The tailored scientific advice and regulatory guidance offered through EMA’s PRIME scheme are a major draw for sponsors.
While it is often assumed that PRIME is focussed on rare diseases, that is not necessarily the case. Sponsors developing products identified by EMA as public health priorities may be eligible. These include treatments for antimicrobial resistance or medicines that offer significant benefits for conditions with high societal burden, such as diabetes and Alzheimer’s disease.
The difficult path to PRIME approval
However, gaining PRIME designation is not easy. Around 75% of applications are rejected. This high rate of refusal has prompted some industry groups to argue that the eligibility criteria are too restrictive to truly foster innovation.
Recent reports from EMA suggest that these challenges persist. At its March 2025 meeting, the CHMP reviewed five recommendations for PRIME eligibility. Of those, two were approved: this included one for a nervous system disorder and another for infections and infestations, while three were denied. Rejected applications included another treatment for infections and infestations, an oncology product and a therapy for a genetic disorder.
These outcomes highlight the critical importance of having a strong regulatory strategy and an experienced partner who can help craft a compelling application that aligns with PRIME criteria.
The benefits for small sponsors
Early entry into PRIME offers particular advantages for small and mid-sized companies. These include guidance on manufacturing development, approaches to scale-up and guidance on anticipating and planning for potential issues. Addressing CMC (Chemistry, Manufacturing and Controls) concerns early in a development-appropriate manner can be a major asset.
Having that manufacturing development plan aligned with your clinical development strategy ensures that both scientific and quality aspects of your dossier meet EMA expectations.
Additionally, EMA and the US FDA have issued joint Q&As to support sponsors working across both regions, especially regarding quality and GMP requirements. This collaborative guidance helps streamline global development for companies navigating complex regulatory landscapes.
As development progresses towards marketing authorisation, there is also a strategic advantage in having a familiar EMA team. They will understand you product, appreciate its potential and have been closely involved in reviewing your data from an early stage.
The challenges sponsors face with obtaining PRIME designation underscores the critical importance of having a good regulatory partner to navigate the process.