Cost, pricing and value of medicines

Cost, pricing and value of medicines
This thesis of Simon van der Schans addresses the crossroads between pharmaceutical innovation, pricing of medicine, and health system sustainability.
Part 1 of this thesis analyses the current state of research and development costs, pricing and value of medicine are assessed in order to determine the current challenges and opportunities.
Part 2 of this thesis concerns a case study of the introduction of onasemnogene abeparvovec for spinal muscular atrophy. This part aims to highlight multiple facets of health technology assessment and related challenges of a one-time administration expensive gene therapy compared to a chronic treatment.
In part 3, novel methods to support future sustainability of health systems and pharmaceutical care, the importance of incorporating dynamic medicine prices and broader fiscal health perspectives into HTA is assessed.
In this thesis, the costs, pricing and value of medicine are explored, based on various analyses related to current methods in HTA as well as novel methods. The values that are included in the reimbursement decision needs to align with the changing health system requirements and expectations from society in order to ensure future affordability and accessibility to innovative pharmaceutical products. In this process, the assessment of medicines should be a dynamic process which includes additional data collection to reduce uncertainty and re-assessment in order to optimize future decision-making. The changing pharmaceutical landscape and evolving market access and reimbursement policies provide opportune moments to implement innovative methods for development, pricing and reimbursement, as suggested in this thesis.