The RESTORE Momentum Workshop, which took place on 17th February 2020 in Berlin, brought together the RESTORE community to reflect on the progress made in the last 12 months and how to continue fulfilling the RESTORE mission in the future. There were thought provoking talks from patient advocates, Bettina Ryll (MPNE), Karolina Hanslik (EURORDIS) and Sabine Dupont (IDF-Europe), on how to make the patient voice heard among the many players in the ATMP development pipeline and the importance of clear communication between all stakeholders.
The importance of clear communication and well-defined procedural guidelines was a theme throughout the day. From the talk of Toni Cathomen (Universitätsklinikum Freiburg), we learnt about the need for clear guidelines and assays for the detection of off target effects of CRISPR/Cas9 products. Similarly, Hildegard Büning (Hanover Medical School) talked about the many barriers for AAV reaching their full potential in gene therapy methods, including the need for defined genotoxicty testing and measures for transduction efficiency and accuracy. Johan Van Eldere, speaking on behalf of EUHA, highlighted the requirement for a new model of communication and cooperation between university treatment centres for CAR T cell therapies. Whilst Stephen Sullivan (GAiT) stressed the importance of standardisation both in the language used to describe different requirements during ATMP development and the quality control methods used for different products. Clear-cut guidelines were again a focus in the talk by Ulrike Köhl (Frauenhofer IZI), in which she stressed the importance of up to date staff training and coherence of this training and information transfer between institutes about manufacturing processes for ATMPs.
A joint survey by CARAT and RESTORE, presented by Michel Apel (Miltenyi) is investigating the views of professionals on ethical, regulatory and technological obstacles for the translation of Advanced Therapies. The results are still coming in (if you want to participate please click here), but already trends are emerging about areas in which more information and clarity is required.
Finally, more than 80% of European citizens support the idea that EU member states should be funding innovation and enabling technologies for Advanced Therapies (preliminary results from survey by RESTORE, presented by Gady Goldsobel). A strong indication to the politicians that healthcare is important to EU citizens and should not be neglected in future budget plans. Perhaps information that is not currently being communicated between the general public and the political sphere and an area where RESTORE hopes to close this communication gap.
The issue of bringing Advanced Therapies to the market is a tricky one. There are a huge number of technical, scientific and economic barriers that must be overcome in order to make Advanced Therapy Medicinal Products (ATMPs) available to all those in need. As was evident from the discussions between speakers and audience members, breaking down these barriers will be no mean feat. However, this is the mission RESTORE set out to fulfil just under 1 year ago, to make the promise of Advanced Therapies a reality for all patients in need to ensure their implementation into clinical practice as affordable, standard of care therapy and to thereby enhance patient outcome with a high impact on European society and economy. RESTORE coordinator, Hans-Dieter Volk (Charité Universitätsmedizin Berlin) summarised the event in his closing remarks “with the enthusiasm and backing of the RESTORE community, we continue to learn and evolve and so will continue to build up a European ecosystem to bolster innovation, communication and translation within the complex realm of Advanced Therapies”.