PRIME is an EMA scheme that was launched in 2016 for priority medicines (PRIority MEdicines) that target an unmet medical need, enabling both additional regulator support during development and expedited evaluation to allow for earlier patient access. The aim is to improve the quality of the data generated during development to allow for a timely and high quality Marketing Authorisation Application (MAA). PRIME is an option for medicines in early development that may offer a major therapeutic advantage over existing treatments or benefit patients without treatment options.
Application for PRIME designation can be made once preliminary clinical data are available to show proof of concept or the potential of the medicine to significantly address an unmet need. Small businesses (SME) or non-commercial applicants can apply earlier, as long as nonclinical data are compelling and first in human studies indicate adequate exposure for the desired pharmacotherapeutic effects and tolerability. A pre-submission meeting to discuss PRIME eligibility with the EMA is possible and the PRIME application is made through the EMA IRIS portal.
The increased EMA support during development for medicines with PRIME designation, compared to those without designation, include:
- A Rapporteur from CHMP/CAT and a Scientific Coordinator from EMA, appointed early on in development for guidance on data requirements and regulatory strategy.
- PRIME kick-off multidisciplinary scientific and regulatory meeting with the CHMP/CAT Rapporteur, relevant EMA experts and the EMA product team. A regulatory roadmap is agreed, with maintenance of a product development tracker that will monitor development, help to identify topics warranting further discussion with regulators and act as a guide during meeting discussions.
- Scientific advice any time, or at key development milestones, with shortened timeframes. It is possible to involve other stakeholders if required (e.g., HTA bodies) to gain valuable input to ensure the correct data is generated for patient access. SMEs can request a fee waiver for scientific advice for a product with PRIME designation.
- Submission readiness meeting approximately 12 months before MAA, to discuss development status and scientific advice to identify any regulatory issues. Plans for post-marketing evidence generation must be presented at the meeting.
- Potential accelerated assessment (150 days instead of 210 days), which must be formally confirmed 2-3 months before MAA.
EMA PRIME metrics show that as of June 2025, 546 PRIME requests for designation were received with 68% of requests denied and only 26% of requests granted (6% were out of scope or withdrawn) (see EMA link). The low success rate for prime designation is because applications are made too early in development, with insufficient clinical data and before there is adequate proof of mechanism for the medicinal product. Applications were most prevalent within the therapeutic areas of oncology (27%), neurology (13%), endocrinology-gynaecology-fertility-metabolism (10%) and infectious diseases (7%). However, the success rate of applications in these therapeutic areas was also low at 35% for oncology (39/110), 24% for neurology (14/59), and 34% for infectious diseases (10/29), but slightly higher at 62.5% for endocrinology-gynaecology-fertility-metabolism (20/32).
The EMA’s 2016 goal for PRIME was to “foster better planning of medicine development to help companies generate the high quality data we need to assess quality, safety and efficacy of medicines,” so that “breakthroughs in medicines reach patients quicker”. The EMA performed a review of the first 5 years of PRIME, for the period March 2016 to June 2021 (see EMA link), and it was concluded that PRIME has had a positive impact in supporting marketing authorisation evaluation review and reducing overall time to marketing authorisation. The 5-year review showed that medicinal products with PRIME designations benefitted from reduced time for MAA due to a reduced ‘clock stop’ during the procedure where the applicant prepares answers to the EMA’s questions.
PRIME designation has a particularly positive impact on the approval of complex medicinal products, such as advanced therapies (ATMPs), as well as medicines with limited datasets, such as orphan products and ATMPs. It was reported that 7 ATMPs with PRIME designation had a shorter average assessment time than the average assessment time of all types of new active substances. Additionally, for orphan medicinal products, a 61-day decrease of the clock stop for PRIME products was observed compared to non-PRIME products.
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