Major objections in EU medicines authorisation applications: an analysis of dossier submissions by small- and
Eglantine Legros1, Leonor Enes1, Constantinos Ziogas1
1European Medicines Agency, Amsterdam, the Netherlands.
Abstract:
Small and medium-sized enterprises (SMEs) are an important source of new and innovative medicines, particularly for rare diseases and unmet medical needs, yet they frequently encounter regulatory challenges. We analysed major objections raised by the EMA's Committee for Medicinal Products for Human Use in the Lists of Questions for 63 EU marketing authorisation applications submitted by SMEs, with outcomes between 2020 and 2023. Most major objections related to quality and clinical aspects, notably trial design or clinical efficacy data. Compared with larger companies, SME applications had lower success rates, although outcomes have improved over time. Early regulatory engagement and scientific advice are key to mitigating these deficiencies.
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