174PHow can we analyze natural history across multiple sites without moving the data? - a feasibility study and early pilot using PUL 2.0

Abstract

Analysis of Real-World Data from patients with DMD has proven pivotal in advancing understanding of natural history, which in turn has led to better clinical trial design and analysis. However, natural history can evolve. With both care standards and the availability of new therapies evolving differently across centers and geographies, it is imperative that comparisons of outcomes are understood in the context in which they were observed. To address these concerns, cTAP engaged The Hyve, a knowledge engineering consultancy, to investigate collaboration via federated analyses, an approach that enables analyses of data without moving data outside organizations and geographies. A feasibility study was conducted through semi-structured interviews with four geographies assessing interoperability along three axes: data, technology, and governance. Feasibility was ranked as Low, Medium, or High based on a qualitative assessment of the extent of changes required to processes, technologies or data. A pilot study adapted analytical code to the data model used at LUMC which was then run against patient data at that center. A federated framework is feasible. For data, sites collect similar data and use the same functional assessments. For technology, there is no existing end-to-end solution, but there are components available that can be assembled. For governance, sites are fully compliant and require no change. cTAP will need to formalize current manual processes for securing iterative site approval of analyses to ensure an auditable governance. Execution of the code in the pilot study generated a prognostic model for PUL 2.0 in a single run with no errors. Enabling important scientific analyses requiring multi-institutional collaboration to be addressed is feasible. A decade of successful multi-institutional collaboration with cTAP lowers the barrier to execution.