Institutional Readiness of Novel Advanced Therapy Medicinal Product Systems
MetadataShow full item record
Chimeric Antigen Receptors for T-cells (CAR-T) technology can be used as a new innovative form of cell therapy treatment with the potential to cure diseases which as of now are considered incurable, like multiple myeloma. However, there are barriers that hamper the development, diffusion and the availability of CAR-T treatments. These barriers arise because the technology and manufacturing processes needed for autologous CAR-T treatments are subjected to a variety of barriers that are not applicable to conventional drugs, such as small molecules. The research is conducted based on the research question “What are the most important barriers in CAR-T’s innovation system in Belgium, the Netherlands and Luxembourg and how can these barriers be overcome especially regarding the institutional readiness for CAR-T cell therapy?” The innovation process of CAR-T is analysed using two different frameworks. First, an event analysis based on the functions of the Technological Innovation System (TIS) framework is performed to map the development of the CAR-T innovation system in the Netherlands, Belgium and Luxembourg from 2011 to 2020. Second, the TIS framework only analyses innovation on a systemic level, while for complex innovative medical technologies barriers also arise on the organisational level. Webster and Gardner’s institutional readiness (IR) framework is applied to cover the barriers of CAR-T innovations on the organisational level. This thesis has an explorative nature, because both CAR-T technology and the IR framework, and its categories, are relatively new and underexplored concepts. To accommodate the explorative character of the thesis, data was collected by desk research on CAR-T innovation. The desk research is complemented with 16 interviews with experts in CAR-T development, including experts from academic hospitals, the pharmaceutical industry, universities and governmental agencies. The event analysis shows that the CAR-T innovation system is currently at the end of its development phase and the beginning of the take-off phase. The main barriers on a systemic level are hampering regulations, unharmonized policies on a European level and a lack of consensus between the pharmaceutical industry and governmental agencies about what constitutes cost-effectiveness for CAR-T treatments. Barriers on the organisational level concern the lack of resources and vein-to-vein infrastructure needed to manufacture autologous CAR-T cells. This research suggests several interactions between the functions of TIS and the categories of IR, such as the interactions between; knowledge development and relative need, strategic focus and guidance of the search and between guidance of the search and evaluation processes. The thesis concludes by providing policy recommendations which are 1) pharmaceutical firms should invest in vein-to-vein infrastructure to perform the manufacturing process. 2) governments and pharmaceutical organisations are advised to find a drug reimbursement system to find consensus about cost-effectiveness of CAR-T while still providing access to treatment for patients. 3) Policy and application of policy on a European level should be actively harmonised by the EMA.