The Speeding Therapy Access Today (STAT ACT), H.R. 1730/S. 670, includes creation of a Rare Disease Center for Excellence within the U.S. Food and Drug Administration (FDA). The legislation was introduced March 2021 and is currently assigned to the House Committee on Energy and Commerce, which held a hearing on March 17 that included the important bill. The rare disease community awaits the bill’s next steps.
But what would a Rare Disease Center for Excellence at the FDA look like? What would it do? How would it function?
If the bill is successful, all of these questions and more would have to be tackled. Who better to help answer these questions than the rare disease community? The people who understand the unique situation facing rare disease patients and drug development for their conditions.
Creating a Vision for the Center
In support of its hopeful creation, a group of rare disease experts and advocates have created a conceptualization of what the Rare Disease Center for Excellence would look like. Among the experts was Cure Sanfilippo Foundation Chief Science Officer Cara O’Neill, MD, FAAP.
Thr group recently published white paper, “A Community Vision for a Rare Disease Center of Excellence at the FDA,” which consolidates the ideas.
The white paper defines the Center as “an administrative mechanism for collecting and organizing all the FDA’s scientific, clinical and regulatory expertise on a therapeutic area.” It states that, “Given that rare diseases often cut across multiple organ systems and share common drug development challenges, rare diseases would be an ideal candidate for such coordination and organization.”
The white paper advocate the Center to aggregate existing rare disease expertise that is currently dispersed throughout the FDA. The Center would also ideally “help organize all FDA resources – such as statisticians, regulatory scientists and experts in clinical trial design for small populations – within a single structure to avoid duplication and disciplinary silos as well as to make concentrated resources available to multiple review divisions.”
Rare Disease in General
“Individually, rare diseases affect relatively-small populations compared to highly-prevalent conditions such as heart disease, but collectively they take a major toll with estimates suggesting one of every 10 Americans is living with a rare disease.”
It is estimated that more than 300 million Americans have a rare disease, which is defined as a condition that affects fewer than 200,000 people. More than 7,000 rare diseases have been identified and more than half of them affect children.