RESTORE Survey on the Public Perception of Advanced Therapies and ATMPs in Europe — Why the European Union Should Invest More!

Advanced therapy medicinal products (ATMPs) are potential game changers in modern medical care with an anticipated major impact for patients and society. They are a new drug class often referred to as "living drugs," and are based on complex components such as vectors, cells and even tissues. The production of such ATMPs involves innovative biotechnological methods. In this survey, we have assessed the perception of European citizens regarding ATMPs and health care in Europe, in relation to other important topics, such as safety and security, data protection, climate friendly energy supply, migration, and others. A crucial question was to determine to what extent European citizens wish to support public funding of innovations in healthcare and reimbursement strategies for ATMPs. To answer this, we conducted an online survey in 13 European countries (representative of 85.3% of the entire EU population including the UK in 2020), surveying a total of 7,062 European citizens. The survey was representative with respect to adult age groups and gender in each country. Healthcare had the highest ranking among important societal topics. We found that 83% of the surveyed EU citizens were in support of more public funding of technologies in the field of ATMPs. Interestingly, 74% of respondents are in support of cross-border healthcare for patients with rare diseases to receive ATMP treatments and 61% support the reimbursement of very expensive ATMPs within the European health care system despite the current lack of long-term efficacy data. In conclusion, healthcare is a top ranking issue for European Citizens, who additionally support funding of new technologies to enable the wider application of ATMPs in Europe

The effect of IM injection of PLX-RAD cells on days 1 and 5 after radiation on the number of nucleated cells from whole BM and the ratio of progenitors within this cell population.

<p>(A) Up to day 9 after irradiation, the number of the nucleated BM cells decreased sharply in both arms, followed by a faster gain in the number of total nucleated BM cells in the PLX-RAD treated animals, as compared to the vehicle controls. By day 30 a full recovery of the number of nucleated BM cells was recorded only in the group of irradiated PLX-RAD treated mice. (B) FACS analysis of the kinetics of changes in the % of CD45⁺/Sca-1⁺ of the nucleated cells (representing the progenitor cells population) was tested in both arms of mice irradiated by 7.7 Gy, PLX-RAD or vehicle control treated. Maximal increase in the proportion of these cells due to the radiation exposure was recorded on day 9 in both groups, before the onset of the critical phase of the hematopoietic syndrome. Significance: * = P<0.05, ** = P<0.01.</p

The effect of IM PLX-Mat and PLX-RAD injection on the survival of 7.7 Gy total body irradiated mice.

<p>The setup of the experiment is described in (A). 2×10⁶ PLX-RAD or PLX-Mat cells were injected IM twice, 1^st injection delivered 24 hrs and 2^nd injection on day 5 after irradiation. The follow-up of mice survival is shown in (B). Weight changes for the group of IM injected mice at day 1 and 5 after irradiation are presented in (C). The BM and blood counts of the surviving animal at the end of the experiment at day 23 are presented in (D). Significance: * = P<0.05, ** = P<0.01*** = P<0.005, **** = P<0.001, ***** = P<0.0001.</p

Time dependent alterations of the profile of the 3 peripheral blood cell lineages following irradiation and PLX-RAD treatment.

<p>Mice were injected with PLX-RAD on day 1 and 5 following 7.7 Gy irradiation. The mice were bled and sacrificed on days 2, 6, 9, 14, 23 and 30 after irradiation. RBC (A), WBC (B) and Platelets (C) were counted in peripheral blood. The data are based on at least 8 mice which were included for each group for the different rime points. For day 23 the data are based on 31 PLX-RAD treated and 55 vehicle treated mice (from repeated experiments). The recovery on day 23 of peripheral blood cells counts of the surviving mice subjected to the different tested arms with different timing of the first IM injections is presented. The more affected animals died before the end of the experiment, mostly in the group of vehicle treated controls, leaving only the few survivors on which the relevant data are based. The profile of the WBC, platelets and RBC are presented. Significance: * = P<0.05, ** = P<0.01, **** = P<0.001, ***** = P<0.0001.</p