Frequency and Impact of Off-Label Biologic Prescriptions in Pediatric Rheumatology

Le résumé fourni par la source

Objectives Over the past 25 years, biologic disease-modifying antirheumatic drugs (bDMARDs) have dramatically altered pediatric rheumatology practice and treatment outcomes. However, many bDMARDs have no on-label indications for pediatric rheumatological conditions in North America. In clinical practice, this gap creates a significant barrier for care. The objectives of this study were to 1) assess the frequency of off-label bDMARD prescriptions across pediatric rheumatological conditions in patients followed at the Stollery Children’s Hospital and the Glenrose Rehabilitation Hospital in Edmonton, Alberta, and 2) assess the impact label indication has on funding approval. Methods A retrospective chart review was conducted on patients with bDMARDs prescriptions at our 2 outpatient pediatric rheumatology clinics. Charts were identified through a list created by nursing staff of all patients on bMARDs. Patients were included if the bDMARD was started at our clinic for prescriptions between January 2015 to June 2023; there was a pediatric rheumatologic indication, and the patient was ≤18 years old at time of prescription. Duplicates, unfilled prescriptions, and non-bDMARD prescriptions were excluded. We tabulated the Health Canada and the FDA online databases on-label indications of prescribed bDMARDs (as of March 2024). Prescriptions and indication status were reviewed individually by the first and senior author and then reviewed together. Descriptive analysis was done with categorical data summarized as frequencies and percentages and numerical data as means and standard deviations (SD). Results 215 charts and 378 prescriptions were included (Table 1). The median age at diagnosis was 8.75 with SD 4.59. Juvenile Idiopathic Arthritis (JIA) was the most common diagnosis (n=158, 73.5%). The most prescribed bDMARDs were adalimumab (n=188, 44.3%), etanercept (n=75, 19.8%) and Tocilizumab (n=35, 9%). Across all included bDMARD prescriptions, 50.5% (n=191) were for use in an off-label indication. 151 patients (70.2%) had no insurance denials for coverage while 64 (29.8%) had ≥ 1 denials. Table 1. Patient diagnoses, rheumatologic medications prescribed, bDMARDs prescribed, and insurance coverage denials for outpatient pediatric rheumatology patients prescribed a bDMARD. Conclusion At our pediatric rheumatology center in Alberta, 50.5% of bDMARD prescriptions were for an off-label indication. 29.8% of our patients had at least 1 denial of medication coverage. This indicates that this widening gap between clinical and labeling indications in daily clinical practice may contribute to challenges in patient care. Further analysis is underway to examine the impact of off-label prescriptions on the approval of different funding streams.

Ce résumé expose les affirmations des auteurs. BNTIC ne l’interprète pas comme une validation indépendante des résultats.