Deliverables
Strategy paper on Regulatory Science for Advanced Therapies
Publications and material for communication second report (flyers, newsletters, videos, seminars)
Strategy paper on iPSC as source for Advanced Therapies - banking haplotyped iPSC and universal allogenic resource - definition of centers of excellence for clinical grade iPSC production - off-the shelf iPSC-derived products and immunogenicity tests - automation and scalable manufacturing platform iPSC-derived products - data base of clinical grade iPSC lines
Strategy paper on viral/non-viral vector development for in vivo/ex vivo gene therapy - Identification of potential non-viral delivery technologies and appropriateness for application to this field. Plan for first RIA projects to assess lead candidates - Viral Vector Manufacture and Analytics - Assessment of anticipated requirements of product developers and gap analysis of current technologies to supply anticipated need
Post Trial Patient Follow-up – develop a proposal to establish best practice for patient follow-up and data capture, this will include considerations relating to post-marketing surveillance and risk- minimization measures, in particular, for those products with a high degree of scientific uncertainty upon point of approval. Develop a plan for first calls on projects to deliver this infrastructure
Defining Key Performance Parameters for the candidate LSRI RESTORE
Strategy paper on benefit and requirements of mechanistic side studies
Strategy paper on Health Economics & Reimbursement – Identify the limitations and opportunities within the existing reimbursement practices within the healthcare systems which do not easily accommodate the potential outstanding value that curative and disease modifying drugs, such as cell and gene therapies, can deliver and develop a plan to define projects to address these to allow rapid European ATMP adoption including the development of innovative payment mechanisms
List of candidates of governing bodies (names)
Position paper of patient´s view to Advanced Therapies by patient advocates
Publications and material for communication first report (flyers, newsletters, videos, seminars)
Publication of the strategy and position papers as well as the White Paper
Draft of Governance (structure and processes) of the candidate LSRI RESTORE
Strategy paper on biomaterial developments and composite products
Strategy paper on innovative early and late clinical trial design and regulatory rules for Advanced Therapies - Pivotal Clinical Trials – Assess current systems for traceability and tracking of ATMPs. Deliver a plan of projects to develop a system that is compatible with current regulations and which will be suitable for applying across Europe - A review will be performed of current reported methodological issues and invalid trial designs (including challenges of patient numbers [e.g. orphan diseases] and nonrandomized trial de-signs without a comparator), inappropriate clinical outcomes and benefit risk assessments - Develop an expert working group to that will assess review and develop proposals for projects that will deliver best practice in late phase clinical trial design this will include clinical consider-ations for the co-development of companion diagnostics - Develop a plan to work with regulators and other stakeholders to ensure a regulatory regime that enables safe and effective ATMP medicines to be trialed and licensed more rapidly and with a level of global compatibility, enabling Europe to be a leading player in clinical delivery
Strategy paper on new clinical indications based on i) improved diagnostics and availability of stem cells for gene editing (rare diseases), ii) new immunotherapeutic options, iii) tissue regeneration vs. tissue replacement approaches
Strategy paper on various issue of manufacturing (including automation) of Advanced Therapy products
Strategy paper on GMP-conform cell sorting approaches
Completion of selection and description of regulatory acceptable “contexts of use” for advanced animal vs human/patient-on-a-chip model developments with fit to preclinical developments and pivotal clinical trials of WP 3
Strategy paper on early Health-Technology Assessment tools for Advanced Therapies
Implementation of working groups addressing the main tasks of the Strategic Research & Innovation Agenda (mo 3)
Map of European´s ecosystem and non-European network activities for Advanced Therapies
Roadmap of the needs and strategy of clinical Research for Advanced Therapies
Roadmap of the needs and strategy of mission-driven basic science and technology development
Roadmap of the needs and strategy of the Implementation of new Advanced Therapies
Report to EC
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Publications
Author(s): Enrico Fritsche, Magdi Elsallab, Michaela Schaden, Spencer Phillips Hey, Mohamed Abou-El-Enein
Published in: Cell and Gene Therapy Insights, Issue 5/11, 2019, Page(s) 1505-1521, ISSN 2059-7800
DOI: 10.18609/cgti.2019.156
Author(s): Enrico Fritsche, Hans-Dieter Volk, Petra Reinke, Mohamed Abou-El-Enein
Published in: Trends in Biotechnology, Issue 38/10, 2020, Page(s) 1099-1112, ISSN 0167-7799
DOI: 10.1016/j.tibtech.2019.12.009