Executives from 15 rare disease-focused nonprofits met on Wednesday with acting FDA Commissioner Kyle Diamantas and senior staff.
In the meeting, Diamantas made it clear he’s headed in a different direction from former Commissioner Marty Makary and his team, particularly when it comes to listening to career staff on new drug approvals. The goal was for the groups to level with the newly installed leadership, which also includes acting CDER Director Mike Davis and acting CBER Director Karim Mikhail, both of whom attended the meeting.
Diamantas told attendees that “he would prevent interference with the science and decision-making of career staff,” James Valentine, a board member at the nonprofit EveryLife Foundation for Rare Diseases who attended Wednesday’s meeting, recalled to Endpoints News in a phone interview.
Valentine said the new acting commissioner also indicated more adcomms will be forthcoming, as the administration has so far significantly reduced the number of adcomms it’s held when compared to previous administrations.
“As a part of our push to usher in reforms and continue our work to end the chronic disease epidemic and accelerate access to life-saving treatments, we will be meeting regularly with key stakeholder groups across the FDA continuum, most namely our trusted partners in the rare disease community,” a spokesperson for the FDA said in a statement.
Both of the acting center directors also said at the meeting that they are working to try to recruit more review staff, following the major staff cuts in April 2025, and are including people who have worked at the FDA before.
While no specific applications were discussed at the meeting, Valentine and two other attendees told Endpoints that the groups were generally pleased with the responsiveness of the FDA leaders.
“The acting commissioner and his team were genuinely engaged and it showed,” Jeff Allen, president and CEO of the nonprofit Friends of Cancer Research, told Endpoints in a statement on Thursday. “We raised specific opportunities around biomarker development, novel trial designs and clinical trial efficiency, and we left with real optimism that this leadership wants to find solutions.”
One of the FDA leaders at the meeting, who asked to remain anonymous, said the meeting went well and that it was a constructive atmosphere.
Other rare disease nonprofit executives who attended the 90-minute meeting included Kara Berasi, CEO of the Haystack Project; Andrea Goodman, CEO of the nonprofit I AM ALS; Cara O’Neill, co-founder of the Cure Sanfilippo Foundation; and Pamela Gavin, CEO of the National Organization for Rare Disorders.
“It’s been heartening to hear directly from the FDA’s current leadership as they prioritize reform and modernization, particularly for rare diseases,” Goodman said. “We’re optimistic about what’s ahead and believe that, together, we can help create a more responsive, rigorous and patient-centered pathway.”
Haystack’s Berasi told Endpoints, “Two things really resounded from the discussion – we need to improve the infrastructure for gathering evidence in rare diseases and also work in the here and now, meeting science where it is and using the tools we have to discern treatment effect even when populations are small and heterogenous. I believe FDA heard that we cannot afford to choose between those goals.”
The meeting follows significant concerns from rare disease advocates, Congress and other stakeholders who sought more consistency in the FDA’s approval process for rare disease drugs, even as Makary and former CBER Director Vinay Prasad proposed a new “plausible mechanism” pathway.
At a Senate Aging Committee hearing in February, lawmakers on both sides of the aisle discussed their frustrations with the FDA, including a recent slate of rejections of rare disease treatments. Annie Kennedy, chief mission officer of the EveryLife Foundation, told the committee at the time that there were 23 rejections of new rare disease drugs since the start of 2025.
The Wall Street Journal editorial board, a frequent critic of Makary, also noted last February that the agency “torpedoed several rare disease drugs for reasons that echo Dr. Prasad’s criticism of accelerated approvals when he was an academic.”