Today’s guest blog comes from Lilly scientist Symantha (Sam) Melemed, Ph.D., a product team leader in the Lilly Oncology group.
As a researcher, it’s the hope that we’ll find “the one” – the treatment that makes a significant step forward and an impactful difference in a patient’s life – that drives my work forward. That’s why I’m constantly striving to provide sound evidence of the promise of emerging cancer therapies. But I do so with the acute awareness that patients’ everyday lives differ greatly from a scientific lab.
Recently, I spoke at an event hosted by the Duke-Margolis Center for Health Policy about how the use of real-world evidence, or RWE, can help us to bridge the gap between research settings and patients’ lives. After the approval of a new medicine, sources like electronic health records, insurance claims or patient registries – data from the “real world” – can act as RWE to show how treatments perform and are used outside of clinical trials.
Real-world evidence, importantly, is not a substitute for data gathered in clinical trials. Instead, it complements these data, adding breadth and depth to our understanding of a treatment. As a researcher in pursuit of “the one,” I believe that RWE elevates the research I do on a daily basis. For example, RWE can more accurately represent the experiences of diverse groups of patients receiving everyday care.
Real-world evidence can also showcase the value and impact of a treatment for a wider range of cancer patients than initially included in trials. It can demonstrate areas where cancer care can be improved or can be used to measure the performance of a certain patient’s therapy. This information can, in turn, allow cancer institutions, from major centers to community clinics, to identify best practices so that cancer patients can have the best possible outcomes.
Since cancer can be a quick and complex disease, our research and knowledge of treatments and diagnostic testing must be constantly evolving, too. RWE allows us to continuously build on initial trial data to show how oncologists are using the treatment for different subgroups of patients or in combination with other treatments. In addition, doctors are incorporating new diagnostic and biomarker testing, so we can learn even more about the complexity of cancer. By creating an FDA review process that incorporates RWE into the data packages upon which regulatory decision are made, we can bring even more robust information about innovative therapies to patients sooner.
This year, Congress reauthorized the Prescription Drug User Fee Act (PDUFA) for the sixth time and provided a path forward on how we can use RWE to actually build this efficient and patient-centric review process. The Cancer Moonshot Task Force importantly proposed a new pilot program for cancer treatments that utilize RWE. These actions highlight the continuously growing importance of RWE to us in the scientific community, as well as to the patients and advocates we’re working to help.
Clear and deliberate steps forward to include RWE in our research of cancer treatments and their regulatory review is essential to ensure patients have the best possible outcomes. Real-world evidence is a crucial asset in our pursuit of “the one” – for researchers, for advocates and for patients.