Looking Back and Ahead, Gottlieb Discusses FDA
Outgoing US Food and Drug Administration (FDA) Commissioner Scott Gottlieb continued his farewell tour of interviews on Friday with a detailed discussion with former FDA Commissioner Mark McClellan at the Pew Charitable Trusts.The wide-ranging conversation, thanks to the Alliance for a Stronger FDA, dovetailed from jokes (Gottlieb said on his resignation at one point: “Is it too late to change my mind?”) to more serious topics like opioids, the regulation of cell and gene therapies, leveraging electronic health record data and whether FDA should allow drug importation to bring down costs.
As for what Gottlieb thinks might be the most enduring change made during his tenure, he pointed to the Center for Drug Evaluation and Research’s evolving application process, which is moving away from a paper-based system to a collaborative, cloud-based document where data submitted can be pulled together and structured in a way that makes different reviews look similar.
He also explained how FDA is updating its guidance on clinical evidence and will look further into how to use real-world evidence and real-world data to evaluate a treatment’s safety and effectiveness. The agency has substantial new budget authority to help build out better access to electronic health record (EHR) data for the purposes of decision making, Gottlieb said. Last July, FDA finalized guidance on using EHR data in FDA-regulated clinical investigations.
“The widespread use of EHR data is coming,” he said, noting it will supplement FDA’s use of administrative claims data with its Sentinel system.
As cell-based regenerative medicine becomes more mainstream, Gottlieb noted that such products are changing the contours of the agency’s mission. And the agency will have to formulate new ways to regulate these new technologies and may have to seek new authorities to regulate them.
But on the topic of using drug importation to try to lower pharmaceutical expenses in the US, Gottlieb was less optimistic. He said he does not think any widespread importation would be feasible under the current system and it’s hard to see anything more than limited importation to ensure safety, citing the agency’s allowance of imports for products in shortage when Puerto Rico was hit by Hurricane Maria.
In January, Sens. Chuck Grassley (R-IA) and presidential hopeful Amy Klobuchar (D-MN) introduced a bill, dubbed the Safe and Affordable Drugs from Canada Act of 2019, that would allow for Americans to import drugs from Canada.
Also on drug pricing, which Gottlieb said he’ll have “more to say in seven days” on when he leaves FDA, the agency is publishing data soon that will show how companies bringing a second novel product to market after the originator are taking longer, and if the market is too small, some companies are slowing or pulling out entirely.
“We hope that product competition means price competition, but sometimes that doesn’t happen,” he said.
Gottlieb also explained how FDA’s lawyers are still looking into whether biosimilar companies might be able to access reference product from Europe for certain pre-market studies, although the issue hinges on whether that change might raise problems with commercially confidential information.
And as for the change he’s most proud of, in terms of the agency’s relationship with a regulatory challenge, Gottlieb singled out FDA’s work with opioids. He said the agency’s view has changed about its obligations and has taken a more affirmative role to reduce the rate of new addiction and to ensure prescriptions written are appropriate. However, he said the challenge now is that the nature of the crisis is changing dramatically as addiction is beginning more with recreational opioid use.