Looking ahead to COVID-19 strategies, Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research (CBER), said vaccine studies might be done not in clinics but in the streets. “By definition, we’re probably not going to be able to vaccinate everyone simultaneously,” he noted. “If you had a situation where you do still have virus circulating, one could imagine that, as vaccine runs off the fill-and-finish line, you might be able to set up a trial whereby essentially what you’re doing is using real-world evidence,” studying groups vaccinated at different times and comparing them to each other.

Marks’ comments came during a webinar, hosted by the Commonwealth Fund’s Alliance for Health Policy, called “Coronavirus Science, Technology, and Policy: Vaccine Development.” Not-vaccinated people, he said, would function as controls under the real-world testing setup. “It’s not perfect,” he said. “I will acknowledge right away that there are certain people at the NIH who, I’m sure, would like to tell me that’s a horrible design.” Officials must be willing to “entertain and discuss in an open fashion” any possibility and not dismiss any out of hand, he said.

“There’s no set number” in terms of efficacy that will be demanded of a vaccine, Marks said, but the target bullseye is herd immunity. “If we don’t get there, well, we would probably take the next best thing. We can’t have perfection be the enemy of good,” though researchers need to adopt “a smart development approach right up front.” He modified a woodworking slogan to recommend that developers “measure quickly twice, and cut quickly once. Right now, there have not been mutations to my knowledge that have occurred that would fundamentally alter vaccine development programs in terms of the targets they’re going after,” he added, though scientists need to stay vigilant about this, too.

Litjen Tan, chief strategy officer of the Immunization Action Coalition (IAC), said regulators must maintain “very stringent and absolute outcome measurements in terms of safety and efficacy, even though the process is being accelerated. We’re going to be seeing some compromise in terms of things like, for example, wanting long-term data,” he said.

Asked about an emergency use authorization (EUA) as suggested by National Institute for Allergy and Infectious Diseases Director Anthony Fauci, Marks said such a move would “depend on the strength of the evidence [for efficacy] that we saw,” and how much virus is circulating at the time. If COVID-19 is still rampant, and clinical data plus reliable findings in animal models were available, decision-makers would “possibly” consider an EUA, he said. “If you didn’t have circulating virus but you’re just trying to get people vaccinated, I don’t think you’d see that.”

On the crystal-ball question of when a vaccine might be available, Marks said “there is what is likely and what is desired. What is desired, if I had a magic wand right now, is that everything would go perfectly and somehow, nine months to a year from now, we would have [a] vaccine that might not be perfectly licensable, but at least” could be given to people under an EUA. “If we look at what is most likely [for] timelines – this is total speculation – we’re probably looking at 12 to 18 months,” he said. The IAC’s Tan estimated between 15 and 18 months. Esther Krofah, director of Faster Cures, pointed out that vaccine makers including Johnson & Johnson, Glaxosmithkline plc, and Sanofi SA are making “a big bet on [roughly] that time frame.”

With 77 COVID-19 vaccines in development and 207 would-be therapies, the FDA is gearing up – and hoping – for what could be an avalanche of submissions. “We’re reassigning bodies” to process BLAs, Marks said, “since some other work has quieted down a little bit. We have plans to staff up some. We’re still actually hiring through this, believe it or not. I’ve had video interviews with people,” as others at CBER have. Adding personnel “should become easier as things loosen a little” in terms of social distancing and shelter-in-place rules, he said.

Meanwhile, “ideating” is the right thing to do, Marks said. “You never know when there could be some diamond in the rough, or some old technology for that matter that comes up and saves the day,” and funders will be watching for the better players to back. “I don’t think we’re going to say, ‘Stop developing’ something,” he said. “Ultimately, the free market will take care of this.”

Coding and reimbursement issues remain to settle. “When these vaccines get approved, who’s going to be paying for them?” Tan asked. “Are we going to be relying entirely on the public sector, which is kind of what we did with H1N1? Or are we going to be more likely relying on private and public-sector payment to happen?” The major vaccine concerns, in any case, are practiced at getting codes in place. Krofah said “we’ve seen with the Centers for Medicare & Medicaid Services [CMS] and their respective counterpart agencies” a willingness to move with dispatch when necessary. “I’m hopeful that they would very quickly be open to these kinds of conversations,” even if the coverage and analysis group at CMS is “quite small. I don’t have a sense to what extent those conversations have started,” she said.

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