Balancing between Science-Technology and Society:

Background: In 2007 the European Medicines Agency (EMA) set up a new regulation, determining a new class of therapeutic products: the Advanced Therapy Medicinal Products (ATMPs). Furthermore, it established a new scientific committee, the Committee of Advanced Therapies (CAT) responsible for the assessment of these products prior to their authorization. This study will try to examine how the regulatory framework was drawn in response to innovative therapies. It is investigated whether the idiom of co-production is applicable. Furthermore, expectations of different stakeholders such as developers, patients and regulators are presented. Lastly, CAT is considered a hybrid forum, since amongst others there are patient representatives as members of the committee. Therefore, it is found beneficial to study the input of patients into the regulatory field and the role of patient participation. Methods: In order to find answers to the aforementioned issues, information is collected from scientific papers and articles. Moreover, public documents, reports or statements of EMA or patient groups are valuable for the analysis. Interviews are also conducted with people from different fields such as industry, academia, EMA and patient groups. Results: It is found that financial incentives are given to companies in order to support their financial sustainability. The input of academia is highly valued by regulators and their involvement in the regulatory process is encouraged. Scientific uncertainty seems to be the reason for the tendency of regulators to have them on board. CAT strives for flexibility and more proactive approaches, fostering dialogue with developers at early stages. However, it is not optimally operated. The identity of regulators gradually changes, moving from the traditional strict model to a more proactive, open and communicative one. Expectations seem to have a dual nature. On the one side, they help establish the practice of ATMP research and therapy. On the other side, they cultivate unwanted hopes in patients. Regulators need to find balance between these two and provide an appropriate framework that would foster innovation and protect patients. The input of patients is highly acknowledged. They give insight into their diseases, symptoms and treatments through their real-life experiences. Patient organizations are created. They raise awareness, fund research and assist in clinical trials. They put pressure in governments or institutions, assert their rights and fight for their benefit. The institutionalization of their role in committees of EMA has strengthened the realization that their voices should be heard. They are considered partners, becoming active players both in research and regulatory process. Conclusion: Co-production seems to be an appropriate approach, describing how a regulatory framework walks in parallel with innovation. Certain deficiencies arise but the triangle of developers-regulators-patients is indicative of the intertwinement of science and technology with society. Different aspirations need to be taken into consideration and a balance between hope and hype should be achieved. Patient empowerment raises legitimacy of regulatory decisions. The challenge of more public participation strategies remains to be further studied.