Digital clinical trials and technologies — as well as an unprecedented collaboration among industry, government and academia — were some of the most critical components to the rapid development of the Pfizer-BioNTech COVID-19 vaccine, health leaders said in the leadup to the vaccine’s long-awaited emergency use authorization.
The Food and Drug Administration’s vaccine advisory committee Thursday granted the authorization of the vaccine for individuals 16 years and older. The move follows FDA's published documents indicating that the vaccine, an mRNA-based candidate, provides effective protection against COVID-19. Seventeen members of the committee voted in favor, while four voted against it, and one abstained a vote.
The approval follows Pfizer’s announcement last month that its two-dose vaccine is 95% effective when each dose is given three weeks apart. The United Kingdom and Canada have also approved the vaccine for emergency use authorization.
As the U.S. marked a new step toward combating the virus with the FDA’s approval, Pfizer CEO Albert Bourla noted that the quick development of his company’s vaccine was largely able to happen because of technological advancements in medical research and development, including a modified RNA platform, data sharing and digital clinical trials, as well as the unprecedented collaboration to combat the pandemic.
“The technology that we used were multiple to be able to make this happen, and I think the basic platform is the modified RNA technology,” Bourla said during the Milken Institute Future of Health Summit this week. “This is by itself cutting edge, but in order to make from this technology [a] product, we needed to use technologies in sequencing, technologies in being able to replicate RNAs, being able to build DNA templates, being able to scale up manufacturing, and also digital technologies were used.”
The digital clinical trials, Bourla added, were particularly effective in making their vaccine candidate trials more efficient.
“The only reason why we were able, in Pfizer, for example, to execute their 44,000-people study that typically takes years to execute in a condensed period of time, this monumental, this requires [Pfizer] to connect the gigantic load of information,” Bourla said. “We were only able to do it because we had invested in the last few years in this technology to digitize our clinical studies, and a lot of the things that we had just implemented at Pfizer were used."
The FDA has been looking to pedestal data to improve its processes and biomedical research and development, showing how the effort has benefited across the public-private partnership that led to quick vaccine development. FDA CIO Amy Abernethy also spoke at the Milken Institute Summit on how real-world data applications and insight have especially been critical amid COVID-19.
“One of the things that we did was acknowledge that moving data around and trying to gather it all up and put it in one place is really difficult, and it’s difficult to work with datasets that you don’t know, and you’re better off really having people who know their data well to be able to work with it and answer questions," she said. "We at FDA really worked together with our public-private partnership at the Reagan-Udall Foundation to advance a community of real-world data scientists to try and start to figure out how do we bring real-world data to bear in the pandemic."
The partnership led to data-driven insights about COVID-19 beyond information gained from clinical trials, she added.
Technology will continue to be critical to the oversight of the vaccine authorization. During the FDA's Vaccines and Related Biological Products Advisory Committee meeting Thursday, FDA Medical Officer Dr. Susan Wollersheim presented the digital adverse event reporting protocol that will track adverse effects and complications from the vaccine upon widespread vaccinations in the U.S.
More specifically, the reporting will occur through the Department of Health and Human Services' Vaccine Adverse Event Reporting System (VAERS), an online portal that will enable vaccine recipients, vaccination providers and vaccine authorization sponsors to report and analyze such data. The system will enable coordinated data sharing between the FDA and the Centers for Disease Control and Prevention.
The reporting system for this vaccine, and for future COVID-19 vaccines, will help inform data abstraction, screening for all incoming serious adverse events, literature review and data mining.
Pfizer and the FDA intend to continue with a longer-term drug safety plan to identify any potential risks associated with the vaccines, missing information in pregnant women and those who are younger than 16 years old. The FDA is also requiring Pfizer to analyze anaphylactic reactions and risks amid recent cases of severe allergic reactions to the vaccine reported in the United Kingdom.
Pfizer has also proposed, with the support of the FDA, to use the COVID-19 HERO registry that surveys 20,000 U.S. health care workers, as well as electronic health records from HHS, the Defense Department and the Veterans Health Administration to continue surveillance studies.
During the public session Thursday, other representatives across the medical field expressed the need to provide more trial data for racial and ethnic minority groups, individuals with conditions like HIV/AIDS, children, individuals in long-term care facilities and individuals who have already tested positive for COVID-19.
As the FDA and Pfizer continue to collect data, the authorization of the vaccine marks a start to the marathon of vaccinations for COVID-19. Pfizer will produce approximately 50 million doses in December and aims to ramp up production to 1.3 billion total doses in 2021, Bourla said. He stressed the importance for other companies to innovate and produce vaccines to help the rest of the world overcome the virus.
Although the authorization will allow the U.S. to start distributing the vaccine, authorization can be revoked at any time if there are widespread complications. Pfizer said it aims to receive biologic license application approval in 2021.
Officials say the creation of this vaccine, as well as the development of other vaccines from companies like Moderna and Johnson & Johnson, was successful because of not only the technological advancements behind the effort, but also because of the collaboration that has occurred across pharmaceutical companies with academia and government agencies — domestically and internationally.
“It’s extremely important that you try to have a global mindset when it comes to this pandemic,” Bourla said. “I think we are going to be much better prepared next time because I think we will learn a lot of lessons, and those lessons are at different levels, different categories. Clearly, I think one of the very powerful lessons is the power of science and how that can play a role.”
Pfizer was a partner in the National Institutes of Health’s "Accelerating COVID-19 Therapeutic Interventions and Vaccines (ACTIV)" public-private partnership to accelerate research and development efforts for COVID-19 vaccines and therapies.
NIH Director Dr. Francis Collins highlighted at the summit the importance of these partnerships both throughout the pandemic and beyond. He highlighted how ACTIV — as well as the Rapid Acceleration of Diagnostics (RADx), an initiative to improve COVID-19 testing — and backing from the 21st Century Cures Act have shown the potential of the U.S.'s ability to collaborate toward innovative solutions to public health crises.