Makary Pushes for Reductions in Regulatory Barriers
FDA commissioner Marty Makary has promised that the agency will listen to stakeholder ideas on reducing regulatory barriers and evolving its processes by “challenging deeply held assumptions on how we do things if it means we can deliver more cures and meaningful treatments, safe and effective to the American people.” Speaking at an FDA June 5 cell and gene therapy roundtable, Makary told the audience that the agency has to create processes that are not in a “receive-only mode,” but it must also partner with developers and inventors so that “we can reduce barriers and cut waste and get it right to what’s meaningful to the public.
“The FDA should have a strong partnership with inventors and scientists and developers and companies,” Makary continued, “and at the same time, preserve scientific independence in the product review process.” Regarding the rare disease community and cutting edge treatments (stem cell therapy, cell and gene therapy), Makary pledged that the agency will “continue the successes of the FDA in facilitating the regulatory process for these conditions and for these products. We are also going to try and improve by creating more efficiencies. We are challenging deeply-held assumptions in the traditional way that we do things. We are taking a look at the application process. We’re asking ourselves, ‘can certain materials be submitted in advance so that approvals can be quicker’? We are asking ourselves, ‘Can we look at products in a way that a patient or the parent of a child looks at a product?’ And can we use post approval monitoring and the assurance of post approval monitoring in the actual decision making process when we decide whether or not to approve a product.’”
Makary also said FDA will continue to build on the successes of priority reviews and accelerated approval pathways, and expanded access.
Roundtable moderator and CBER director Vinay Prasad reiterated some of his guiding principles for advancing and approving promising therapies discussed earlier this week at a rare disease meeting (see earlier story). “Of course, at FDA, we want what transformational therapies and cures and major advances, but we are also willing, and of course, will strongly consider any step forward in these conditions. We understand that progress is not always made in a single leap, and we will consider incremental steps forward, because those add up. … We will rely on gold standard science and common sense.”
During a closing panel session, HHS secretary Robert F. Kennedy Jr. asked the audience to provide a list of regulations that should be withdrawn to help speed innovations and approvals. “President Trump has given us an executive order that says for every regulation that we put in place, we have to get rid of 10,” the secretary said. “So we would solicit, right now, a list from all of you of any regulations you think we ought to be getting rid of.”
Kennedy also took the opportunity to defend cuts occurring at FDA. “There’s a lot of people who criticize us, 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 to spawn and foster and fortify innovation and unleash it.”