Former FDA Commissioner, Scott Gottlieb, MD, addressed attendees of the Asembia 2019 Specialty Pharmacy Summit as keynote speaker where he shared the core principals by which he governed in the FDA, offered insight into open projects and initiatives, as well as spoke about what the agency should focus on going forward.
“I was fortunate when I came into the agency, I had the tailwind of new resources and new authorities that have just been implemented… a lot of new authorities trying to accelerate the development of breakthrough medical technologies,” said Dr Gottlieb, who has been praised for his focus on the patients and consumers.
“The landscape we were looking at was a lot of novel technology, a lot of opportunity for gene therapy, regenerative medicine, targeted medicine, and digital health tools,” reflected Dr Gottlieb. “So when I came in I knew that we were going to be there for a period of time when we wrote the modern rules and frameworks for how these new technologies would advance, and whether or not we would discover new opportunities for patients with these paradigms.”
Reflecting on his time spent at the FDA, Dr Gottlieb mentioned a number of core values that he embodied, including his firm belief that no one can run out of political capitol. He recalled when someone would say, “I don’t want to go to bat for that policy or fight for that provision” because that would require using and calling in favors, and stated that there is no such thing as spending political capitol.
“I think you replenish your capitol and build your stature, deterring people from trying to tread on your authority and take your ground by taking on fights and winning,” Dr Gottlieb continued. “FDA is an agency of immense authority. We make decisions where there are finite winners and losers and there are always other agencies that want to have pieces of our authority…we have to preserve the framework of that authority.”
Other core principles Dr Gottlieb said guided him during his time as commissioner included owning your problems in a very public way—in the media and in your position, stressing that the media will come after you if it believes there is a story you are not telling.
This is why Dr Gottlieb came out so aggressively on a number of issues. He testified 19 times before Congress and would only miss if he or his team believed someone else was a stronger choice.
“You saw us lean really hard into the data on the danger of e-cigarette products. I received that data on August 30 of 2018 and within 10 days was out giving a speech that we now know that there is an epidemic in the use of these vaping products—and we used the word epidemic.”
Dr Gottlieb emphasized the importance of focusing on the first step to solve a problem and take a tactical view. He also stressed the significance of maintaining a consistent theme in your values and goals and ensure that are well-articulated.
Among the most significant developments that came about during Dr Gottlieb’s time as commissioner include laying the architecture for new technological frames and paradigms related to health care, increasing research in gene therapy and regenerative medicine, and the innovation of countless digital tools implemented by those in the health care sector.
“As the agency builds its capabilities, it’s going to build its confidence in these data paradigms and open up more chances to move forward with these new kinds of developments and approaches. There is nothing more powerful for the FDA than changing its culture and enabling opportunities.”
Dr Gottlieb explained that the FDA budget increased by $1 billion in two years, funding projects in building databases for electronic health records, and artificial intelligence research for gene sequencing, among others.
“The world we live in today is very different from the world that was envisioned many years ago,” said Dr Gottlieb, as he noted that FDA has the opportunity to create projects for change and provide resources for innovation.
He also spoke on reimbursement challenges for costly treatments, how payment models need to be adjusted but how that will be complicated for treatments like gene therapy. “Once you get a gene therapy to market and treat everyone that has that disease, the small market of people left that are diagnosed with the disease isn’t large enough to drive a second product to market,” which Dr Gottlieb explained limits competition and drive to lower prices. Understanding how to create payment models and structures for new kinds of technology and medicine is something the FDA will continue to work toward.
“Ten years ago, if I said we’re going to cure some of the most vexing pediatric inherited disorders, that would have been highly irresponsible but now, speaking about cures isn’t that far off,” said Dr Gottlieb. “We have the technology here to enable that.”
Lastly, Dr Gottlieb commented on the concept of Medicare for All, stating that it is “much more possible than conventional wisdom would ascribe to believe.” He said because it is such a public issue, legislation to change anything will be slow, however, there are ways to make a difference without writing pages and pages of policy.
“This is a wonderful time to be working in this enterprise. I think the kinds of opportunities that we’re going to see in a years to come are going to be deeply profound,” said Dr Gottlieb. “I hope that the frameworks that we have set in motion at the FDA, for trials, development, and approval is going to make these more available to patients.”—Edan Stanley