Publications by authors named "T Beukelman"
Generating Real-World Evidence From the Excellence Network in Rheumatology.
Pharmacoepidemiol Drug Saf
December 2024
Purpose: The Excellence Network in RheumatoloGY (ENRGY) was founded in 2021 and encompasses data from more than 700 private practice rheumatology providers throughout the United States, forming a practice-based research network (PBRN).
Methods: Electronic health record (EHR) data from participating practices are aggregated, including structured data (e.g.
Article Synopsis
- The CRISPR gene editing therapy exagamglogene autotemcel (exa-cel) was recently FDA-approved for patients with severe sickle cell disease (SCD) due to its 97% efficacy in reducing vaso occlusive crises (VOCs) over a year demonstrated in a phase III trial.
- A study using Medicaid claims data found that only 7.7% of a cohort of severe SCD patients remained VOC-free over a one-year period in routine clinical care, highlighting a significant difference from the trial results.
- The findings suggest that if exa-cel's efficacy is replicated outside of clinical trials, it could greatly improve health outcomes and public health for those living with severe SCD.
Purpose: To facilitate claims-based research on populations with juvenile idiopathic arthritis (JIA), we sought to validate an algorithm of new medication use as a proxy for worsening JIA disease activity.
Methods: Using electronic health record data from three pediatric centers, we defined new JIA medication use as (re)initiation of disease-modifying antirheumatic drugs or glucocorticoids (oral or intra-articular). Data were collected from 201 randomly selected subjects with (101) or without (100) new medication use.
Article Synopsis
- The study aimed to compare the effectiveness of a second tumor necrosis factor inhibitor (TNFi) versus a non-TNFi biologic in children with polyarticular-course juvenile idiopathic arthritis (pJIA) after discontinuing the first TNFi due to ineffectiveness.
- 216 patients were analyzed, with 85% starting a second TNFi, and the most common biologic switches being adalimumab for TNFi and tocilizumab for non-TNFi.
- Results showed no significant differences in disease activity after six months, suggesting that both treatment options may be equally effective for managing pJIA.
Objective: The objective of this study was to determine the proportion of new medication prescriptions observed in electronic health records (EHR) that represent true incident medication use, accounting for undocumented previous prescriptions (prevalent medication use) and failure to initiate treatment (primary nonadherence) with linked administrative claims data as the reference standard.
Methods: Using single-specialty rheumatology EHR data from more than 700 community practices in the United States linked to administrative claims data, we identified first (index) EHR prescriptions and assessed the positive predictive value (PPV) of different EHR-derived new user definitions to identify true incident use (no prior claims). We then assessed how often index EHR prescriptions that met a definition of new use resulted in primary nonadherence (no subsequent claims).