Assessment of off-label use of baricitinib in the paediatric population (Paediatric use of baricitinib)

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/44308
• EU PAS number:
  EUPAS24371
• Study ID:
  44308
• Official title and acronym:
  Assessment of off-label use of baricitinib in the paediatric population (Paediatric use of baricitinib)
• DARWIN EU® study:
  No
• Study countries:
  United Kingdom
• Study description:
  Rationale and background: Baricitinib is approved in Europe for the treatment of rheumatoid arthritis (RA) in adults. Although the potential for off-label use in paediatric patients is considered low, baricitinib has not yet been studied in children and adolescents and hence its use in this population is classified as a safety concern (missing information) in the EU-Risk Management Plan for baricitinib. Lilly has proposed this study in order to provide a more systematic evaluation of use in children and adolescents in Europe. Understanding the proportion of baricitinib prescribing that is off-label to children and adolescents will help quantitate the level of this safety concern. Research question and objectives: This study’s primary objective is to evaluate the proportion of baricitinib prescribing that occurs off-label in paediatric patients (defined as less than 18 years of age). As a secondary objective, if paediatric use is ≥5 patients, this study aims to describe paediatric patients who receive a prescription for baricitinib in terms of total number of patients, demographics (age and sex) and select baseline diagnoses codes. Study design: This observational study will use a descriptive cohort design to characterize off-label prescribing of baricitinib in United Kingdom (UK) paediatric patients. Population: The setting is general care practices within the UK, limited to the practices that contribute to the Clinical Practice Research Datalink (CPRD). Patients will be retrospectively selected from the CPRD (both GOLD and Aurum) based on first occurrence of baricitinib within the database from April 2017 (date of baricitinib launch in the UK) to June 2020.
• Study status:
  Ongoing

Research institution and networks

Institutions

Eli Lilly and Company

Study timelines

Date when funding contract was signed

Planned: 30/06/2018

Actual: 30/06/2018

Study start date

Planned: 01/10/2019

Actual: 29/08/2019

Date of final study report

Planned: 31/03/2023

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Eli Lilly and Company

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  EU RMP category 3 (required)