Real-world evidence (RWE) studies are crucial for newly approved medications. Randomized controlled trials (RCTs) are the gold standard for evidence when testing efficacy. Real-world studies allow understanding of how the medication is used in everyday clinical practice and how adherence may impact effectiveness. Real world studies complement RCT data by addressing gaps and offering insights that are critical for patients, healthcare providers, regulators, and policymakers.

With support from Ardelyx, Arbor Research is investigating several aspects of real-world utilization of tenapanor (Xphozah®), a medication recently approved by the United States Food and Drug Administration (FDA) to treat hyperphosphatemia in dialysis patients. With access to large databases of electronic health record (EHR) data representing over 45,000 dialysis patients and multiple dialysis providers in the US, Arbor Research is well-positioned to identify and analyze over 1,000 tenapanor initiators within one year of approval. The goals of this study are two-fold:

Part 1: In a single arm study of tenapanor initiators, characterize real-world utilization - including rate of uptake, characteristics of initiators, dose trajectories, time on therapy, concomitant phosphate binder utilization, and overall phosphate-lowering pill burden - and investigate short-term effectiveness in lowering serum phosphorus levels.
Part 2: Compare real-world effectiveness of tenapanor to traditional phosphate binder therapy by utilizing an active comparator arm of patients already using a traditional phosphate binder and initiating use of another non-tenapanor phosphate binder.

Arbor Research has the technical capabilities to not only carry out these analyses reliably and efficiently, but to do so with a very large database reflecting a unique combination of US dialysis provider EHRs with minimal lag time (<1 month) from measurement to analysis. Arbor Research is well-experienced working with sponsors to investigate a variety of aspects of real-world utilization for newly approved medications, including treatment patterns (Karaboyas 2022 AJKD) and comparative effectiveness (Karaboyas 2022 Kidney Medicine).

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