Blog July 22, 2019

Real World Evidence Replacing Some Clinical Trials?

evidencePharmaceutical companies have long used real-world data on drug performance to help justify their pricing decisions to payers. But now such real-world evidence (RWE), gathered from electronic medical records (EMRs), national patient registries and other patient databases, is increasingly being looked at as a way to make the case for new supplemental approvals and label extensions.


This spring, Pfizer received an FDA approval for the use of Ibrance as part of a combination treatment for men with HR-positive, HER-2 negative advanced or metastatic breast cancer based largely on RWE. Breast cancer is rare in men, affecting an estimated 2,610 U.S. patients in 2019. Thus it would have been difficult to conduct a clinical trial. Instead, the regulators studied data from EMRs, post-marketing reports from the IQVIA Insurance database, the Flatiron Health Breast Cancer database, and Pfizer’s Ibrance global safety database to support their approval of the new indication.


Ned Sharpless, the acting head of the FDA, told BIO attendees that the agency is “open for business” in considering RWE in its approvals of new uses for cancer drugs. The agency has been helping to drive the use of RWE to learn more about a drug’s safety and efficacy as it enters clinical usage, especially as cancer patient populations become more precisely defined and thus, the recruitment of patients in clinical trials becomes more challenging.


In April, the FDA teamed with Brigham and Women’s Hospital to test whether RWE analyses are good enough to replace clinical studies for supplemental drug approvals. The collaborators are using a group of seven clinical studies in a pilot project to see if RWE could predict the results of randomized clinical trials that have not yet been completed. Interim results from the project are expected soon, with full results to be reported at the end of 2020. If the project is successful, RWE studies using existing patient data may negate the need for many lengthy and expensive clinical trials for supplemental drug approvals.


The FDA also recently announced a partnership with the precision medical technology company COTA to help fuel a deeper understanding of cancer treatment through RWE. Starting with a two-year collaboration focused on breast cancer, they aim to create a repository of RWE, including treatment and outcome differences among the general population of breast cancer patients, that can help support the agency’s guidance and decision making. In addition to this government-driven initiative, the industry is witnessing the success of a growing number of businesses that are collecting, storing and analyzing RWE data, such as IQVIA, Flatiron and Aetion.  


Could the increased use of RWE in regulatory decision-making help reduce the high costs of clinical trials, at least for supplemental approvals? What sorts of studies might be most appropriate for the use of RWE?