gms | German Medical Science

Background: Children and adolescents are disadvantaged in terms of drug treatment. 42-90% of hospitalised paediatric patients are treated with medications outside their product licence (off-label use); in the outpatient sector, 46-64% of patients are affected [1], [2]. The aim of the present study was to analyse the licensing status of drug prescriptions in the outpatient clinics of a paediatric university hospital in Germany and to determine differences.

Materials and Methods: A retrospective, monocentric cross-sectional study was conducted to assess drug prescriptions regarding their licensing status in one general (GPOC) and nine specialised (SPOCs) outpatient clinics of a university children’s hospital in Germany. All patients less than 18 years of age, who received at least one electronic drug prescription in one of the 10 outpatient clinics in the period from 1st to 31st October 2019, were included. The prescription data were systematically retrieved from the patients records and manually assessed for their licensing status. In-label drug use was defined as drug utilisation according to its marketing authorisation as described in the relevant Summary of Product Characteristics (SmPC). The definitions for off-label and unlicensed use were adopted from Neubert et al. [3]. Relative frequency and prevalence of off-label and unlicensed prescriptions were calculated and stratified by GPOC and SPOCs. Furthermore, the reasons for off-label prescribing were determined.

Results: 1438 prescriptions (GPOC n=333, SPOCs n=1105) of 786 patients (GPOC n=222, SPOCs n=564) were assessed and classified according to their licensing status.

The overall relative frequency of off-label prescriptions was 43.9%, 3.1% of prescriptions were unlicensed. The proportion of off-label prescriptions was significantly higher in the SPOCs than in the GPOC (45.5% vs. 38.4%, p=0.022).

Overall, a total of 53.1% of patients received at least one off-label prescription. The prevalence of off-label prescriptions was 50.5% in the GPOC and 54.1% in the SPOCs, respectively (p=0.359).

The substances most commonly prescribed off-label in the GPOC were antiinflammatory and antirheumatic products (ATC: M01), antibacterials for systemic use (ATC: J01) and nasal preparations (ATC: R01), while antiepileptics (ATC: N03), vitamines (ATC: A11) and drugs for obstructive airway diseases (ATC: R03) dominated in the SPOCs.

The comparison revealed the same high-ranking reasons for off-label use: Dosing, indication and missing paediatric information.

Conclusion: Off-label prescribing plays an important role in everyday clinical practice in paediatrics. While data from the GPOC are comparable to outpatient treatment, off-label prescribing in the SPOCs appears to be equivalent to inpatient treatment due to the complexity of diseases treated there. Further efforts are therefore needed to generate more evidence-based knowledge in the field of paediatric pharmacotherapy to provide children with the best possible treatment in outpatient settings, too.