TransCelerate, a non-profit research and development organization backed by big pharma, is developing a framework for implementing real-world data.
The framework aims to increase the use of real-world data and real-world evidence in regulatory decision-making and provide companies with tools to implement it into their own organizations. It will include an Audit Readiness Checklist, which will form a list of best practices for those managing real-world studies.
The nonprofit will also produce a tool to make it easier for companies and researchers to engage with health authorities.
Cathy Critchlow, vice president and researcher at Amgen, a Thousand Oaks, California-based biotech company, said there was no other framework for implementing real-world data. As real-world data grows in popularity, she said it was important to ensure this data is being used properly
“Patients bear the brunt if you get a decision wrong,” Critchlow said at Tuesday’s Drug Information Association conference in Chicago. “What is the cost of getting a wrong answer? [We] are trying to use the data for a purpose other than why it was originally collected.”
Amgen is a member of TransCelerate and Critchlow is an active participant in developing this framework.
Real-world data leverages existing data sources, such as electronic health records, claims data, wearables and patient surveys, to draw hypotheses in patient populations. Experts say it is an alternative to traditional clinical trials and could bring drugs to market faster.
While still nascent, real-world data has resulted in at least one approval. The U.S. Food and Drug Administration approved Prograf, a drug which can limit organ rejection in adult and pediatric patients receiving lung and other transplants. When approving this drug, the FDA said it was the first example of how a "well-designed, non-interventional (observational) study relying on fit-for-purpose (i.e., reliable and relevant) real-world data."
Experts say that by using real-time data, clinicians can acquire a full, 360-view of the patient beyond the walls of the hospital. This can inform healthcare organizations about the benefits and risks associated with various interventions.
Panelists at the Drug Information Association conference agreed the space is developing quickly, but a key hurdle facing real-world data is successfully pulling large amounts of information from existing datasets.
“There are multiple ways to pull out large amounts of data, but those tools are in their infancy,” said Irene Nunes, vice president of regulatory affairs at Flatiron Health, a health tech company focused on developing cancer treatments.
TransCelerate said it plans to publish its findings from the initial rollout of the framewokr in a peer-reviewed format by 2023.