Dr Kim on the Background of a Real-World Study of the Use of Regorafenib in mCRC

Richard Kim, MD, service chief, Medical Gastrointestinal Oncology, senior member, Department of Gastrointestinal Oncology, Moffitt Cancer Center; professor, oncology, the University of South Florida College of Medicine, discusses the rationale for launching a real-world investigation of long-term responses with regorafenib (Stivarga) in patients with metastatic colorectal cancer (mCRC), highlighting the implications of this research that were presented at the 2024 Gastrointestinal Cancers Symposium.

The purpose of real-world evidence extends beyond mere validation of findings from phase 3 clinical trials, Kim begins. It serves as a complement to phase 3 data, offering a nuanced perspective on the long-term safety and efficacy of treatments in routine clinical settings, he says. A real-world analysis of regorafenib in patients with mCRC underscores the enduring utility and benefit of the agent in this patient population, particularly in specific subsets of patients who continue treatment for extended periods, often surpassing 4 or 5 months, Kim explains. What emerges from these real-world data is a reaffirmation of the positive outcomes observed in controlled clinical trials, he elucidates, which demonstrate continued effectiveness and durability of response over time.

Although real-world research has inherent limitations, notably the absence of clear correlations between treatment response and certain biomarkers, such as KRAS or BRAF, it nonetheless sheds light on key determinants of treatment success, according to Kim. Notably, in this real-world study, the influence of patient performance status emerged as a significant predictor of outcomes, emphasizing the importance of holistic patient care beyond mere molecular profiles, Kim explains.

Furthermore, this real-world evidence serves as a testament to the enduring efficacy of regorafenib in mCRC, Kim continues. Despite the 2012 FDA approval of regorafenib for patients with advanced CRC that has progressed following prior therapy, the drug continues to demonstrate tangible benefits for a notable proportion of patients, Kim says. This sustained efficacy, coupled with the absence of new safety concerns over extended periods, underscores the drug's enduring relevance and clinical utility in patients with mCRC, he imparts. In essence, this real-world evidence validates the findings of phase 3 trials and enriches understandings of treatment outcomes in diverse clinical scenarios, Kim notes. These findings provide reassurance to clinicians regarding the efficacy and safety of regorafenib and support its use in routine clinical practice, Kim concludes.