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The European Medicines Agency adopted their Geriatric Medicines Strategy more than a decade ago. The strategy aims at elucidating the evidence basis for marketing authorization of new medicines which will be used in the older population, and at ensuring the appropriate communication of findings to the patient and healthcare provider. During the past decade new tools and data sources have emerged to support the strategy goals, and their use should be considered. Possible concrete actions are presented to improve the design of clinical trials, the data collection both pre‐ and post‐approval, the assessment of the findings, and the communication to assist informed prescription and safe medicine taking. Implementation and prioritization of these actions should be done from the perspective of addressing the needs of patients while maximizing efficient use of resources, with the aim of integrating geriatric aspects into routine medicines development and assessment. [ABSTRACT FROM AUTHOR]

Background: Over the last twenty years of orphan drug regulation in Europe, the regulatory framework has increased its complexity, with different regulatory paths and tools engineered to facilitate the innovation and accelerate approvals. Recently, the proposal of the new Pharmaceutical Legislation for the European Union, which will replace at least three Regulations and one Directive, was released and its new framework is raising many questions. The aim of this study was to present a characterisation of the Orphan Medicinal Products (OMPs) authorised by the European Commission (EC), between 2010 and 2022, looking into eighteen variables, contributing to the ongoing discussion on the proposal and implementation of the new Pharmaceutical Legislation proposed., Methods: Data of the OMPs identified and approved between 2010 and 2022 were extracted from the European Public Assessment Reports (EPARs) produced by the European Medicines Agency. Information regarding legal basis of the application, applicant, protocol assistance received, type of authorization, registration status, type of molecule, ATC code, therapeutic area, target age, disease prevalence, number of pivotal clinical trials supporting the application, clinical trial designs, respective efficacy endpoints and number of patients enrolled in the pivotal clinical trials were extracted. A descriptive statistical analysis was applied., Results: We identified 192 OMPs approved in the period between 2010 and 2022. 89% of the OMPs have legal basis of "full application". 86% of the sponsors received protocol assistance whereas 64% of the MAA benefited from the accelerated assessment. 53% of the active substances are small molecules; about 1 in 5 molecules are repurposed. 40% of the OMPs have oncological therapeutic indications and 56% of the OMPs are intended to treat only adults. 71% of the products were approved based on a single pivotal trial., Conclusions: This analysis of OMPs approved between 2010 and 2022 shows that a shift has occurred in the rare disease medicine development space. Through the period studied we observe an increase of non-small molecules approved, accelerated assessment received and non-standard MA's granted., (© 2024. The Author(s).)

Background: Understanding variability in prescribing patterns through comparative drug utilization studies can contribute to improve an efficient, effective and safe use of medicines., Objectives: To perform a cross-country comparison of consumption patterns of ambulatory high expenditure therapeutic groups between Portugal and six European countries and simulate potential cost-saving scenarios through the adoption of the different prescribing patterns of studied countries., Methods: Cross-country comparison of 2019 drug consumption patterns between Portugal, Denmark, England, Finland, the Netherlands, Norway, and Spain. Analysis comprised antihypertensive drugs, glucose lowering drugs (GLD), insulins, lipid lowering drugs (LLD) and oral anticoagulants. Cost-saving analysis were performed using the Portugal average annual cost/daily defined dose and the potential reduction in expenditure simulating other European countries consumption pattern scenarios., Results: Portugal had the lowest consumption uptake of metformin and the highest consumption of GLD (30.1%) and LLD (8.5% vs <3%) fixed-dose combinations. Annual cost-savings scenarios showed that Portugal would have saved between 53 M€ and 305 M€ if it had the same prescribing patterns than Norway or the Netherlands, respectively., Conclusions: Different utilization patterns across countries were found. Although Portugal has the lowest gross domestic product per capita among the countries studied, it had the highest uptake of newly and costly drugs.