In March 2020, the COVID-19 pandemic began to shut down normal day-to-day activities in the U.S. No industry was spared, including drug discovery. During her keynote address at the 2020 Veeva R&D Summit, Amy Abernethy, principal deputy commissioner & acting CIO for the FDA, discussed how the agency responded.
“There was so much we didn’t know,” says Abernethy. “We were talking about clinical trials being done in China and lessons that could be learned from other countries. We were observing what was happening in New York City and in Italy, but the real question was how do we make sense of it all and how can we learn as quickly as possible from the data around us.”
Understanding Real-World Data
The FDA wanted to explore how it could use real-world data collected out of EHRs, claims data, patient sensors, and social media to understand what it did and did not know about the pandemic. That is when the agency announced its participation in the COVID-19 Diagnostics Evidence Accelerator. The Accelerator is a multi-stakeholder collaborative project to advance the development of diagnostics. It was organized by the Reagan-Udall Foundation for the FDA, in collaboration with Friends of Cancer Research.
“The Accelerator started as an idea of how we create a place where we can quickly gather the totality of the information and make sense of it,” says Abernethy. “We realized that in order to use all of the information available, we are going to have to bring everybody to the table and give them the tools to perform high quality work as quickly and as confidently as possible.”
The FDA is an active participant in the Accelerator but can also provide technical advice. Abernethy notes the collaboration quickly learned how data can help solve the pandemic. The first thing developed was a list of questions that FDA required an answer to. A list of prioritized common data elements was developed that could be pulled from outside data systems such as CDISC or the VA. Translation tables were created that allow for translation between common data models that would allow work to be performed more quickly. Finally, safe spaces were created for researchers across the ecosystem to work together to solve the prioritized problems.
“What I think is most important about the Evidence Accelerator is who is involved with it,” adds Abernethy. “It is over 600 people and over 100 organizations representing government, academics, non-profits, industry, health systems, payers, and the health technology companies. Health systems and payers historically do not get involved in these types of conversations. We are figuring out how all of us can learn from each other and work very quickly.”
Another goal of the Accelerator was to do the research in a way that reduces innovation cycles. This was done by using what Abernethy refers to as ruthless transparency. Those involved in the Accelerator would showcase their results and others would comment on them. This allowed all members of the Accelerator to figure out what works and not have to redo any of the work performed by others. Abernethy notes that ruthless transparency allowed for the quick development of high-quality science. After that science is adjudicated and cross-checked, it is put forward and built upon.
Innovation Assists Trial Execution
In addition to taking over one million lives worldwide, COVID has also disrupted the clinical trial process. The FDA put out emergency guidance to help companies, and sponsors have managed to continue to execute trials. The execution has certainly been aided by innovations that were fast-tracked because of COVID.
“There has been innovation in service of COVID,” states Abernethy. “Some of this is activity the FDA and others had already been championing, such as decentralized clinical trials. But part of the challenge of getting new trial innovation to happen is figuring out how to put our toe in the water. COVID created the urgency and the practical reality that we had to figure out how to do it. People couldn’t leave their homes, but clinical trials still had to persist.”
COVID created the need for companies to innovate with respect to remote monitoring, decentralized trials, and the use of telemedicine solutions. Those solutions allowed sites to interface with patients where they are and keep them safe while understanding their condition. At the same time companies learned how to use real-world data to fill in gaps in clinical trial data sets.
As these new innovations have come forward, Abernethy has been asked what she is excited about and what she believes will persist. With COVID, the industry has learned a lot about how these innovations can be used. But the industry is also seeing the implications of innovation on patient safety and the integrity of the data.
“What will persist beyond COVID will depend on what we learn is safe for patients and maintains the integrity of the data set,” says Abernethy. “The answer to the persistence question will have to do with everyone having the discipline to learn from what happened, seeing what worked, and having the confidence to continue to use it going forward.”
The Role Of Investigators
While decentralized trials will be a wonderful opportunity for patients and reduce the burden placed upon them, it will also change the role of site investigators. Abernethy has served as a site physician herself, and understands the role played by those physicians.
“Investigators need to keep the study participants safe,” she says. “They also have to generate the high-quality data sets that will help answer the question at hand. Practically speaking, the site is critical to that entire set of activities. I think about clinical trials as a series of solutions that will help physicians involved in the clinical trial keep watch over the health of the participant while also having technical solutions in place that will ensure the integrity of the data. The investigators involved in a clinical trial will continue to have these central functions, even if the trial happens to be decentralized.”
Abernethy will continue to drive the Technology Modernization Action Plan (TMAP) within FDA. She notes the plan was announced in September 2019 and consists of three parts. The first part addresses shoring up the technical infrastructure of the FDA and ensuring there is a cloud-forward infrastructure the agency can rely on. The second part is building a more modern agency through a series of use cases or living examples. That will allow the agency and its stakeholders in the industries it regulates to imagine the possible as it is built and figure out what else needs to be added. The third part of TMAP is opening the communication channels with stakeholders in the data and technology industries. Abernethy notes that effort will help the FDA and those stakeholders imagine together where that landscape goes.
“TMAP came before COVID, but at the end of 2019 we were having conversations about vaping-related lung illness,” adds Abernethy. “Within FDA, there were discussions about how important our technical infrastructure would be to perform surveillance, understand where the problems are, and get out in front of medical issues that arise. In January 2020 we brought forward our data modernization plans and began embarking on them ahead of schedule. The pandemic has also accelerated that story and hopefully you will hear more about that in the next month or two.”