OBJECTIVES: A patient-centric approach is key for managing rare/orphan kidney diseases. In this disease area, the identification and use of sensitive biomarkers, beyond initial diagnoses, is crucial for improving patient care [1]. This research study aims to identify suitable biomarkers for potential development of personalized treatment algorithms. By identifying and select biomarkers that can sensitively and rapidly detect meaningful changes of laboratory signs and patient-most burdensome clinical symptoms, the management of those already treated diseases could be significantly enhanced. The approach could lead to improvement of disease and patient outcomes [2-4].

METHODS: Clinical trials focusing on rare/orphan nephrology diseases from 2018 onwards were screened and jointly reviewed with Climedo Health GmbH, resulting in over 200 potential biomarkers. To prioritize these biomarkers for feasibility assessment, a two-fold approach was employed. Firstly, the frequency of biomarker usage across trials was analyzed to identify commonly employed biomarkers. Secondly, post-market clinical trials were selected to assess biomarkers used in trials addressing available therapies.

RESULTS: Through leveraging public domain data, Google, and ChatGPT, complementing the search from Dr.Evidence, PubMed, Medline, Embase, Cochrane, ClinicalTrials.gov, nine biomarkers were prioritized to be considered for further analysis ("shortlist"). The preliminary shortlist is a combination of biomarkers search through data from their highest usage in clinical trials and from those used across prioritized indications.

CONCLUSIONS: In orphan/rare kidney diseases area the development and use of personalized treatment algorithms is still lacking, whereas this is common in other diseases, such as diabetes mellitus showing the benefits of ‘patient empowerment' and disease management. This research project intends to explore if a path is feasible through identified/selected biomarkers to generate personalized treatment algorithms. The outcome would be highly beneficial for people suffering from those poorly treated rare/orphan kidney diseases.