Significant progress has been made in data collection efforts to support use of real-world evidence (RWE) in regulatory settings; however challenges remain, chiefly with combining, organizing, and analyzing data from various information sources. On July 10th, Friends of Cancer Research (Friends) hosted a meeting to present data from a pilot project assessing the use and efficacy of immune checkpoint inhibitors in advanced non-small cell lung cancer (aNSCLC). The pilot project comprised of data collected from six leading healthcare data organizations with the goal of developing a dataset curation process and exploring potential real-world endpoints extracted from electronic health records (EHRs) and claims data that may be fit for regulatory purpose as well as assessing long-term benefits of a product. The meeting comprised of data presentations and three panel discussions.
Jeff Allen, President and CEO of Friends, opened the meeting by presenting results from the pilot project, which demonstrated that despite differences in data sources, population characteristics were similar among the datasets. Moreover, several endpoints extracted from EHR and claims data, such as time to treatment discontinuation (TTD) and overall survival (OS), fell within the range of median times observed in clinical trials demonstrating the potential utility of real-world data (RWD).
“No matter the source and no matter the size the similarity and ease of extractability was similar and astounding” – Jeff Allen, Friends of Cancer Research
The first panel consisted of representatives from the pilot project’s six healthcare data partners. Elad Sharon from the National Cancer Institute moderated the discussion that covered topics such as data collection and methodological gaps; challenges associated with differing levels of available data and consistency across provider types and settings; and opportunities to address challenges and encourage the collection and curation of high-quality data. Some challenges identified by the panelists included the difficulty to assess progression events that are often defined inconsistently across practice settings and an inability to access up-to-date mortality data to produce timely data for regulatory and reimbursement decisions.
“If we’re going to use real-world evidence, we need to make sense of the data sitting in front of us” – Amy Abernathy, Flatiron
FDA’s Gideon Blumenthal and Rajeshwari Sridhara presented additional data assessing TTD and time to treatment failure (TTF) using a pooled analysis of clinical trial data of tyrosine kinase inhibitors (TKI), immune checkpoint inhibitors, and chemotherapy. The data revealed that physicians are often treating patients past progression and that TTD correlated closely with progression-free survival, a more traditional clinical trial endpoint. The results of the study indicate a need for further exploration of TTD as an endpoint particularly in RWE studies where progression events are measured inconsistently.
“There is a gap in understanding the actual impact of what patients go through, and we need to do a better job incorporating how patients feel and survive” – Janet Woodcock, FDA
The second panel focused on the implications of RWE and the roles it can play within cancer drug development. A discussion led by Michael Maitland from Inova Schar Cancer Institute revealed that drug sponsors utilize RWE at varying levels during the different stages of drug development. All the panelists agreed the pilot project data highlighted the potential utility of RWD and the roles it can play in clinical trial designs, measuring real-world performance, and discussions with payers. Panelists also discussed how RWE can further contribute to the understanding of therapies by providing additional information on safety and implications of concomitant medications, which are not completely captured within a clinical trial. Panelists stated that standardizing the definitions for real-world endpoints as well as extraction methods will be critical as the RWE field moves forward.
“Patients want meaning from their experience and for it to help other patients” – Jane Perlmutter, Gemini Group
Michael McCaughan with Prevision Policy moderated the final panel, which consisted of a broad group of stakeholders discussing topics ranging from the implications of RWE for collecting patient-generated data, regulatory and payer decision-making, and assessing new uses and the value of therapies over time. Panelists discussed how RWE can provide a greater insight into patient experience by capturing hospitalization rates, late stage side effects, and patient reported outcomes to determine optimal value in care. Real-world data also provides the opportunity to address other important questions that are often not answered including identifying the most optimal dose and duration of treatment.
Throughout the meeting, panelists and meeting attendees identified potential next steps and questions that could be addressed in a pilot project 2.0 including capacity and methodology for data linkage; identifying late stage side effects; occurrence of a subsequent cancer; use of RWE for control arms or to supplement clinical trial data; and assessing efficacy of approved therapies in rare cancers. Exploration of these and other key questions will be further examined to evaluate additional uses of RWE and the potential for these data to inform on-going policy discussions and guidance development at the FDA.
For more information on this project, please click here.