Real-World Evidence Portfolio

Real-World Data (RWD) is patient data routinely collected in electronic health records (EHRs), claims data, and registries that can provide valuable information about the real-world performance of cancer therapies and diagnostics. Unlike traditional clinical trials, which collect data at predefined timepoints and under standardized protocols, RWD often vary in timing, completeness, and format within and across sources. Inconsistent definitions and data missingness present challenges to using RWD for evaluating treatment effectiveness, such as assessing a therapy’s impact on survival or its use in certain patient populations. Strategies and methodologies for mitigating these factors and aligning RWD are needed to fully realize its potential. 

Since 2017, Friends has facilitated collaboration among RWD vendors, pharmaceutical companies, government officials, and academics to advance the understanding and applications of RWD and real-world evidence (RWE) in oncology. These multistakeholder partnerships support alignment on best practices, provide a venue to develop and test methodologies for aligning and analyzing RWD, and help identify opportunities to proactively strategize on future use of RWD/E in oncology:  

• RWE Pilot 1.0 created a framework for aligning data sources and showed that implementing a harmonized protocol and set of definitions for necessary data elements supports identification of similar patient populations across data sources and extraction of several real-world endpoints. 
• RWE Pilot 2.0 applied the framework developed in Pilot 1.0 and showed that RWD can generate similar results to clinical trials when measuring the treatment effect of chemotherapy compared with immunotherapy in patients with advanced non-small cell lung cancer (aNSCLC). The group developed a list of considerations for the design, conduct, and interpretation of RWD studies from different data sources.  
• rw-Response established a framework for measuring overall response rate to treatment outside of a clinical trial and found relative consistency of the measure across RWD sources in first-line chemotherapy regimens for patients with aNSCLC.  
• rw-Care developed a framework for the use of RWD at point of care to improve patient outcomes from immunotherapy by mitigating treatment-related adverse events.  
• The External Control Arms (ECA) Pilot will identify and refine best practices for using historical clinical trial data and RWD to construct rigorous ECAs or augment single arm trials. When randomized controlled trials are not feasible, ECAs have the potential to improve the quality and efficiency of clinical research by expanding access to investigational treatments and supporting more timely regulatory decision-making for patients with high unmet needs. 

Traditional clinical trials, such as randomized controlled trials (RCTs), can take a long time and typically enroll narrowly defined patient populations that represent only a fraction of those who will likely receive the product in the real world. RWD can generate supplemental evidence that reflects a broader, more diverse patient population and address timely clinical questions, including use of different treatments in standard of care practice and long-term safety. Additionally, RWD can be used to evaluate treatment where it may be unethical or otherwise infeasible to enroll patients to a control arm. Without coordinated efforts like those led by Friends and support from collaborative sponsors, the field may struggle to establish aligned best practices and frameworks for aggregating and analyzing RWD effectively.