TGDOC Annual Curators Meeting 2019

Is your registry a two-way street? The importance of giving something back to your registry participants – and how you can do it

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It is a universally acknowledged truth that the patient perspective is crucial at every stage of registry development and governance; from identifying the purpose of the registry, to designing the registration experience, to defining how the data will be used. We discuss regularly and at length the importance of actively seeking input and feedback from patients, patients’ families, and patient groups – or even better including them as partners from the outset. Certainly in the neuromuscular field we are fortunate to have active, engaged and expert patient communities who willingly give their time and energy to shape these projects.

But the scenario above describes a one-way street. Knowledge, input and feedback is flowing from the patient community into the registries, and often not much is flowing back again. This can have a negative effect on patients’ willingness to participate and their motivation to keep their data up to date. Here we suggest some ideas that we feel will increase the sense of engagement for participants:

1. Many registries produce newsletters to inform participants about registry activities, relevant news and events, clinical trials and drug developments, standards of care and so on. We would strongly encourage all registries to adopt this approach; even if resources are tight and it is limited to a brief email summary once a year.
2. Registry participants could be offered data insights whenever possible, by providing them (if they wish) with comparisons of their data against mean values for relevant sample groups. This is obviously dependent on the IT platform used and/or the resource available (the plan is for the TREAT-NMD Universal Registries Platform to have this functionality, for those who wish to use it).
3. Telling participants about the impact of their data is a powerful way of demonstrating that the time and effort they commit to the registry has tangible and important outputs. What has your registry data been used for? Which projects have benefited from it? At which events have the data or registry been presented? Has the registry produced or been cited in any publications (and can you summarise the content or even produce a lay version)? What enquiries have been made into the data?

On the subject of enquiries; what of the data that your registry provides for TGDOC global enquiries? We want to be better at tracking the impact of those data and feeding that back to you for inclusion in your own impact reports and patient newsletters. We have established a TGDOC Publications Committee with the remit to track and encourage publications and academic research using TGDOC affiliated registries, and to ensure appropriate acknowledgement for registries who contribute data to the global enquiries. Please forward any 2019 publications and abstracts from your registry work to the Committee, so we can showcase them to our network and wider stakeholders.

The Committee is also undertaking a series of systematic reviews to determine the academic output from neuromuscular disease registries and to understand the registry factors that contribute to successful academic productivity. If you joined us in Leiden in December, you may have seen the poster (at the TREAT-NMD Conference) or talk (at the TGDOC Curators’ Meeting) about this work, presented by Tran Nugyen from Western University Canada. The poster can be viewed and downloaded here and you are encouraged to share this with your registry participants in your next newsletter.  The main call-to-action from this first systematic review is that as TREAT-NMD Registry group members, it is important that we always make reference to TREAT-NMD within the manuscript or abstract.

Following on from this, the TGDOC Publications Committee aim to provide an impact summary at each annual TGDOC Curators’ Meeting (which will also be circulated to all Curators) and we would encourage you to also pass these summaries on to your participants in future. Any questions about the work of the Publications Committee can be directed to the Chair, Dr Rasha El Sherif.

And finally, we will soon be providing registries with a letter of thanks from the TGDOC Chairs, to pass on to registry participants who have consented (or who consent in future) for their data to be shared with the global registry network. This will also include information about data protection and confidentiality.

TGDOC Publications Committee Update - Dr Rasha El Sherif

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As TREAT-NMD registries network expands, to include more countries bringing diversity and high impact to the data collected, acknowledging the efforts made in response to global enquires as academic work would be of importance to all of us as curators and scientists. The Publication Committee was established with the following specific goals:

 1- To identify opportunities for academic publications derived by the collective data from the TREAT-NMD Global registry enquiries. Such publications have proved in the past to be of great impact to the NMD literature.

2- To collect and showcase the academic work done by individual and collective registries that make up our TREAT-NMD registry network,

3- To ensure that the people behind this work are acknowledged in the right way.

4- To highlight the academic impact of the registries.

Our role is not to review articles in an academic way but to help emphasise this cooperative work done by TREAT-NMD registries. In the last year, we started a series of systematic reviews to determine the output from neuromuscular disease registries and to understand the registry factors that contribute to successful academic productivity.  Tran Nugyen from Western University Canada presented the first review in a poster during the 6th TREAT-NMD meeting in Leiden. This work will continue in 2020.

TGDOC is truly a global network and as more registries from outside Europe become affiliated the impact of our global data will only increase.

The committee is committed to providing a summary at the end of each year during the annual TGDOC face to face meeting.

For more information or if you have any suggestions for the committee please contact Dr Rasha El Sherif. 

TGDOC Global Enquiries 2019 Summary

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Last year was a very eventful year for TGDOC, with five Global Registry Enquiries completed. The Chairs would like to take this opportunity to thank every curator who has assisted with these and helped generate the funds to run the Annual Curators Meeting. All successful 2019 enquiries were in the Duchenne disease area; these consisted of three feasibility studies and two recruitment enquiries.

Feasibility studies were undertaken for three of the largest pharmaceutical companies in the Neuromuscular field, and curators were asked to provide information on nonsense mutations and exon skip amenable deletions, alongside core data items such as ambulation, steroid status and clinical trial involvement.

Two successful recruitment enquiries were also completed last year, the first since 2013. The process has now been streamlined and both enquiries were incredibly successful, with one of the trials reaching capacity within only a few weeks of the patient information being disseminated. We anticipate a higher number of recruitment enquiries will be completed in 2020 as they have proven to be a highly efficient way of reaching out to potentially eligible patients in specific countries.

Please be reminded that in order for us to understand which enquiries your registry should be included in, it is crucial that a representative for your registry has completed the Registries Review. It is also essential to have a Confidential Disclosure Agreement with TREAT-NMD in order to be included in any enquiries. We have had great successes in 2019 with an increasing the number of registries with an active CDA, special thanks to TREAT-NMD Communications Coordinator Hannah Murray for her tireless efforts with these! If your registry does not currently have a CDA, or you are unsure of your CDA status please contact Hannah.

2020 TGDOC Registries Review Survey Coming Soon

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2018 saw the launch of the TGDOC Registries Review survey to be completed by all TGDOC affiliated registries. This asked for essential information including the registry's contact information, the disease area(s) and dataset(s) collected and the IT system used. 

Over the course of 2019 sixty-one of our affiliated registries completed the review. Although this is a good response, in future, we need to ensure that all affiliated registries complete the review in line with the TGDOC Charter. 

By the end of February all affiliated registries will receive an invitation to complete the updated survey. If your registry completed the review in 2018 all you will need to do is click on the link and review the answers you provided only amending where appropriate. You may notice that some new questions have been added to the 2020 survey. It is really important that TGDOC obtains your views on these topics as these responses will help inform the committee's work moving forward. 

If you'd like further information about the review please contact Hannah.

Expanded Dataset for SMA Registries: Project Update

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Background

In 2017-2018 we completed a pilot project to expand the Core Dataset for TREAT-NMD SMA Registries, and the Expanded SMA Dataset (v1) was confirmed in September 2018. This work was done so that the real word data in 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; and
• support post marketing surveillance (PMS) for new treatments.

We are now in year one of a three-year Implementation Project (May 2019 to May 2022) which aims to support the remaining registries in the network to start collecting the Expanded Dataset. Bursaries are available to help fund this work and a workshop is held each year to support Registry Curators with implementing the Dataset.

The Dataset documents are available on the SMA Dataset project page on the TREAT-NMD website.

Latest developments

1. A Dataset Manual is now available (on the project webpage) to support the Dataset, containing a data dictionary and guidance and information on: identifiable data, data submissions, Dataset compliance, data sharing and publications, text for consent and ethics, and feedback, harmonisation and revisions.

3. On 13^th December 2019 (the day after the annual TGDOC Curators’ Meeting) we held the first SMA Dataset Workshop. The purpose of workshop was to provide project updates, guidance to curators on implementation of the new Dataset, information about support available, to discuss progress and issues, and to share solutions and best practice between registries. It was primarily aimed at SMA Registry Curators taking part in Year 1 of the Implementation Project but was also open to others. There was excellent attendance and engagement at the workshop, and we have had fantastic feedback. We will shortly be circulating a ‘light-touch’ Workshop Report along with the presentation slides which will also be available on the project webpage.

4. Feedback on the Dataset can be submitted to the Project Team at any time. The first formal revision will begin with a wide stakeholder consultation in March 2020, and version 2 of the Dataset will be confirmed in June 2020. Feedback is welcomed on the revision process itself, which is also available on the project webpage.

We are currently contacting registries to confirm participation in Years 2 and 3 of the Implementation Project. If you have not been contacted about this work but would like to be involved, please contact Jo or Joanna (contact details below)

Coming soon

February 2020: As mentioned above, a summary report from the Year 1 Dataset Workshop held on 13 December 2019 will be circulated and made available on the website.

May 2020: To support the SMA Dataset we are developing an Outcome Measure Toolkit which will include:

• Summary information about each outcome measure listed in the SMA Dataset.
• Guidance on appropriate patient populations and resources/facilities needed for use.
• Conditions of use (licensing etc.).
• Link to the questionnaire/scale if available.
• Signposting to training resources and/or trainers.
• Links to publications and further information.

June 2020: As mentioned above we will begin our first formal dataset revision process in March 2020, and version 2 of the Expanded SMA Dataset will be confirmed in June 2020.

Contacts

Please feel free to contact us with any questions, feedback, or if you would like to be involved (or more involved) in this work. We would love to hear from you!

Project Manager: Jo Bullivant
Project Coordinator: Joanna Das 

TGDOC Affiliated Registries - We need you!

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We would love to include articles in our newsletter from our affiliated registries and the wider NMD community. Do you have a regular newsletter you'd like to highlight? Perhaps you have a blog you'd like to make known to the wider community? If so please contact Hannah Murray.