The 7^th International TREAT-NMD Conference is fast approaching! This fantastic in person event will take place from 7-9^th December 2022 in Vancouver, Canada.  It will be a fantastic opportunity for patients, academics, clinicians, patient registry curators and industry representatives, to get together to network, learn and exchange ideas about translational research.

Some topics that will be covered include: patient engagement; Clinical trial considerations in ultra-rare patient groups; Global Neuromuscular Care and Access to Treatment; Emerging Therapeutics options in NMD and many more! View the full programme here.

There will be lots of opportunities for discussion and networking during the conference, at our networking dinner and welcome drinks reception.  At our conference you are not just part of the audience, you are part of the discussion!

Tickets: Reserve your ticket NOW!

In conjunction with the Conference, The Annual Curators meeting will also take place in Vancouver on Saturday the 10th of December and approximately 50 curators have already confirmed their attendance.

We will be discussing the best ways to address challenges in implementing new datasets, ways to avoid duplication of data collection/reporting and methods to maintain patient engagement with a registry. In addition, alongside the ultra-rare Subgroup Leads, we will be discussing a standard/common dataset that could be applied to any rare neuromuscular disease.

Look out for the final  agenda which will be circulated at the end of November 2022 and we look forward to seeing you in Vancouver!

We are pleased to welcome two new TGDOC members:

Associacao Brasileira de Facio-Escapulo-Umeral Registry (ABRAFEU) – a new Brazilian registry with nearly 300 FSHD patients.

Russian FSHD Registry   – a registry established 2 years ago which is already collecting data on 114 genetically confirmed FSHD patients.

We are also pleased to announce that the Global Registry Network has just completed it’s first ever enquiry in FSHD.  This provided data to a pharmaceutical company developing plans for future clinical trials in the condition; the results of the enquiry will help the company to understand the patient population and the feasibility of running clinical trials in that group.

If you would like to find out more about TGDOC Membership please get in touch: info@treat-nmd.com or visit our website

The TREAT-NMD Global Data-systems Oversight Committee (or TGDOC) oversees the TREAT-NMD Global Registry Network. The Committee includes curators from all member registries and is led by a troika of Chairs who are elected for a total term of 6 years (2 years as chair-elect, 2 as chair, and 2 years as outgoing chair).

The TGDOC chairs hold regular meetings with staff at TREAT-NMD to offer their expert advice; this ensures that the Global Registry Network runs efficiently and within the terms of the TGDOC Charter.

The next election for a TGDOC Chair will take place later this year, with nominations opening after the curators meeting in December. We’d encourage potential candidates to start thinking about standing – it’s a position that plays a vital role in the TREAT-NMD Global Registry Network. For more information please contact: info@treat-nmd.com.

We’d like to remind all curators that we are currently conducting the annual survey of registries. This year the survey is in two parts

• The Registry Review Survey collects essential information about your registry (e.g. contact info, disease area and dataset collected), patient consent, and data sharing and should take around 50 minutes to complete. It is an important part of being a member of the TREAT-NMD Global Registry Network, ensuring you can take part in enquiries and other opportunities for collaboration.

 

• A request for basic aggregate data on the patients within your registry (e.g. the number using a wheelchair or ventilatory support). This survey has been developed alongside the TGDOC Chairs, Subgroup Leads and Patient Reps and will allow us to produce a high-level overview of the data within the Global Registry Network. We aim to publish the overview in a scientific journal, and the curator/PI of registries taking part will be included in the list of authors.

The Global Registry Network team continues to engage with registries to progress the adoption of the latest DMD dataset (version 1.2) & LGMD dataset (version 1.0). There continues to be a steady increase of registries adopting the core DMD & LGMD datasets. The datasets are very concise and capture the important aspects of the inclusion criteria required for clinical trials. The datasets have been created by key opinion leaders and tailored with the insight of DMD & LGMD registries and associated stakeholders. Collecting an agreed set of data allows the data to be comparable and anonymously aggregated which ensures a coordinated approach to clinical trial readiness.

We have had a couple of new adopters of DMD v1.2 & LGMD v1.0. Currently 9 registries have adopted the DMD dataset into their existing platforms (in-direct) and 8 have adopted the dataset via the GRP (direct). Overall, 17 registries have adopted v1.2 of the DMD dataset. Currently 3 registries have adopted the LGMD dataset into their existing platforms (in-direct) and 7 have adopted the dataset via the GRP (direct). Overall, 10 registries have adopted v1.0 of the LGMD dataset.

Both core datasets can be implemented in a registries current IT platform or can be adopted via the TREAT-NMD Global Registries Platform (The GRP). The GRP is a free-to-use cloud-based system providing a user-friendly environment for patient registries to collect patient data. The GRP enables even the smallest registries to collect standardised information on their patients.

If we’ve emailed you about the dataset, please don’t hesitate to get in touch and we will set up a Zoom call at a mutually convenient time to discuss your registry’s needs and what we can do to support you in the adoption of the expanded core dataset. To discuss the above or any other points, please contact the Global Registry Network Manager Neil Bennett via neil.bennett@treat-nmd.com or Global Registry Network Administrator Farjana Ali via farjana.ali@treat-nmd.com.

There have been a number of interesting developments and events planned in TREAT-NMD spinal muscular atrophy (SMA) projects over the past few months:

SMA Annual Report

 The Annual Report (Year 3) is now complete and has been submitted to Biogen (project sponsor ) for review. This will be available for registries to view on the TREAT-NMD website by early November. We wish to express our sincere thanks to all registries who took the time to participate in the survey and provided valuable data to inform this report.

Curator Drop- In sessions

 This is a reminder that monthly drop-in sessions are available for all curators and PI’s involved in SMA data collection. We can provide registries with support and guidance with regards to the core dataset and it is an opportunity for you to chat through any questions you may have. If you’re unable to attend the sessions listed below, then please contact us directly to discuss any issues or concerns you may have and we will be happy to work with you (Julie.bohill@ncl.ac.uk / jess.page@ncl.ac.uk)

The schedule for the 1hr drop-in sessions are as follows :

• Tues 1 Nov, 2022 – 14.00 (GMT)
• Wed 4^th January 2023 – 14.00 (GMT)
• Tues 7^th Feb 2023 – 14.00 (GMT)
• Wed 8th March 2023 –  10.00 (GMT)
• Wed 5^th April 2023 – 10.00 (GMT)

3^rd International Scientific Congress on SMA, Barcelona 2022

We are pleased to have been invited to present a poster at this year’s conference on the 21-23 October. The poster will then be made available to registries to view on the TREAT-NMD website.

SMA Dataset Workshop – 7^th December 2022, Vancouver

 We are excited to be holding our first in-person workshop with registry curators since 2019 and the TREAT-NMD conference will then follow on 8-9 December. We have been impressed with the very high level of registration and are looking forward to a great networking opportunity and chance to discuss key issues facing registries around patient engagement, dataset collection, outcome measures and sharing best practice.  Following the workshop, a report will be produced to share the outputs from this meeting and is expected to be available to view on the TREAT-NMD website, January 2023.

Since late last year, TREAT-NMD has been working towards EMA Qualification for our spinal muscular atrophy (SMA) core dataset and Global Registries Platform/Central Data Warehouse.

The Qualification process provides a regulatory opinion on a registry’s technology, processes and dataset. It assesses whether a registry demonstrates the ability to provide consistent, quality assured data that can support regulatory decision-making for a set of specified scenarios (e.g. post marketing surveillance or provision of natural history data).

Working closely with SMA stakeholders including clinicians, physiotherapists, patient representatives and registry curators, TREAT-NMD sought EMA advice covering our dataset and also our data management, data quality, and data security processes for a range of regulatory uses earlier this year.

After several meetings with the EMA, the final Scientific Advice was received from the EMA’s CHMP in September; this will be followed by a public letter of support from the EMA.

The Advice gives an in-depth response to our application and sets out a number of details that should be considered in our application for Qualification Opinion. This is the final step in the Qualification process, and we hope to address the points raised as soon as possible so an application can be submitted during 2023. This will create a resource that meets EMA requirements in SMA, and we are already working towards applications in DMD and LGMD as well.