Analysts at Jefferies see Makary as a positive for the rare disease space, given his support for accelerated approvals and openness to “customizing regulatory pathways for rare diseases.”
FDA Commissioner nominee Marty Makary played it safe in front of the Senate’s Health, Education, Labor and Pensions Committee on Thursday, distancing himself from recent high-profile disruptions at the agency, while also reassuring lawmakers that, if approved, he would continue the FDA’s tradition of following good science.
A key focus of the hearing was the cancelled meeting of the FDA’s vaccines advisors about the upcoming flu vaccination season.
According to reporting from Healio, HELP Chairman Bill Cassidy (R-LA) flagged the lack of transparency with the cancellation, pointing out that the move seemed to go against the promises made by Robert F. Kennedy Jr—recently confirmed as Secretary of Health and Human Services—of boosting transparency in the health department.
Makary asserted that he wasn’t involved in the canceled meeting and reassured senators that, if approved, the FDA’s Vaccines and Related Biological Products Advisory Committee would continue to meet. Makary was non-committal about the canceled flu meeting in particular, refusing to promise that he would reschedule it.
“The VRBPAC committee will be meeting if confirmed and I’m commissioner, yes,” Makary said during the hearing, as reported by CNN, however qualifying that he “would re-evaluate which topics deserve a convening of the advisory committee members of VRBPAC and which may not require a convening.”
Makary similarly distanced himself from the recent firings at the FDA, saying that he hadn’t been involved in those decisions. If approved, “you have my commitment that I will do an assessment of the staffing and personnel at the agency,” he continued, according to NPR. Prior to the hearing, the FDA had laid off around one thousand of its employees, of which it now plans to ask some 300 to return.
Makary also said that he would root out conflicts of interest at the FDA’s advisory committees, “because people see things that appear to be a cozy relationship between industry and the regulators that are supposed to be regulating the products,” as per the CNN report.
Meanwhile, analysts at Jefferies pointed out in a Thursday investor note that if confirmed, Makary would be a “clear” positive for the broader disease space. During his hearing, the prospective FDA head insisted that he wanted the life sciences industry to “thrive,” and also expressed support for expedited reviews, particularly for generics, biosimilars and rare disease therapies.
“Dr. Makary clearly voices his support of streamlining rare disease drug development,” the analysts wrote, noting that in this space specifically, Makary “hopes to customize the regulatory process to the condition.”
