A Patient-Centric Approach to Evaluate Rare Disease Treatments

A “one-size-fits-all” approach to drug pricing and value assessment frameworks undermines development of therapies for people – very often children – with rare diseases. As rare disease companies, we champion policies that help people with high unmet needs by developing innovative cutting-edge medicines and working with federal and state policymakers to address access and affordability.

Learn more about the importance of restoring equity and patient focus when valuing rare disease treatments in this overview.