Clinical Trial Landscape: Rare Disease Patient Matching in CKD

Background: Slow recruitment of patients to clinical trials significantly increase overall study costs. Recruitment for rare kidney disease clinical trials is particularly slow due to difficulty in identifying eligible patients. Clinical trial support services (CTSS) and patient-centric trial designs can improve enrollment success. Here we compared how CTSS aided recruitment for two clinical trials (Trial A and Trial B) investigating treatment for the same rare kidney disease. Methods: CTSS, performed by Natera (Austin, TX) utilized an internal database of renal genetic test results to identify and clinically review potentially eligible patients. Providers of identified patients who provided consent for outreach at the time of testing were contacted via phone, fax or email, to further assess patient eligibility. Trial A used CTSS to select study site locations based on potentially eligible patient volume and proximity. Trial A provided travel arrangement assistance and required <10 in-person study visits. Trial B selected study sites independent of CTSS, did not provide travel arrangement assistance and required >12 in-person study visits. Both clinical trials used CTSS to target referral of 20 patients, to locate potential participants ≤50-miles of study sites and to facilitate ≥3 outreach attempts to each referring physician or patient. Results: Trial A had fewer study sites compared to Trial B (6 vs 14). CTSS reached 20 referrals in 6 months for Trial A and 9 months for Trial B, despite similar patient referral rates (Trial A: 1.3% [20/1552]; Trial B: 1.4% [49/3450]). Of the patients CTSS referred, Trial A had a lower rate of screen failure (31% [4/13] vs 89% [34/38]), shorter average time from referral to screening (73 days vs 113 days) and higher enrollment rate (69% [9/13] vs 11% [4/38]) than Trial B. Conclusion: CTSS, utilizing a database of patients who underwent renal genetic testing, successfully identified patients eligible for enrollment in clinical trials for rare kidney disease treatment. While CTSS provided similar screening and provider/patient contacts to each study, Trial A had a higher enrollment rate, lower screen fail, and faster referral time, suggesting that using CTSS to guide site selection and the incorporation of patient-centric features helps optimize study implementation. Funding: Commercial Support - Natera, Inc.