Frequency and Impact of Off-Label Biologic Prescriptions in Pediatric Rheumatology
Notice bibliographique
Résumé
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.
Récupéré en direct depuis OpenAlex et désinversé. Les résumés ne sont pas conservés dans cette base de données : les index inversés représentent 8,6 Go des 9,3 Go de texte de la base, et le serveur dispose de 13 Go libres.
Comment cette classification a été obtenuedéplier
Prédiction machine sur la base complète
Imitation des enseignantsNi prévalence calibrée, ni vérité terrain. Validation humaine à venir. Le volet Gemma est une étiquette directe du modèle pour chaque travail de la base, lue sur la notice réduite au titre. Le volet Codex est un classifieur appris des 10 348 étiquettes directes de Codex et calibré sur les taux pondérés de l'échantillon; les champs sans appui suffisant ne portent aucun appel Codex. Le mode candidate est l'union des deux volets; le consensus est leur intersection. Ces sorties portent le statut machine_predicted_unvalidated et ne sont pas des étiquettes humaines.
Scores du classifieur distillé par catégorie (deux têtes)
| Catégorie | Codex | Gemma |
|---|---|---|
| Métarecherche | 0,002 | 0,012 |
| Méta-épidémiologie (sens strict) | 0,000 | 0,000 |
| Méta-épidémiologie (sens large) | 0,000 | 0,000 |
| Bibliométrie | 0,002 | 0,003 |
| Études des sciences et des technologies | 0,000 | 0,000 |
| Communication savante | 0,001 | 0,001 |
| Science ouverte | 0,000 | 0,001 |
| Intégrité de la recherche | 0,000 | 0,001 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,002 | 0,000 |
Scores machine (provisoires)
Les deux têtes enseignantes du modèle étudiant, lues sur ce travail. Un score ordonne la base pour la relecture; il n'affirme jamais une catégorie, et le statut de validation accompagne chaque rangée tel quel.
Scores de référence d'un modèle non mature (critères de maturité non atteints, 7 itérations). Un score ordonne; il n'affirme jamais une catégorie.
score_only:v0-immature-baseline · tel quel depuis la passe de notation : score_only signifie que le nombre peut ordonner les travaux, et qu'aucune étiquette de catégorie n'en découleClassification
machine, non validéePrédiction automatique; un appel candidat d’une seule source (Gemma direct ou Codex distillé), pas un consensus.
Le détail, modèle par modèle et score par score, se trouve en fin de page sous « Comment cette classification a été obtenue ».