Periodic Reporting for period 2 - EuroGCT (European consortium for communicating gene- and cell-based therapy information.)
Okres sprawozdawczy: 2022-02-01 do 2023-07-31
Cell and gene-based therapies have the potential to treat many debilitating diseases and conditions. However, their development and use raises ethical and practical concerns that vary across Europe, including around the complexity and costs of product development, fragmented regulatory positions and procedures for reimbursements, and around patient and public understanding of which conditions may realistically be treated by cell and gene-based therapies and the processes which must be navigated for a new scientific discovery to develop into a new clinical treatment. Furthermore, while the field is moving steadily towards its anticipated translational goals, unscrupulous operators are already offering patients unproven treatments, often at high financial cost. As a result, this research is the subject of great debate among scientists, clinicians, patients and people affected by conditions, politicians, religious groups, business interests, educators and students, and the public in general, and both high-quality information and engagement with fields related to cell and gene therapy development are required to foster better understanding and evidence-based decision-making among European stakeholders.
The European Consortium for Communicating Gene and Cell Therapy Information (EuroGCT) unites 47 partner organisations and institutions across Europe, including the major European advanced therapies learned societies, with the common goal of providing reliable and accessible information related to cell and gene therapy development to European stakeholders.
EuroGCT has two major objectives:
• To provide patients, people affected by conditions, healthcare professionals and citizens with accurate scientific, legal, ethical and societal information and with engagement opportunities, and thus to support better informed decision-making related to cell and gene-based therapies.
• To facilitate better decision-making at key points in development of new therapies and thus enable improved product development, by providing the research community and regulatory and healthcare authorities with an information source on the practical steps needed for cell and gene therapy development.
The European Consortium for Communicating Gene and Cell Therapy Information (EuroGCT) unites 47 partner organisations and institutions across Europe, including the major European advanced therapies learned societies, with the common goal of providing reliable and accessible information related to cell and gene therapy development to European stakeholders.
EuroGCT has two major objectives:
• To provide patients, people affected by conditions, healthcare professionals and citizens with accurate scientific, legal, ethical and societal information and with engagement opportunities, and thus to support better informed decision-making related to cell and gene-based therapies.
• To facilitate better decision-making at key points in development of new therapies and thus enable improved product development, by providing the research community and regulatory and healthcare authorities with an information source on the practical steps needed for cell and gene therapy development.
In January 2022 the consortium launched a website at www.eurogct.org with sections targeted for patients and the public, and for the research and regulatory communities. This is the digital framework on which the remainder of the project will build.
The ‘Discover Gene and Cell Therapy’ section is targeted to patients, people affected by conditions, healthcare professionals and citizens. Content can be filtered by condition or theme, searched by keywords or browsed in sections on Cell and Gene Fundamentals, Methods and Tools, Current and Potential Uses, and Clinical Trials and Patient Involvement. Published resources include an initial set of new fact sheets addressing topics identified as a priority by the consortium and through discussion with target audience members. These include conditions that can currently be treated, CAR-T Therapy, clinical trials and unproven therapies. For a range of specific conditions, introductory text has been prepared about the state of play. The information hub also integrates selected collated resources developed or recommended by project partners. These include a suite of fact sheets about how stem cell research may benefit specific conditions, which will be brought up to date to reflect the latest developments across the field of gene and cell therapy in the next stage of the project.
The ‘Research Pathways’ section is tailored information for the research and regulatory communities on the development of gene and cell therapy from lab to patients. Introductions and links to resources are available for each of the subsections: Research and Innovation, Therapy Classification, Manufacturing, Commercialisation, Actors and Networks, and Public Involvement and Data. A nested tree structure is in development for detailed entries within each subsection covering the regulatory aspects and practical steps at each stage along the development pathway. These entries will include what happens at the relevant stage, stakeholders involved, terminology definitions, challenges, opportunities and incentives, interactions with regulators, practical steps, and information on regulations, including applicable binding texts, guidelines and other support.
The ‘Discover Gene and Cell Therapy’ section is targeted to patients, people affected by conditions, healthcare professionals and citizens. Content can be filtered by condition or theme, searched by keywords or browsed in sections on Cell and Gene Fundamentals, Methods and Tools, Current and Potential Uses, and Clinical Trials and Patient Involvement. Published resources include an initial set of new fact sheets addressing topics identified as a priority by the consortium and through discussion with target audience members. These include conditions that can currently be treated, CAR-T Therapy, clinical trials and unproven therapies. For a range of specific conditions, introductory text has been prepared about the state of play. The information hub also integrates selected collated resources developed or recommended by project partners. These include a suite of fact sheets about how stem cell research may benefit specific conditions, which will be brought up to date to reflect the latest developments across the field of gene and cell therapy in the next stage of the project.
The ‘Research Pathways’ section is tailored information for the research and regulatory communities on the development of gene and cell therapy from lab to patients. Introductions and links to resources are available for each of the subsections: Research and Innovation, Therapy Classification, Manufacturing, Commercialisation, Actors and Networks, and Public Involvement and Data. A nested tree structure is in development for detailed entries within each subsection covering the regulatory aspects and practical steps at each stage along the development pathway. These entries will include what happens at the relevant stage, stakeholders involved, terminology definitions, challenges, opportunities and incentives, interactions with regulators, practical steps, and information on regulations, including applicable binding texts, guidelines and other support.
The EuroGCT website will be iteratively developed over the remainder of the project. Key content will be published in at least six other European languages. Robust procedures are in place for information management, ensuring ongoing reliability and accessibility. The update of existing condition-specific information is underway, alongside the development of new resources. The consortium is working with end users such as patient groups to co-create resources that meet their needs, and with relevant experts to populate the detailed entries for the lab-to-patient development pathways. Beyond the website, through a series of communication events and other engagement opportunities, fostering relationships with the media and multiplier organisations, and supporting capacity building among researchers and science communicators around patient and public engagement with cell and gene therapy research, we will contribute to increased awareness of scientifically viable information related to the development of gene and cell therapies, thus enabling informed decision-making. Through bringing together into a collated hub the complex and dispersed information to support researchers, regulators and healthcare authorities along the therapy development pathway, we will improve its accessibility and uptake, promoting knowledge exchange within and between these communities, with the ultimate aim to improve product development.