Improving the data quality of spontaneous ADR reports: a practical example from Malta.

BACKGROUND: Adverse drug reaction (ADR) reporting rates and high-quality data within case summary reports are crucial to detect emerging safety concerns and implement regulatory action. In this study we aimed to improve the data quality and reporting rates of ADR reports in Malta through a series of national activities. RESEARCH DESIGN AND METHODS: Between April 2018 and July 2019, we carried out the following activities: i) a review of wholesale dealers ADR reporting forms; ii) a series of educational workshops targeting physicians and pharmacists; iii) a quality system audit of the Authority's ADR management process. RESULTS: Twelve wholesaler dealer forms were reviewed, and 155 improvements were identified. Incident reporting forms modified to capture ADRs had the most opportunities for improvement. Five workshops were organized and in total 62 physicians and 22 pharmacists attended. Although feedback from participants was positive, in our case, an increase in reporting was not observed following the workshops. The quality system audit resulted in the introduction of the 'four-eye principle' to the Authority's ADR management process. CONCLUSIONS: The implementation of such activities is expected to contribute to the overall pharmacovigilance systems in Malta and our experience could benefit other entities involved in spontaneous ADR reporting.

Treatment of Leber's hereditary optic neuropathy: An overview of recent developments.

Leber's hereditary optic neuropathy (LHON) is a rare, maternally-inherited optic neuropathy caused by mitochondrial DNA point mutations and which can cause blindness. Currently, Raxone (idebenone) is the only available medicinal product authorised to treat LHON within the European Union and LHON remains an unmet medical need. The aim of this article was to summarise interventional clinical trials published over the past 5 years (between 2014 and 2019) with the primary purpose of treating LHON. Therapeutic approaches discussed include modulating agents of the mitochondrial electron transport chain such as Raxone, cysteamine bitartrate and KH176, inhibitors of apoptosis such as elamipretide, gene therapy medicinal products such as GS010 and scAAV2P1ND4 and retinal tissue regeneration medicinal products such as bone marrow-derived stem cells.