Flatiron Health Announces Three Publications Studying a Feasible, Reliable, Scalable and Meaningful Real-World Progression Endpoint for Oncology Research
Today, Flatiron Health announced a series of three research publications that developed and evaluated a new approach to capture when a patient’s cancer has worsened based on information contained in the electronic health record (EHR). These manuscripts illustrate the potential impact for research conducted from data collected as part of routine clinical care (real-world data).
Cancer researchers and regulators, including the U.S. Food & Drug Administration, have typically relied on measurements from imaging tests to document cancer shrinkage or growth. When conducting research from real-world data, access to these radiology studies and measurements may be limited and another measure of disease status is needed. The authors define a new measure, “real-world progression,” based on the documents contained in a patient’s chart that does not require the scans themselves. Results from these three studies provide the tools and evidence needed to generate high-quality data and facilitate interpretation of the evidence obtained from future real-world studies in oncology.
“Progression, or worsening of cancer, is one of the most important points in a cancer patient’s journey because it indicates the cancer is no longer controlled. For patients and their families, progression impacts everything from treatment side effects to vacation plans. For doctors, progression informs next steps in care and treatment decisions. For cancer researchers, progression identifies where we need to do better.” said Dr. Rebecca Miksad, senior medical director at Flatiron Health. “Through this research, generating evidence from real-world datasets will help us achieve our goal of learning from the experience of patients treated in real-world settings.”
Published in collaboration with the U.S. Food & Drug Administration (FDA), Genentech and Dana-Farber Cancer Institute in Advances in Therapy (May 2019), the first manuscript, “Generating real-world tumor burden endpoints from electronic health record data: Comparison of RECIST radiology-anchored and clinician-anchored approaches for abstracting real-world progression in non-small cell lung cancer,” evaluates different methods to curate rwP from documents available in the EHR. Researchers found that applying strict radiology interpretation rules (called “RECIST”) was not viable for assessing progression of cancer in patients treated outside of clinical trials, in the real world; however, capturing the doctor’s written assessment of the patient’s cancer status corroborated with radiologic evidence was both feasible and reliable for understanding when disease progressed in cancer patients treated in routine clinical settings.
A follow-up study, “Characterizing the feasibility and performance of real-world tumor progression end points and their association with overall survival in a large advanced non-small-cell lung cancer dataset,” published in JCO Clinical Cancer Informatics (August 2019), with the FDA, Duke University, Dana-Farber and Genentech, tested this approach in the de-identified records of more than 30,000 patients with advanced non-small cell lung cancer (aNSCLC). Researchers tested curating rwP events from the EHR for a large group of patients treated in real-world settings and determined the length of time until a tumor worsened, both of which are fundamental treatment metrics in oncology. This approach that anchors to the clinician’s assessment of the patient’s cancer status proved to be meaningful and scalable for understanding disease progression in real-world patient cohorts large enough and recent enough to potentially impact clinical decision making.
After identifying the most effective approach to assessing rwP as an endpoint, investigators at Flatiron and the FDA sought to derive meaningful insights from a de-identified dataset. Building on prior research studies (see here and here), the third study in the rwP manuscript series, “Real-world progression, treatment, and survival outcomes during rapid adoption of immunotherapy for advanced non-small cell lung cancer,” published in Cancer (August 2019), expands and deepens our understanding of how immunotherapy treatment impacts patients with aNSCLC treated in the real world by evaluating rwP-based outcomes. Notable findings from this manuscript include shifts in practice patterns in recent years, outcomes in patient groups underrepresented in clinical trials, and relationship of rwP events to the patient’s overall care and survival.
By enhancing our understanding of progression for cancer patients treated in the real-world, this research continues to develop a solid foundation for generating real-world evidence; research that may supplement and complement results from clinical trials and may help inform patient and clinician decision-making.
For a synthesis of our approach to our rwP research across all three publications, please see our blog post, “Is the Cancer Better or Worse? Our Journey to Curate Tumor Progression From the Electronic Health Record.”
Flatiron Health is a healthtech company dedicated to helping cancer centers thrive and deliver better care for patients today and tomorrow. Through clinical and data science, we translate patient experiences into real-world evidence to improve treatment, inform policy, and advance research. Cancer is smart. Together, we can be smarter. Flatiron Health is an independent affiliate of the Roche Group. Flatiron.com @FlatironHealth