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UNEW_FY2013 [UNEW_FY2013] [20123307] - Operating Grant
Mission and vision of the operating grant holder

TREAT-NMD is an international network for rare inherited neuromuscular disorders (NMDs). Its mission is to provide an infrastructure to increase collaboration between clinicians, scientists, and patients, accelerate therapy development for these incurable conditions, improve patient care through publication and dissemination of best-practice consensus guidelines, and provide reliable information for patients and professionals. TREAT-NMD was established in 2007, and until the end of 2011, was funded through FP6 to bring together stakeholders representing all NMDs (>200 diseases). Although NMDs are individually rare, TREAT-NMD activities address ~200,000 patients in the EU, and ~3 million worldwide.
Since 2007 TREAT-NMD has increased the collaborating capacity of the field, raised the profile of NMDs within the wider rare disease (RD) field, become a model for RD collaboration, and increased the profile of European NMD research and healthcare globally. A public consultation in 2010 strongly endorsed the continuation and further development of TREAT-NMD`s core activities.
The TREAT-NMD Alliance has extended beyond the original network members to integrate new participants from around the world. The original number of ~350 scientists and clinicians that were integrated into the network through the 22 partners has now more than doubled and constitutes a critical mass for innovation and harmonisation in NMD care and research globally.
TREAT-NMD outputs:
-highly developed communication/dissemination infrastructure with extensive website and newsletter
-network of over 270 NMD care and trial sites
-global patient registries for several NMDs with >20,000 patients
-range of preclinical resources
-tools supporting translational & clinical research
TREAT-NMD vision:
-enhance European excellence in the NMD field
-increase visibility & recognition of NMDs in national health policy
-sustain core resources and added value of networking developing tools that cannot be maintained by individual research projects
-extend networking activities and resources to new experts worldwide

Strategic objectives and specific activities

The TREAT-NMD Alliance`s overarching objective is to be an international platform for improving care, advancing research and collaboration and providing information to the neuromuscular stakeholder community.
In uniting NMD specialists and other key stakeholders, the TREAT-NMD Alliance has become a comprehensive network for implementing the objectives of EU RD health policy for the NMDs internationally. The Alliance contributes to the development and dissemination of knowledge on NMDs from basic research through to support of healthcare professionals and empowerment of patients, contributing to improvements in access to quality services and care.
Its critical mass of expertise has a major focus on the publication of consensus guidelines and standards covering basic research (e.g. standards for animal model assessment), translational research (outcome measures validation/protocol harmonisation) and care (international consensus guidelines for NMD care) ; all vital prerequisites for progress that can only be achieved by collaboration. It has also become a significant catalyst for new research projects, which take advantage of the translational research platform offered by the Alliance.
The TREAT-NMD Alliance will continue to facilitate activities and serve as the international coordination and communication platform to ensure research funders maximise their investment.
Ongoing coordination activities provide the essential infrastructure or platform on which additional separately funded projects can be built. Maintenance of the core infrastructure provides enormous added value as it ensures the ongoing availability of key resources for patient care and therapy development.
-Facilitating translational research and collaboration
-Implementing national and international networking in countries across Europe and the world to share and develop best practice and care guidelines in the field of rare inherited NMDs
-Establishing, maintaining and extending global patient registries for the collection of natural history data, recruitment of patients into clinical trials and disseminating information to patients
Provide assistance, governance and oversight to neuromuscular patient registries to ensure best legal and ethical practice according to the registries charter, effective use of data for clinical research and safe-guard patients and their families.
Maintain close contact and connectivity with international initiatives in RD registries (EPIRARE, IRDiRC, PARENT, RD-CONNECT, EUCERD etc).
-Developing and validating outcome measures for NMDs to accelerate clinical trials
-Engaging with regulatory authorities (EMA/FDA)
-Promoting and extending the Care and Trial Site Registry
-Advisory support for clinical trial sponsors through the utilisation of the above mentioned tools and services
-Provide advisory and educational support on taking promising preclinical candidates to clinical trial and subsequent market authorisation
-Integrating characterised biobanks of cells, tissue and DNA for NMD research
-Developing preclinical research resources and standard operating procedures for the assessment of animal models for NMDs
The TREAT-NMD Alliance is committed to bringing real patient benefit through partnerships between academic leaders in the field and advocacy groups to develop the resources for therapy development and delivery. Close relationships with the major NMD advocacy groups has been key to the success of TREAT-NMD.
Through the Project Ethics Council (PEC) with members comprising clinicians, scientists, parents, ethicists, legal academics, patients and representatives of parent organisations, the TREAT-NMD Alliance identifies and examines ethical and social aspects of research taking place in the context of the network.

Expected outcomes

The work programme serves to maintain the core operations carried out by the secretariat of the TREAT-NMD Alliance to integrate and harmonise the translational research activities that are necessary to prepare the NMD field for clinical trials and deliver new therapies and care for NMD patients.
OBJECTIVES FOR 2013 (refer to work plan for 2012-2014)
1) Provide over-arching secretariat support for the TREAT-NMD Alliance
2) Provide coordination and support for international NMD projects developed by TREAT-NMD Alliance members
3) Maintain and enhance the communication/information resources of the network for patients and professionals
4) Ensure the international management/coordination of the global patient registries and facilitate their expansion and further development
5) Extend networking for NMDs through advice and support for activities of national networks
TREAT-NMD Alliance core operations and activities:
-Organise 1 annual Executive Committee meeting
-Organise 1 annual Registry Curator/TGDOC meeting
-Establish external Scientific Advisory Board of experts from outside the NMD field
-Obtain ethical input for Alliance activities from Project Ethics Council
-Manage the Alliance administrative, legal and financial aspects
-Assist Executive Committee in coordination of the Alliance (follow-up on planning, implementation, task initiation or completion)
-Provide progress monitoring and results assessment for attention of Executive Committee and Alliance members
-Prepare all project deliverables for dissemination
-Coordinate integration of international NMD projects and activities developed by research and healthcare professionals across the Alliance
-Provide a forum for leaders of the field to come together to develop new projects in NMD research and care
-Act as a platform to support NMD projects funded through other mechanisms with tools and services that will accelerate the achievement of objectives
-Integrate additional experts and NMD patient groups into the Alliance from countries currently underrepresented
-Inform members about funding opportunities
-Maintain and extend TREAT-NMD website
-Maintain monthly TREAT-NMD electronic newsletter
-Provide an intranet platform for Alliance participants
-Provide communication hub for Alliance participants
-Disseminate educational and healthcare documents
-Target general public through press releases, media articles and interviews
-Increase patient registration in existing patient registries by raising awareness (publicising, disseminating information)
-Provide advice and support to national curators to help ensure that data are updated and quality controlled
-Extend registries to new countries & diseases
-Publish aggregate data from the patient registries to aid understanding of prevalence, natural history and variations in disease care between countries
-Conduct registry enquiries to facilitate recruitment of patients into clinical trials
-Harmonise the patient registries with the Care and Trial Site Registry
-Provide administrative support for organisation of annual registry curator meeting
-Support and manage the TREAT-NMD Global Database Oversight Committee
-Continue to promote creation of national NMD networks
-Act as international hub for interaction of national networks with the international community
-Provide information and advice for those wishing to set up national networks
-International harmonization of the NMD field (additional collaborations, less duplication of effort, improved cross talk between separately funded NMD projects)
-Additional patient registries for NMDs and more patients linked within existing registries
-Level playing field for patients
-Better informed patient community
-Easier access to information for NMD stakeholders
-United NMD field with a stronger voice
-Greater trial readiness and accelerated clinical trial development for NMD patients

Start date: 01/01/2013
End date: 01/01/2014
Duration: 12 month(s)
Current status: Finalised
Programme title: Second Programme of Community action in the Field of Health 2008-2013
EC Contribution: € 199 681,00
Portfolio: Rare diseases