in lung cancer
Introduction:
Randomized clinical trials (RCTs) of novel treatments for solid malignancies typically measure disease progression using the Response Evaluation Criteria in Solid Tumors (RECIST) with precisely timed patient assessments relative to treatment administration.
However, structured information on disease progression is not typically available in data routinely collected to monitor health status or healthcare delivery such as electronic health records, medical claims, disease registries and other patient-reported data (“real-world data”). Thus, limiting our ability to advance clinical effectiveness of cancer therapies outside of RCTs.
Novel approaches to estimate cancer outcomes at scale may be useful to advance the use of real-world data for the acceleration of cancer research.
A literature review was conducted to summarize the current methodologic approaches to approximate real-world disease progression using lung cancer as an illustrative example.
Here we describe the approaches used, discuss challenges and limitations of each approach, and propose future directions.
Methods:
A methodical literature review was conducted in PubMed to identify approaches of estimating disease progression in patients with lung cancer.
Only English articles were considered between 2008-2022.
The search terms used included: “lung cancer”, “non-small cell lung cancer”, “real world disease progression”, “methods to estimate progression”, “volumetric delineation”, “tumor volume”, “radiomics”, “image segmentation”, and “tumor response”.
Data abstracted from each article (if available) included source of clinical data used, description of the method used to estimate real-world progression, whether the real-world endpoints were compared to a gold standard and the results of that comparison, and the definition of the gold standard.
Results:
Figure 1. Distribution of articles by type of method identified.
Table 1. Examples of definitions to estimate real-world disease progression.
Figure 2. Comparison of real-world disease progression to proposed gold standard (n=27 studies).
Table 2. Advantages and disadvantages of the approaches to estimate real-world disease progression.
Conclusions
A significant amount of literature exists relating to methods for curating real—world disease progression in patients with lung cancer.
However, additional research is needed to further develop, validate, harmonize, and scale these measures to truly drive discovery in the analysis of real-world data.
Consensus on gold standard definitions beyond RECIST is needed to create manually curated data sets for training and validating across centers.
Studies are needed to compare different methods within the same patient population in order to identify an optimal method for estimating real-world progression with high effectiveness and accuracy.
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