In a roundtable event on Thursday, HHS Secretary Robert F. Kennedy Jr. said his office will work to eliminate barriers that keep cell and gene therapies from the market.
Health and Human Services Secretary Robert F. Kennedy Jr. backed his health appointees, who in recent weeks have expressed openness to regulatory flexibility for investigational cell and gene therapies, particularly those for rare diseases.
“We’re going to do everything in our power to sweep away the barriers from you getting those solutions to market and getting them funded and do everything that we can to support you,” Kennedy said Thursday during an FDA roundtable on cell and gene therapies. “We are going to continue to figure out new ways . . . of accelerating approvals for drugs and treatments that treat rare diseases.”
Kennedy also directly addressed the recent budget cuts at HHS, claiming that instead of totally taking away these funds, his office will instead reallocate them to other efforts, with an eye toward boosting efficiency.
“There’s a lot of people who criticize us because they say we cut the budget of the agency,” he said. “We didn’t cut any of the budget of the agency. We have that money and we are taking away from the administrative costs and redundancies and we’re going to reapply it, spawn and foster and fortify innovation, and unleash it.”
In April, The Washington Post revealed that President Donald Trump was considering reducing HHS’ budget by $40 billion, citing leaked internal documents. These cuts would slash the NIH’s budget by some 40% and the CDC’s by 44%.
The Thursday event, hosted by the FDA’s Center for Biologics Evaluation and Research (CBER), functioned largely as a dialog between HHS and experts in the field. Representing the government were Trump’s top health appointees, including newly appointed CBER director Vinay Prasad, FDA Commissioner Marty Makary and NIH head Jay Bhattacharya. Several industry and academic experts were also part of the roundtable, including cell therapy pioneer Carl June and Terence Flotte, president of the American Society of Gene and Cell Therapy.
Health authorities in recent weeks have publicly expressed support for more regulatory flexibility for cell and gene therapies—an encouraging signal for developers in the space.
In April, for instance, Makary said on the Megyn Kelly Show that the FDA is open to considering what he called “plausible mechanism” as a basis for approval, particularly for therapies meant to treat diseases affecting “a small number of people.” Such an approach, he explained at the time, would look at whether a drug candidate “makes sense physiologically”—but would only grant approval “on a conditional basis.”
Prasad has backed his boss up, saying on Tuesday at an event organized by the National Organization for Rare Disorders that his office “will take action at the first sign of promise for rare diseases. We’re not going to wait.”
Further spurring momentum in the cell and gene therapy/rare disease space, Sarepta Therapeutics announced earlier this week that it was granted the FDA’s first-ever publicly recorded Platform Technology Designation for its viral vector rAAVrh74, which the biotech uses for its gene therapies. The designation, which was conceived during the Biden administration, is designed to promote predictability for drug applications and streamline the review process, ultimately easing the development burden for drugmakers.
