(A-235) Evaluation of Effectiveness of Additional Risk Minimisation Measures (aRMMs): perspectives from a UK non-interventional post authorisation safety study (PASS)

Background: Glycopyrronium Bromide (GB) 1mg/5ml oral solution is licensed for the treatment of severe sialorrhoea in children aged ≥ 3 years with chronic neurological conditions. Additional risk minimisation measures (aRMMs) were proposed to minimise the important risks, including off-label use. These aRMMs included a healthcare professionals (HCPs) checklist and a reminder card for caregivers, distributed by the Marketing Authorisation Holder to potential prescribers. As the product is not intended to reduce serious morbidity or mortality, the threshold for an acceptable level of risk is low when considering the benefit-risk profile of the drug.

Objectives: Monitoring the effectiveness of aRMMs for GB in a non-interventional prospective cohort study

Methods: Patients (pts) < 18 years initiated on GB 1mg/5ml as part of routine clinical care for sialorrhoea were identified by paediatric teams in participating hospital trusts. Pt/parent/guardian consent was obtained, and information extracted from the pt’s medical notes onto electronic data capture forms (eDCFs) at baseline and at 3, 6, 9 and 12 months post 1st prescription (i.e., secondary data collection). Pts or their parents/guardian/carer were invited to submit eDCFs at 6 and 12 months (i.e., primary data collection) and the pt’s GP at 12 months. Data on drug utilisation, adverse events, and receipt and use of aRMMs were collected.

Results: Twelve sites participated in the study and of these eight sites recruited 18 eligible pts. Three of the eight sites (37.5%) reported receiving the checklist for HCPs and/or the reminder card for caregivers. The HCPs that had received the reminder card for caregivers reported giving this to the parent/carer/pt. Although all pts were invited to contribute information relating to their experience of using GB, including AE data, only 3 pts (or parents/guardian/carer) submitted this information. Off-label use was identified in seven patients (38.9% of cohort): n < 5 pts were < 3 years old at GB initiation and n < 5 pts had been prescribed GB for the off-label indication of mild to moderate sialorrhoea. At the last data lock point the most frequently reported important identified risk was constipation (n=7, 38.9%).

Conclusions: This PASS provides quantitative measures for evaluating aRMM effectiveness and highlights challenges faced. Low rates of receipt of educational tools were reported by HCPs illustrating the difficulties of successful dissemination. The Revision 3 of GVP module XVI emphasises the need for pt reported outcomes however in this study pt responses were low. Qualitative research methods such as HCP and pt focus groups may help to better understand the barriers for effective aRMMs.