Real-World Data and Evidence in Lung Cancer: A Review of Recent Developments

Simple Summary The use of real-world data (RWD) to generate real-world evidence (RWE) is increasing in oncology. RWD studies provide valuable information to regulators, sponsors, and clinicians. RWD studies rely on collecting and analyzing observational data, offering insights into the practical application of cancer treatment in real-world settings. However, the quality of RWD can compromise the reliability of the RWE. Hybrid methodological analyses that combine the strengths of RCTs and RWD studies, known as R2WE, are being conducted to address these challenges. RWD sources include patient registries and electronic health records (EHRs). High-quality data are essential for generating credible RWE. To obtain RWD, it is necessary to obtain data from relevant sources, clean and harmonize the data, and ensure compliance with the laws and regulatory requirements.Abstract Conventional cancer clinical trials can be time-consuming and expensive, often yielding results with limited applicability to real-world scenarios and presenting challenges for patient participation. Real-world data (RWD) studies offer a promising solution to address evidence gaps and provide essential information about the effects of cancer treatments in real-world settings. The distinction between RWD and data derived from randomized clinical trials lies in the method of data collection, as RWD by definition are obtained at the point of care. Experimental designs resembling those used in traditional clinical trials can be utilized to generate RWD, thus offering multiple benefits including increased efficiency and a more equitable balance between internal and external validity. Real-world data can be utilized in the field of pharmacovigilance to facilitate the understanding of disease progression and to formulate external control groups. By utilizing prospectively collected RWD, it is feasible to conduct pragmatic clinical trials (PCTs) that can provide evidence to support randomized study designs and extend clinical research to the patient's point of care. To ensure the quality of real-world studies, it is crucial to implement auditable data abstraction methods and develop new incentives to capture clinically relevant data electronically at the point of care. The treatment landscape is constantly evolving, with the integration of front-line immune checkpoint inhibitors (ICIs), either alone or in combination with chemotherapy, affecting subsequent treatment lines. Real-world effectiveness and safety in underrepresented populations, such as the elderly and patients with poor performance status (PS), hepatitis, or human immunodeficiency virus, are still largely unexplored. Similarly, the cost-effectiveness and sustainability of these innovative agents are important considerations in the real world.