Reliable and accessible information on cell and gene-based therapies

Proposals should offer well-structured and detailed strategies to convey accurate and up-to-date information on cell and gene-based therapies using multiple contemporary modalities, including a website. The consortium should consist of diverse actors and could include experts in science communication, patients’ representatives, industry, SMEs, clinical and academic researchers as well as the major European learned societies in the field. They should provide expertise across the field of human stem cells, regenerative medicine, genome-editing and gene therapy. All communication material/information should be translated to English and proposals should provide a detailed strategy on the linguistic approach of dissemination in order to reach a large EU audience. The website should be user-friendly and should contain tailored sections dedicated to at least researchers, patients, and the public.

For broader audiences proposals should create a reliable, transparent, accessible resource for patients to make informed decisions and for citizens to have access to scientifically viable information on cell and gene-based therapies, including sex and gender aspects when relevant. Proposals should provide state-of-the-art strategies to engage the public and foresee regular evaluation of whether they reach the targeted audiences. In addition, a series of communication events should be organised, also open to the public, where innovative technologies could be presented and discussed.

For the research community, proposals should create an information source on the practical steps needed for cell and gene-based therapy development. Proposals should provide a one-stop shop on where to seek further information and guidance relating to manufacturing guidelines, regulatory requirements, intellectual property rights, market acceptability and ethical matters. Proposals should provide a strategy on how they will liaise with regulatory agencies (e.g. national agencies, the European Medicines Agency (EMA[[European Medicines Agency: http://www.ema.europa.eu/en]]), the Heads of Medicines Agencies (HMA[[The Heads of Medicines Agencies is a network of the Heads of the National Competent Authorities whose organisations are responsible for the regulation of medicinal products for human and veterinary use in the European Economic Area http://www.hma.eu/]]) network, EUnetHTA[[EUnetHTA Joint Action 3 is a European network of national/regional HTA bodies under the EU Third Health Programme http://www.eunethta.eu]] network). Finally, proposals should include a realistic sustainability plan which explores how the ownership of the information will be structured, and propose a defined organisation to take responsibility, manage and administer the information, and to which authorities/organisations the information will be delivered at the end of the project. Sustainability should be ensured for at least 5 years after the end of the project.

The Commission considers that proposals requesting a contribution from the EU between EUR 1.5 and 2 million would allow this specific challenge to be addressed appropriately. Nonetheless, this does not preclude submission and selection of proposals requesting other amounts.

Cell and gene-based therapies have the potential to treat many debilitating diseases and conditions. However, the pace of their clinical development does not meet public expectations. They face difficulties reaching patients because inter alia the complexity and costs of product development, regulatory hurdles and the non-harmonized procedures for reimbursements. In addition, there are concerns over patient safety due to the use of unproven treatments[[https://blogs.scientificamerican.com/news-blog/embryonic-stem-cells-cause-cancer-i-2009-02-19/

]] [[https://www.senato.it/application/xmanager/projects/leg17/file/repository/relazioni/libreria/novita/XVII/IC-41_stamina.pdf]] .

• Better informed decision making by patients and the public, due to objective, accurate and transparent communications of the latest developments and actual treatments available in the field in order to avoid misconceptions
• Better informed decision making by regulatory and healthcare authorities, due to better access to reliable and updated information, and to stronger synergies and knowledge sharing between decision-makers and other stakeholders including advanced therapies learned societies.
• Improved products development, by providing the research community and patients with a high-quality information source.