McKesson’s Sarah Alwardt, Ph.D, recently wrote in OncLive about the importance of real-world evidence (RWE) in the field of cancer treatment research.
Fewer than 5 percent of adults with cancer enroll in clinical trials, but sufficient enrollment is necessary in order to successfully complete randomized controlled trials (RCTs) and bring new oncology drugs to market sooner. But because the oncology community is not likely to be able to recruit enough participants for many of these trials, she says it’s time to rethink how trials are populated and how data is gathered for regulatory decisions.
While RCTs remain the gold standard for evaluating the safety and efficacy of drugs, the complexity of the field of oncology and the growth of personalized medicine provide an opportunity to incorporate RWE.
Real-world cohorts can become part of RCTs through the use of “pragmatic trials,” which offer the opportunity to test treatment options on realistic patient populations—typical RCT participants are younger and healthier than those in the real world, and they also tend to lack gender and ethnic diversity. The resulting real-world data can be used for standalone analysis or as part of the RCT.
Synthetic control cohorts can also support drug development and approval. Using real-world data to generate a synthetic control arm could reduce the number of patients assigned to the control arm of an RCT or allow for a smaller randomized trial.
“Standard of care” control groups can be synthesized by using historical real-world evidence to generate safety and effectiveness data for current cancer treatments versus those of the new treatments being tested in RCTs.
Dr. Alwardt also suggests a two-step regulatory approval process, so biopharma companies could receive conditional approval from the FDA on the basis of early-phase (phase II or III) RCT data. Once conditional approval is granted, the company would have to conduct more comprehensive trials for final approval of a new drug application.
Real-world evidence will play an important role in drug development and testing because it provides critical information about how new treatments perform in real clinical settings.
Read the full article here.