The PRIority MEdicines (PRIME) scheme is an initiative from the European Medicines Agency (EMA) that provides early, proactive dialogue and enhanced support to drug developers for promising new medicines addressing significant unmet medical needs, so that development generates a robust data package for regulatory approval and market access. The scheme is voluntary and subject to an application based on adequate data to justify a potential major public health interest, with support tailored to meet the needs of development at different stages. PRIME does not lower the evidentiary standard for marketing authorisation and does not guarantee a positive regulatory outcome.
What is PRIME?
The PRIME scheme was launched in 2016 to facilitate an early, proactive dialogue between applicants and the EU regulatory network, ensuring the generation of a robust data package to address marketing authorization application requirements and enable accelerated assessment of new medicines targeting an unmet medical need.
The PRIME scheme is limited to medicines under development which are not authorized in the EU and for which the applicant intends to apply for an initial marketing authorisation application (MAA) through the centralized procedure.
Eligibility criteria
The eligibility criteria for PRIME are identical to EMA's MAA accelerated assessment criteria but are applied at an early stage of development with a higher degree of uncertainty compared to the time of accelerated assessment requests. Eligibility for the PRIME scheme considers both the magnitude of treatment effect and the clinical relevance of observed outcomes that predict effects on morbidity, mortality, or disease progression.
Products eligible to PRIME support shall:
- Target conditions where there is an unmet medical need, i.e., for which there exists no satisfactory method of diagnosis, prevention, or treatment in the Community or, even if such a method exists, in relation to which the medicinal product concerned will be of major therapeutic advantage to those affected.
- Demonstrate the potential to address the unmet medical need to a significant extent, for example, by introducing new methods of therapy or improving existing ones.
- Have the potential to provide a significant therapeutic advantage to patients with a specific medical condition based on available data and clinically meaningful outcomes.
- Provide evidence of clinical response in patients, substantiated by exploratory clinical studies addressing an unmet medical need, including human safety data supporting tolerability.
PRIME is not suitable for products that are in advanced stages of development, in the pre-submission phase of an MAA, or already authorized in the EU with a new indication being sought.
EMA recommends that most applicants apply during the exploratory clinical development phase when proof-of-concept data are available. SMEs and applicants from academic research may be eligible earlier where compelling non-clinical and early clinical data support the request.
How PRIME Works
The drugs or products must address an unmet medical need and target a seriously debilitating or life-threatening disease or a rare disease, or they must be intended for use in emergencies in response to a public health threat. It is important to emphasize that the expedited pathways will not lower the bar for approvals, and the intention is to deepen the partnership between the authorities and the drug developing companies. These interactions will support the generation of the most relevant data and will allow Health Authorities to prioritize the review of applications for drugs that may be crucial for patients with serious conditions.
In its guidelines, the EMA recommends that the applicant applies for PRIME while in the exploratory clinical trial phase and can demonstrate proof of concept that the investigational drug works. Micro, small, and medium-sized enterprises, and academic research may qualify for PRIME designation at an earlier stage of development if they have compelling non-clinical data in a relevant model that provide early evidence of promising activity (proof of principle) and first-in-human studies indicate adequate exposure for the desired pharmacotherapeutic effects and tolerability. In addition, PRIME support may be withdrawn if emerging data indicate that the criteria no longer apply. The CHMP will grant or deny PRIME designation within 40 days of the start of the PRIME application assessment.
Enhanced interaction and early dialogue
One of the main benefits of PRIME for drug developing companies is the potential for accelerated assessment, which can help to expedite the regulatory approval process and bring new treatments to patients more quickly. Another is the early and ongoing regulatory support provided by the EMA, including access to scientific advice, guidance on the overall development plan, and the opportunity to engage with relevant EMA committees and working parties. EMA has also introduced expedited scientific advice with shortened timelines, a submission readiness meeting, a regulatory roadmap, and a product development tracker as part of the enhanced support available through PRIME.
If we break down the key benefits, the drug developers are entitled to:
- Early appointment of a CHMP/CAT Rapporteur: Request for the appointment of a Rapporteur from the CHMP or CAT as early as possible in the development process to ensure continuity in the life-cycle approach.
- Kick-off meeting with the CHMP/CAT Rapporteur and a multidisciplinary group of EMA experts to provide preliminary guidance on the overall development plan and discuss key development steps, and the recommended regulatory strategy.
- A PRIME Scientific Coordinator, who will serve as a dedicated point of contact and coordinate the support offered throughout the scheme.
- Iterative scientific advice on the overall development plan at major development milestones and on key issues with the possibility to involve additional stakeholders such as health technology assessment (HTA) bodies, patients, and the US FDA.
- Access to expedited follow-up scientific advice under certain criteria with shortened timelines for receiving the adopted CHMP advice.
- A submission readiness meeting approximately one year before the MAA filing date to discuss the status of the development for key development areas, the maturity of the dossier, and plans for post-marketing evidence generation and any expected regulatory challenges.
- A dedicated toolbox to support the quality data package development.
PRIME remains a highly selective scheme. According to EMA's 2024 annual report, the Agency received 58 requests for PRIME eligibility and completed its assessment of 56 requests. Fifteen medicines were granted access to the scheme, corresponding to 27% of the requests assessed.
Conclusion
By engaging with the regulatory authorities early on, medicine developers can benefit from structured dialogue, scientific advice, and proactive support during development, with the potential for accelerated assessment at the marketing authorisation stage.
At SSI Strategy, we have experience of PRIME assessments from both client and agency perspective. Drop us a line by using the form below to discuss your PRIME strategy.
References
EMA, PRIME: priority medicines: https://www.ema.europa.eu/en/human-regulatory-overview/research-development/prime-priority-medicines
EMA, Guidance for applicants seeking access to PRIME scheme
EMA, Human medicines, Annual Report 2024: https://www.ema.europa.eu/assets/en/annual-report/2024/human-medicines/index.html
