• During the COVID-19 pandemic, real-world data has helped answer pressing queries that lengthy clinical trials are slow to resolve — most notably, the effectiveness of the malaria drug hydroxychloroquine for treating the novel virus.
  • “Real-world data can help us understand the story of a disease,” says Dr. Abernethy of the FDA. “It can help us prioritize treatments that might work. It can help us understand and put those treatments into context.”
  • Compliance and scientific rigor must remain paramount even as researchers work quickly to learn about, find treatments for and develop a vaccine against COVID-19 — Dr. Abernethy says the issue calls for “deliberate agility.”

COVID-19 continues to spread throughout the United States. Safe and effective treatments remain elusive, and a vaccine is yet to be approved.

But there is reason to remain hopeful.

The pandemic has facilitated unprecedented global collaboration among health care organizations, providers, drug manufacturers, academic institutions, and government and regulatory institutions. What these institutions are learning about how we respond to and analyze diseases has incredible potential for the future of these fields.

Among those bodies collaborating on every aspect of the pandemic response is the U.S. Food and Drug Administration, which relies on real-world data, among other information, to evaluate potential COVID-19 treatments as well as the disease’s impact on ongoing research.

“There certainly has been a whole of government response, but I would really call it a whole of health care and whole of life sciences response to COVID-19,” says Dr. Amy Abernethy, principal deputy commissioner and acting CIO of the FDA.

For this episode of the “Real World Talk” podcast, COTA’s Zoe Li spoke with Dr. Abernethy, and COTA’s president and CEO, Mike Doyle, about how the FDA is responding to COVID-19 and how it uses real-world data.

Real-World Data in a Pandemic

With the immediacy of the COVID-19 crisis, practitioners have not had the luxury of waiting for results from extensive longitudinal clinical trials to teach about the disease and potential treatment.

“We have more questions in front of us than we can possibly answer in such a short period of time,” says Dr. Abernethy.

In this situation, real-world data becomes an important practical solution to quickly determine the value of experimental treatments.

“Real-world data can help us understand the story of a disease,” says Dr. Abernethy. “It can help us prioritize treatments that might work, it can help us understand and put those treatments into context.”

COVID-19 Evidence Accelerator

The COVID-19 Evidence Accelerator creates a space for data providers, government organizations, academic researchers, and health systems to collaborate to ask questions and share results. Through the accelerator, COTA works together with Friends of Cancer Research and the Reagan-Udall Foundation for the FDA to examine new data from real patients with COVID-19 to understand treatment outcomes and potential risk factors.

For example, researchers in the accelerator were able to look at the effectiveness of hydroxychloroquine, a malaria drug believed to be potentially effective in treating COVID-19 symptoms.

“There was a lot of different evidence to say it might be effective, it might not be effective,” says Doyle, “but there wasn’t the type of rigor and the type of study that we were able to do with our partners that allowed all of us to take a look at it in a very objective manner.”

By looking at real-world data from partners at Hackensack Meridian Healthcare, COTA was able to determine the drug would not be effective in treating COVID-19 and could, in fact, be harmful.

“The evidence accelerator allows us to bring together a large community of data holders and analytic teams beyond the groups that were historically worked with,” says Dr. Abernethy, “in order to figure out who can answer which questions with what speed and how.”

Maintaining Agility and Accuracy

Despite the benefit of expediency, the use of real-world data can pose a challenge for ensuring scientific rigor, compliance, and accuracy.

“We need deliberate agility,” says Dr. Abernethy. “We need to acknowledge that we have to work fast, but we can’t work sloppy.”

To determine which data sets to use; and how to analyze them, work quickly and maintain reliability, she suggests:

  • Find data sets that are ready.  Work with the data that’s available to address questions. Prioritize based on when data sets will be ready for analysis to make the most of the time you have.
  • Understand the quality of the data. What do you know about the data holders’ experience with data curation? How complete is the data? 
  • Convergence of results. To avoid making decisions based on erroneous results, ensure that you see consistent results when a study is replicated.
  • Ability to course correct. Researchers need to stay open to adjusting direction as they learn more, even when it happens very quickly. “We should all acknowledge that the story is changing,” says Dr. Abernethy.
  • Replication and accountability. “We have to have ruthless transparency,” says Dr. Abernethy, to ensure trust from patients. Other researchers must be able to see your work, replicate and cross-check the findings.
  • Peer review. Before information makes its way to the general public, it should be validated by peer-review to ensure it’s credible and complete.
  • Practice. Engage experienced researchers and reviewers to ensure you have a sufficient understanding of a study’s methods and limitations.

What the FDA Is Doing About COVID-19

The FDA’s reach amid this pandemic is wide-ranging.

“The FDA’s role is as an actor in all of the efforts [to combat COVID-19] as well as a specific role as it relates to protecting and promoting public health, and regulating a large number of products that are directly related to COVID-19,” says Dr. Abernethy.

COVID-19 Treatment

In addition to its higher-profile involvement in the development of a COVID-19 vaccine, the FDA is involved in the approval and monitoring of diagnostics, drug treatments and biologic treatments for the disease.

Dr. Abernethy says the FDA’s role in development of these treatments and preventions is “as a regulator, as a thought leader and as a partner” with private companies.

Food Supply

Amid a health crisis, it’s easy to forget the FDA’s role in the food supply chain.

During the pandemic, it continues to monitor for foodborne illness and other issues that could affect the food supply in the U.S., and it must remain agile to the ways the pandemic could affect how it performs that duty.

What We Can Learn From Pandemic-Era Trials

Researchers should not design clinical trials specifically to accommodate pandemic conditions — generally, this environment is still exceptional.

Still, there’s a lot to learn from the ways COVID-19 has affected clinical trials. This extended disruption of ongoing and planned trials can inform improvements and new trial designs going forward, including:

  • Using more external comparators.
  • Running more single-arm trials.
  • Simplifying requirements for participation to alleviate the patient’s burden.

“We are going to need to plan future trials based on a lot of new knowledge that’s happened in the last three months about the pandemic,” says Dr. Abernethy, “and about how health care has changed and how this may impact disease.”

An open question is whether real-world data can fill in gaps resulting from clinical trials that were paused to slow the spread of the virus. On the other hand, real-world data could offer insight into how contracting COVID-19 affects patients with other diseases using previously approved treatments.

“FDA has been, I think, quite forward-leaning,” says Dr. Abernethy. “We’re going to work together across the community to understand which data are fit for purpose, what are the right analytic plans and what are the right use cases.”