Gottlieb, who declined to comment on the nomination, is unlikely to up-end the FDA in the way O’Neill might have, but he is nonetheless expected to bring significant change, including moving the agency to increase flexibility in the clinical trial development process.
In this, he will be supported by the recently passed 21st Century Cures Act, which instructs the FDA among other things to consider the use of “real world evidence” to support new drug applications. This could include anecdotal data, observational studies and patient reports
“People don’t want to take chances with safety, but there’s increasingly some clamor to be more flexible on the efficacy side,” says Kathleen Sanzo, who leads the FDA practice at the law firm Morgan, Lewis & Bockius. “You need to have some signal of efficacy. The question is, how much?”
The FDA has attempted to push back against moves to sideline randomized clinical trials, long considered the gold standard. In January, it issued a report documenting 22 cases in which drugs that appeared to show promise in early trials turned out to be either ineffective or unsafe or both in larger trials.
One of Gottlieb’s priorities will likely be to streamline the process for approving generic versions of complex, difficult-to-copy therapeutics. He has stated publicly that he does not believe the FDA has good tools or policies to move such products and has advocated the creation of different approval standards.
“He’s a thoughtful and nuanced kind of guy, and not solely an industry shill,” says Jim Shehan, head of Lowenstein Sandler’s FDA regulatory practice.
A survey conducted by Mizuho Securities USA Inc of 53 pharmaceutical executives found that 72% favored Gottlieb over other potential candidates. Many described him as knowledgeable, experienced and balanced.
“Gottlieb is someone who the industry and investors view as an incremental positive,” says RBC Capital Markets analyst Michael Yee. “The industry and investors need rational scientific logic and an understanding of risks and benefits.”
Patient advocates welcomed the news.
Gottlieb “has firsthand experience at the FDA and as a physician that has treated patients understands the breadth of work that needs to be achieved on their behalf,” says Ellen Sigal, founder of Friends of Cancer Research.