August 26, 2019
Three former FDA commissioners unveiled a report last week that proposes a dozen ways to expand the use of real-world evidence (RWE) and real-world data (RWD).
The commissioners, on behalf of the Bipartisan Policy Center, urged the FDA to issue guidance on how sponsors can use RWE for new indications for existing drugs and support postapproval requirements.
“Real world data and evidence are also necessary for the implementation of many novel development and approval pathways,” says the report, which was endorsed by Robert Califf, Mark McClellan and Andrew von Eschenbach. As medicine becomes more personalized, they said, “randomized controlled trials will become increasingly less feasible.”
According to a survey of sponsors conducted by the Tufts Center for the Study of Drug Development, use of RWD/RWE by drug sponsors already is widespread. Of the 30 companies responding to the survey, 97 percent said they use RWD in a variety of ways, including in support of drug applications.
Respondents reported having very little direct experience with regulatory agencies accepting RWE for labeling (17 percent) or labeling changes (13 percent), but more experience using RWE for postapproval safety assessments (55 percent) and in postmarketing studies (57 percent).
Twenty percent of companies said they spend more than $5 million on RWE analytics, 60 percent said they spend more than $5 million on research studies and data, and 86 percent plan to increase their RWD budgets by 11 percent to 25 percent in coming years.
The commissioners’ report also points to increased support for adaptive approval pathways, in which data and evidence generated on a smaller, distinctively defined patient population, allows regulatory approval for those specific subpopulations.
The former commissioners also endorsed leveraging technology to gain input directly from patients to help conduct clinical trials and expanding the use of AI to support trial development.
Read the full report here: https://bit.ly/30parRL.
Data Sources Used in Support of an NDA by Sponsor
Source: Tufts CSDD