On September 11, 2023, Friends of Cancer Research (Friends) hosted a public meeting, “Supporting the Use of RWD in Oncology Drug Development”, in Washington, DC. The meeting included a presentation of findings from Friends’ Real-world Response (rw-Response) Pilot and two panel discussions focused on the findings’ regulatory implications for use of RWD and real-world evidence (RWE), and additional uses of RWD in oncology drug development. This meeting builds on Friends’ multi-year portfolio of projects on Real-World Data.
Morning Keynote Featuring Amy Abernethy, President, Product Development & Chief Medical Officer, Verily, with Moderator Steve Usdin of BioCentury
The meeting began with an opening keynote conversation between Steve Usdin of BioCentury and Amy Abernethy of Verily. Dr. Abernethy discussed how the understanding around leveraging RWD in oncology has evolved over time. She noted that while experience using RWD to date has helped to identify best practices and understand what information can be obtained from RWD sources (e.g., real-world endpoints), there is still a lot of work to be done. While discussing the future of RWD, Dr. Abernethy described the need for approaches to integrate passively and prospectively collected data from various data sources such as clinical trial data, multi-omics data, sensor data from digital health technologies, and other long-term follow-up data to paint a longitudinal picture of a person’s health and life. Dr. Abernethy also noted the importance of patient consent, data privacy and security, and on the need for transparency about how patient data are being leveraged.
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Session 1: Establishing a Framework for Evaluating Real-world Response
Presentation — Assessment of rw-Response in an Aligned Patient Population Across RWD Sources
Brittany McKelvey of Friends opened the session presenting findings from Friends’ rw-Response Pilot. The collaborative partnership across 7 RWD partners demonstrated the feasibility of obtaining rw-response endpoints based on clinician assessments included in RWD sources and highlighted the opportunity to use rw-response to inform drug effectiveness evaluation.
Panel 1 Discussion
Following the presentation, panel moderator Elizabeth Garrett-Mayer of the American Society of Clinical Oncology, introduced panelists Amanda Bruno of Syneos Health (formerly Bayer Pharmaceuticals), Pallavi Mishra-Kalyani of the U.S. Food and Drug Administration (FDA), Yanina Natanzon of ConcertAI, Nicholas Robert of Ontada, and Lawrence Schwartz of Memorial Sloan Kettering Cancer Center. The panelists delved into the value of conducting a pilot study on measuring response in RWD, highlighting the differences between clinical trials and RWD sources, and the need to understand how rw-response is captured and whether it can be reliably measured across data sources. They discussed the need to integrate RWD throughout drug development from early discovery to post-marketing surveillance. Appropriate trial designs, particularly those that incorporate RWD, were highlighted as important for successfully using RWD in oncology drug development. The speakers also discussed the value of clinician assessments to ascertain rw-response as it allows for assessment informed by radiology reports while not needing the images or reports themselves, given the heterogeneity in data availability. Panelists emphasized the importance of collecting images and follow-up data to enhance RWD while also acknowledging the limitations in using imaging data to measure rw-response. They suggested that aggregating imaging data and analyzing these data with the help of AI could potentially overcome these limitations. The speakers also discussed the potential of using liquid biopsies to evaluate rw-response, highlighting the changing landscape of clinical documentation and the need for objective measurements.
Lunch Keynote with Dr. Robert Califf, U.S. FDA Commissioner, and moderator Kate Rawson, Prevision Policy
During the lunch keynote, Kate Rawson of Prevision Policy opened the discussion highlighting previous remarks by Dr. Califf, U.S. FDA Commissioner on the “disaggregated fragmented system” for evidence generation in pre-market and post-market drug development. Dr. Califf went deeper into the intricacies of the FDA’s three-phase approval system, shedding light on the key contributors to data quality issues. He provided insights into the pathways that potential drugs navigate, emphasizing that more than 85% of drugs in phase one fail to make it to market. He underscored the critical juncture of product approval as the beginning of disarray within the American healthcare system and how that is reflective of the status quo. By comparing the U.S. healthcare system and life expectancy with Singapore, of interest given his upcoming visit after the meeting, Dr. Califf stressed the importance of effectively bridging social determinants of health and efficient technologies with product approvals to positively impact the overall health outcomes of the American population.
Regarding structuring and management of clinical data and information within the healthcare system, Dr. Califf emphasized the public health system’s critical role in establishing an efficient and properly aligned healthcare system. He noted that RWE initiatives should not be dismissed, as they hold immense potential, but their full realization hinges on addressing the systemic issues within the American healthcare system itself. In concluding the lunch keynote, Dr. Califf emphasized the necessity of cross-sector collaboration involving key entities such as the FDA, National Institutes of Health (NIH), Centers for Medicare and Medicaid Services (CMS), and other stakeholders. He stated,
“There’s a lot of aggregation going on for the financial benefit of elements of the system. Where is it integrated for the benefit of the patient? It wouldn’t take that much give from every element of the system.”
Session 2: Lessons Learned from Real-world Endpoint Analyses and Opportunities for Use in Drug Development
Panel 2 Discussion
The second panel, moderated by Irene Nunes of Flatiron Health, featured a discussion on “Lessons Learned from Real-world Endpoint Analyses and Opportunities for Use in Drug Development.” The panelists included Ashita Batavia of Johnson & Johnson Innovative Medicine (formerly Janssen R&D), Thomas Brown of Syapse, Laura Fernandes of COTA, Jane Perlmutter, a Patient Advocate, and Donna Rivera of the FDA. The discussion started with highlighting Friends’ initiatives to characterize rw-endpoints and establish aligned methodologies.
The panelists engaged in a robust conversation about the feasibility of collecting RWD from diverse sources. They stressed the importance of aligning clinically based assessments of response while acknowledging the nuances between rigorous clinical trials and observational data, such as RWD. Moreover, the panelists collectively emphasized their commitment to building a high-quality RWE system. This system would be characterized by source standardization, design justification, and method validation to ensure drug safety and effectiveness, underpinned by robust evidence.
In their closing remarks, the panelists highlighted the need for an open-minded approach to embracing new technologies, including artificial intelligence, as well as the imperative of fostering more cohesive cross-sector collaborations. Their ultimate goal is to leverage RWD and precision medicine to improve patient health outcomes.
Friends also published a pre-meeting discussion document titled “Considerations for Leveraging Real-World Endpoints in Oncology Drug Development”: An Analysis of Pilot Results by the Real-World Response Working Group.
