European standards for harmonization of myasthenia gravis registries and emerging digital solutions

278^th ENMC International Workshop

Location: Hoofddorp, The Netherlands

Title: European standards for harmonization of myasthenia gravis registries and emerging digital solutions

Date: 20-21^st September 2024

Organisers: Prof. Sabrina Sacconi (France), Prof. Andreas Meisel (Germany), Prof. Renato Mantegazza (Italy)

Translations of this report by:
Norwegian:  Nils Erik Gilhus
Italian: Fiammetta Vanoli
French: Sabrina Sacconi
Spanish: Elena Cortes Vicente
Dutch: Jan Verschuuren
Czech: Stanislav Vohanka
Swedish: Susanna Breuner
German: Frauke Stascheit

Participants:

Anja Hoffmeister (patient representative Germany), Lidgarde Allard (patient representative Belgium), Peter-Bram Hoen (Netherlands), Nils Erik Gilhus (Norway), Lorenzo Maggi (Italy), Adela Della Marina (Germany), Emmanuelle Salort Campana (France), Stanislav Vohanka (Czech Republic), Fiammetta Vanoli (Italy), Renato Mantegazza (Italy), Sabrina Sacconi (France), Andreas Meisel (Germany), Elena Cortes Vicente (Spain), Jan Verschuren (Netherlands), Ernestina Santos (Portugal), Terenshina Evangelista (France), Suanna Brauner (Sweden), Pascal Laforet France), Matthieu Lusignan (France), Mohammad Ashragi (UK), Frauke Stascheit (Germany)

Online: Raqual Pardo (Spain), Marjan Cosyns (Belgium)

Summary

Background information

Myasthenia Gravis (MG) is a rare neurological autoimmune disorder that recently gained growing attention due to the discovery of new therapeutic approaches. In order to better assess the value of the new treatments in the context of standard treatments and the ever-improving pathophysiological understanding, large multinational data sets obtained in the real world of MG care are needed. An essential prerequisite for this is the harmonization of data collection in order to create a solid ground for data exchange and research.

In this workshop patients, clinicians and other stakeholders were involved to discuss the interest of data harmonization among different European MG registries to facilitate data sharing, collaboration and ultimately improve standard of care.

As a preparatory activity before the meeting, the organizers implemented a Delphi questionnaire to reach a consensus on mandatory and optional data that should be included in all national MG registries. Results of each round were then discussed among all participants. Moreover, organizers collected a survey on existing MG national European registries to identify potential technical, ethical, and economical issues that could interfere with data collection and data sharing. Furthermore, patients were also involved in the creation of an informative document that provides knowledge on registry participation and its implications.

Workshop aims

The main aim of the 278^th ENMC meeting was to promote a collaboration between patients, clinician and other stakeholders to identify which tools are needed to build a functional MG registry initiative. The ultimate goal of a MG registry is to improve quality of care for patients, which is achievable through the collection of meaningful large-scale and high-quality data on the European MG population. Moreover, establishing common data elements among different registries is critical for data harmonization which is essential for collaboration and research initiatives, including epidemiological and clinical studies, clinical trials, pharmacovigilance as well as providing real world data on novel therapies.

Workshop outcomes/deliverables:

Through the Delphi process, participants reached a consensus on a list of mandatory and optional data to be included in a MG registry to facilitate data sharing and collaboration. The importance of a well-structured and comprehensive informed consent form was also taken into account, which must be easy to understand as well as address important issues like data ownership, data anonymity ad purpose of data use.

The survey showed that in Europe 8 MG national registries are already active and 2 are in the process of being activated, with data of more than 7.000 MG patients being currently available and potentially sharable.

Nevertheless, to optimize future collaborations at a European level, several aspects need to be addressed and improved:

• The first key aspect is to integrate the patient perspective and improve patient communication. Patients bring a unique expertise of their condition, which must be taken into account when building a disease registry. Patients’ representative underlined that current tools fail to capture some critical aspects of MG, thus their participation as stakeholders in the development of a national MG registry could improve data collection, ensuring their real needs and issues are addressed. Furthermore, MG patients requested to have access to their own data, for self-monitoring purposes, as well as participate in data entry (e.g. for patient reported outcomes). This model of active involvement on multiple levels will likely encourage the continuous engagement of patients in data collection.
• The second key aspect is that national registries need a clear governance structure. This should ideally include a Steering or Data Committee, responsible for managing the registry, a Medical Advisory Board, that supervises the activity of the Steering/Data Use Committee, and a Patient Advisory Board. The governance structure is responsible for defining the methods of data sharing as well as who is responsible for managing any data-sharing initiative, while ensuring that the interests of the people involved in the creation of the registry are well represented.
• The third aspect requires that the registry is be based on a robust infrastructure and a harmonized core data set. The database should be built as close as possible to the FAIR principles (findability, accessibility, interoperability, and reusability), as it ensures consistency and reproducibility of data collection, which are essential to establish common data elements and set the basis for future collaborations. To reach this goal and establish harmonization of data, the registry should include the results of the Delphi process in terms of mandatory and optional data, as well as consider the principles of the EMA Patient Registry Initiative.

Digital solutions, such as smartphone apps, can be optional additional tools useful for more detailed and frequent data collection (e.g. patient reported outcome measures).

• The fourth aspect concerns financial sustainability. Ideally a registry should be financed by public bodies or patient organizations, that ensure continued commitment and the long-term viability of the registry. When funding comes from private entities, it is important to guarantee transparency on funding sources, data ownership and meaning of commercial use. A multi-source funding approach is also preferable
• The fifth aspect is long-term The success of a registry is also based on long-term quality data collection. This strongly depends on the engagement of people involved in the registry, including patients and healthcare providers, which can be encouraged by giving periodic feedback on registry initiatives.

Next steps

Thanks to the participation of clinician, registry managers, patients and other stakeholders, this workshop has allowed to set the principles for a functional MG registry. A common ground on what are the core elements that need to be included in the MG registry has been reached through a Delphi process. Furthermore, emphasis was put on how to create FAIR data that can easily allow data sharing between different national registries and collaboration for future research initiative. In a future prospective, this could ultimately lead to the creation of a European registry, which would allow to work on large scale numbers which is essential for new research initiative, with the ultimate goal of improving quality of care for patients.

A further meeting is planned to follow-up on the achieved goals.

A full report will be published in Neuromuscular Disorders (PDF)