Extension of therapeutic indication and marketing authorization refusal – key aspects in EMA procedures during the period 2022-2024

European pharmaceutical legislation includes the assessment of key quality, efficacy, and safety parameters during CHMP meetings. Marketing authorization refusals are an important element of the Committee's work, and an analysis of these refusals for the period 2022-2024 clearly shows deficiencies in the design and conduct of clinical trials as a major cause. The low proportion of refusals (4%) compared to positive opinions testifies to the good communication between the Agency and the applicants, as well as to the effective implementation of various initiatives such as scientific advice and others. The extension of therapeutic indications, as an approach of the CHMP for timely patient access to treatment, supports the process of drug repositioning. The study on extension recommendations for the same period reports a dominant number versus positive opinions for new drug molecules not yet authorized in the EU. This overall leads to several advantages, with the main ones being the reduction of drug development time and costs.