Initiatives driving accelerated access to medicines in Europe: Review of recent concepts and developments:

摘要

Improving timely patient access to new medicines, particularly in areas with high unmet need, has been a healthcare priority during the past 5–10?years, with several new regulatory initiatives from the European Medicines Agency, as well as on national level within the European Union. Nevertheless, evidence suggests that medicines going through these regulatory initiatives experience variable reimbursement outcomes due to uncertainties in the clinical or economic evidence base. New initiatives, including the adaptive pathways concept, have therefore been introduced that embrace a holistic view of a medicine’s route to patient access. These involve expanded clusters of stakeholders working together to prospectively influence and design evidence generation strategies, including use of real-world evidence, to ensure that development plans meet the needs of multiple stakeholders including regulatory agencies and health technology assessment bodies. Multi-stakeholder dialogues, provided through scientific advice, are already available for medicines in Europe in various forms and are important tools for regulators, health technology assessment bodies and pharmaceutical companies to develop evidence generation plans optimised to support decision-making on marketing authorisation and reimbursement of new medicines. Multiple stakeholder groups have been actively engaged in advancing developments of initiatives driving timely access and it is likely to continue due to the need to balance this with affordability. The aim of this article is to provide a review of the latest, as well as a future perspective on, developments with respect to accelerated access of medicines in the European Union with a particular focus on procedures for formal scientific advice.