Historically the trove of medical and para-medical information known as “real-world evidence,” including health records but also insurance claims and patient registries, has mostly been used as a source for additional evaluation of the efficacy of existing drugs after they have already been approved. But now the situation has changed dramatically with the advent of massive computing power, which allows researchers to analyze these vast data pools for forward-looking purposes, including the development of new drugs. Eli Lilly cancer researcher Kimberly Blackwell details some of these futuristic applications.
- One of the most important applications is in crafting personalized cancer treatments based on genetic markers, Blackwell explains: “As cancer treatments become more individualized, researchers have begun to rely on molecular testing to understand which patients will benefit from a treatment — rather than use a ‘one size fits all’ approach. When developing new targeted therapies, RWE helps us match the genetic markers we find in some in tumors with the potential treatments most likely to make an impact.”
- At the same time, RWE and genetic information can help rule out treatments, allowing doctors to focus on other approaches that will be more beneficial: “Working backwards, RWE can also help identify the patients who don’t respond to standard treatment options, and more quickly focus our research on opportunities that may work better for them.”
- Analysis of RWE also lets researchers distinguish between the treatment’s actual effects and random artifacts due to other factors: “RWE can be used to help identify patterns of response earlier, which helps differentiate whether a potential therapy is truly effective at treating disease, or if the changes we observe during the study are the result of other causes, such as genetics, environment, lifestyle factors, etc.”
- There are still shortcomings and limitations in RWE, including “sporadic” medical records and regulatory barriers, but overall the FDA has been supportive of the process, Blackwell writes.
