A new publication in the European Journal of Health Economics identifies potential challenges of ATMPs in view of current health technology assessment (HTA) methodology and explores potential solutions to these challenges. Three key topics are explored: uncertainty, discounting, and health outcomes and value. A series of recommendations are proposed.
Orphan drugs inherently face a number of challenges in their development and value assessment. In this blog post, we describe the Mechanism of Coordinated Action (MoCA) initiative, and discuss why it can be beneficial to all parties involved and how it can improve decision making for – and access to – orphan drugs in European countries.
When should progression-free survival be used as a surrogate for overall survival in oncology? Uneven reporting of results – different definitions and methods of analysis, lack of rigour in applying methodology – make progress difficult.