Project
Framework for value driven market access of gene therapy
Gene therapy makes the treatment of serious, incurable diseases possible, such as cancer and genetic diseases. These therapies may cause promising health benefits, but their long-term effects have not yet been established. Additionally, the development of gene therapies is advanced which leads to high costs. Therefore, this PhD project will investigate and ensure sustainable market access so gene therapies may reach the patient. First, we will investigate the gathered clinical evidence of gene therapies and research the preferences of patients that will receive them. Second, we will examine if the current assessment framework of the government to determine the price and reimbursement is ready for gene therapies and propose a new, value-driven framework that incorporates patients' preferences. Lastly, due to the high prices asked for gene therapy, the government is in need for novel payment models and mechanisms for the collection of real-world evidence. Therefore, we will explore the current barriers for the adoption of such payment models and propose relevant solutions for both payment and evidence collection. Furthermore, we will also determine the roles and responsibilities of all parties involved in gene therapy market access such as the roles of physicians, pharmaceutical industry, regulatory authorities, etc. In conclusion, this project will lead to sustainable market access of gene therapy by providing a value-driven framework for government, industry and others.