Real-world evidence for coverage determination of treatments for rare diseases

Health technology assessment (HTA) decisions for pharmaceuticals are complex and evolving. New rare disease treatments are often approved more quickly through accelerated approval schemes, creating more uncert...

Source: Orphanet Journal of Rare Diseases - February 7, 2024 Category: Internal Medicine Authors: Victoria W. Dayer, Michael F. Drummond, Omar Dabbous, Mondher Toumi, Peter Neumann, Sean Tunis, Nelson Teich, Shadi Saleh, Ulf Persson, Johann-Matthias Graf von der Schulenburg, Daniel C. Malone, Tay Salimullah and Sean D. Sullivan Tags: Position statement Source Type: research