P12.52.A REAL-WORLD DATA COLLECTION FOR LOWER GRADE GLIOMA: EXPERIENCE FROM AROUND THE WORLD

Abstract BACKGROUND The study of rare diseases remains a scientific challenge. While randomized clinical trials (RCT) are the gold standard design for the evaluation of treatment strategies, they are expensive and limited by a small number of selected participants and endpoints. Lower grade glioma (LGG) is a rare brain tumor of young to middle aged adults. The disease is generally fatal and has one of the highest mean years of potential life lost. Over the past 40 years, survival times have remained unchanged and optimal treatment plans remain unclear. The paradigm of an RCT is difficult to implement in LGG given the slow disease course and the small number of patients. Real-world registries collect clinical, epidemiological, and biological data and have the potential to generate new evidence as they benefit from a broader and more representative patient population. They represent a cost and time efficient pragmatic study design uniquely suited to address questions of interest to patients and researchers. They help to understand the incidence and natural history of rare diseases, as well as treatment selection and outcomes by geography. Method: A Real-world evidence (RWE) Glioma Council was formed with representatives of established brain tumor registries in the US, Australia, Spain, Germany and Austria in 2024. These international registries include a broad range of study design, source data (e.g. hospital, patient organization, social media) and variables. Data are captured either from direct interviews, online questionnaires, or electronic medical records, and include clinical, epidemiological, and quality of life data. The RWE Glioma council assembled to share their experiences, and to discuss the aims and structure of their registries. The BRAIN (Australia), LGG (USA), and RETSINE (Spain) registries’ data dictionary were compared to identify and harmonize common data elements. RESULTS A core group of variables that were felt critical and likely able to be collected across countries was identified (including demographics, treatment, and survival outcomes). A secondary group of variables that were of value but more difficult to collect were also noted. For each variable, the RWE Glioma Council suggested data collection time point, format and entry values. They also identified best practices (e.g. methods of data collection, programmed questionnaires, software packages) relevant for registry build. CONCLUSION To our knowledge, this is the first combined effort across RWE registries to engage, enroll, and study patients with glioma. The harmonized data dictionary resulting from this work provides a robust basis for researchers aspiring to create their own registries and sets a relevant benchmark for glioma registries in development. Further research into compiling existing data sets is underway as is the development of toolkits for those wishing to undertake such registries in the future.