Rare Disease Advocates Stage ‘Funeral’ at FDA Headquarters
More than 100 mothers and advocates affected by a group of rare metabolic disorders known as mucopolysaccharidoses (MPS) held a “funeral” in front of the FDA headquarters 3/18 to “mourn the futures” of their children following the agency’s rejection of various rare disease treatments. Newsweek reports that the protestors, who dressed in black, held signs, and carried a coffin outside the headquarters, want treatments for MPS conditions such as Sanfilippo syndrome, Hurler syndrome, and Hunter syndrome.
Kim Stephens, whose son has Hunter syndrome, told the magazine that the protest “was important for our families to stand up for their children and their futures. FDA won’t approve safe and effective drugs, and their children will die because of it. FDA needs to see the faces of the children they are condemning to death. We want the president to see them and help us.”
The article says the agency did not respond to an out-of-working-hours request for comment, but previously had said, “FDA is diligently committed to delivering cures and treatments to families in the safest way possible.”
According to Newsweek, “FDA has come under increasing pressure in recent months over its denial of certain rare disease treatments. Rare disease advocates have said the agency’s rejections show signs of inconsistencies around regulatory requirements, slowing down the process of getting treatments across the line for suffering Americans, while FDA has maintained that approvals in general have not gone down.”
The protestors called on the agency to implement regulatory flexibility for ultrarare diseases with no approved treatments and accelerate pathways and biomarker-based endpoints for MPS conditions. They said Congress should oversee FDA’s decision-making, while including meaningful patient and family engagement throughout drug development and review.
Ryan Foundation volunteer executive director Mark Dant told the magazine that part of the reason the drugs have been denied is because of FDA’s “dislike” of the accelerated approval pathway. “This pathway, codified by Congress, is the only way to keep the science that can treat our children moving forward to approval, and yet because of personal dislikes by [CBER] director [Vinay] Prassad, our children continue to suffer and die,” he said. “We can no longer stand by and wait to plan their funerals. For decades we waited for science to find our tomorrows. Now, it has, and bureaucrats within the agency we pay for are keeping these treatments from our children. We know they are there. We just can’t reach them.”
Stephens, the mother of a Hunter syndrome child, told Newsweek the MPS community has sent over 10 letters to FDA commissioner Marty Makary requesting a meeting. “There is no answer,” she said. “We received a form letter. I even put a request in Makary’s hand in a bright orange envelope. No answer. We heard today that a senior leadership official at FDA told them at FDA to not go out and engage with us because they don’t want to reward bad behavior.” She said the community would keep speaking up.