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1 | TREAT-NMD 17 th September 2020 AGENDA 1. Welcome and - PowerPoint PPT Presentation

Nov 25, 2023 •788 likes •1.28k views

LGMD CORE DATASET PROJECT 1 ST MEETING 1 | TREAT-NMD 17 th September 2020 AGENDA 1. Welcome and introduction from participants 2. Housekeeping and ground-rules 3. TREAT-NMD and TGDOC overview 4. Introduction to TREAT-NMD LGMD Global

  1. LGMD CORE DATASET PROJECT 1 ST MEETING 1 | TREAT-NMD 17 th September 2020 AGENDA 1. Welcome and introduction from participants 2. Housekeeping and ground-rules 3. TREAT-NMD and TGDOC overview 4. Introduction to TREAT-NMD LGMD Global Registry and Core dataset development experiences in other diseases 5. Q&A 6. Break 7. Stakeholders presentations (Patients' representative, clinicians and registry curators, and pharmaceutical companies) 8. Breakout room discussions (mixed groups) 9. Feedback from groups 10. Curated questions sessions from group sessions 11. Break 12. Introduction to Meeting 2 and 3 13. Close and thank you

  2. 2 | TREAT-NMD HOUSEKEEPING AND GROUND RULES

  3. 1 | TREAT-NMD TREAT-NMD AND TGDOC INTRODUCTION LGMD CORE DATASET PROJECT 1 ST MEETING 17 th September 2020 Ben Watling – CEO TREAT-NMD Enterprise Anna Ambrosini – Incoming TGDOC Chair

  4. 2 | TREAT-NMD What is TREAT-NMD? • A Global Membership Network for the rare neuromuscular field, providing an infrastructure for ‘collaboration’ to ensure that the most promising new therapies reach patients as quickly as possible. • Launched in 2007, we have focussed on the development of networks and tools that industry, clinicians and scientists need, in order to bring novel therapeutic approaches through preclinical development and into the clinic, and on establishing best-practice care for neuromuscular patients worldwide..

  5. 3 | TREAT-NMD What is TREAT-NMD? • Promising research results are starting to be translated into treatments For Patients TREAT-NMD addresses • Work on standardized care guidelines aims to improve numbers of patients numerous issues, uniting the receiving optimal care stakeholders in the community and providing an infrastructure • Supporting clinical trial readiness by providing disease specific natural that is accelerating research and history data from our associated For Industry Member Registries therapy development, increasing • Accessing the appropriate patient collaboration, improving patient cohorts for clinical trial planning and recruitment care and helping to support ‘clinical trial readiness’ on an • Working on support tools such as For international scale. validated clinical outcome clinicians & measures and standard operating procedures for research protocols researchers to aid therapeutic development

  6. 4 | TREAT-NMD Who are the Executive Committee? Chair - Annemieke Aartsma-Rus • Interim Vice-Chair – James Dowling • - Fabiola Bertinotti - Annamaria De Luca - Marie-Christine Ouillade - Gillian Butler-Browne - Volker Straub - James Dowling - Andoni Urtizberea - Linda P Lowes - Elizabeth Vroom - Patient Representative TBC

  7. TREAT-NMD Structure 5 | TREAT-NMD TREAT-NMD Executive TREAT-NMD TEC Committee Enterprise Board • Masterclasses Disease Specific Advisory • Standards of • Operational Boards (KOLs) Care • Procedural • Family Guides • Legal/Contracting • Financial TACT TGDOC General • • • Membership Registry Membership Clinical Trial Enquiries & Affiliation Planning, • • TREAT-NMD Registry Data Enquiries Development, • Conference (Academic Research, Protocol • Network Trial Feasibility & Advice • Enquiries Recruitment) Panel Review

  8. 6 | TREAT-NMD TREAT-NMD Global Data systems Oversight Committee ( TGDOC )

  9. 7 | TREAT-NMD Composition and role Responsible for reviewing the requests for data from the TREAT-NMD Global Registries Network TGDOC Annual Established Chaired 79 affiliated face to in 2007 by a troika registries face meeting TREAT-NMD TREAT-NMD registry alliance curators representatives Patients organizations

  10. 8 | TREAT-NMD TGDOC Chairs TGDOC Support & Management Helen Walker Emma Faid Anna Ambrosini Craig Campbell Nathalie Goemans Incoming chair Current chair Outgoing chair Ben Watling​ CEO, TREAT-NMD Services

  11. TGDOC Structure 9 | TREAT-NMD

  12. 10 | TREAT-NMD Wider Registries Community TGDOC Affiliated Member Registries LGMD CMD New TGDOC Core LGMD Dataset ALL CMT Project LGMD NMDs Member Registries CMS FSHD MTM LGMD LGMD FSHD LGMD ALL CNM DM1 NMDs DMD GNE FSHD DMD FSHD DMD FSHD DMD ALL SMA DM1 SMA NMDs DM1 SMA DM1 SMA DM1 DMD SMA DMD SMA Dataset Dataset Expansion Expansion Project Project Core Members are registries that participate Affiliated members do not participate in global enquiries, but are otherwise Wider Registries Community includes all active patient in global enquiries (collecting core dataset(s), engaged with TGDOC. Can take part in internal surveys & dataset projects. registries we are aware of, who do not participate directly in updated regularly, CDA in place, correct Includes international ultra-rare disease-specific registries (with own any TGDOC projects. We list them on website & encourage consent, etc.) enquiry processes) and registries working towards TGDOC affiliation. them to join in dataset creation/expansion projects.

  13. 11 | TREAT-NMD Aims: usually to establish where patients are located for clinical trial feasibility / recruitment • Patient numbers can be stratified by any dataset items • Can request data from all disease-specific registries, or just for certain countries Only Active, TGDOC Core member registries can participate in formal enquiries • Must be collecting Core Minimum Dataset for specified disease area(s) • Must have patients’ consent to share data • Must have Confidential Disclosure Agreement in place • Patient data must be updated at least annually • Patients must have a genetically confirmed diagnosis

  14. Global Data Enquiry Process 12 | TREAT-NMD Enquiry received from National industry or academia registry National National registry registry National registry National TGDOC registry Governance Global National Registry registry Network National National registry registry TREAT-NMD National registry requests Data returns Data

  15. New TGDOC Membership Criteria 13 | TREAT-NMD TGDOC Non-TGDOC Affiliation Criteria Core Member Registry Affiliated Member Registry Unaffiliated Registry Disease subgroups DMD / SMA / DM1 / FSHD/ LGMD Any / all NMDs Any / all NMDs Allow registry info on TREAT-NMD website Essential Essential Strongly Preferred Abide by Charters & data protection regulations Essential Essential Strongly Preferred Must complete annual Registries Review Survey Essential Essential Strongly Preferred Collect all mandatory items in Core Dataset(s), and HE if possible Essential Strongly Preferred Strongly Preferred Participate in dataset revision process Essential Strongly Preferred Strongly Preferred Participate in disease subgroups Essential Strongly Preferred n/a Attend TGDOC Annual Curators’ meeting Essential Strongly Preferred n/a Engage with TGDOC Publications Committee Essential Essential n/a CDA in place and DoI submitted Essential Strongly Preferred n/a Participate in Surveys / Non-Core dataset Queries Strongly Preferred Strongly Preferred n/a Participate in Enquiries Essential Only relevant for Core Members Only relevant for Core Members Abide by Voting SOP Essential

  16. 14 | TREAT-NMD Any questions?

  17. 1 | TREAT-NMD TREAT NMD LGMD Global Registry LGMD CORE DATASET PROJECT 1 ST MEETING 17 th September 2020 Sonia Segovia - TREAT-NMD Project Manager

  18. 2 | TREAT-NMD TREAT-NMD actions in LGMD field • Masterclasses Educational • Guidelines Standards • PROMs of care Registries

  19. 3 | TREAT-NMD LGMD registries actions Phase A . Phase B . Rol out of Phase C . Development of LGMD Core Dataset Development LGMD Core Dataset + development of extended LGMD and network URP dataset

  20. 4 | TREAT-NMD Why now? • Acceleration of therapeutic • The current LGMD registry developments landscape is fragmented. • Recruitment for clinical trials • Multiple small, subtype • registries Post-marketing surveillance • General neuromuscular diseases registries LGMD CORE DATASET NO CORE DATASET FOR FOR LGMD registries LGMD

  21. 5 | TREAT-NMD Objectives. Phase A. Development of LGMD Core Dataset and network Establish the LGMD global registry network as affiliates of TREAT- NMD and reach consensus on a feasible core data set , that all members agree to collect. Implementation Core Dataset Data dictionary Plan

  22. 6 | TREAT-NMD Project team Ben Watling Heather Hildsen Michela Giugleri Sonia Segovia CEO TREAT- LGMD project Principal Project Manager NMD liaison Investigator

  23. 7 | TREAT-NMD TREAT-NMD LGMD Advisory Board Institutional Support Jordi Diaz Manera Newcastle University Michela Guglieri Newcastle University Volker Straub Newcastle University TGDOC chair Johanna Palmio Tampere, Finland Craig Campbell Linda Lowes Children's Nationwide Columbus Ohio Craig Campbell University Western Ontario Nathalie Goemans Nathalie Goemans Leuven University Anna Ambrosini Andoni Urtizberea Hendaye Maggie Walter Munich University Nick Johnson VCU Richmond USA Elena Pegoraro University of Padova Tanja Stojkovic Institute of Myology, Paris Anna Mayhew Newcastle University Edmar Zantoneli Sao Paolo, Brazil John Vissing Rigshopitalet, Copenhagen Laura Rufibach Jain Foundation Jenn Levy Coalition Cure Calpain 3 Jordi Diaz-Manera Newcastle University

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