From Advocacy to Impact: Policy Strategies to Accelerate Rare Disease Cures

On February 19, the Vasculitis Foundation hosted a virtual rare disease policy briefing that brought together leaders from patient advocacy organizations, research institutions, and congressional offices.

The discussion focused on a critical question for the rare disease community: how can policy help accelerate the development of treatments for patients who urgently need them?

Speakers included Beth Westbrook of the Vasculitis Foundation, Annie Kennedy of the EveryLife Foundation for Rare Diseases, and Kit Devine, Legislative Assistant to Representative Kevin Mullin. The conversation was moderated by Brett McReynolds, Penn Quarter Partners.

Throughout the conversation, panelists emphasized that the rare disease community is at an important moment. Scientific innovation is accelerating, public awareness is growing, and patient advocates are playing an increasingly influential role in shaping policy conversations.

The Reality for Vasculitis Patients

Beth Westbrook opened the briefing by outlining the challenges facing people living with vasculitis.

She described vasculitis as “a complex family of roughly 20 conditions with up to 11 approved treatments, none of which offer a cure.” While existing therapies can help manage symptoms, many patients remain concerned about the slow pace of innovation and the limited number of treatment options available today.

At the same time, she highlighted emerging scientific opportunities. For the first time, several companies are exploring genetically targeted technologies in vasculitis research. These approaches may lead to more precise therapies and improved outcomes for patients in the future.

Turning scientific breakthroughs into treatments, however, requires more than research alone. Supportive public policy is essential to help innovative therapies move efficiently through development and reach the patients who need them.

Progress Across the Rare Disease Community

Annie Kennedy broadened the conversation to the national rare disease landscape. “10% of Americans live with a rare disease,” she explained, “yet 95% percent still lack an FDA-approved treatment.”

Despite this challenge, Annie highlighted recent policy progress driven by coordinated advocacy efforts. Advances such as the reauthorization of the Priority Review Voucher program and improvements supporting cross-state Medicaid access demonstrate how policy can make a meaningful difference for patients and families.

She also emphasized that the rare disease community now has stronger tools to advocate effectively, from improved data resources to growing networks of patient advocates.

Policy Opportunities Ahead

Kit Devine underscored the importance of a stable federal research environment supported by strong investment in the NIH and FDA.

She also pointed to a key legislative opportunity currently before Congress: the MINI Act (H.R. 1672). The bill would strengthen development pathways for innovative therapies, particularly those aimed at rare diseases where treatment options remain limited.

Advancing legislation like the MINI Act depends on continued engagement from the patient community. Lawmakers rely on hearing directly from constituents about why policies supporting medical innovation matter.

Take Action

For many patients, the most urgent need is progress toward treatments that improve their quality of life.

As one recent patient survey response captured: “I do not need to worry about drug costs until there is a treatment that truly benefits me.”