Preliminary Attainability Assessment of Real-World Data for Answering Major Clinical Research Questions in Breast Cancer Brain Metastasis: Framework Development and Validation Study
- The study proposes the PAR framework for data attainability screening in clinical research, particularly useful for addressing unmet clinical needs in BCBM.
- A survey showed clinical questions had a mean score of 4.37, indicating the significance of these questions.
- RWE generation is crucial in BCBM due to limited clinical trials, but challenges like incomplete gold standards and unpredictable "hidden labor" exist.
- The PAR framework helps assess research feasibility by matching research variables with data fields, refining the data scope before analysis.
- The framework enhances reproducibility and reusability of research processes, especially when using common data models like OMOP and i2b2.
- Challenges identified include deficiencies in exact data variables and difficulties in extracting key variables from EHRs, suggesting the need for surrogate definitions or integrating other data types.
- The study's limitations include its focus on a single clinical domain (BCBM) and data source (SMC BCR), indicating the need for further application in different domains.
- The PAR framework is associated with improved efficiency and quality in RWD-based clinical research by enhancing transparency and reproducibility.
Brain metastasis (BM) is a major cause of mortality in patients with breast cancer, and it increases the difficulty of treatment. Advancements in treatment and the development of brain imaging technology have increased the survival of patients with metastatic breast cancer, leading to an increased incidence of BM. Nonetheless, the opportunity to participate in prospective randomized clinical trials (RCTs) is typically only available to a limited number of patients with breast cancer brain metastasis (BCBM).
Design challenges, including heterogeneity of patients, varying definitions of clinical end points, and different methods to assess these end points, have led to excluding most patients with BCBM from RCTs. Consequently, while the incidence and survival duration have expanded, clinical research methods for BCBM remain limited...
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