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The TGDOC Executive Board are working through the suggestions, feedback and comments received from the TGDOC disease subgroup breakout meetings and are using this valuable information to develop a TGDOC Strategy document. This will be shared with the Global Registries Network at the earliest opportunity.

Latest Newsletters from UK Registries

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The latest newsletters from the UK Myotonic Dystrophy and FSHD Patient Registries are now available. These include updates on recent registry enquiries, drug developments, upcoming and previous events, and new developments for the UK registries and their websites.

You can also find the UK Myotonic Dystrophy Patient Registry website here and the UK FSHD Patient Registry here.

The COL6 and International GNE Myopathy registries have also published their newsletters for Spring 2021. To view the newsletter, or to learn more about the registries, you can go to their respective websites.