A Reflective Gottlieb Touts Drug Review Reform
FDA commissioner Scott Gottlieb believes that the most “enduring” accomplishment at the agency during his almost two-year stint is CDER’s ongoing new drug approval process reform and changing the nature of the application process to move away from a paper-based submissions. In a 3/29 sit-down chat with former FDA commissioner Mark McClellan (co-hosted by the Alliance for a Stronger FDA and Pew Charitable Trusts), short-timer Gottlieb (his last day at the agency is 4/5) highlighted drug review changes that will eliminate reviewers working individually to file “consultant reports” to the review process, and instead they will engage a “collaborative document” that is cloud-based. He said that instead of the agency taking information/data from tables submitted by companies, FDA will have access to the raw data to formulate it in “reproducible tables in a much more structured approach so that each review looks the same.” Those working to formulate these tables are experts in data analysis and not medical reviewers, he said.
Asked to elaborate on the optimism surrounding real-world evidence, Gottlieb said FDA is currently updating agency guidance on “how we define clinical evidence of effectiveness, and we are going to incorporate constructs of how we can use real-world data and real-world evidence as the basis for determining effectiveness.” He said the agency has been using those data constructs to look at drug safety issues, and the agency has received substantial resources in the budget over the past two years to help it “build out better access” to electronic health records (EHR) data for purposes of using real-world evidence and real-world data for making effectiveness determinations for medical devices and drugs.
“This is the first time we will have widespread access to EHR data in the same way we have access to [insurance] claims data in the Sentinel system,” Gottlieb said. “So it is a profound change in the agency’s ability to access data for purposes of regulatory decision making.” On the real-world evidence side, Gottlieb said FDA is using new resources to “go back and see if we can replicate clinical studies just using real-world evidence so we can determine how predictive it is. So we are doing a number of studies to look at that and to develop a paradigm for how predictive real-world evidence is and how confident we can be in conclusions drawn from it for purposes of determinations of effectiveness.”
On drug importation as a means to curb pharmaceutical prices for consumers, Gottlieb said he still maintains that any kind of drug reimportation is not feasible in the current system given increases in counterfeit products coming into the country. “It is hard to conceive of a system where you have anything more than very limited importations in very exceptional circumstances where you can provide anything close to the assurance of safety that we have with our closed supply chain,” he said. The agency, however, will consider limited importation in settings of drug shortages, such as when two hurricanes wiped out manufacturing in Puerto Rico in 2017, he said.
As for his next role when he leaves the agency next month, Gottlieb told McClellan that he has no plans other than to immediately take his wife and three young daughters to Disney World.