Acting FDA Commissioner Kyle Diamantas Meets with Rare Disease Groups to Repair Strained Relations
Key Insights
Acting FDA Commissioner Kyle Diamantas met with rare disease advocacy organizations on June 3 to rebuild trust following former Commissioner Marty Makary's tenure, which saw several setbacks for rare disease drugmakers.
Jeff Allen, CEO of Friends of Cancer Research (search), described the meeting as a "breath of fresh air" and praised Diamantas for exhibiting "real leadership" by listening to the rare disease community.
Advocacy groups pressed for greater regulatory clarity, novel clinical trial approaches, and consistency from the FDA for treatments targeting small patient populations.
Acting U.S. FDA Commissioner Kyle Diamantas met with representatives from rare disease advocacy organizations on Wednesday, June 3, in a concerted effort to mend relations that had frayed under the leadership of his predecessor, former Commissioner Marty Makary. The meeting, confirmed by both attending groups and a U.S. Department of Health and Human Services official, signals a potential shift in the agency's engagement with stakeholders focused on treatments for small patient populations.
Representatives from organizations including Friends of Cancer Research (search) and the Foundation for Angelman Syndrome Therapeutics (search) attended the meeting, pushing for greater certainty and support for therapies aimed at rare diseases.
Jeff Allen, CEO of Friends of Cancer Research (search), described the meeting as a "breath of fresh air" in a phone interview with Reuters. "The Acting Commissioner is exhibiting real leadership by listening to people," Allen said. "It was clear they are repairing the organization, and they are supporters of science and career staff."
A Strained Relationship Under Makary
The rare disease community's relationship with the FDA had deteriorated during Makary's tenure, during which the agency dealt a series of setbacks to drugmakers in the space. The FDA declined to approve Biohaven (search)'s experimental treatment for a brain disorder, placed a serious safety warning on a Sarepta Therapeutics gene therapy (search), and a top agency official publicly described UniQure's Huntington's disease (search) treatment as a "failed product."
These actions, combined with broader upheaval at the agency following an overhaul of the federal bureaucracy under the Trump administration that saw many top experts leave the FDA, left rare disease groups seeking greater regulatory clarity. In April, a coalition of rare disease organizations pressed President Donald Trump and other top health leaders for more certainty amid the leadership changes and uncertainty at the agency.
No Promises, But a Willingness to Listen
Allen noted that no specific promises for action were made by FDA officials during the meeting. Instead, agency representatives predominantly listened as attendees advocated for novel approaches to clinical trials, engagement with the broader public, and consistency and predictability from the FDA.
For diseases with small patient populations, regulatory clarity is essential to overcoming challenges including complex disease histories and limited options for measuring a treatment's safety and efficacy, according to the Foundation for Angelman Syndrome Therapeutics (search), an advocacy group for patients with a rare neurogenetic disorder.
An official from the U.S. Department of Health and Human Services confirmed the meeting and characterized it as part of a broader push to work with "key stakeholder groups across the FDA continuum, most namely our trusted partners in the rare disease community."
Leadership Uncertainty at the Agency
Diamantas, who serves as the agency's food chief, does not have the traditional background for the FDA's top role but is viewed by some inside and outside the agency as a steady hand. He is under consideration to be nominated for the role permanently, along with other candidates, according to two sources familiar with the process. However, Endpoints News reported that Diamantas is not interested in stepping up to the role on a permanent basis.
CNN reported that Norman "Ned" Sharpless, who briefly served as FDA commissioner during President Trump's first term, is being considered again for the position. Sharpless has been an outspoken critic of Health and Human Services Secretary Robert Kennedy Jr.'s vaccine policies.
Rare disease advocacy organizations have previously demonstrated their political influence, having successfully swayed Trump during his first term to back federal legislation giving terminally ill patients access to experimental treatments, which he signed into law in 2018.