Value-Based Reimbursement Decisions for Orphan Drugs: A Scoping Review and Decision Framework

摘要

Background The rate of development of new orphan drugs continues to grow. As a result, reimbursing orphan drugs on an exceptional basis is increasingly difficult to sustain from a health system perspective. An understanding of the value that societies attach to providing orphan drugs at the expense of other health technologies is now recognised as an important input to policy debates.