This paper is part 5 in Blue Matter’s series on the “rare disease ecosystem.” It outlines key questions and considerations for biopharmaceutical companies as they develop pricing, access, and reimbursement strategies for rare disease therapeutics.

Other installments in the series can be accessed via these links:

Part 1 – Understanding the Rare Disease Ecosystem

Part 2 – Optimizing Clinical Trial Design

Part 3 – Continuous Evidence Generation

Part 4 – Clinical Trial Implementation