If Peter Marks had a magic wand to perfect the sophisticated process that takes a new vaccine from idea to reality, the US could have a way to prevent some people from COVID-19 infection in nine months to one year.
In the real world, the journey to a vaccine OK’d under emergency guidelines is more likely to take 12 to 18 months, said Marks, who runs the FDA’s Center for Biologics Evaluation and Research, on a Friday teleconference hosted by the Commonwealth Fund and the Alliance for Health Policy Teleconference for Media and Policymakers.
Marks said factors that will play a role in FDA’s issuance of an emergency use authorization—not an approval—will include the level of coronavirus circulating, how well the vaccine works, and how safe it appears. Some of the data to back up safety and efficacy claims are likely to come from less traditional sources, such as adaptive trials, or those designed to allow changes along the way in response to early results, and real-world evidence.
Facilitating the development of a vaccine for COVID-19 is an agency priority now and for the foreseeable future since the US will likely “see a second wave or even third wave [of infections] if we don’t get it right and get it right quickly,” he said.
The agency aims to ensure vaccine makers move forward the candidates with the most potential, he said.
“What that will really mean from the FDA perspective is working with the various sponsors of the vaccines to most efficiently look at their preclinical data, understand as much as we can about the characteristics of these vaccines, (and) help people develop plans that are most efficient at understanding whether their candidates will get to the level of protection that would really warrant carrying them forward, because, frankly, vaccine candidates that have very low efficacy or not tremendous efficacy, in this particular case, could just distract from capacity for more robust candidates to come forward,” he said.
Marks described the careful calculus of ensuring that any vaccine distributed is safe and effective while removing roadblocks to the fastest possible authorization and distribution.
“We may not have the long-term data that we normally might like, so we may be putting real-world evidence into practice in this case by vaccinating relatively larger populations of individuals—it may be individuals at higher risk for things like this—then gathering data in the real world,” he said.
Choosing which candidates to advance is an especially necessary and high-stakes effort in the face of a pandemic, Marks said.
“My unique perspective on this is that it’s time to really mobilize as best we can in our animal modeling to try and compare things, to the extent that we can, and try and sort things out before we get to humans, knowing it’s never perfect to make that transition … There is an opportunity cost to taking any candidate forward, and so you want to take your best candidates forward because, by definition, they will occupy capacity over one another,” he said.
Esther Krofah, who heads the Milken Institute’s FasterCures center, said drug companies appear to be operating with a similar timeline in mind, given some recent announcements about plans to ramp up manufacturing of their vaccine candidates in the event they prove safe and efficacious.
In recent weeks Johnson & Johnson (NYSE: JNJ) announced it had selected a lead vaccine candidate to advance against the novel coronavirus, and committed to boosting its manufacturing capacity to be able to make more than one billion doses of such a vaccine. The company anticipates human tests of its candidate to start “at the latest” by September, and said it expects batches could be ready for use under emergency guidelines in early 2021.
That’s a “substantially accelerated timeframe in comparison to the typical vaccine development process,” the company noted. Krofah says Milken is tracking 86 active vaccine projects, including six clinical trials; two dozen more are slated to enter the clinic in summer or early fall.
LJ Tan, chief strategy officer at the Immunization Action Coalition, emphasized the speed at which COVID-19 vaccine development is occurring compared to typical timelines for new vaccine innovation.
Tan previously spent about 16 years in leadership roles at the American Medical Association, most recently as director of medicine and public health and as director of infectious disease, immunology, and molecular medicine for more than a decade.
“Traditional vaccine development, through all its phases, can take 10, 15, sometimes 20 years, and can cost at least $800 million dollars or even more,” Tan said. “When you build in all the costs to build the manufacturing facility, the equipment, the quality control that goes into this, the figure can go way over $1 billion to bring a vaccine from development all the way through to market.”
Moderna (NASDAQ: MRNA), whose vaccine candidate was the first to start human testing in the US, said Thursday that it anticipated the start of a Phase 2 study this quarter, if safety data from the National Institutes of Health-led Phase 1 trial supports continuation. The Cambridge, MA-based biotech also announced that the US Biomedical Advanced Research and Development Authority (BARDA) had agreed to award it up to $483 million to fund the vaccine’s development through FDA review and ramp up its manufacturing to allow it to be produced at scale this year.
The company said it plans to add as many as 150 new employees by 2021.
Some vaccine candidates are being advanced in multiple geographies. This week Inovio Pharmaceuticals (NASDAQ: INO) announced a $6.9 million award from the Coalition for Epidemic Preparedness to move its vaccine into a Phase 1/2 trial in South Korea. Working with International Vaccine Institute and the Korea National Institute of Health, the trial will parallel Inovio’s ongoing Phase 1 in the US, which started April 6.
“Remember, ultimately, we’re not talking about just vaccinating a few hundred thousand individuals, we’re taking about vaccinating a few hundred million people in this country alone and a few billion people globally, so one really would like to try to get it right to the extent they can, obviously working at a very rapid pace,” Marks said.