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

Abstract

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.

Keywords: European Union; advanced therapies; advanced therapy medicinal products (ATMPs); healthcare; public perception; survey in Europe.

Graphical Abstract

Graphical abstract summarizing the main points.

Figure 1

General assessment of important social topics. Relevance of important societal topics as viewed by people in different European countries or viewed by different age groups (Scale 1—very important to 5—least important), (A) Geographical heat map representation of the ten studied topics, with impact scale bar shown to the right and top ranking issue ‘Healthcare' depicted in yellow-orange color tones, while the lowest ranking issue ‘Migration' is depicted in purple-blue color tones. (B,C) Numerical depiction of priority scoring sorted according to issue and country (shown in B) or according to issue and population age (shown in C).

Figure 2

Knowledge about ATMPs prior to survey. Results on survey topic A (see Table 1) sorted according to questions A1-A4. (A–C) Question A1-A3 geographical heatmaps shown to the left and corresponding numerical depiction shown to the right, with pie charts displaying the (%) agreement across Europe and bar charts displaying (%) answered yes per country. (D) Results of question A4 is displayed as bar chart (%) answered yes per country.

Figure 3

Opinions on public funding in healthcare. (A,B) Results on survey topic B sorted according to questions B2-B3 (Question B1 is show separately as introductory topic in Figure 1) with geographical heatmaps shown to the left and corresponding numerical depiction shown to the right, with pie charts displaying the (%) agreement across Europe and bar charts displaying (%) answered yes per country. Interestingly, an overwhelming 84% of European citizens agree that EU- and state-funding should be invested in the development of future medical innovations, while 83% agree that EU and member states should fund enabling technologies for cell and gene therapies, with only 4% of EU citizens answering ‘No' and 12-13% answering ‘Not Sure', indicating a strong support of EU citizens for funding future medical innovations and enabling technologies for cell and gene therapies.

Figure 4

Opinions on reimbursement of ATMPs. (A–C) Results on survey topic C sorted according to questions C1-C3, with geographical heatmaps shown to the left and corresponding numerical depiction shown to the right, with pie charts displaying the (%) agreement across Europe and bar charts displaying (%) answered yes per country. Only 61% of European citizens agree that the state should pay for expensive therapies although evidence for long-term benefit has not been shown yet, while 74% agree that in the case of rare diseases cross-border health-care (e.g. traveling abroad) is the best way to provide the most beneficial treatment for patients, and 70% agree that non-medical costs (e.g. travel and accommodation) should be covered in cross-border healthcare, clearly indicating that a majority of European citizens is in support of European cross-boarder health care for rare diseases and for support for non-medical costs, such as travel and accommodation.

Figure 5

Relationship between various themes and how they affect overall acceptance of cell and gene therapies, adapted from Aiyegbusi et al. (21). In our “ATMP EU Survey,” the highlighted four main groups (emphasized larger circles) were found to be of key importance.