Background: Medication errors are a widespread problem which can, in the worst case, cause harm to patients. Errors can be corrected if documented and evaluated as a part of quality improvement. The Danish community pharmacies are committed to recording prescription corrections, dispensing errors and dispensing near misses. This study investigated the frequency and seriousness of these errors. Methods: 40 randomly selected Danish community pharmacies collected data for a defined period. The data included four types of written report of incidents, three of which already existed at the pharmacies: prescription correction, dispensing near misses and dispensing errors. Data for the fourth type of report, on adverse drug events, were collected through a web-based reporting system piloted for the project. Results: There were 976 cases of prescription corrections, 229 cases of near misses, 203 cases of dispensing errors and 198 cases of adverse drug events. The error rate was 23/10 000 prescriptions for prescription corrections, 1/10 000 for dispensing errors and 2/10 000 for near misses. The errors that reached the patients were pooled for separate analysis. Most of these errors, and the potentially most serious ones, occurred in the transcription stage of the dispensing process. Conclusion: Prescribing errors were the most frequent type of error reported. Errors that reached the patients were not frequent, but most of them were potentially harmful, and the absolute number of medication errors was high, as provision of medicine is a frequent event in primary care in Denmark. Patient safety could be further improved by optimising the opportunity to learn from the incidents described.

Background: Medication errors can have serious consequences for patients, and medication safety is essential to pharmaceutical care. Insight is needed into the vulnerability of the working process at community pharmacies to identify what causes error incidents, so that the system can be improved to enhance patient safety. Methods: 40 randomly selected Danish community pharmacies collected data on medication errors. Cases that reached patients were analysed, and the most serious cases were selected for root-cause analyses by an interdisciplinary analysis team. Results: 401 cases had reached patients and a substantial number of them had possible clinical significance. Most of these errors were made in the transcription stage, and the most serious were errors in strength and dosage. The analysis team identified four root causes: handwritten prescriptions; ''traps'' such as similarities in packaging or names, or strength and dosage stated in misleading ways; lack of effective control of prescription label and medicine; and lack of concentration caused by interruptions. Conclusion: A substantial number of the medication errors identified at pharmacies that reach patients have possible clinical significance. Root-cause analysis shows potential for identifying the underlying causes of the incidents and for providing a basis for action to improve patient safety.

Background Utility studies enable preference-based quantification of a disease’s impact on patients’ health-related quality of life (HRQoL). It is often difficult to obtain utility values for rare, neurodegenerative conditions due to cognitive burden of direct elicitation methods, and the limited size of patient/caregiver populations. CLN2 disease (neuronal ceroid lipofuscinosis type 2) is an ultra-rare, progressive condition, for which there are no published utility data fully capturing all disease stages. This case study demonstrates how utility values can be estimated for ultra-rare paediatric diseases by asking clinicians to complete EQ-5D-5L questionnaires based on vignettes describing the stages of CLN2 disease. Methods An indirect elicitation method using proxy-reporting by clinical experts was adopted. Eighteen vignettes were developed, describing nine progressive disease stages as defined by motor and language domain scores of the CLN2 Clinical Rating Scale, in individuals treated with cerliponase alfa or standard care. Eight clinical experts with experience of treating CLN2 disease with cerliponase alfa and current standard care completed the proxy version 2 EQ-5D-5L online after reading these vignettes. Resulting scores were converted to EQ-5D-5L utility values for each disease stage, using UK, German and Spanish value sets. Results Utility values, which are typically anchored by 0 (equivalent to death) and 1 (full health), decreased with CLN2 disease progression (results spanned the maximum range of the utility scale). Assigned utility values were consistently higher for patients receiving cerliponase alfa than standard care; differences were statistically significant for the 6 most severe disease stages (p < 0.05). Analysis of the individual dimensions of the EQ-5D-5L showed that greatest differences between patients treated with cerliponase alfa and standard care occurred in the pain dimension (differences in mean scores ranged between no difference and 1.8), with notable differences also observed in the anxiety/depression dimension (differences in mean scores ranged between 0.1 and 1.0). Conclusions This study demonstrates a feasible methodology for eliciting utility values in CLN2 disease, indicating HRQoL declines with disease progression. Vignettes describing patients receiving cerliponase alfa were consistently assigned higher utility values for the same disease state, suggesting this treatment improves HRQoL compared with standard care. Trial registration NCT01907087, NCT02485899.

Background Cerliponase alfa, an enzyme replacement therapy for neuronal ceroid lipofuscinosis type 2 (CLN2), is currently available in England through a managed access agreement (MAA). It is administered every 2 weeks via an intracerebroventricular device. Here we report qualitative research with families of children with CLN2 disease and healthcare professionals (HCPs) who run the MAA, to understand how access to cerliponase alfa via the MAA at Great Ormond Street Hospital (GOSH) in London, and the overall management of CLN2 disease, was affected during the coronavirus disease 2019 (COVID-19) pandemic. Methods Telephone interviews were conducted with nine families, representing 11 children with CLN2 disease, and two HCPs in November and December 2020. Results Children had received cerliponase alfa treatment for a mean (SD) of 23.1 ± 24.7 months (7.1 ± 4.6 months in the MAA). Families travelled 7–398 km for treatment (mean 210 ± 111 km). Treatment with cerliponase alfa was designated “essential” by GOSH and continued as normal during the pandemic but with extra safety precautions, and no children missed any treatments. Families were highly motivated to continue treatment, despite considerable anxiety about the risk of coronavirus infection from travelling and staying overnight but were reassured by communications from GOSH and the safety precautions put in place. Support therapy services were widely compromised, causing families concern about deterioration in their children’s condition. Families were confused about COVID-19 testing and shielding, and were unclear whether children with CLN2 disease were vulnerable to COVID-19. Conclusions Looking forward, advice for children with CLN2 disease should be specific and tailored, taking into account the family unit. Support therapies should be considered essential alongside cerliponase alfa treatment.