Summary
Despite the rigor of clinical trials, further understanding of a therapy’s effectiveness is often still needed. Real-world data (RWD) that generates real-world evidence (RWE) has been used to fill these gaps and support evaluation of therapeutic effectiveness, and has been promoted across major regulatory initiatives, including the FDA’s 21st Century Cures Act, Prescription Drug User Fee Act, Food and Drug Omnibus Reform Act, and President Biden’s Cancer Moonshot.
Convened by Friends of Cancer Research (Friends), a collaboration between Friends, the FDA, and seven electronic health record data companies aimed to develop an aligned methodology for assessing real-world response to treatment across disparate data sources. This pilot project sought to: 1) understand the availability and feasibility of using specific RWD elements to assess real-world response, 2) evaluate the potential to ascertain real-world response using available data elements from the EHR, and 3) evaluate the consistency of these measures across data sources. Common protocols were used to assess and report real-world response endpoints, including real-world response rate (rwRR), real-world duration of response (rwDOR), and the association of these with real-world overall survived (rwOS), real-world time to next treatment (rwTTNT) and real-world time to treatment discontinuation (rwTTD).
The researchers demonstrated the feasibility of aggregating data from various RWD sets to generate RWE and showed reasonable consistency in real-world response endpoints derived from these disparate data sources in patients with metastatic non-small cell lung cancer (mNSCLC). The study also highlights RWD’s ability to support specific use cases through real-world response, including when specific populations are underrepresented in clinical trials or to identify populations for future trials or label expansions on already approved therapies.
Why this matters
As use of real-world data to support drug development and regulatory decision making continues to be prioritized across the industry, aligning on methodologies for aggregating and analyzing this data is critical to ensure it is a reliable and consistent source of information. Additionally, this research demonstrates how cross-industry collaboration with health authorities like the FDA can advance RWE’s role in regulatory submissions and drive meaningful learnings for people with cancer.