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P25 Unlicensed thalidomide in paediatrics: ensuring pregnancy prevention programme compliance

bmjpo · 2026-04-09 · canonical JSON source

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Aim Thalidomide is a known teratogen, licensed in the UK for treating adults with multiple myeloma. 1 2 Despite its severe teratogenic potential, it is also used off-label in paediatric patients with dermatological and gastrointestinal disorders. According to manufacturer requirements, all females, including pre-pubertal girls, must be treated as women of childbearing potential (WCBP), necessitating full adherence to the Pregnancy Prevention Programme (PPP) unless they have an exemption. Historically, compliance with the PPP in some paediatric tertiary hospital was inconsistent, particularly in ensuring timely completion of the Prescription Authorisation Form (PAF) and adherence to safety steps.3 The aim of this project was to improve PPP compliance in paediatric patients who were prescribed thalidomide and strengthen safeguards in this vulnerable population.Method In a single centre paediatric hospital a multidisciplinary pharmacy focus group, including technicians, dispensary pharmacists, deputy chief pharmacist, and clinical leads in dermatology and gastroenterology was convened to review current workflows and identify barriers to PPP compliance. Key issues included delays in receiving completed PAFs, inconsistent documentation of pregnancy tests, and lack of clear prescribing pathways.As a result, a streamlined workflow, and Standard Operating Procedure (SOP) were developed. This ensured that prescriptions were processed in accordance with the manufacturer’s PPP, with safeguards built into the electronic prescribing system. These included mandatory documentation of negative pregnancy test results within 3 days of prescribing and PAF completion before a prescription could be activated.The proposed workflow and SOP were subsequently reviewed at the Trust’s Pharmacy Clinical Governance meeting, gaining approval from the chief pharmacist and wider stakeholders. Targeted education sessions were then delivered to pharmacy and clinical teams to ensure awareness and engagement across the medication pathway from prescription to dispensing.Results Implementation of the new workflow led to significant improvements in compliance and patient safety:100% of patients received their thalidomide within 7 days of an active prescription.All WCBP patients had documented negative pregnancy tests within 3 days of prescribing.No prescriptions were dispensed without a clinically screened and approved PAF.Every patient was counselled on the risks of thalidomide at each point of dispensing.Conclusion Full compliance with the thalidomide PPP has been achieved in a paediatric tertiary care setting through multidisciplinary collaboration and system-based interventions. Despite thalidomide’s unlicensed status in children, regulatory safeguards must be applied, particularly for pre-pubertal females considered WCBP. Digital prescribing systems have been instrumental in embedding mandatory checks, ensuring no prescription bypasses PPP requirements. Engagement from prescribers and the wider MDT remains essential to maintain high safety standards for paediatric patients receiving high-risk medications. This project highlights the critical role of pharmacy teams, from procurement to clinical screening in enforcing safe medication practices. In particular, the involvement of pharmacists is vital, given their expertise in navigating unlicensed medicine use, coordinating care, and ensuring that safety protocols are applied across all stages of medication pathways.References National Institute for Health and Care Excellence (BNFc). Available online at: BNFC (British National Formulary for Children) | NICE. [Accessed date: 03/07/2025].Electronic medicines compendium (EMC). Thalidomide BMS 50mg Hard capsules. Available online at: Thalidomide BMS 50 mg Hard Capsules - Summary of Product Characteristics (SmPC) - (emc) | 6317. [Accessed date: 06/07/2025].Cooper AM, Horwitz M, Becker ML. Improving the safety of teratogen prescribing practices in a pediatric rheumatology clinic. Pediatrics 2019;143(4).