Advocacy Update
Rare disease advocates gathered during Rare Disease Week on Capitol Hill 2020.
COVID-19 Raises Urgency for FDA Rare Disease Center of Excellence Britta Dornan
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n late February 2020, shortly before COVID-19 became our country’s headlining healthcare concern, more than 900 rare disease advocates, including pemphigus and pemphigoid (P/P) patients, traveled to Washington, DC., to participate in Rare Disease Week on Capitol Hill. The event, hosted by the Rare Disease Legislative Advocates (RDLA), a program of the EveryLife Foundation for Rare Diseases, was designed to give rare disease advocates the opportunity to make their voices heard by members of Congress. Among the policy issues that the army of advocates championed was the establishment of a Rare Disease Center of Excellence (COE) at the US Food and Drug Administration (FDA).
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Quarterly | Summer 2020
Advocates explained how the process of developing therapies for small patient populations presents unique challenges, which a COE would address. They maintained that a COE would not create new bureaucracy, but instead would remove communication barriers across FDA divisions and facilitate collaboration. They pointed out that the FDA already has authority under the 21st Century Cures Act (signed into law in 2016) to establish a COE. They further stressed that 93 percent of the 7,000 known rare diseases have no FDA-approved therapies, making improvements to our regulatory system an urgent need. In the days, weeks, and months that followed Rare Disease Week, COVID-19 invaded our communities,
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