Real-world data-driven drug development has been a hot topic the last few years. Recently, Peter Loftus of the Wall Street Journal (WSJ) showcased recent examples of big pharma working to win drug approvals by parsing vast data sets of electronic medical records in the hopes of depending less on random controlled trials—which can be lengthy and expensive.
RWE can Save Time & Money
Loftus reports that the movement to real-world evidence can be used in some situations as an alternative to a clinical trial’s control arm offering a way to compare outcomes for patients who received the standard treatment as compared to those who are taking a new drug in a clinical trial—the difference between in years and in an industry that can count one day as a $1 million that mean a lot of savings.
There are Pros and Cons: Perhaps RWE can Compliment RCT?
TrialSite News has offered a number of overviews of RWE-based studies. The WSJ offers some pros and cons as to the use of real-world evidence and reminds the reader of the 2016 law requiring the FDA to consider encourage more industry use of real-world data. The agency established some preliminary guidance and standards. An FDA official, Amy Abernethy (formerly with real-world data venture Flatiron Health which was acquired by Roche) noted that real-world evidence is used as a way to add to existing standards which falls in line with the TrialSite News understanding.
The WSJ covers some recent real-world evidence market data points such as $5 billion worth of Roche acquisitions (Flatiron Health and Foundation Medicine) and IQVIA’s dozen projects associated with real-world evidence. Major biotech company Amgen used real-world data derived from leukemia patient records to augment evidence for a new drug application for Blincyto which the FDA did accept as it approved a new use for the dug. According to Elliott Levy, senior vice president of global development, Amgen saved enormous time as they would have had to enroll at least 50% more patients in the clinical trial to have a standard control arm reports Loftus.
J&J used Roche’s Flatiron and Foundation data to identify patients with specific genetic traits that would not benefit from certain immune-boosting drugs and rather supported an efficient augmentation of a separate clinical trial in which benefited patients with the specific genetic trait. These results were included in J&J’s Balversa’s approval by the FDA in 2019.
Meanwhile, Pfizer has used Flatiron to mine for data to determine how men who were described a breast cancer drug fared compared to those who hadn’t taken the drug. According to Chris Boshoff, Pfizer’s chief development officer for oncology noted the use of the real-world data saved them a lot of time and cost them “a fraction” of what a normal trial would cost. However, the FDA review of the Pfizer’s application conveyed the data size was too small to prove effectiveness.
Call to Action: Check out the WSJ piece from Peter Loftus.