Rationale and design of the rheumatoid arthritis non-responders to treatment (RANT) study: Use of a bioinformatics platform and “decentralized” clinical recruitment design

Abstract

The Rheumatoid Arthritis Non-responders to Treatment (RANT) study (https://clinicaltrials.gov/study/NCT05447182) uses a novel bioinformatics platform to facilitate a “decentralized” patient oriented clinical study. The goal is to identify genetic factors associated with inadequate response to biologic and targeted synthetic rheumatoid arthritis (RA) medications. The study was advertised through social media, research website postings, and targeted recruitment of potentially eligible patients in academic practices and existing RA research registries. Interested RA patients who failed two or more biologic or targeted synthetic RA therapies completed a self-directed online informed consent and questionnaires on RA treatment history and granted access to electronic health records through a third-party vendor. After confirming eligibility by reviewing participant responses, participants received a blood sample kit by mail, had blood sample tubes drawn locally, and returned the kit by mail to the study genomic lab for whole genome sequencing. The study enrolled 164 eligible patients. The majority of eligible participants were identified through an established RA research registry (n = 101, 61.6 %) and through clinic recruitment at academic practices (n = 47, 28.7 %). Of the participants who were mailed a blood kit, 118/130 (90.7 %) returned the kit with blood sample by mail. Clinical and genetic factors of RA treatment non-responders will be compared to RA treatment responders from established research cohorts. This study demonstrates the feasibility of decentralized patient-oriented clinical studies in medicine, and highlights the importance of “traditional” recruitment methods like patient registries and academic practices. Future study results will identify potential clinical and genetic predictors of treatment refractory RA.