BetaEntity Annotation Prototype
← Back to institutions

Annotated abstract

4CPS-068 Eighteen-month descriptive analysis of the paediatric population included in early access authorisation, compassionate use authorisation, compassionate program in a pharmacy at a cancer centre

ejhpharm · 2026-03-18 · canonical JSON source

12 visible annotations · policy: published · automated confidence ≥ 75.00%

Document resource

Background and Importance Early Access Authorisation (EAA), Compassionate use authorisation (CUA) and compassionate program (CP) are key regulatory pathways to provide innovative medicines to paediatric patients. Thanks to these programs many patients at our cancer centre have benefited from innovative treatments.Aim and Objectives The aim was to describe the characteristics of paediatric patients and medicines managed under EAA, CUA and CP in our hospital pharmacy over 18 months.Material and Methods We retrospectively collected all drugs provided by our pharmacy under EAA, CUA, CP between January 2024 and June 2025. Data included drug name, route of administration, regulatory status (EAA, CUA, CP) at dispensation and on 30 June 2025, indication. Dispensations were grouped by therapeutic area and analysed descriptively.Results Over the study period, 453 dispensations were done for 76 patients in the paediatrics committee (PC). This represents 8.6% of our population treated in these programs. A total of 10 drugs were used, mainly oral (9/10).PC had the most indications of all our committee with 19 different indications. The second was chest disease (CD) (18 indications for 103 patients) and haematology the third (16 indications for 80 patients).Regarding the regulatory status of dispensing, 12 indications were in CUA for PC (nine for CD , seven for haematology). This represented 12.2% of all our indications. As of 30 June 2025 we had still 12 indications in CUA (seven for CD, six for haematology).Two indications were in EAA at dispensation (2% of all our indication) (five for CD and seven for haematology) and only one on 30 June 2025.In PC, only one indication obtained reimbursement (two for CD and three for haematology).Conclusion and Relevance Our analysis highlights the major difficulties for access market in the paediatric population. PC is the only committee in our hospital that has not seen a decrease in the number of indications. Plus, only 5.2% and the indication in PC obtained a reimbursement on 30 June 2025. This highlights the need to collect more real-world data in paediatrics to facilitate assessments and market access. The development of new pharmaceutical forms adapted to paediatrics by our pharmacy may also be a lever to facilitate market access.Conflict of Interest No conflict of interest