Recently, I sat in the audience of an oncology congress and heard a patient survivor and advocate describe critical transitions in his experience living with cancer: At diagnosis, he went from a “person” to a “patient,” and during clinical trial enrollment, he went from a “patient” to a “subject.”
But this is the age of personalized medicine. We have made, and continue to make, amazing strides in the scientific understanding and application of that knowledge in the fight against cancer. The proliferation of biomarkers, advanced genetic screening and approved targeted therapies, and the exciting horizon of cellular and gene therapies are among the advancements that have enabled us to get closer to the right treatment for the right patient. It's no longer just about what the physician can see with her eyes but also what she can’t see: the patient's DNA. These tools allow us to acknowledge that each patient is complex and unique, and merits a personalized care plan.
The discrepancy highlighted in this patient’s story, however, is that “personalized” means much more than any clinical definition can reflect. It’s much more than the tumor type being treated, much more than biomarker expression, and much more than the burden of disease or any other marker of “who” a patient is in context of his cancer. “Personalized” also is about the context of his life as a person living with cancer, and how a treatment can enable him to keep living that life.
While clinical trials attempt to elucidate how a typical patient may react to a given treatment, none of us would want to be considered “typical” patients. At another recent event—the 2017 Annual Society of Clinical Oncology (ASCO) Annual Meeting—one question that I brought with me was, Is real-world evidence part of the solution of “personalization” and putting patients at the center of evidence?
At ASCO, I sat down with Dr. Yousuf Zafar, associate professor of medicine and public policy at Duke University, to talk about the definition of real-world evidence and its role in getting us closer to individualized patient treatment. In short, real-world evidence offers a tremendous opportunity to make choices that take into account the demographic and situational elements of a patient—more than clinical evidence can do alone.
Dr. Zafar defines real-world evidence as "collecting data from patients and from the health systems in a way that’s representative of the environment in which those patients were treated." He enthusiastically describes the potential to treat cancer in a way that considers what’s important to the patients, themselves, not just the disease they have.
As the ability to harness and use real-world evidence increases, it will be important that, as an industry, we’re considering how and when we’re capturing the type of information that will allow for specificity in treatment decisions. For oncology manufacturers in particular, Dr. Zafar’s comments remind us of the important role that real-world evidence will play in actual practice.