On October 24, 2018, as part of the Milken Institute Future of Health Summit, Scott Gottlieb gave a detailed interview on his vision for the FDA, interviewed by Tanisha Carino, executive director of Faster Cures.
See the Milken agenda here. See a detailed transcript and links to the Youtube video interview here.
Gottlieb, a physician with a background ranging from federal policy to investing, and a cancer survivor himself, dicussed keeping FDA focused on a "values oriented strategy" and one that incorporates patient perspectives.
He noted that FDA will soon release new guidance on incorporating digital apps and monitors into drug approvals. (See also an open access article at ISPOR, here.) He noted that digital tools could also have post-market or post-regulatory roles. (For a November 2018 trade journal article on pharma investments in digital, here.)
He discussed the new for new regulatory tools in areas like neurologic diseases, and improving cognitive tools in Alzheimer's.
He noted that FDA was restructuring the drug pipeline and wants to move things like filling spreadsheets away from expert medical reviewers and let them focus on high-impact medical decision making during the review process. He also noted that the FDA needs much better knowledge management systems in house. This is the classic dilemma in organizations, "How do we know what we know?" The agency is also committed to more objective public disease-specific guidances (less need to rely on tacit experience or to reinvent the wheel.) The agency will create "problem based review memos based around "critical questions."
He discussed the wave of pending retirements and that the FDA is a highly technical working agency, not just one that assigns and passes through money.
He referred to the agency's commitment to areas of self-regulation or self-certification (e.g. in some areas of digital health) and to the 70 pages of comment FDA recently submitted to Congress proposing new legislation for regulating LDTs.
There was a discussion, pivoting off of a Harvard economist Amitabh Chandra presentation, the R&D in pharma wasn't well aligned to major public health needs (e.g. too directed to orphan diseases rather than pneumonia and heart disease). Some disease areas are piled high with "good-enough generics" that are a barrier to new research.
He noted, as he has in several recent speeches, that sometimes we don't get reimbursement right, and that is a barrier to progress as well. He raised the concern that some areas may get stuck with "natural monopolies" of one or two drugs and it's too hard for more diverse new entrants to get a foothold.