Gottlieb: There Couldn't Have Been a Better Time to Be at the FDA

When Scott Gottlieb, MD, joined the FDA as the 23rd commissioner, it was on the tailwind of new resources and authorities being implemented and new rules needed to be written for innovations coming to market. He knew even then, and he still believes, that there couldn’t have been a better time to be at the FDA.

During his keynote speech at Asembia’s 15th annual Specialty Pharmacy Summit, held April 30 to May 2 in Las Vegas, Nevada, Gottlieb reflected on governing principles he learned during his time as FDA commissioner, highlighted secular trends for which FDA wrote modern rules, and outlined ongoing reimbursement challenges. He even discussed the likelihood of some form of Medicare for All passing.

At the time he joined FDA, at the beginning of 2017, the landscape had a lot of novel technology with things like digital health tools, gene therapy, and targeted medicine.

“When I came in, I knew that we were going to be there over a period of time when we wrote the modern rules and frameworks for how those new technologies would advance and whether or not we would realize opportunities of the applications growing out of these new paradigms,” he said.

From his time at the agency, he highlighted a number of efforts, such as navigating the crisis on Puerto Rico after Hurricane Maria brought manufacturing capabilities offline and the long government shutdown at the end of 2018 into the beginning of 2019. He also discussed his work on e-cigarettes and vaping, noting how quickly the agency implemented new policy.

Gottlieb explained that while the agency and public health groups were getting anecdotes from teachers about youth use of e-cigarettes, it wasn’t until August 30, 2018, when FDA got the results of a survey and saw the dramatic rise in youth use. Just over a week later, he was out giving a speech that FDA would have to regulate some of these products, particularly the flavored products, and by November the agency had outlined its policy. March 2019 it was out in draft form and the policy is now being implemented.

“That really was a record time for the implementation a policy to address public health crisis, but it’s still too slow to effect that public health crisis,” Gottlieb said. “The trends are still underway; I suspect youth use of e-cigarette is still going to continue to go up. The lesson there is you’re constantly making policy in an imperfect data environment and…if there’s a burgeoning crisis it’s going to be hard to address in time frame that can actually get in front of it.”

During his tenure at FDA, reimbursement became an even bigger issue with costly life-saving drugs, like chimeric antigen receptor (CAR) T-cell therapies coming to market. Gottlieb believes the country is at a crossroads, trying to structure reimbursement that is “capable of compensating for all the downstream costs being pulled forward.” Drug payments used to, effectively, be an annuity, with costs being paid out over time as the disease was treated, but the new paradigm is that of a one-time administration to cure a disease, which requires recouping the investment into creating that treatment upfront.

Medicare tends to struggle with new payment models, which has been part of the problem with CAR T-cell therapy. Gottlieb said that the agency is trying to work through a payment structure for the therapy, but there is a structural challenge. CMS is currently trying to figure out what a Diagnosis Related Group would be for the inpatient administration for CAR T-cell treatment.

“I worry that as we start to contemplate the reimbursement for gene therapy and CAR T, if we don’t get this right, we can see…early investment pull out of these areas,” Gottlieb said. “And in fact, if you look at the early pipeline for CAR T, you’re seeing less early investment…”

There seems to also be fewer people trying to bring CAR T-cell therapy to solid tumors, he added, While there are some scientific reasons for that, he thinks there might also be some trepidation of making investment bets in products with a lot of uncertainty about how they’re going to be paid for.

Finally, Gottlieb, touched upon Medicare for All, which is gaining more interest and attention as the Democratic presidential candidates push legislation on it. The day before his keynote, advocates for Medicare for All testified at a hearing before Congress. Gottlieb worries that extending the government reimbursement model into the larger marketplace might create headwinds for new technologies. However, he doesn’t think it is impossible for a Medicare for All bill to pass.

Most of the bills that have been proposed have a lot of policy in them that would fundamentally alter the insurance model and would require 60 votes to pass the Senate. Instead, if they decide to just change benefit design to expand Medicare to a broader population and eliminate private coverage that can be done through budget reconciliation, which only needs 50 votes to pass.

If a bill simply wants to extend Medicare to 40 year olds, 30 year olds, or even 20 year olds, then Medicare will have regulatory latitude to make determinations for that population, he explained. That language doesn’t need to be in the legislation, and if it is, it won’t get through budget reconciliation.

“The challenges we face in the current model, that’s fueling, some of it, consumer desire for what’s perceived as simplicity of a Medicare for All model,” Gottlieb said, although he added that is a perception he doesn’t necessarily agree with.

“I think the future’s really bright,” he said. “I saw, when I was at FDA, unlimited opportunity for what we were capable of doing.”