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.
Just published are two new reports that explore the use of real world evidence for coverage and formulary decisions. The publications represent 1) the background paper to the 2017 ICER Membership Policy Summit and 2) a follow up paper based on the discussion at the Summit.