RDCC Proposes Policy Solutions to Remove Barriers for Rare Disease Patients Seeking Treatment Options and Increase Equity of Access

The Rare Disease Company coalition (RDCC) is pleased to share this new resource for federal and state policymakers and regulators to help inform legislative actions that can bolster patient access to life-changing, FDA-approved rare disease therapies.

Today, over 95% of rare diseases lack an FDA-approved treatment, and while the FDA continues to add to the number approved therapies, treatments are only as valuable as they are accessible.

Once a rare disease treatment is approved by the FDA, patients seeking to benefit from the approved therapy are often faced with challenges to access due to complex interstate credentialing, limited coverage, delays in review, inaccurate value frameworks, and insufficient medical necessity determinations.

In this new resource, the RDCC outlines six areas for improvement with specific policy solutions that will improve outcomes for the rare disease community.

2023 Access Campaign Report by Rare Disease Company Coalition
Advancing Patient Access to Rare Disease Therapies